PHAR
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Investor releaseQuarter not tagged2026-08-03Pharming Group (ENXTAM:PHARM) Could Be 63% Undervalued After Guidance Cut And Half Year Results
Simply Wall St.
Pharming Group (ENXTAM:PHARM) Could Be 63% Undervalued After Guidance Cut And Half Year Results
Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply Wall St. Pharming Group (ENXTAM:PHARM) has drawn fresh attention after lowering its 2026 revenue guidance by US$30 million and reporting half year 2026 results that showed reduced sales but a smaller net loss. See our latest analysis for Pharming Group. Pharming Group’s guidance cut and half year 2026 results appear to have fed into weaker sentiment, with the stock’s share price down 26.76% over 30 days and a 1 year total shareholder return of 5.37% in decline from prior levels. If this kind of news has you reassessing where growth or resilience might come from next, it could be worth scanning for opportunities in AI driven health stocks through the 128 healthcare AI stocks The guidance reset and sharp share price fall now leave Pharming Group trading at a steep discount to both analyst targets and an estimated fair value. Is that a clear mispricing, or a fair reflection of the risks ahead? The most followed narrative on Pharming Group compares a fair value of €2.36 to the last close of €0.88. It frames a wide gap that hinges on rare disease growth drivers and long term cash flows. Read the complete narrative. Curious what has to happen for that gap between price and fair value to close. The narrative leans heavily on multi year revenue compounding, margin uplift and a rich future earnings multiple. Want to see exactly which assumptions do the heavy lifting in this €2.36 figure. Result: Fair Value of €2.36 (UNDERVALUED) Have a read of the narrative in full and understand what's behind the forecasts. However, the Pharming Group narrative still faces meaningful risk if Joenja label expansions are delayed or if hereditary angioedema competition gradually eats into RUCONEST volumes. Find out about the key risks to this Pharming Group narrative. The earlier Pharming Group narrative leans on discounted future cash flows and points to a wide gap between price and fair value. On current numbers though, the stock trades on a P/E of 76.9x versus 35.5x for peers and a fair ratio of 44.8x, which suggests a high bar if earnings stumble. For investors, that raises a simple question: Is this a rare chance to buy misunderstood growth, or just paying up for forecasts that may not fully play out? See what the numbers say about this price — find out in our valuation b…Read full documentShow less
Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply Wall St. Pharming Group (ENXTAM:PHARM) has drawn fresh attention after lowering its 2026 revenue guidance by US$30 million and reporting half year 2026 results that showed reduced sales but a smaller net loss. See our latest analysis for Pharming Group. Pharming Group’s guidance cut and half year 2026 results appear to have fed into weaker sentiment, with the stock’s share price down 26.76% over 30 days and a 1 year total shareholder return of 5.37% in decline from prior levels. If this kind of news has you reassessing where growth or resilience might come from next, it could be worth scanning for opportunities in AI driven health stocks through the 128 healthcare AI stocks The guidance reset and sharp share price fall now leave Pharming Group trading at a steep discount to both analyst targets and an estimated fair value. Is that a clear mispricing, or a fair reflection of the risks ahead? The most followed narrative on Pharming Group compares a fair value of €2.36 to the last close of €0.88. It frames a wide gap that hinges on rare disease growth drivers and long term cash flows. Read the complete narrative. Curious what has to happen for that gap between price and fair value to close. The narrative leans heavily on multi year revenue compounding, margin uplift and a rich future earnings multiple. Want to see exactly which assumptions do the heavy lifting in this €2.36 figure. Result: Fair Value of €2.36 (UNDERVALUED) Have a read of the narrative in full and understand what's behind the forecasts. However, the Pharming Group narrative still faces meaningful risk if Joenja label expansions are delayed or if hereditary angioedema competition gradually eats into RUCONEST volumes. Find out about the key risks to this Pharming Group narrative. The earlier Pharming Group narrative leans on discounted future cash flows and points to a wide gap between price and fair value. On current numbers though, the stock trades on a P/E of 76.9x versus 35.5x for peers and a fair ratio of 44.8x, which suggests a high bar if earnings stumble. For investors, that raises a simple question: Is this a rare chance to buy misunderstood growth, or just paying up for forecasts that may not fully play out? See what the numbers say about this price — find out in our valuation breakdown. With sentiment on Pharming Group split between concern and optimism, it may be useful to review the full picture for yourself using the 4 key rewards and 2 important warning signs. If Pharming Group has you rethinking where to focus next, it makes sense to broaden your watchlist using structured stock ideas rather than chasing headlines. Use the Simply Wall St Screener to quickly compare potential opportunities and keep your shortlist aligned with your own goals and risk comfort. Target potential upside by scanning companies highlighted in the 259 high quality undervalued stocks. These combine quality fundamentals with prices below their estimated worth. Strengthen your income stream by shortlisting stocks from the 433 dividend fortresses, which focuses on higher yields with an emphasis on resilience. Prioritise capital preservation by filtering companies in the 303 resilient stocks with low risk scores, which emphasises stability over aggressive growth. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include PHARM.AS. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]
Investor releaseQuarter not tagged2026-07-30Pharming Group's Q2 Earnings, Revenue Fall; 2026 Revenue Outlook Updated
MT Newswires
Pharming Group's Q2 Earnings, Revenue Fall; 2026 Revenue Outlook Updated
Pharming Group (PHAR) reported a Q2 earnings Thursday of $0.002 per diluted share, compared with $0.
Investor releaseQuarter not tagged2026-07-30Pharming reports second quarter and first half 2026 financial results and revises year-end guidance; strong Joenja momentum and near-term clinical readouts to support broader therapeutic use
GlobeNewswire
Pharming reports second quarter and first half 2026 financial results and revises year-end guidance; strong Joenja momentum and near-term clinical readouts to support broader therapeutic use
For investor audiences and media only Second quarter 2026 total revenues decreased by 3% to US$90.2 million, compared to the second quarter 2025 RUCONEST® revenue was US$72.3 million, a 10% decrease compared to the second quarter 2025 and a 24% increase compared to the first quarter 2026, with active patient base 93% of year-ago and strong new patient enrollments Joenja® revenue was US$17.9 million, a 40% increase compared to the second quarter 2025, reflecting continued strong U.S. and international growth, with first European launch in Germany after quarter-end Second quarter operating profit amounted to US$1.3 million compared to US$10.8 million in the second quarter 2025, impacted by manufacturing-related inventory impairments, lower revenue and France site closure Plans to report clinical data with leniolisib in significantly larger CVID patient populations in the fourth quarter 2026 Updates 2026 total revenue guidance to US$375 million - US$395 million, reflecting a US$30 million reduction, and improves operating expense guidance by US$15 million to US$315 million - US$320 million Pharming to host a conference call today at 13:30 CEST (7:30 am EDT) Leiden, the Netherlands, July 30, 2026: Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the second quarter and first half year ended June 30, 2026. Chief Executive Officer, Fabrice Chouraqui, commented:“While we have lowered our full-year revenue guidance, we are encouraged by the resilience of RUCONEST, a year after the launch of the first oral on-demand HAE treatment. The active patient base remained at 93% of year ago levels, and RUCONEST continues to be a cornerstone on-demand treatment for high-burden HAE patients. Supported by strong new patient enrollments in the second quarter, we expect RUCONEST revenues to stabilize and return to growth during the second half of 2026. Joenja (leniolisib) continued to build momentum in APDS, supported by expansion into additional geographies. Beyond APDS, we are encouraged by the potential for leniolisib in broader populations with primary immunodeficiencies, including CVID, where the PI3Kδ pathway is seen as a key driver of immune dysregulation, and we look forward to reporting data from two clinical trials in the fourth quarter of 2026. As we continue to implement a more disciplined operating m…Read full documentShow less
For investor audiences and media only Second quarter 2026 total revenues decreased by 3% to US$90.2 million, compared to the second quarter 2025 RUCONEST® revenue was US$72.3 million, a 10% decrease compared to the second quarter 2025 and a 24% increase compared to the first quarter 2026, with active patient base 93% of year-ago and strong new patient enrollments Joenja® revenue was US$17.9 million, a 40% increase compared to the second quarter 2025, reflecting continued strong U.S. and international growth, with first European launch in Germany after quarter-end Second quarter operating profit amounted to US$1.3 million compared to US$10.8 million in the second quarter 2025, impacted by manufacturing-related inventory impairments, lower revenue and France site closure Plans to report clinical data with leniolisib in significantly larger CVID patient populations in the fourth quarter 2026 Updates 2026 total revenue guidance to US$375 million - US$395 million, reflecting a US$30 million reduction, and improves operating expense guidance by US$15 million to US$315 million - US$320 million Pharming to host a conference call today at 13:30 CEST (7:30 am EDT) Leiden, the Netherlands, July 30, 2026: Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the second quarter and first half year ended June 30, 2026. Chief Executive Officer, Fabrice Chouraqui, commented:“While we have lowered our full-year revenue guidance, we are encouraged by the resilience of RUCONEST, a year after the launch of the first oral on-demand HAE treatment. The active patient base remained at 93% of year ago levels, and RUCONEST continues to be a cornerstone on-demand treatment for high-burden HAE patients. Supported by strong new patient enrollments in the second quarter, we expect RUCONEST revenues to stabilize and return to growth during the second half of 2026. Joenja (leniolisib) continued to build momentum in APDS, supported by expansion into additional geographies. Beyond APDS, we are encouraged by the potential for leniolisib in broader populations with primary immunodeficiencies, including CVID, where the PI3Kδ pathway is seen as a key driver of immune dysregulation, and we look forward to reporting data from two clinical trials in the fourth quarter of 2026. As we continue to implement a more disciplined operating model, reflected in our improved full-year operating expense guidance, Pharming is evolving into a more diversified rare disease company with a high-value pipeline that has the potential to materially increase our scale and strengthen our long-term growth profile.” Second quarter and first half 2026 highlightsCommercialized assetsRUCONEST marketed for the treatment of acute HAE attacks RUCONEST revenue in the second quarter of 2026 was US$72.3 million, a 10% decrease compared to the second quarter of 2025 and a 24% increase compared to the first quarter of 2026. Revenue for the first half of 2026 was US$130.7 million, a 12% decrease compared to the same period in 2025. RUCONEST revenue in the current quarter compared to the second quarter of 2025 was impacted by market dynamics in the U.S. (reducing revenue by 6%), inventory drawdowns at U.S. specialty pharmacy customers, and the completion of the planned withdrawal from non-U.S. markets. With its differentiated efficacy, reliability and rapid onset of action via IV administration, RUCONEST remains a trusted on-demand treatment option for high-burden patients experiencing more severe or frequent attacks who have failed other on-demand medications, despite increasing competition. The overwhelming majority of RUCONEST patients remain on therapy with the active patient base at 93% of year ago levels. Notably, we achieved a significant increase in new patient enrollments and continued to add new prescribers in the current quarter, indicating continued physician confidence and improvement in underlying performance. Joenja (leniolisib) marketed for the treatment of APDS Joenja revenue increased to US$17.9 million in the second quarter of 2026, a 40% increase compared to the second quarter of 2025. Revenue for the first half of 2026 was US$32.0 million, a 37% increase compared to the same period in 2025. Year-over-year revenue growth in the current quarter was driven by a strong increase in patients on paid therapy in the U.S., inventory normalization at U.S. specialty pharmacy customers following greater drawdowns in the first quarter, and increased demand in international markets. The U.S. market contributed 86% of second quarter revenues, while the EU and Rest of World contributed 14%. As of June 30, 2026, 132 patients were on paid therapy in the U.S., representing a 16% increase from the 114 patients at the end of the second quarter of 2025 and an increase of 5 patients during the quarter. APDS patient finding As of June 30, 2026, we have identified 1,042 diagnosed APDS patients of all ages globally, including 298 patients in the U.S. and 387 in core markets outside of the U.S. Of the identified patients in the U.S., 198 patients are 12 years of age or older and currently eligible for treatment with Joenja, while 60 are between 4 and 11 years of age. Joenja (leniolisib) developmentLeniolisib for APDS As of June 30, 2026, there are 188 APDS patients in either a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a paid access program. Pediatric label expansion On June 4, 2026, we announced that the U.S. Food and Drug Administration (FDA) had accepted our resubmitted supplemental New Drug Application (sNDA) seeking approval for Joenja as a treatment for children aged 4 to 11 years with APDS. Following the Complete Response Letter (CRL) received on January 30, 2026, and a subsequent Type A meeting with the FDA on March 26, 2026, the resubmission seeks approval of 40 mg and 50 mg twice-daily dosing for pediatric patients weighing 27 kg or more, who represent a meaningful proportion of the identified pediatric patient population. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 24, 2026. We plan to submit a separate sNDA in the coming days, seeking approval for lower doses in patients weighing 13 - 27 kg. Japan We expect to launch Joenja for the treatment of APDS in adult and pediatric patients aged 4 years and older in the third quarter of 2026. European Economic Area (EEA) On May 22, 2026, we announced that the European Commission (EC) had granted Marketing Authorization for Joenja as the first and only approved treatment of APDS in adult and pediatric patients 12 years of age and older. Joenja was commercially launched in Germany on July 1, 2026, marking the first European launch following EC approval, with additional launches anticipated pending completion of national reimbursement negotiations. Other countries On July 8, 2026, Joenja was approved by Health Canada for the treatment of APDS in adult and pediatric patients 12 years of age and older. On July 10, 2026, Joenja was approved by the South Korea Ministry of Food and Drug Safety for the treatment of APDS in adult and pediatric patients 12 years of age and older. Leniolisib for additional primary immunodeficiencies (PIDs) Two Phase II clinical trials are evaluating leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, including genetically identifiable PIDs linked to altered PI3Kδ signaling and common variable immunodeficiency or CVID, which represent substantially larger patient populations than APDS. Patient enrollment in both clinical trials is complete and we anticipate trial read-outs in the fourth quarter of 2026, consistent with prior guidance. The PI3Kδ pathway is seen as a key driver of immune dysregulation in many PIDs, and we currently anticipate conducting a single registrational Phase III trial in the broader CVID indication, incorporating patient populations from both studies. A presentation at the 2026 Annual Meeting of the Clinical Immunology Society (CIS), which took place May 6-9, included clinician expanded access experience with leniolisib to treat immune dysregulation in patients with CVID and CVID-like disorders. Clinician-reported outcomes demonstrated improvements, with no progression, in clinical manifestations of immune dysregulation as well as improvements in patients’ quality of life. Other events As we continue to improve operating efficiency in line with our strategy, we have decided to close our production-support site in Évry, France, with the closure expected to take effect in the fourth quarter of 2026. As a result, we recognized a one-time charge in the current quarter to reflect the estimated costs associated with the planned closure.Financial Summary Figures may not add up due to rounding. Financial highlightsSecond quarter 2026 For the second quarter of 2026, total revenues decreased by US$3.0 million, or 3%, to US$90.2 million, compared to US$93.2 million in the second quarter of 2025. RUCONEST revenues amounted to US$72.3 million, a 10% decrease compared to the second quarter of 2025. The decrease in RUCONEST® revenues was primarily driven by a decrease in volume. Joenja revenues amounted to US$17.9 million in the second quarter of 2026, a 40% increase compared to the second quarter of 2025. This increase in Joenja revenues was primarily driven by an increase in volume. Gross profit decreased by US$8.5 million, or 10%, to US$75.7 million, compared to US$84.2 million in the second quarter of 2025, mainly due to manufacturing-related impairments of inventory (US$4.9 million) and the decrease in revenues. The operating profit amounted to US$1.3 million compared to US$10.8 million in the second quarter of 2025. Excluding US$4.9 million in one-time manufacturing-related impairments of inventory and US$1.7 million in expenses associated with the planned closure of the production-support site in Évry, France, adjusted operating profit1 in the second quarter 2026 amounted to US$7.9 million. Excluding US$2.1 million of non-recurring Abliva acquisition-related expenses, adjusted operating profit in the second quarter 2025 amounted to US$12.9 million. The operating result was primarily impacted by manufacturing-related impairments of inventory and a decrease in revenues, while operating expenses remained similar to the second quarter of 2025. The finance result (net) and share of result in associates amounted to a gain of US$1.8 million compared to a loss of US$3.7 million in the second quarter of 2025. This improvement was mainly driven by favorable EUR/USD exchange rate movements, resulting in a foreign currency gain of US$2.9 million in 2026, compared to a loss of US$1.9 million in the second quarter of 2025. The Company had a net profit of US$1.6 million, compared to US$4.6 million in the second quarter of 2025. The effect of the aforementioned drivers was partially offset by a favorable change in the net finance result. Cash used in operations amounted to US$9.7 million, compared to US$11.7 million cash generated from operations in the second quarter of 2025. Cash and cash equivalents, restricted cash and marketable securities decreased from US$171.8 million at the end of first quarter of 2026 to US$159.5 million at the end of the second quarter of 2026, primarily driven by unfavorable working capital movements, mainly a decrease in trade and other payables and an increase in inventories, as well as income tax payments, partially offset by collections of trade and other receivables. 1 Adjusted Operating Profit is a non-IFRS measure used by management to assess underlying operating performance and provides additional insight into the Company's core operating profitability. It excludes certain non-core items. First half year 2026 Total revenues decreased 6% during the first half of 2026 to US$162.7 million, compared to US$172.3 million during the first half of 2025. For the first half of 2026, total RUCONEST revenues were 12% lower at US$130.7 million, compared to revenues of US$149.0 million for the first half of 2025. The decrease in RUCONEST revenues was primarily driven by a decrease in volume. Joenja revenues amounted to US$32.0 million in the first half of 2026, a 37% increase compared to the first half of 2025. This increase in Joenja revenues was primarily driven by an increase in volume. Gross profit decreased by US$13.5 million, or 9%, to US$141.5 million, compared to US$155.0 million in the first half of 2025, mainly due to the decrease in revenues and manufacturing-related impairments of inventory (US$4.9 million). Further details on revenue and gross profit segmentation is provided in Note 7. Segment information in the Notes to the condensed consolidated interim financial statements of this press release.The operating loss amounted to US$3.6 million compared to an operating profit of US$3.8 million in the first half of 2025. Excluding US$4.9 million in one-time manufacturing-related impairments of inventory and US$1.7 million in expenses associated with the planned closure of the production-support site in Évry, France, adjusted operating profit in the first half 2026 amounted to US$3.0 million. Excluding US$9.9 million of non-recurring Abliva acquisition-related expenses, adjusted operating profit in the first half 2025 amounted to US$13.7 million. The deteriorated operating result was primarily driven by a decrease in revenues and manufacturing-related impairments of inventories in 2026. The finance result (net) and share result in associates amounted to a gain of US$1.9 million compared to a loss of US$8.5 million in the first half of 2025. This improvement was mainly driven by favorable EUR/USD exchange rate movements, resulting in a foreign currency gain of US$5.3 million in the first half year of 2026, compared to a loss of US$4.5 million in the first half year of 2025.The Company had a net loss of US$3.6 million, compared to a net loss of US$10.3 million in the first half of 2025. In addition to the aforementioned drivers, the net result was positively impacted by a lower tax expense of US$1.9 million compared to US$5.6 million in the first half of 2025. Cash used in operations amounted to US$7.7 million, compared to US$12.0 million of cash generated from operations in the first half of 2025. Cash and cash equivalents, restricted cash and marketable securities decreased by US$21.6 million to US$159.5 million from US$181.1 million at the end of 2025, primarily driven by negative working capital movements, increased income tax payments and US$12.3 million settlement of the lease liability following the early termination of the DSP facility lease at Pivot Park in Oss, the Netherlands. Outlook/Summary For 2026, the Company anticipates: Total revenues between US$375.0 million and US$395.0 million (0% to 5% growth), updated to reflect a US$30 million reduction compared with prior guidance. Total operating expenses between US$315.0 million and US$320.0 million (1% to 3% growth), including over US$40 million incremental R&D investment to advance the pipeline and US$9 million structural G&A cost reductions based on the plan announced in October 2025, reflecting an improvement of US$15 million from prior guidance. RUCONEST revenue stabilization and return to growth during second half of 2026, and significant and accelerating annual Joenja U.S. and ex-U.S. growth. Additional regulatory approvals and commercial launches for leniolisib for APDS patients 12 years of age or older and for pediatric label expansion in key global markets. Top-line data readouts for the two ongoing leniolisib Phase II clinical trials in PIDs with immune dysregulation, including CVID, to expand the asset’s addressable patient population. Completion of enrollment in the pivotal FALCON clinical study for napazimone (KL1333) in mitochondrial DNA-driven primary mitochondrial diseases. Enhancing capital efficiency to drive growth and build a leading global rare disease company. Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases. Financing, if required, would come via a combination of our strong balance sheet and access to capital markets. No further specific financial guidance for 2026 is provided. TrademarksJoenja® and RUCONEST® are registered trademarks owned by or licensed to Pharming Group N.V. or its affiliates. Additional informationPresentation The conference call presentation is available on the Pharming.com website from 07:30 CEST today. Conference Call The conference call will begin at 13:30 CEST/07:30 EDT on Thursday, July 30. A transcript will be made available on the Pharming.com website in the days following the call. Please note, the Company will only take questions from dial-in attendees. Webcast Link: https://edge.media-server.com/mmc/p/m35zejc7 Conference call dial-in details: https://register-conf.media-server.com/register/BId285845cff504c39931913d279f646ea Additional information on how to register for the conference call/webcast can be found on thePharming.com website. Financial Calendar 2026 3Q 2026 financial results November 5, 2026 For further public information, contact: PharmingMichael Levitan, VP Investor Relations and Capital MarketsT: +1 (908) 705 1696E: [email protected] Saskia Mehring, Head of Corporate CommunicationsT: +31 6 28 32 60 41E: [email protected] Media RelationsJulia Deutsch (Lyra Strategic Advisory on behalf of Pharming)E: [email protected] Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)T: +31 6 53 81 64 27 About Pharming Group N.V. Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations. For more information, visit www.pharming.com and find us on LinkedIn. Auditor’s involvement The Condensed Consolidated Interim Financial Statements have not been audited by the Company’s statutory auditor. Responsibility Statement The Board of Directors of the Company (the “Board”) hereby declares that to the best of its knowledge, the condensed consolidated interim financial statements, which have been prepared in accordance with IAS 34 (interim financial reporting), give a true and fair view of the assets, liabilities, financial position and profit or loss of the Company, and this interim Board report includes a fair review of the information required pursuant to section 5:25d(8) and (9) of the Dutch Financial Supervision Act (Wet op het financieel toezicht). Leiden, July 30, 2026 Fabrice Chouraqui, Chief Executive Officer and Executive DirectorRichard Peters, Non-Executive Director and Chairman of the Board of DirectorsMark Pykett, Non-Executive DirectorBarbara Yanni, Non-Executive DirectorLeonard Kruimer, Non-Executive DirectorJabine van der Meijs, Non-Executive DirectorElaine Sullivan, Non-Executive Director Forward-looking Statements This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information. Inside Information This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. Pharming Group N.V. Condensed Consolidated Interim Financial Statements in US Dollars (unaudited) For the period ended June 30, 2026 Condensed consolidated interim statement of income Condensed consolidated interim statement of comprehensive income Condensed consolidated interim balance sheet Condensed consolidated interim statement of changes in equity Condensed consolidated interim statement of cash flow 2 Comparative presentation updated for consistency. Notes to the condensed consolidated interim financial statements For the period ended June 30, 2026 1. Company information Pharming Group N.V. is a limited liability public company which is listed on Euronext Amsterdam (PHARM) and on the NASDAQ (PHAR), with its headquarters and registered office located at: Darwinweg 24 2333 CR Leiden The Netherlands Pharming Group N.V. is a global biotechnology company that develops and commercializes innovative therapies for rare and ultra-rare diseases with significant unmet need. We focus on immunological and genetic conditions where our scientific and commercial expertise can help advance care over the long term. 2. Statement of compliance The consolidated interim financial statements for the six-month period ended June 30, 2026, have been prepared in accordance with International Accounting Standard IAS 34, Interim financial reporting. The condensed consolidated interim financial statements should be read in conjunction with the annual financial statements for the year ended December 31, 2025. They do not include all of the information required for a complete set of financial statements in accordance with IFRS Accounting Standards. However, selected explanatory notes are included to explain events and transactions that are significant to an understanding of the changes in the Group’s financial position and performance since the last annual financial statements. These condensed consolidated interim financial statements were authorized for issue by the Board of Directors on July 29, 2026. The published figures in these condensed consolidated interim financial statements are unaudited. 3. Accounting policies Accounting policies are consistent with those of the financial statements for the year ended December 31, 2025. The following exchange rates have been applied: 4. Estimates and judgements The preparation of interim financial statements in conformity with IAS 34 and Book 2 Title 9 of the Dutch Civil Code requires the use of certain critical accounting estimates. It also requires management to exercise its judgement in the process of applying the Company’s accounting policies. In preparing these condensed consolidated interim financial statements, the significant judgements made by management in applying the Company’s accounting policies were the same as those applied to the consolidated financial statements for the year ended December 31, 2025. 5. Going concern In preparing and finishing the interim financial statements the Board of Directors of Pharming have assessed the Company’s ability to fund its operations for a period of at least twelve months after the date the interim financial statements are issued. Based upon the assessment on a going concern basis, the Company has concluded that funding of its operations for a period of twelve months, after the date the interim financial statements are issued, is realistic and achievable. Overall, based on the outcome of this assessment, the interim financial statements have been prepared on a going concern basis. 6. Seasonality of operations Seasonality has no material impact on Company’s interim financial statements. 7. Segment information Operating segments are components of the Company that engage in business activities from which it may incur expenses, for which discrete financial information is available and whose operating results are evaluated regularly by the Company’s Chief Operating Decision Maker (“CODM”) to make decisions about resources to be allocated to the segment and assess its performance. The Executive Members of the Board of Directors are considered the CODM. CODM reviews the Company’s results under four operating segments based on a combination of the products that the Company has launched - RUCONEST and Joenja, and the main geographies where sales are consummated - focused on the US and reporting, in aggregate, EU and Rest of the World (“RoW”). The four operating segments correspond to each of its four reportable segments for financial reporting purposes. The CODM reviews revenues and gross profit to assess the performance of their operating segments, which are the sole performance measures on segment level which are provided regularly to the CODM. No other information, such as operating expenses, assets or other performance indicators on segment level are provided regularly to the CODM. Total revenues and gross profit per each operating and reportable segment for the period ended June 30 are: There are no intersegment sales. 8. Other income Other income decreased by US$0.4 million in the first half of 2026 to US$1.8 million as compared to US$2.2 million the first half of 2025. 9. Expenses by nature Costs of sales Costs of inventories recognized as expenses in the first half year of 2026 were US$12.7 million versus US$13.2 million for the first half of 2025 and relates to actual product sales of RUCONEST and Joenja. Pharming expensed royalty fees to Novartis on Joenja sales, amounting to US$3.4 million in the first half of 2026 (first half of 2025: US$2.4 million). Inventory impairments amounted to US$5.1 million (1H 2025: US$1.7 million) and stems from the valuation of the inventories against lower net realizable value and mainly relates to material no longer expected to be used for commercial sales. Other operating costs Other operating costs decreased to US$146.9 million in the first half of 2026 compared to US$153.4 million in the first half year of 2025. Employee benefits are charged to research and development costs, general and administrative costs, or marketing and sales costs based on the nature of the services provided. Employee benefits of production related employees have been included in the value of inventories. Depreciation and amortization charges amounted to US$6.0 million in the first half of 2026 compared to US$5.3 million the first half year of 2025, and related to the following: 10. Finance income (expenses) Foreign currency results primarily reflect movements in the EUR/USD exchange rate. The strengthening of the U.S. dollar against the euro during the first half of 2026 (compared with a weakening during the first half of 2025) generated foreign exchange gains. These gains were mainly attributable to the revaluation of the U.S. dollar-denominated cash and marketable securities balances held by euro functional currency entities and euro-denominated monetary assets and liabilities held by the U.S. dollar functional currency entity. Interest income increased compared with the first half of 2025 due to a higher average balance of cash and marketable securities following the completion of the Abliva acquisition in the first half of 2025, as well as higher effective yields earned on cash and cash equivalents. 11. Income tax (expenses) Income tax expenses are recognized in each interim period based on the best estimate of the weighted average annual effective income tax rate expected for the full financial year. 12. Investments Investments accounted for using the equity method The asset relates to an investment in the ordinary shares of BioConnection Investments B.V. (“BioConnection”). In the Board of Directors’ judgement, the investment in BioConnection constitutes an investment in an associated company and is therefore not consolidated. Pharming has significant influence but does not have control of BioConnection and is embargoed by a shareholder’s agreement between the shareholders of BioConnection from influencing any activity between the two parties which is in any significant way different from the relationship which existed between the two prior to the investment. The carrying amount of this investment has changed as follows: Investment in debt instruments designated as at FVTPL The asset relates to the preference share in BioConnection Investments B.V. The Board of Directors made an assessment on the accounting treatment of the preference share obtained. The Board concluded that the asset should be recognized as a financial asset (debt instrument) measured at initial recognition at fair value, subsequently measured at fair value through profit and loss. The fair value is calculated on a yearly basis using the forward-looking Black-Scholes-Merton (“BSM”) financial instrument pricing framework. No events or matters are known as of the date of this report which would lead to a significant impact in the fair value of the asset, compared to December 31, 2025. The carrying amount of this investment has changed as follows: 13. Deferred tax assets The deferred tax assets decreased mainly due to foreign exchange effects and changes in temporary differences. 14. Inventories Inventories include batches of Joenja and RUCONEST and related work in progress. Changes in the adjustment to net realizable value: The inventory valuation at June 30, 2026, of US$65.4 million (December 31, 2025: US$64.9 million) is stated net of an impairment of US$16.6 million (December 31, 2025: US$13.1 million). The impairment relates to the write down of inventories to their net realizable value. Inventories are available for use in commercial, preclinical and clinical activities. Estimates have been made with respect to the ultimate use or sale of product, taking into account current and expected sales as well as preclinical and clinical programs. These estimates are reflected in the additions to the impairment. The costs of materials used in research and development activities are presented under the research and development costs. The main portion of inventories at June 30, 2026, have expiration dates starting beyond 2026 and are all expected to be sold and/or used before expiration. Cash, cash equivalents and marketable securities Since April 1, 2026, the marketable securities are considered to be cash equivalents as they are managed from that date as part of the Group’s total liquidity position and are held to meet potential short-term cash commitments rather than for investment purposes. The money market funds consist of LVNAV money market funds regulated under the EU Money Market Fund Regulation, which imposes liquidity, maturity, diversification and credit quality requirements broadly comparable to those applicable to SEC Rule 2a‑7 money market funds. The carrying value includes accrued interest of US$0.3 million as of June 30, 2026 (December 31, 2025: US$0.1 million). Cash is free at disposal of the Company. 16. Equity The Company’s authorized share capital amounts to €10.56 million (US$12.0 million) and is divided into 1,056,000,000 ordinary shares with a nominal value of €0.01 each. All 707,781,240 shares outstanding at June 30, 2026, have been fully paid-up. Other reserves include those reserves related to currency translation, share-based compensation expenses and other equity-settled transactions. Please refer to the Condensed consolidated interim statement of changes in Equity. The other reserves are made up as shown in the below table. 17. Convertible bonds In April 2024, the Company issued €100.0 million (US$113.9 million, based on the EUR/USD exchange rate as of June 30, 2026) aggregate principal amount of 4.50% convertible bonds due 2029. The movements of the convertible bonds were as follows: 18. Earnings per share and diluted shares Basic earnings per share is calculated based on the weighted average number of ordinary shares outstanding during the period. Diluted earnings per share in the case of a profit is computed based on the weighted average number of ordinary shares outstanding including the dilutive effect of shares to be issued in the future under certain arrangements such as option plans. However, as the net result represents a loss, the diluted earnings per share are equal to the basic earnings per share. For 1H 2026 and 1H 2025, the basic and diluted earnings per share are: Diluted shares The composition of the number of shares and share rights outstanding as well as authorized share capital as per June 30, 2026 is provided in the table below: 19. Financial risk management and fair value Financial risk management Pharming is exposed to several financial risks: market risks (being currency risk and interest rate risk), credit risks and liquidity risks. The Board of Directors and the Executive Committee are responsible for the management of currency, interest, credit and liquidity risks and as such ultimately responsible for decisions taken in this field. The Group’s exposure to financial risks has not materially changed during the period. Fair value The following table provides information on the fair value of financial instruments not measured at fair value as at the reporting date: For financial instruments not included in the table above, the carrying amount is a reasonable approximation of fair value. The following table presents information on the fair value measurement hierarchy of financial instruments measured at fair value: Further information on the investments in debt instruments designated as at FVTPL is included in note 12. During the six-month period ended June 30, 2026, there have been no changes related to the fair value hierarchy. Comparative information was updated for consistency. 20. Related party transactions There are no material changes in the nature, scope, and scale in this reporting period compared to last year. More information is included in note 24 to the consolidated financial statements as at and for the year ended December 31, 2025. 21. Events since the end of the reporting period There were no significant events since the end of the reporting period. Attachment Pharming Group reports 2Q_1H26 results_EN_30JULY2026
Investor releaseQuarter not tagged2026-07-30Pharming Group (PHGUF) (Q2 2026) Earnings Call Highlights: Joenja Surges 40% as Ruconest Faces ...
GuruFocus.com
Pharming Group (PHGUF) (Q2 2026) Earnings Call Highlights: Joenja Surges 40% as Ruconest Faces ...
This article first appeared on GuruFocus. Release Date: July 30, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Ruconest active patient base remained resilient at 93% of prior year levels despite oral competition, with new patient enrollments recovering to near pre-competitive levels. Joenja delivered strong 40% year-over-year revenue growth, driven by U.S. momentum and accelerating European expansion. Two Phase 2 studies for leniolisib in broader primary immunodeficiencies (CVID and genetic PIDs) are fully enrolled, with top-line results expected in Q4 2026, representing a potential multi-billion-dollar opportunity. Operating expense guidance was reduced by $15 million to $315-$320 million, reflecting disciplined cost management and improved operational efficiency. Near-term catalysts include U.S. pediatric label expansion for Joenja (PDUFA date October 24, 2026) and upcoming launches in Germany and Japan. Total Q2 2026 revenue declined 3% year-over-year, driven by a 10% drop in Ruconest revenue due to competitive dynamics and inventory normalization. Full-year 2026 revenue guidance was lowered by $30 million to $375-$395 million, reflecting ongoing market share pressure from oral on-demand HAE treatments. Operating cash flow was negative $9.7 million in Q2, impacted by strategic inventory build and lower revenues. A $4.9 million impairment of manufacturing inventory for Ruconest was recorded, indicating potential production or quality issues. The planned closure of a production support site in France and associated restructuring costs highlight ongoing operational challenges. Here are the key highlights from Pharming Group's Q2 2026 earnings call, focusing on the most significant Q&A exchanges. Warning! GuruFocus has detected 5 Warning Sign with LNC. Is PHGUF fairly valued? Test your thesis with our free DCF calculator. Q: Regarding the upcoming Phase 2 data for leniolisib in CVID and genetic PIDs, what are the next steps assuming a positive outcome? A: (Anurag Relen, Chief Medical Officer) We expect results in Q4. The studies have similar patient populations with significant overlap. We anticipate conducting a single registrational Phase III trial in the broader CVID indication, likely a randomized control study similar to the one performed in APDS. We will review the data and dose regimen with th…Read full documentShow less
This article first appeared on GuruFocus. Release Date: July 30, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Ruconest active patient base remained resilient at 93% of prior year levels despite oral competition, with new patient enrollments recovering to near pre-competitive levels. Joenja delivered strong 40% year-over-year revenue growth, driven by U.S. momentum and accelerating European expansion. Two Phase 2 studies for leniolisib in broader primary immunodeficiencies (CVID and genetic PIDs) are fully enrolled, with top-line results expected in Q4 2026, representing a potential multi-billion-dollar opportunity. Operating expense guidance was reduced by $15 million to $315-$320 million, reflecting disciplined cost management and improved operational efficiency. Near-term catalysts include U.S. pediatric label expansion for Joenja (PDUFA date October 24, 2026) and upcoming launches in Germany and Japan. Total Q2 2026 revenue declined 3% year-over-year, driven by a 10% drop in Ruconest revenue due to competitive dynamics and inventory normalization. Full-year 2026 revenue guidance was lowered by $30 million to $375-$395 million, reflecting ongoing market share pressure from oral on-demand HAE treatments. Operating cash flow was negative $9.7 million in Q2, impacted by strategic inventory build and lower revenues. A $4.9 million impairment of manufacturing inventory for Ruconest was recorded, indicating potential production or quality issues. The planned closure of a production support site in France and associated restructuring costs highlight ongoing operational challenges. Here are the key highlights from Pharming Group's Q2 2026 earnings call, focusing on the most significant Q&A exchanges. Warning! GuruFocus has detected 5 Warning Sign with LNC. Is PHGUF fairly valued? Test your thesis with our free DCF calculator. Q: Regarding the upcoming Phase 2 data for leniolisib in CVID and genetic PIDs, what are the next steps assuming a positive outcome? A: (Anurag Relen, Chief Medical Officer) We expect results in Q4. The studies have similar patient populations with significant overlap. We anticipate conducting a single registrational Phase III trial in the broader CVID indication, likely a randomized control study similar to the one performed in APDS. We will review the data and dose regimen with the FDA and other regulators to finalize the plan. Q: New Ruconest enrollments recovered to 84 in Q2, close to pre-competitive launch levels. Has this trend continued into Q3? A: (Levern March, Chief Commercial Officer) Yes, the momentum is consistent into July. While there is some seasonality in the HAE market, the trend is holding. We continue to add new prescribers, reinforcing Ruconest's entrenched position in high-burden HAE patients. Q: Can you provide more color on the commercial launch efforts for Joenja in the EU and the magnitude of the opportunity? A: (Levern March, Chief Commercial Officer) We have identified about 387 potentially eligible APDS patients across our core 8 markets. The UK launch is proceeding at pace, the German launch is underway since July 1st, and we are preparing to launch in Japan in August. We are assuming a similar conversion rate to what we've seen in the U.S. as we secure reimbursement. Q: For the 84 new Ruconest patients, what do they look like in terms of severity and attack rate? Are they all new to therapy? A: (Levern March, Chief Commercial Officer) These are high-burden patients with high-frequency attacks, more severe attacks, and concerns about high-risk attack locations like laryngeal attacks. The 84 figure represents pure new enrollments (first time on Ruconest) and does not include patients returning to therapy. Q: Regarding the larger CVID and PID patient populations, how well characterized are these patients and how accessible are they commercially? A: (Anurag Relen, Chief Medical Officer) This is very different from APDS. CVID is clinically diagnosed, and these patients are largely already identified. They are almost always on immunoglobulin replacement therapy. This means the diagnosis mechanics are different and less reliant on genetic testing compared to APDS. Q: With the upcoming CVID data, could it provide any incremental boost to Joenja revenue prior to potential approval? A: (Fabrice Chouraqui, CEO) No. We will engage with the FDA and assume we need to conduct a Phase III trial. Until we receive proper approval, we cannot promote the data and do not expect any sales from CVID. However, the many opportunities in APDS, including pediatric expansion and geographic expansion, will fuel Joenja's growth in the coming years. Q: On the on-demand HAE market, how do you expect new patient initiations to play out? Will patients still find Ruconest after trying other drugs? A: (Levern March, Chief Commercial Officer) The vast majority of new enrollments come from patients who have had suboptimal responses to other therapies and are switching to Ruconest. We expect this to continue because patients with high severity or frequent attacks (two or more per month) require a more reliable, faster-onset treatment, where Ruconest is uniquely positioned. Q: Could you provide more color on the manufacturing impairment of inventory? Which product does it relate to? A: (Kenneth Linnert, Chief Financial Officer) This relates to manufacturing inventory for Ruconest. The matter is not concluded, but indicators suggest some inventory may not be usable for commercial purposes. We impaired it according to our normal process and will conclude on the matter before the end of Q3. Q: For the genetic PID Phase 2 study, how did you select the specific genetic PIDs? A: (Anurag Relen, Chief Medical Officer) The genes selected for the basket study were all chosen based on published data showing a connection to the PI3K delta pathway. The selection was driven by evidence that abnormalities in these genes could lead to driving that specific pathway. Q: When might we see pediatric approval for Joenja in the European Union? A: (Anurag Relen, Chief Medical Officer) We have identified a significant number of pediatric APDS patients outside the U.S. We plan to bring the same pediatric data set to Europe, beginning with the UK as we did with the adult population, and then continue across Europe. For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Investor releaseQuarter not tagged2026-07-30Pharming Group Q2 Earnings Call Highlights
MarketBeat
Pharming Group Q2 Earnings Call Highlights
Interested in Pharming Group N.V. Sponsored ADR? Here are five stocks we like better. Pharming cut its 2026 outlook, forecasting revenue of $375 million to $395 million after second-quarter revenue fell 3% to $90.2 million. The company also lowered its operating-expense forecast by $15 million, while expecting RUCONEST sales to stabilize and resume growth in the second half of 2026. Joenja remained the growth driver, with second-quarter revenue up 40% to $17.9 million. Pharming is advancing launches in Germany and Japan, pursuing additional pediatric doses in the U.S., and expects high-30% Joenja revenue growth in 2026. Pharming expects fourth-quarter Phase II results for leniolisib in broader primary immunodeficiency conditions, including CVID, which management estimates could represent an addressable population up to 40 times larger than APDS. Despite positive operating profit, cash and marketable securities declined to $159.5 million at quarter-end. Pharming Group (NASDAQ:PHAR) reported second-quarter revenue of $90.2 million, down 3% from a year earlier, as growth from its Joenja treatment partly offset lower sales of hereditary angioedema therapy RUCONEST. The company reduced its full-year revenue outlook by $30 million but said it expects RUCONEST sales to stabilize and return to growth during the second half of 2026. The company now expects 2026 revenue of $375 million to $395 million, representing approximately flat to 5% growth versus 2025. It also lowered its operating-expense forecast by $15 million to a range of $315 million to $320 million. → Why SK hynix Could Be the Best AI Chip Stock to Buy Now Management said the revised outlook reflects a clearer view of the competitive hereditary angioedema, or HAE, market nearly a year after the launch of the first oral on-demand treatment. Pharming continues to position RUCONEST as an on-demand option for patients with frequent or severe attacks, while seeking to expand Joenja in activated phosphoinositide 3-kinase delta syndrome, or APDS, and potentially in broader immune-deficiency indications. RUCONEST revenue fell 10% year over year to $72.3 million in the second quarter, though sales rose 24% sequentially. Chief Commercial Officer LaVerne Marsh said the year-over-year decline reflected U.S. competitive dynamics, the completion of Pharming’s planned withdrawal from international markets, and temporar…Read full documentShow less
Interested in Pharming Group N.V. Sponsored ADR? Here are five stocks we like better. Pharming cut its 2026 outlook, forecasting revenue of $375 million to $395 million after second-quarter revenue fell 3% to $90.2 million. The company also lowered its operating-expense forecast by $15 million, while expecting RUCONEST sales to stabilize and resume growth in the second half of 2026. Joenja remained the growth driver, with second-quarter revenue up 40% to $17.9 million. Pharming is advancing launches in Germany and Japan, pursuing additional pediatric doses in the U.S., and expects high-30% Joenja revenue growth in 2026. Pharming expects fourth-quarter Phase II results for leniolisib in broader primary immunodeficiency conditions, including CVID, which management estimates could represent an addressable population up to 40 times larger than APDS. Despite positive operating profit, cash and marketable securities declined to $159.5 million at quarter-end. Pharming Group (NASDAQ:PHAR) reported second-quarter revenue of $90.2 million, down 3% from a year earlier, as growth from its Joenja treatment partly offset lower sales of hereditary angioedema therapy RUCONEST. The company reduced its full-year revenue outlook by $30 million but said it expects RUCONEST sales to stabilize and return to growth during the second half of 2026. The company now expects 2026 revenue of $375 million to $395 million, representing approximately flat to 5% growth versus 2025. It also lowered its operating-expense forecast by $15 million to a range of $315 million to $320 million. → Why SK hynix Could Be the Best AI Chip Stock to Buy Now Management said the revised outlook reflects a clearer view of the competitive hereditary angioedema, or HAE, market nearly a year after the launch of the first oral on-demand treatment. Pharming continues to position RUCONEST as an on-demand option for patients with frequent or severe attacks, while seeking to expand Joenja in activated phosphoinositide 3-kinase delta syndrome, or APDS, and potentially in broader immune-deficiency indications. RUCONEST revenue fell 10% year over year to $72.3 million in the second quarter, though sales rose 24% sequentially. Chief Commercial Officer LaVerne Marsh said the year-over-year decline reflected U.S. competitive dynamics, the completion of Pharming’s planned withdrawal from international markets, and temporary inventory normalization. → 3 Value ETFs to Consider as Growth Stocks Lag Behind Marsh said the active RUCONEST patient base remained at approximately 93% of its level a year earlier. The company recorded 84 new patient enrollments during the quarter, compared with about 50 in the first quarter and close to the level reported in the second quarter of 2025. Pharming also added 17 new prescribers. “The resilient patient base, improvement in new enrollments, and continued addition of prescribers support our belief that revenue will stabilize,” Marsh said, adding that RUCONEST is expected to return to growth in the second half. → 5 AI Stocks Are Pulling Back—Which Growth Catalysts Still Look Strongest? Management said new RUCONEST enrollments increasingly involve patients with high disease burdens, including those with frequent or severe attacks and concerns about high-risk attack locations. Marsh also said the new-enrollment figure excludes patients restarting therapy. In response to an analyst question, Chief Financial Officer Kenneth Lynard said the company expects inventory patterns to be within normal seasonal ranges for the rest of 2026 after inventory reductions earlier in the year. Joenja revenue rose 40% year over year to $17.9 million. U.S. revenue increased 31% to $15.4 million, while international revenue climbed 150% to $2.5 million. At the end of the quarter, 132 patients were on paid Joenja therapy in the U.S., up 16% from a year earlier and by five patients from the first quarter. The number of identified U.S. APDS patients rose by 16 during the quarter to 298, including 60 patients between ages 4 and 11. Pharming is awaiting an October 24 PDUFA date for U.S. approval of higher 40-milligram and 50-milligram pediatric doses of Joenja. Marsh said the company submitted a supplemental new drug application for lower 20-milligram and 30-milligram doses on the day of the call. Outside the U.S., the company launched Joenja in Germany on July 1 and said it is preparing to launch in Japan during August after progressing through pricing and reimbursement steps. Marsh said Pharming had identified approximately 387 potentially eligible APDS patients across eight core markets outside the U.S. Management expects Joenja’s annual revenue to grow in the high-30% range in 2026, supported by Germany, Japan and the anticipated U.S. pediatric expansion. Lynard said this compares with 29% growth in 2025. Chief Medical Officer Anurag Relan said Pharming’s two fully enrolled Phase II studies of leniolisib, the active ingredient in Joenja, are expected to report top-line results in the fourth quarter. The programs are evaluating the treatment in broader primary immunodeficiency populations, including common variable immunodeficiency, or CVID, with immune dysregulation. The CVID study includes 20 patients across multiple centers, while a second 12-patient basket study at the National Institutes of Health is examining genetically defined primary immunodeficiencies associated with immune dysregulation. The studies are assessing lymph node and spleen size, blood-cell counts, liver and lung involvement, patient and clinician-reported outcomes, and biomarkers. Relan said Pharming currently anticipates that positive Phase II results could support a single registrational Phase III study in a broader CVID population. The company said the potential CVID addressable population could be up to 40 times larger than APDS, though management said it does not expect CVID sales before a Phase III trial and regulatory approval. Pharming also expects results in 2027 from the pivotal FALCON study of napazimone in patients with primary mitochondrial disease. Lynard said reported and adjusted operating profit were positive in the second quarter despite lower revenue and nearly $9 million in incremental investments compared with the prior-year quarter. Adjusted operating profit declined by $5 million year over year, primarily due to lower revenue and higher research and development spending. Operating cash flow was an outflow of $9.7 million in the quarter, driven by working-capital changes, including a strategic inventory build, and operating performance. Cash and marketable securities totaled $159.5 million at quarter-end, down $12.3 million from the end of the first quarter. For the first half, revenue totaled $162.7 million, down 6% year over year. Joenja revenue increased 37%, while RUCONEST revenue declined 12%. The company increased research and development investment by $13 million in the first half while adjusted operating expenses rose 1%. Pharming expects cost of goods sold, including a $4.9 million manufacturing-inventory impairment recorded during the second quarter, to be approximately 11% of full-year revenue, implying a gross margin of about 89%. Lynard said the impairment related to RUCONEST manufacturing inventory that may not be available for commercial use, with the company expecting to conclude its assessment before the end of the third quarter. Pharming Group N.V. is a clinical-stage biopharmaceutical company headquartered in Leiden, the Netherlands, with a primary focus on developing and commercializing innovative protein replacement therapies for patients living with rare diseases. The company employs a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, enabling scalable and cost-efficient manufacturing of complex therapeutic proteins. The company's lead product, RUCONEST (recombinant human C1 esterase inhibitor), is approved for the treatment of acute hereditary angioedema (HAE) attacks in multiple markets, including the United States and Europe. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Pharming Group Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for July 2026.
TranscriptFY2026 Q22026-07-30FY2026 Q2 earnings call transcript
Earnings source - 82 paragraphs
FY2026 Q2 earnings call transcript
Good morning and good afternoon, everyone, welcome to our Q2 2026 earnings call. I will be joined on this call today by Leverne Marsh, our Chief Commercial Officer, Anurag Relan, our Chief Medical Officer, and Kenneth Lynard, our Chief Financial Officer. In this call, we will be making forward-looking statements that are based upon our current insights and plans. You know, these may differ from future results. You saw in today's press release, while we lowered our full-year revenue guidance, we are encouraged by the resilience of RUCONEST with robust underlying demand indicators. Joenja continued to deliver strong growth, we are approaching a number of significant near-term catalysts. Before I go into more detail on the quarter, let me take a step back and put these developments into context of the ongoing transformation of Pharming.
We are evolving Pharming into a more diversified rare disease company with an increasingly attractive long-term growth profile. This is supported by three key pillars. RUCONEST, which provides a durable source of cash flow. Joenja, a high-growth asset still early in its life cycle with significant commercial opportunities ahead. A high-value pipeline with two potential billion-dollar opportunities. Each of these pipeline programs has the potential to materially increase our scale and are important steps forward in our ambition to making Pharming a leading global rare disease company. We have made important progress across the business during the second quarter, despite a 3% year-over-year decline in total revenue. RUCONEST revenue declined to $72.3 million, compared with $80.4 million in the second quarter of last year. However, we are encouraged by the underlying performance indicators, which demonstrate the resilience of RUCONEST in an evolving on-demand HAE market.
One year after the launch of the first oral on-demand HAE treatment, the RUCONEST active patient base remained at 93% of levels a year ago. We are also seeing strong new patient enrollments, which have returned to levels close to those of a year ago. We expect this to support momentum over the coming months as these patients initiate treatment. We also continue to see new prescribers using RUCONEST, which underscore RUCONEST differentiated value proposition for high-burden patients. Leverne will provide further detail on the RUCONEST performance later in the call. Turning to Joenja, we delivered another quarter of strong growth, with revenue increasing by 40% to $17.9 million. We remain focused on expanding the opportunity for Joenja in APDS through both label and geographic expansion, we are pleased to see commercial momentum accelerating in Europe.
Beyond APDS, we see a potentially significant opportunity for leniolisib in broader primary immunodeficiencies, including CVID. This indication represents an addressable patient population up to 40x larger than APDS. Anurag will provide more detail on the mechanistic rationale underpinning our excitement in these additional indications. With greater visibility into the current HAE market dynamic, which is much clearer a full-year after the launch of the first oral on-demand treatment, we have decided to lower our full-year revenue guidance by $30 million. Nevertheless, based on the underlying trends we are seeing, we expect RUCONEST revenue to stabilize and to return to growth during the second half of 2026. Importantly, our increasingly disciplined operating model has enabled us to maintain positive cash flow from operations.
We have also reduced our full-year operating expense guidance by $15 million to a range of $315 million-$320 million. As we position Pharming for an important second half of 2026, our priorities are clear. First, we will continue to reinforce RUCONEST differentiated value proposition for high disease burden patients and build on the solid underlying performance indicators that we have seen in the second quarter. Second, we will continue to drive the growth of the Joenja franchise, supported by the potential pediatric label expansion in the U.S. and further geographic expansion into new markets, including Japan. Importantly, we expect to advance our pipeline at pace with the readout in Q4 of the two phase II studies for leniolisib in much broader CVID patient populations.
Success in this indication could expand the annual sales potential of Joenja to more than $1 billion and establish Joenja as a blockbuster franchise. With that, I will turn the call over to Leverne to discuss our progress on the commercial front. Leverne?
Thank you, Fabrice. Good morning, good afternoon, everyone. Let me begin with RUCONEST. This quarter, RUCONEST revenue was $72.3 million, down 10% year-over-year, but up sequentially by 24%. The year-over-year decline reflected continued competitive dynamics in the U.S., completion of our planned withdrawal from international markets, and a temporary inventory normalization. I am pleased to report that nearly one year after the launch of a new oral on-demand treatment, the active RUCONEST patient base remains highly resilient at approximately 93% of the prior year level. While some patients are evaluating new treatment options, the vast majority have continued on RUCONEST, as this drug remains especially important for patients who have more frequent attacks or severe attacks who need reliable on-demand treatment. Also notable, we have not seen a reduction in RUCONEST utilization amongst patients receiving newer prophylactic treatments.
This reinforces that preventive and on-demand therapies play complementary roles in managing HAE. We're also encouraged by the upticks in new demand. During the quarter, we recorded 84 new patient enrollments, up substantially from approximately 50 in the first quarter and close to the high level we received during 2025, quarter two. We also added 17 new prescribers, suggesting continued physician confidence even following the addition of a new competitor. Importantly, we see underlying U.S. demand improving. The resilient patient base, improvement in new enrollments, and continued addition of prescribers support our belief that revenue will stabilize and RUCONEST is expected to return to growth during the second half of the year. Next slide. These patient retention and demand dynamics reflect the distinct role that RUCONEST continues to play in high-burden HAE patients.
HAE is challenging to treat as it affects patients differently and no single treatment meets every patient's needs. Convenience matters, but for patients with more frequent attacks, with more severe attacks, reliability, rapid onset, and confidence in their treatment are critical, particularly when attacks occur in high-risk locations or other therapies have not provided adequate control. RUCONEST is well-suited to meet these needs. By replacing deficient or dysfunctional C1 esterase inhibitor, it helps restore control of the pathways that drive HAE attacks while providing the efficacy and reliability of a self-administered IV treatment. This differentiated clinical profile explains why physicians could continue to prescribe RUCONEST, including for new patients with more severe disease. Separately, RUCONEST's highly specialized manufacturing process limits the potential for direct biosimilar competition, supporting its durable commercial position well into the future. Next slide, please. The data shown here bring that differentiated profile into focus.
In clinical studies, 97% of acute attacks required only one dose of RUCONEST, and RUCONEST stopped 93% of attacks for at least three days. Importantly, RUCONEST also demonstrated a median time to complete attack resolution of approximately four and a half hours, a key point of differentiation versus competition. Taken together, HAE patients with a high disease burden, this combination of efficacy, reliability, and rapid and complete attack resolution provides the confidence to both patients and physicians in choosing RUCONEST as their on-demand treatment. These data reinforce our belief that RUCONEST will remain an important on-demand therapy for high-burden HAE patients and a durable part of Pharming's commercial portfolio. Next slide, please. Turning now to Joenja. We delivered another strong quarter and continue to build momentum across the U.S. and international markets.
Global revenue increased 40% year-over-year to $17.9 million, while the U.S. revenue grew 31% to $15.4 million and international revenue increased 150% to $2.5 million, reflecting growing uptake and high adherence. In the U.S., new patient starts continue to drive sales growth. By the end of the quarter, 132 patients were on paid therapy, up 16% year-over-year, and five patients sequentially over quarter one. We are also continuing to expand the diagnosed APDS population. During the quarter, the number of identified U.S. patients increased by 16 to 298, including 60 patients between the ages of four and 11, which is a key catalyst for future growth in APDS. As we identify more patients, we see meaningful room for continued growth in patients on therapy, both in the U.S. and internationally. Next slide, please. Looking ahead, we see several opportunities to continue expanding Joenja.
In the U.S., sources of growth in APDS include continued patient identification, increased genetic testing, and continued efforts to reclassify VUSs. Pediatric expansion is the next important growth opportunity. We are awaiting our October 24 PDUFA date for approval of the higher 40 mg and 50 mg doses. I am pleased to report the filing of the sNDA for the lower 20 mg and 30 mg doses will be submitted to the FDA today. We are also expanding internationally. Our first European launch is now underway in Germany, and we are preparing to launch in Japan in the third quarter. Together with continued growth in the U.K. and other markets, these launches significantly increase the number of patients we are positioned to reach. Finally, our phase II studies in genetic PIDs and CVID with immune dysregulation are evaluating substantially larger patient populations for Joenja beyond APDS.
Together, these opportunities provide multiple sequential drivers of growth for Joenja, supporting our ambition to build a broader immunology franchise and positioning Joenja for potential blockbuster status. With that, I will now turn over to Anurag.
Thank you, Leverne. I'll start by reviewing the science supporting leniolisib's potential beyond APDS and the scientific foundation supporting our two phase II programs. Let's begin by reviewing the relevant biology, where PI3K delta is a central immune signaling molecule. Specifically, it regulates lymphocyte activation, proliferation, differentiation, trafficking, and survival via the AKT, mTOR, and FOXO pathways. Consequently, imbalance in the pathway drives immune dysregulation, which can be clinically manifest as lymphoproliferation, autoimmunity, and inflammatory end-organ disease, specifically in the lung, liver, and the GI tract. This mechanistic understanding forms the scientific rationale for Joenja or leniolisib in APDS and our development programs in more prevalent PIDs. Before turning to these broader patient populations currently under investigation, it's worth reviewing the clinical foundation we've already established in APDS with PI3K delta inhibition using leniolisib.
Leniolisib has demonstrated consistent and durable reductions in lymph node and spleen volume, showing sustained benefits with up to seven years of therapy in an open-label extension follow-up relevant to other PIDs. The safety profile is also well-established. Joenja has truly delivered life-changing benefits for patients, including fewer infections and hospitalizations, and a substantial reduction in treatment burden. What we have achieved in APDS gives us confidence in the potential for leniolisib to deliver the same meaningful benefits via PI3K delta inhibition to many more primary immune deficiency patients with immune dysregulation. Other genetically defined PIDs, as well as CVID with immune dysregulation, have a similar underlying PI3K delta-driven biology. Because of this PI3K delta involvement, these patients share many of the same clinical manifestations I just mentioned: lymphoproliferation, autoimmunity, GI disease, and lung disease.
In addition to the mechanistic basis and our APDS experience, as we presented at the CIS conference in May, we have seen encouraging results in a small cohort of CVID patients who were treated in an expanded access program. In this clinician-reported experience, the breadth and consistency of improvement across cytopenias, lymphoproliferation, and end-organ disease provides a compelling early signal. Our two phase II studies are designed to determine whether the same targeted approach can benefit a broader patient population and significantly expand the addressable market. Both phase II studies are now fully enrolled, with top-line results expected in the fourth quarter. The CVID study includes 20 patients and is being conducted across multiple centers. The second study is a 12-patient basket study at the NIH involving genetically defined PIDs associated with immune dysregulation. These studies are designed to answer two fundamental questions.
Can leniolisib produce clinically meaningful improvements across the key manifestations of immune dysregulation? Which dose regimen provides the appropriate balance of clinical activity, safety, and pathway modulation to support further development? To answer these questions, we are evaluating lymph node and spleen size, blood cell counts, liver and lung involvement, as well as patient and clinician-reported outcomes and key biomarkers. With the endpoints in these two studies strategically very similar to those in our APDS clinical program. Improvement across these measures would establish proof of concept and guide the next stage of development, including a registrational phase III trial endpoint in these substantially larger patient populations. Given that the PI3K delta pathway is seen as a shared driver of immune dysregulation. We currently anticipate conducting a single registrational phase III trial in the broader CVID indication, incorporating patient populations from both studies.
With that, I'll turn it over now to Kenneth to review our financial results.
Thank you, Anurag. I will now cover our Q2 and H1 2026 results, along with our updated full-year outlook. Q2 revenue was $90.2 million, down 3% year-on-year. RUCONEST declined, as you heard earlier, by 10% versus Q2 of last year, and Joenja increased 40% year-on-year. Our reported operating profit was positive in the quarter, despite lower revenues and incremental investments of nearly $9 million compared with the same quarter last year. Adjusted operating profit was also positive and declined by $5 million year-on-year, primarily impacted by the decrease in revenues and the higher R&D investments.
It is important to note that the adjusted Q2 2025 figure excludes $2.1 million of non-recurring Abliva acquisition-related costs, and the adjusted Q2 2026 result excludes $6.5 million of non-recurring items, comprising $4.9 million related to the impairment of manufacturing inventory and $1.7 million associated with the planned closure of our production support site in Évry, in France, in the Q4 of 2026. Operating cash flow for the quarter was an outflow of $9.7 million, primarily driven by changes in net working capital, including a strategic inventory build to further strengthen supply security as well as operating performance. As a result, cash and marketable securities at quarter end were $159.5 million, a decrease of $12.3 million compared with the end of Q1 2026. Looking now at our performance for the first half of 2026, the trends we saw in the second quarter are also reflected in our year-to-date results.
Revenue for the first six months was $162.7 million, representing a 6% decline compared with the first half of 2026. For RUCONEST, revenue was 12% lower than the prior year period, and Joenja continued to deliver strong growth, with revenue increasing 37% year-on-year. Reported operating profit was impacted by a decrease in revenue impacted by RUCONEST demand and inventory destocking at specialty pharmacy level, predominantly in Q1 2026, and the manufacturing-related impairment of inventories in the first half of 2026. Adjusted operating profit was $10.7 million, lower than in the first half of 2025. For comparability, the adjusted H1 2025 result excludes $9.9 million of non-recurring Abliva acquisition-related costs, while the adjusted H1 2026 result excludes $6.5 million, comprising of the $4.9 million impairment of manufacturing inventory and $1.7 million provision related to the site closure in France.
Compared with the first half of last year, we increased investments in R&D by $13 million. We maintained strong cost discipline. On an adjusted basis, operating expenses increased by just 1% versus the first half of 2025, despite these incremental R&D investments. Finally, operating cash flow for the first half was an outflow of $7.7 million, reflecting the combined effect of net working capital changes, primarily the strategic inventory build mentioned, and lower revenues. Turning now to our outlook for the full-year. We have revised our 2026 revenue guidance to $375 million-$395 million, a reduction of $30 million from our previous outlook, reflecting the RUCONEST stabilization and return to growth and strong Joenja growth as discussed earlier. This represents expected growth of approximately 0%-5% versus 2025.
As Fabrice mentioned earlier, nearly a year after the launch of the first oral on-demand HAE treatment, we have much clearer understanding of the evolving market dynamics, giving us greater confidence in both our forecasting and our revised outlook. At the midpoint of our guidance range, RUCONEST U.S. revenue is expected to decline approximately 3% for the full-year. For Joenja, we continue to expect annual revenue growth in the high 30%s as compared to 29% growth in 2025, with Germany, Japan, and the anticipated U.S. pediatric label expansion contributing. We remain committed to disciplined capital allocation to efficiently balance investments in our pipeline, combined with a strong focus on operational efficiency, driving short and long-term value creation. Accordingly, we have reduced our full-year operating expense guidance by $15 million to $315 million-$320 million, representing growth of 1%-3% versus 2025.
This includes more than $40 million of incremental R&D investment to advance our pipeline and the approximate $9 million benefit from the 20% structural reduction in G&A headcount that we announced last year in October. Marketing and sales expenditure are expected to be broadly stable. For the full-year, we expect cost of goods sold, including the $4.9 million manufacturing inventory impairment recorded in the second quarter, to be approximately 11% of revenue, corresponding to a gross margin of around 89%. Finally, we remain confident that our existing cash resources, together with future operating cash flows, are sufficient to fund our current development pipeline and all associated pre-launch activities. With that, I'll hand the call back to Fabrice.
Thank you, Kenneth. Let me close by putting our updated outlook into context. While we have revised our revenue expectations for 2026, the fundamentals of our business remain solid, and we have several important growth opportunities and clinical milestones ahead. We continue to believe that RUCONEST will remain a cornerstone on-demand treatment for high-burden HAE patients and a durable cash engine over the long term. This is supported by the performance we began to see this quarter, specifically the limited erosion of the active patient base, the renewed strength in patient enrollments, and the continued adoption by new prescribers. Joenja, our key growth driver, is maintaining strong sales momentum and remains early in its commercial life cycle with significant near-term commercial opportunities ahead through geographic expansion and pediatric label expansion. We are also operating the company with much greater discipline.
We have reduced our full-year operating expense guidance while continuing to invest in our most important commercial and clinical growth opportunities. We must continue to transform the organization and significantly increase our level of execution to fully capture the many opportunities in front of us. Looking ahead, we have a compelling series of near-term clinical catalysts. As you've seen in the fourth quarter of 2026, we expect to report results from two phase II studies evaluating leniolisib in broader primary immunodeficiency populations, including CVID. Positive results could significantly expand the opportunity for Joenja and support its potential to become a blockbuster franchise. This will be followed in 2027 by the readout of the FALCON pivotal study of napazimone in patients with primary mitochondrial disease. Another program with the potential to materially increase Pharming's scale.
While we have rebased our near-term revenue outlook, we remain confident in the strength of our commercial foundations, the quality of our pipeline, and the multiple catalysts that can drive our next phase of growth. I believe these combinations position Pharming well to continue building a leading global rare disease company. Thank you. We'll now open the floor for questions. Operator?
Thank you. To ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Our first question is going to come from Jeff Jones with Oppenheimer. Your line is open.
Good afternoon, guys. Congrats on the strength of RUCONEST and the quarter. Could you perhaps provide some color around the commercial launch efforts for Joenja in the E.U. and how you're anticipating these to build? Maybe speak a little bit more to the magnitude of the opportunity as you expand country to country. Then for a second question, can you speak to assuming a positive outcome from the two PIDs trials, what that looks like and next steps moving ahead into that pivotal study? Thank you.
Thank you, Jeff. I'll ask Leverne to answer the first part of your question and Anurag for the second part.
Thank you. Thank you, Jeff, good morning, good afternoon. In terms of commercial launch preparation outside of the U.S. for Joenja, we have made substantial progress in patient identification across our Core 8 markets. We've identified Core 8 markets that will be key for us to launch in the next couple of years. To date, we've identified about 387 potentially eligible APDS patients across these markets. We have launched in the United Kingdom, where we have gained some scale in our capability, both from a commercial execution perspective. Our market access capabilities has grown, which we've scaled into the German launch and are preparing for the launch in Japan. The U.K. launch is proceeding at pace, and we're making good progress. German launch is underway since the 1st of July, and we have made substantial progress in acquiring funded patients on therapy in Germany.
From a Japan perspective, we are in the final stages of securing our pricing and reimbursement following approval in the second quarter. We look forward to launching Joenja in Japan during the August timeframe.
Jeff, on your question about the two phase II studies. Again, we're expecting those results in the fourth quarter. The studies, as I've reviewed today, they have very similar patient populations in terms of one being a group of patients that are genetically defined and the other are more clinically defined. However, they have similar clinical manifestations, and we see significant overlap in the enrolled population, especially from the first study into the broader CVID study, which again, is a patient population that's about 40x the size of APDS. When thinking ahead now to what we can expect, once we have the results, we'll review those results. We're collecting a broad spectrum of endpoints to evaluate.
We're also evaluating the dose regimen. Then we'll come up with a plan to review with FDA, starting with FDA, also with regulators across Europe as well as Japan to see what a registrational study could look like. We do anticipate right now, again, pending the results of those two studies, that we would do a combined CVID study, and this would likely be a randomized control study similar to what we performed in APDS. I think we have a good blueprint based on the work that we've done in APDS, and again, now anxiously awaiting those final results.
Thank you, guys.
Thank you. The next question will come from Ilana Stepwysik with Jefferies. Your line is open.
Hi. Thanks for taking my questions. Firstly, on RUCONEST, enrollments appear to have recovered to around pre-competitor launch levels. I was wondering if you could give a sense of what you've seen so far the first month of this quarter and whether the trend has continued. Secondly, I just wanted to ask on Joenja. There appears to be roughly, I think, 30 identified pediatric patients in the U.S. who could be eligible under the expanded dosing schedule, if approved. How do you think we should think about the pace of converting those patients onto reimbursed therapy following that approval? Thank you.
Leverne?
On RUCONEST enrollments, Ilana, thank you for the question. You're 100% right. We've seen certainly Q2 with 84 enrollments getting fairly close to what we had in Q2 2025, which neared about 90 new enrollments. We are seeing consistency in the momentum into the third quarter. In the HAE market overall, there is some seasonality. If you look back at new enrollments in the previous year or the year before that, you do see some changes in the rate of new enrollments as we go into the third quarter. As I said, it's quite consistent with what we're experiencing deep into July so far. Importantly, we continue to add new prescribers. What we're seeing in these new enrollments is this entrenchment of the RUCONEST position in high-burden HAE patients.
These are typically either HAE normal patients, where the burden of disease is quite high, or patients with more frequent and more severe attacks. That's where we anticipate having a really durable position, even four quarters after competitor launch, to continue into the third quarter.
Joenja?
From a conversion perspective, as I mentioned before, we have a substantial number of patients identified in these core markets, including certainly the U.K., Germany we're launching now, and Japan launch coming. We are assuming a fairly similar conversion rate that we've seen in the U.S. as we secure reimbursement and the commercial teams execute against our plan in the various centers, both in Germany and upcoming in Japan. Part of that will be conversion of patients that are on our EAP programs in some markets, but also pretty clear identification for us in where those patients are sitting in these respective markets and what we need to do to convert them as soon as we have reimbursement.
Specifically for U.S. pediatric?
For U.S. pediatrics, similarly, what we have today is 60 identified patients between four and 11 years old. As Anurag mentioned earlier, the submission is divided into the higher dose and the lower dose. What we see in the split of those identified patients, it is about 50/50. 50 patients in the higher dose category, 50% rather, and 50% in the lower dose category. Again, we are assuming a similar uptake what we have seen with the adult population 12 and above, converting first those EAP patients in the first few months and then continuing to convert the remaining patients that we have identified in the centers that we have.
Thank you, Ilana.
Thank you. Our next question is going to come from Whitney Ijem with Canaccord. Your line is open.
Hi, all. My congrats on the quarter. Just to go back to the 84 new RUCONEST patients, can you talk a little bit more about those patients in terms of what they look like, severity, attack rate, is that consistent with how the RUCONEST patient has looked historically? Are they all new to therapy or some returning back from maybe trying something else?
Thank you, Whitney. Leverne?
Thanks, Whitney. The 84 new enrollments, as I said before, the patient population for us in terms of new enrollments have evolved over time. Now we're seeing a very clear trend emerging for these high-burden patients that have high-frequency attacks, more severe attacks, patients who are more worried about high-risk attack locations like laryngeal attacks, GI attacks. These are just the new enrollments. The new enrollment number does not include restarts, as you've identified, i.e., patients who are returning back to therapy. Pure new enrollments, unique new for the first time on RUCONEST.
Got it. That's really helpful. Then moving over to CVID and PIDs. In terms of the larger patient number, clearly, very large TAM assuming success there. I guess, can you talk about any efforts in terms of patient identification or just as we think about the diagnosis rate in those, given they are larger but not genetic, I guess how well-characterized are those patients and under the care of physicians currently, in terms of being able to access them commercially?
Sure. Hi, Whitney. This group of patients, these CVID patients, is largely already identified, and that's very different from what we experienced with APDS. With APDS, it was a newly defined disease just in the last 15 years. It required genetic testing, and it required a considerable effort in terms of educating doctors also on the importance of genetic testing. CVID, however, is clinically diagnosed, and these group of patients that we're talking about are, again, already diagnosed. They're almost always on immunoglobulin replacement therapy because that's part of one of the key manifestations is that they have low IgG levels. Therefore, really, it's a different, let's say, mechanics in terms of diagnosis versus what we've seen with APDS.
Very helpful. Thank you so much.
Thank you. The next question is going to come from Joe Pantginis with H.C. Wainwright. Your line is open.
Hey, everybody. Thanks for all the updates today. Two questions. First on RUCONEST and then on Joenja. With regard to the current commercial profile coming into the second half, it was mentioned in the prepared comments, with the three factors. One of them was inventory normalization. I just wanted to know any anticipation for further optimization here or anything we can expect with regard to inventory and distribution going forward that might be relatively obvious to you.
Good morning, Joe. This is Kenneth here. We expect that we are in a year with a normal seasonality where we saw some inventory reduction in the first part of the year, and there will be a normal cycle with a bit of inventory build also in the year to go, but all within normal frame. Remember that when we commented on Q1, we said that 2025 was the unusual part. We had a normal year when it comes to the inventory.
Got it. Then for Joenja, I'm going to phrase my question very carefully. With the upcoming PIDs CVID data, which is obviously a major event for you guys, obviously you cannot market these data. However, you can leave or present these data to physicians as just sort of like a supplemental information concept. Do you have any views towards these data providing any sort of incremental boost to revenue prior to potential approval? Obviously, from an off-label standpoint.
No, Joe. Obviously, as Anurag said, we have those two phase II open label study that we read out in Q4, and we would be engaging with the FDA and our base assumption that we're going to have to conduct a phase III trial. Until the phase III trial read out and we receive proper approval from FDA, we won't be able to promote the data, and we don't expect any sales from CVID. This being said, there are a lot of opportunities in APDS, as we disclosed, the many opportunities in APDS in the U.S. in adult as well with the expected upcoming pediatric and the geographic expansion should allow us to fuel the growth of Joenja very significantly in the coming years as we wait for these new indications to materialize.
Thank you.
Thank you. The next question is going to come from Sushila Hernandez with Van Lanschot Kempen. Your line is open.
Yes. Thank you for taking my questions. On RUCONEST and the on-demand HAE market, how do you expect that these dynamics on new patient initiations will play out? Do you expect that patients will still find RUCONEST after they have tried other drugs?
Certainly. Thanks, Sushila. From a new enrollment perspective, we think about inflows in two different ways, right? New enrollments or brand new patient starts and restarts. When we look at new enrollments, the vast majority of those come from, in fact, patients who've had suboptimal responses to other therapies and now switching to RUCONEST. We expect that to continue because what we're seeing in patients who have high severity of attacks, patients who have more frequent attacks, two or more attacks per month, they do require a more reliable, faster onset HAE treatment and that is where RUCONEST is uniquely positioned compared with competitive set to continue to have a position.
Okay, that's clear. On Joenja, you mentioned the eight core markets outside of the U.S. Could you also break down or give us more color on which of the markets will be key drivers for growth ex-U.S.? On the VUS reclassifications, what are the next steps and when could this meaningfully add to Joenja opportunity?
On the Core 8 markets in the very short term, we are especially excited about the progress we're making in Germany. We're one month into that launch, that is going to be key for us as our next ex-U.S. market, our first European launch. Subsequent to that, the Japan launch that we are planning for August will be another key growth driver for us in the short term. From a U.K. perspective, we continue to make progress in adoption in APDS within the U.K. One additional piece that I'll add is especially excited about the Japan market because we do have an approval for ages four and above, which is our first pediatric launch that we will have before we anticipate U.S. approval later this year.
Sushila, on the VUS reclassification efforts, we actually have a number of efforts that are ongoing. We have the efforts that are trying to help clinicians reclassify VUSs on a one-by-one basis. That's ongoing now. That involves collecting additional data, looking at literature, family testing that can also be helpful in resolving these VUSs. That's happening on an ongoing basis, and we're seeing patients getting reclassified as a result of that. We have the much larger project that we talked about in the past that we had partnered with Columbia on, that work has also been initiated. Once I have a little more clarity on when that's getting wrapped up, we'll be able to give you more guidance on what the timing of that. That work is well underway.
Okay, thank you for this additional color. Just the final question. For the genetic PID phase II study, could you remind me again, how did you select these specific genetic PIDs as there are so many? Which ones are more directly or indirectly involved in a PI3K delta-driven immune dysregulation?
The genetic PID study, the genes that were selected for it were all selected on the fact that there was a connection to this pathway and that these abnormalities in these genes could lead to driving of that pathway, and there was published data on that. That's how that group or that basket of genes was selected.
Okay, thanks for that color.
As a reminder to ask a question, please press star one one on your telephone. Our next question comes from Simon Scholes with First Berlin. Your line is open.
Yes, hello. Good morning and good afternoon. I've got three or four questions, if I may. The first one is on the new enrollments. You've pointed out that with 84 new enrollments with RUCONEST in Q2, I mean, you're quite close to the level you were seeing in the first half of last year, which is over 90. Of course, in the first half of last year, you were seeing U.S. growth in RUCONEST of 30%, and now it's retreating somewhat. I think it would be helpful if you could give us the net figure, i.e., the new enrollments less switches and drop-offs. Also, I was wondering if I'm correct in thinking that the next EU launch for Joenja after Germany will not be until 2027.
I was also wondering if you could give us an idea of when you might see pediatric approval in the European Union. Just lastly, I was wondering if you could give us some more color on the manufacturing impairment of inventory, what exactly was happening there, and which product does that relate to. That's it from me.
Thank you, Simon. I'll start with the new enrollment and the outlook, the growth outlook for RUCONEST, as I understand your question well. As we've said, based on the robust underlying key performance indicator related to the resilience of RUCONEST, 93% of the active patient base maintained year-over-year, as well as the solid new enrollment that you pointed out. We expect to stabilize RUCONEST and have it return to growth. Yet, as I've explained, we see RUCONEST as a durable cash engine for Pharming with Joenja being the growth drivers for the years to come. That's how we are approaching our portfolio.
That's how the company is evolving from becoming a one asset company to becoming a more diversified rare disease company with a longer-term growth profile, given the number of growth catalysts for Joenja, as well as very significant pipeline catalysts for Joenja as well as napazimone. When it comes to geographic expansion, I will let Leverne provide her perspective.
In the short term, of course, we are in our peri-launch phase in the U.K., German launch is underway, we're looking forward to the Japan launch in the August timeframe in ages four and above. For 2027, as we look into that timeframe, of course, we have announced both approvals in Canada and South Korea, we are anticipating that we continue launch progress toward Australia and Italy in 2027. When we look in totality at the potential for continued growth and expansion in 2027, we plan to make good progress across those markets for Joenja pediatric and as well as Joenja adult. The second Joenja question, if you could repeat that.
Was about pediatric indication in Europe. I'll let Anurag.
Yes, that's right.
Hi, Simon. Obviously, as Leverne mentioned earlier, we've identified a large number of APDS patients outside of the U.S. in these core markets, and a significant number of those, similar to what we've seen in the U.S., are pediatric patients. The unmet need is very similar across Europe. We're planning to bring the same data set that we have from these pediatric studies to Europe as well. We're going to do that by beginning in the U.K., as we did with the adolescent and adult population, then we'll continue to do that across Europe.
When it comes to the manufacturing impairments, Kenneth?
Yeah. This is a matter where we have impaired for manufacturing inventory throughout the manufacturing process related to RUCONEST. This is a matter that is not concluded upon, but the indicators are that we have inventory that may not be possible to use for commercial use. We will conclude on the matter before the end of Q3. According to our normal process, we impair for such kind of issues when we are made aware of them.
Okay, thanks very much. That's very helpful.
Thank you. I am showing no further questions in the queue at this time. I will now turn the call back over to Fabrice for closing remarks.
Thank you, operator. Thank you so much for all the relevant questions. We hope that we provided additional color on our Q2 earnings. Not only about the revised revenue guidance, but also about the business fundamentals and the fact that we continue to manage a much tighter operating model that has allowed us to make sure that we allocate capital more efficiently and fully capture the number of growth opportunities and pipeline catalysts that are ahead of us. We look forward to updating you on our plans as those catalysts will unlock in the coming weeks and months. Thank you very much.
This concludes today's conference call. Thank you for participating, and you may now disconnect.
Investor releaseQuarter not tagged2026-07-29Earnings To Watch: Pharming Group (XAMS:PHARM) Q2 2026 -- GF Value Sees 36% Upside
GuruFocus.com
Earnings To Watch: Pharming Group (XAMS:PHARM) Q2 2026 -- GF Value Sees 36% Upside
This article first appeared on GuruFocus. Pharming Group (XAMS:PHARM) is set to release its Q2 2026 earnings on Jul 30, 2026. The consensus estimate for Q2 2026 revenue is 88.28 million, and the earnings are expected to come in at 0.02 per share. The full year 2026's revenue is expected to be $367.66 million and the earnings are expected to be $0.05 per share. More detailed estimate data can be found on the Forecast page Warning! GuruFocus has detected 3 Warning Sign with MRVL. Is XAMS:PHARM fairly valued? Test your thesis with our free DCF calculator. Over the past 90 days, revenue estimates for Pharming Group (XAMS:PHARM) have declined from $376.20 million to $367.66 million for the full year 2026, while estimates for 2027 have increased from $392.36 million to $409.01 million. Earnings estimates have increased from $0.03 per share to $0.05 per share for 2026, and from $0.04 per share to $0.06 per share for 2027. In the previous quarter of 2026-03-31, Pharming Group's (XAMS:PHARM) actual revenue was $63.56 million, which missed analysts' revenue expectations of $82.24 million by -22.72%. Pharming Group's (XAMS:PHARM) actual earnings were $-0.01 per share, which missed analysts' earnings expectations of $0.01 per share by -200%. After releasing the results, Pharming Group (XAMS:PHARM) was down by -25.77% in one day. Based on the one-year price targets offered by 4 analysts, the average target price for Pharming Group (XAMS:PHARM) is $2.21 with a high estimate of $2.60 and a low estimate of $1.80. The average target implies an upside of 101.33% from the current price of $1.10. Based on GuruFocus estimates, the estimated GF Value for Pharming Group (XAMS:PHARM) in one year is $1.50, suggesting an upside of 36.36% from the current price of $1.10. Based on the consensus recommendation from 6 brokerage firms, Pharming Group's (XAMS:PHARM) average brokerage recommendation is currently 1.3, indicating a "Buy" status. The rating scale ranges from 1 to 5, where 1 signifies Strong Buy, and 5 denotes Sell.
Investor releaseQuarter not tagged2026-07-16Pharming Group to report second quarter and first half 2026 financial results and provide business update on July 30
GlobeNewswire
Pharming Group to report second quarter and first half 2026 financial results and provide business update on July 30
Leiden, the Netherlands, July 16, 2026: Pharming Group N.V. (“Pharming”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) confirms that it will report its preliminary (unaudited) financial results for the second quarter and first half 2026 and provide a business update on Thursday, July 30, 2026. Management will host a conference call and webcast for analysts and investors on the same day at 13:30 CEST/07:30 am EDT. To participate in the conference call or to watch the live webcast, please register in advance using the links below. Conference call registration:https://register-conf.media-server.com/register/BId285845cff504c39931913d279f646ea Once registered, dial-in information and a unique PIN will be provided, allowing access to the call. Please note, the Company will only take questions from dial-in attendees. Webcast registration: https://edge.media-server.com/mmc/p/m35zejc7 The webcast will also be accessible on the Pharming website at Investors/Financial Documents & Reports, and a replay will be available shortly after the event. About Pharming Group N.V.Pharming Group N.V. (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations. For more information, visit www.pharming.com and find us on LinkedIn. For further public information, contact:PharmingMichael Levitan, VP Investor Relations and Capital MarketsT: +1 (908) 705 1696E: [email protected] Saskia Mehring, Head of Corporate CommunicationsT: +31 6 28 32 60 41E: [email protected] RelationsJulia Deutsch (Lyra Strategic Advisory on behalf of Pharming)E: [email protected] Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)T: +31 6 53 81 64 27 Attachment Pharming Group to report 2Q_1H26 results_EN_16JULY2026
Investor releaseQuarter not tagged2026-05-28Pharming Group announces results of 2026 Annual General Meeting of Shareholders
GlobeNewswire
Pharming Group announces results of 2026 Annual General Meeting of Shareholders
Leiden, the Netherlands, May 28, 2026: Pharming Group (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) today announced that shareholders approved all proposals presented at its Annual General Meeting of Shareholders (AGM), held earlier today. KPMG Accountants N.V. was appointed as the Company’s independent external auditor for the financial years 2026 through 2028 (agenda item 3). Shareholders also approved the proposals to amend the Remuneration policy for the Board of Directors regarding the fees to be paid to the Non-Executive Directors (agenda item 4), to renew the authorizations for the Board of Directors to issue shares (agenda item 5) and to repurchase shares (agenda item 6). A recording of the webcast, the AGM presentation slides, voting results and additional information on the agenda items are available on the Company’s website under Investors/Shareholder Meetings. About Pharming Group N.V.Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations. For more information, visit www.pharming.com and find us on LinkedIn. Inside InformationThis press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. For further public information, contact:Investor RelationsMichael Levitan, VP Investor Relations and Capital MarketsT: +1 (908) 705 1696E: [email protected] Media RelationsGlobal: Saskia Mehring, Head of Corporate Communications T: +31 6 28 32 60 41E: [email protected] U.S.: Christina Skrivan (Precision AQ on behalf of Pharming)T: +1 (636)-352-7883 Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)T: +31 6 53 81 64 27 Attachment Pharming Group announces results 2026 AGM_EN_28MAY26
Investor releaseQuarter not tagged2026-05-09Pharming Group Q1 Earnings Call Highlights
MarketBeat
Pharming Group Q1 Earnings Call Highlights
Interested in Pharming Group N.V. Sponsored ADR? Here are five stocks we like better. RUCONEST revenue fell (down ~15% Y/Y) largely due to specialty pharmacy inventory drawdown and the company’s exit from certain non‑U.S. markets, but management expects inventory normalization in H2 and highlights continued demand with ~50 new patient enrollments and 23 new prescribers. Joenja is a key growth driver—revenues rose 34% Y/Y (about $14.1M) with U.S. paid patients up 25% Y/Y—and Pharming has resubmitted a pediatric sNDA for ages 4–11 with an FDA decision expected within six months and a second lower‑dose submission planned for the summer. Pharming reiterated 2026 revenue guidance of $405–$425 million despite Q1 revenue of EUR72.4M (down 8%), reported positive operating cash flow (~EUR2M) and EUR171.8M in cash/marketable securities, while keeping 2026 operating expense guidance and adding R&D investment ahead of two Phase II leniolisib readouts later this year. Pharming Group (NASDAQ:PHAR) executives said first-quarter 2026 results reflected an expected decline in RUCONEST revenue tied largely to specialty pharmacy inventory movements and the company’s planned exit from certain non-U.S. markets, while Joenja continued to post strong growth and the company advanced regulatory and clinical milestones across its pipeline. CEO Fabrice Chouraqui said quarterly revenue fell primarily due to RUCONEST, a decline he said was “largely expected due to inventory drawdown at specialty pharmacy,” which the company previously discussed on its fourth-quarter 2025 call. Chouraqui also pointed to the “commercial exit from non-U.S. markets” as a contributor to the year-over-year decline, a decision the company announced last year “as part of our renewed financial discipline since the commercialization of RUCONEST in this market was not financially sustainable.” → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% Chief Commercial Officer Leverne Marsh said RUCONEST revenue was down 15% year-over-year, driven by three factors: inventory dynamics that reduced quarterly revenue by 8%, the ex-U.S. exit contributing about 3%, and what she described as a measured impact from competition in the U.S. hereditary angioedema (HAE) market. Despite the headline decline, Marsh emphasized continued demand indicators underneath revenue. She said the company added about 50 new patient…Read full documentShow less
Interested in Pharming Group N.V. Sponsored ADR? Here are five stocks we like better. RUCONEST revenue fell (down ~15% Y/Y) largely due to specialty pharmacy inventory drawdown and the company’s exit from certain non‑U.S. markets, but management expects inventory normalization in H2 and highlights continued demand with ~50 new patient enrollments and 23 new prescribers. Joenja is a key growth driver—revenues rose 34% Y/Y (about $14.1M) with U.S. paid patients up 25% Y/Y—and Pharming has resubmitted a pediatric sNDA for ages 4–11 with an FDA decision expected within six months and a second lower‑dose submission planned for the summer. Pharming reiterated 2026 revenue guidance of $405–$425 million despite Q1 revenue of EUR72.4M (down 8%), reported positive operating cash flow (~EUR2M) and EUR171.8M in cash/marketable securities, while keeping 2026 operating expense guidance and adding R&D investment ahead of two Phase II leniolisib readouts later this year. Pharming Group (NASDAQ:PHAR) executives said first-quarter 2026 results reflected an expected decline in RUCONEST revenue tied largely to specialty pharmacy inventory movements and the company’s planned exit from certain non-U.S. markets, while Joenja continued to post strong growth and the company advanced regulatory and clinical milestones across its pipeline. CEO Fabrice Chouraqui said quarterly revenue fell primarily due to RUCONEST, a decline he said was “largely expected due to inventory drawdown at specialty pharmacy,” which the company previously discussed on its fourth-quarter 2025 call. Chouraqui also pointed to the “commercial exit from non-U.S. markets” as a contributor to the year-over-year decline, a decision the company announced last year “as part of our renewed financial discipline since the commercialization of RUCONEST in this market was not financially sustainable.” → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% Chief Commercial Officer Leverne Marsh said RUCONEST revenue was down 15% year-over-year, driven by three factors: inventory dynamics that reduced quarterly revenue by 8%, the ex-U.S. exit contributing about 3%, and what she described as a measured impact from competition in the U.S. hereditary angioedema (HAE) market. Despite the headline decline, Marsh emphasized continued demand indicators underneath revenue. She said the company added about 50 new patient enrollments in the quarter and brought on 23 new prescribers. Marsh framed these trends as evidence that clinicians continue to use RUCONEST for a “high attack, high severity” subset of patients. → Light Speed Returns: Corning Cashes In on NVIDIA Growth On competitive dynamics, Marsh said that nine months after the launch of a new oral competitor in the U.S., the “overwhelming majority” of RUCONEST patients have remained on therapy. She added that among those who explored alternatives, “many high-burden patients are returning to RUCONEST” when response to new treatments is not adequate. During Q&A, Marsh explained why the company expects additional RUCONEST pressure in the second quarter, citing the lag needed to see trialing and switching behavior through “three to four reorder cycles.” CFO Kenneth Lynard added that the company expects inventory patterns to normalize, saying Pharming anticipates an inventory build in the second half of the year “to basically reflect the demand.” → Years in the Making, AMD’s Upside Movement Has Just Begun Chouraqui clarified that the roughly 50 new patient enrollments discussed in the quarter represent patients “in the pipes” who have received a prescription but are not yet necessarily on therapy, noting there is typically a delay between enrollment and treatment start. Pharming highlighted Joenja as an early-stage growth driver. Chouraqui said Joenja revenues grew 34% year-over-year with “strong momentum both in the U.S.” and in international markets. Marsh reported Joenja revenue of $14.1 million globally in the quarter and said that by quarter-end the company had 127 patients on paid therapy in the United States, a 25% increase over the first quarter of 2025. Marsh said Pharming added seven net new U.S. patients on paid therapy in the quarter, an acceleration compared with the prior two quarters. She also cited an 85% U.S. fill rate, which she attributed to reimbursement support and patient services. Beyond patients already on therapy, Marsh said the company has identified 187 U.S. APDS patients older than 12 who are eligible, plus 57 eligible patients in the four-to-11 age group, calling pediatrics “the next frontier for growth in the U.S.” On international progress, Marsh said Pharming is seeing strong uptake in the U.K. and growth in government-supported access programs elsewhere. Looking ahead, she said the company is positioning for launches in Europe and Japan later this year. In response to an analyst question, Marsh said the first European launch is expected in Germany “toward the second quarter,” and she said Pharming anticipates commercial, paid patients in the second quarter. She also said the company expects to launch in Japan in August. Management provided an update on its efforts to expand Joenja’s U.S. label into pediatrics (ages four to 11) following a complete response letter (CRL) received in January. The company said it held a Type A meeting with the FDA at the end of March, which included two APDS expert physicians. Executives described the interaction as constructive and said the FDA recognized the unmet need and challenges of recruiting young children into trials for an ultra-rare disease. Chief Medical Officer Anurag Relan said Pharming and the FDA aligned on a two-step approach. The company resubmitted the sNDA in April for the 40 mg and 50 mg doses, which Relan said cover “a meaningful proportion” of children ages four to 11. He said an FDA decision is expected within six months or sooner, and the company plans to issue a press release upon FDA acceptance. Relan said a second sNDA for the lowest-weight patients is planned for the summer, also expected to follow a six-month review timeline. Responding to questions about dosing, Relan said the Type A discussion did not change Pharming’s overall dosing strategy and that the planned low-dose submission does not require an additional clinical trial. Marsh said roughly half of the identified four-to-11 population would be eligible for the higher-dose resubmission, with the other half expected to fall into the lower-dose group. Pharming also discussed lifecycle expansion opportunities for leniolisib (Joenja) beyond APDS, focusing on primary immunodeficiencies (PIDs) with immune dysregulation, including common variable immune deficiency (CVID) with immune dysregulation. Relan said Pharming has two phase II, proof-of-concept studies that are now fully enrolled: A multicenter CVID study enrolling 20 patients A genetic PID study conducted at the NIH enrolling 12 patients Relan said both are single-arm, open-label dose range-finding studies, and readouts are expected later this year. He added that one study is a month shorter in duration, so results could become available slightly earlier for one program than the other. At the Clinical Immunology Society meeting, Relan said Pharming and collaborators are presenting seven abstracts, including data from an expanded access program in which six CVID or CVID-like patients with immune dysregulation received leniolisib for a median of 1.4 years. Relan said clinicians reported improvement and “no patient showing progression” across manifestations including cytopenias, splenomegaly, lymphadenopathy, liver disease, and lung disease. He also cited immune profile changes, including reduced transitional and CD21 low B cells, which he said are consistent with PI3K delta pathway modulation seen in APDS. Relan cautioned the dataset was clinician-reported and not from a prospective clinical study, but he called the consistency of improvement across endpoints an encouraging early signal ahead of formal study readouts in the second half of the year. Chouraqui also pointed to napazimone (formerly KL1333) for primary mitochondrial disease as a major pipeline program, saying the registrational study is expected to complete enrollment this year with a readout next year. CFO Kenneth Lynard said first-quarter revenue totaled EUR 72.4 million, down 8% year-over-year. He reiterated RUCONEST revenue declined 15% and attributed most of the change to the expected U.S. inventory normalization (an 8% headwind, consistent with the 7% to 9% headwind discussed on the prior call) and the ex-U.S. exit (3%). He also noted that the first quarter is typically the lowest seasonal quarter for RUCONEST due to ordering patterns. Joenja revenue increased 34% year-over-year, and Lynard said revenue was “modestly affected by inventory timing,” adding that excluding this effect growth would have been $1 million to $2 million higher. Lynard said operating expenses fell 9% year-over-year, and on an adjusted basis—excluding non-recurring acquisition costs tied to Abliva in the prior year—expenses were flat, which he said reflected the company’s ability to increase pipeline investment without increasing overall costs. Pharming generated EUR 2 million in positive operating cash flow in the quarter. Total cash and marketable securities fell EUR 9.3 million to EUR 171.8 million, driven primarily by a EUR 12.3 million payment related to early termination of a facility lease. Management reaffirmed 2026 revenue guidance of $405 million to $425 million, which the company said implies 8% to 13% growth versus 2025. Lynard said the outlook assumes low single-digit RUCONEST growth at the midpoint, with pressure expected in the second quarter and growth anticipated in the second half. He said guidance now includes expected U.S. pediatric label revenues later in the year, which previously had been excluded. Lynard added that hitting the upper end of the range would be most influenced by faster-than-expected pediatric approval and launch timing. Pharming reiterated an operating expense outlook of $330 million to $335 million for 2026, including $60 million in incremental R&D investment to advance the pipeline and “up to $30 million additional for the development of napazimone.” Lynard also referenced a previously announced 20% structural headcount reduction in G&A, which he said delivers a $9 million benefit, while marketing and sales spending remains stable. Gross margin is expected to be about 90%. In closing remarks, Chouraqui said the company believes it is making “important progress across the business” while maintaining financial discipline, and he pointed to upcoming catalysts including the two phase II leniolisib readouts later this year and completion of enrollment for the napazimone registrational study. Pharming Group N.V. is a clinical-stage biopharmaceutical company headquartered in Leiden, the Netherlands, with a primary focus on developing and commercializing innovative protein replacement therapies for patients living with rare diseases. The company employs a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, enabling scalable and cost-efficient manufacturing of complex therapeutic proteins. The company's lead product, RUCONEST (recombinant human C1 esterase inhibitor), is approved for the treatment of acute hereditary angioedema (HAE) attacks in multiple markets, including the United States and Europe. The article "Pharming Group Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.
Investor releaseQuarter not tagged2026-05-07Pharming Group reports first quarter 2026 financial results; on track for Joenja® U.S. pediatric label expansion and launches in Japan and Europe in 2026
GlobeNewswire
Pharming Group reports first quarter 2026 financial results; on track for Joenja® U.S. pediatric label expansion and launches in Japan and Europe in 2026
First quarter 2026 total revenues were US$72.4 million, an 8% decrease compared to the first quarter 2025 RUCONEST® revenue was US$58.4 million, a 15% decrease compared to the first quarter 2025, mainly due to anticipated inventory drawdowns and the planned exit from non-U.S. markets Joenja® revenue was US$14.1 million, a 34% increase compared to the first quarter of 2025, reflecting strong U.S. and international momentum Reaffirmed 2026 total revenue guidance of US$405 - US$425 million (8% - 13% growth) Generated positive net cash flow from operations of US$2.0 million in the quarter Joenja® approved in Japan and received positive CHMP opinion for APDS Resubmitted pediatric sNDA to the FDA for Joenja® (leniolisib) for highest doses; plan additional sNDA this summer for lowest doses Pharming to host a conference call today at 13:30 CEST (7:30 am EDT) Leiden, the Netherlands, May 7, 2026: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary unaudited financial report for the three months ended March 31, 2026. Chief Executive Officer, Fabrice Chouraqui commented: “The first quarter demonstrated important progress across the business while also reflecting revenue variability for RUCONEST®. Joenja® delivered strong revenue growth of 34% year over year, driven by robust patient uptake, reinforcing its role as an important growth driver still early in its lifecycle. We also made meaningful regulatory progress, including approval in Japan for APDS patients aged 4 and older and a positive CHMP opinion in Europe. In the U.S., constructive dialogue with the FDA following receipt of the CRL enabled us to already resubmit our pediatric sNDA for the two highest doses, which cover a meaningful proportion of children aged 4 to 11, and plan an additional sNDA submission for the lowest doses this summer. First‑quarter RUCONEST® revenue was impacted by several factors we had largely anticipated and incorporated into our full-year guidance, notably specialty pharmacy inventory drawdowns and our strategic exit from non-U.S. markets. We continue to see the overwhelming majority of patients stay on RUCONEST® nine months after the launch of a new oral treatment. New patient enrollments and growing prescriber engagement further validate RUCONEST®’s strong value proposition for high-burden patients. We also advanced ou…Read full documentShow less
First quarter 2026 total revenues were US$72.4 million, an 8% decrease compared to the first quarter 2025 RUCONEST® revenue was US$58.4 million, a 15% decrease compared to the first quarter 2025, mainly due to anticipated inventory drawdowns and the planned exit from non-U.S. markets Joenja® revenue was US$14.1 million, a 34% increase compared to the first quarter of 2025, reflecting strong U.S. and international momentum Reaffirmed 2026 total revenue guidance of US$405 - US$425 million (8% - 13% growth) Generated positive net cash flow from operations of US$2.0 million in the quarter Joenja® approved in Japan and received positive CHMP opinion for APDS Resubmitted pediatric sNDA to the FDA for Joenja® (leniolisib) for highest doses; plan additional sNDA this summer for lowest doses Pharming to host a conference call today at 13:30 CEST (7:30 am EDT) Leiden, the Netherlands, May 7, 2026: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary unaudited financial report for the three months ended March 31, 2026. Chief Executive Officer, Fabrice Chouraqui commented: “The first quarter demonstrated important progress across the business while also reflecting revenue variability for RUCONEST®. Joenja® delivered strong revenue growth of 34% year over year, driven by robust patient uptake, reinforcing its role as an important growth driver still early in its lifecycle. We also made meaningful regulatory progress, including approval in Japan for APDS patients aged 4 and older and a positive CHMP opinion in Europe. In the U.S., constructive dialogue with the FDA following receipt of the CRL enabled us to already resubmit our pediatric sNDA for the two highest doses, which cover a meaningful proportion of children aged 4 to 11, and plan an additional sNDA submission for the lowest doses this summer. First‑quarter RUCONEST® revenue was impacted by several factors we had largely anticipated and incorporated into our full-year guidance, notably specialty pharmacy inventory drawdowns and our strategic exit from non-U.S. markets. We continue to see the overwhelming majority of patients stay on RUCONEST® nine months after the launch of a new oral treatment. New patient enrollments and growing prescriber engagement further validate RUCONEST®’s strong value proposition for high-burden patients. We also advanced our key pipeline value drivers, including enrollment in the pivotal napazimone (KL1333) trial in primary mitochondrial disease and preparations for Phase II readouts later this year for leniolisib in broader primary immunodeficiencies. The encouraging compassionate-use experience with leniolisib in patients with CVID and immune dysregulation, which will be presented today at the Clinical Immunology Society Annual Meeting, further supports our view on the program’s potential. Finally, our focus on financial discipline also delivered tangible results, including positive net cash flow from operations in the quarter despite quarterly revenue variability. Taken together, our commercial execution, regulatory progress, pipeline advancement, and disciplined financial management position Pharming well for sustained long-term growth and value creation.” First quarter 2026 highlights Commercialized products RUCONEST® marketed for the treatment of acute HAE attacks RUCONEST® revenue in the first quarter of 2026 was US$58.4 million, a 15% decrease compared to the first quarter of 2025. RUCONEST® revenue in the current quarter compared to the first quarter of 2025 was impacted by inventory drawdowns at U.S. specialty pharmacy customers, which were anticipated and reduced revenue by 8%, our previous decision to withdraw the product from non-U.S. markets, which reduced revenue by 3%, and competitive market dynamics in the U.S. With its efficacy, reliability and rapid onset of action via IV administration, RUCONEST® remains a trusted on-demand treatment option for patients experiencing more severe or frequent attacks who have failed other on-demand medications. We continued to enroll new patients and add new prescribers in the quarter, with the vast majority of RUCONEST® patients showing limited interest in trialing or switching to alternative treatment options. Among patients who have trialed the new oral treatment option, we have observed that a meaningful proportion, particularly those experiencing more frequent attacks, have already made the decision to return to RUCONEST®. Joenja® (leniolisib) marketed for the treatment of APDS Joenja® revenue increased to US$14.1 million in the first quarter of 2026, a 34% increase compared to the first quarter of 2025. Revenue growth was driven by a significant increase in patients on paid therapy in the U.S., offset by greater inventory drawdowns in the current quarter, and increased demand in international markets, including strong patient uptake in the U.K. following the April 2025 launch and significant growth in the number of patients on government-supported access programs. The U.S. market contributed 82% of first quarter revenue, while the EU and Rest of World contributed 18%. As of March 31, 2026, 127 patients were on paid therapy in the U.S., representing a 25% increase from the 102 patients at the end of the first quarter of 2025 and an increase of seven patients during the quarter. APDS patient finding As of March 31, 2026, we have identified 1,016 diagnosed APDS patients of all ages globally, including 282 patients in the U.S. and 385 in core markets outside of the U.S. Of the identified patients in the U.S., 187 patients are 12 years of age or older and currently eligible for treatment with Joenja®, while 57 are between 4 and 11 years of age. Joenja® (leniolisib) development Leniolisib for APDS As of March 31, 2026, there are 180 APDS patients in either a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a paid access program. Pediatric label expansion On January 30, 2026, we received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding our supplemental New Drug Application (sNDA) for Joenja® (leniolisib) for the treatment of APDS in children aged 4 to 11 years. We held a Type A meeting with the FDA on March 26, 2026, to discuss the issues outlined in the letter and align on a path forward for resubmission. Upon the receipt of written feedback received from the FDA in the form of meeting minutes, we resubmitted the sNDA for the 40 mg and 50 mg twice-daily doses which would cover a meaningful proportion of the identified patient population, and plan to file a separate sNDA this summer for patients requiring a lower dose. European Economic Area (EEA) On March 26, 2026, the CHMP adopted a positive opinion recommending marketing authorization for Joenja® (leniolisb) in adult and pediatric patients aged 12 years and older. A final decision by the European Commission (EC) on the marketing authorization is expected in the second quarter of 2026. If approved, Joenja® would become the first approved treatment for APDS in the European Union. The centralized marketing authorization would be valid in all 27 European Union Member States, as well as Norway, Iceland and Liechtenstein. Japan In March 2026, the Japanese Ministry of Health, Labour and Welfare (MHLW) granted marketing authorization for Joenja® for the treatment of APDS in adult and pediatric patients aged 4 years and older. Joenja® is the first approved treatment for APDS in Japan and this approval is the first anywhere globally for children aged 4 to 11. Leniolisib for additional primary immunodeficiencies (PIDs) Two Phase II clinical trials are evaluating leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, including genetically identifiable PIDs linked to altered PI3Kδ signaling and common variable immunodeficiency or CVID, which represent substantially larger patient populations than APDS. Patient enrollment in both clinical trials is complete and we anticipate trial read-outs in the second half of 2026, consistent with prior guidance. A presentation at the 2026 Annual Meeting of the Clinical Immunology Society (CIS), taking place May 6-9, includes clinician expanded access experience with leniolisib to treat immune dysregulation in patients with CVID and CVID-like disorders. Clinician-reported outcomes demonstrated improvements, or no change or progression, in clinical manifestations of immune dysregulation and improvements in patients’ quality of life. Organizational updates On January 1, 2026, Leverne Marsh joined the Company as Chief Commercial Officer, succeeding Stephen Toor. Financial summary Underlying figures are unrounded. Therefore, totals may differ slightly from the sum of individual items due to rounding effects in the presentation of this press release. Financial highlights For the first quarter of 2026, total revenues decreased by US$6.6 million, or 8%, to US$72.4 million, compared to US$79.1 million in the first quarter of 2025. RUCONEST® revenues amounted to US$58.4 million, a 15% decrease compared to the first quarter of 2025. This decrease in RUCONEST® revenues was primarily driven by volume decreases in the U.S. and internationally, following our decision to withdraw from all non-U.S. markets. Joenja® revenues amounted to US$14.1 million in the first quarter of 2026, a 34% increase compared to the first quarter of 2025. This increase in Joenja® revenues was primarily driven by an increase in volume. Gross profit decreased by US$5.0 million or 7% to US$65.8 million (1Q 2025: US$70.8 million), mainly due to the decrease in revenues. The operating loss amounted to US$4.9 million compared to an operating loss of US$7.0 million in the first quarter of 2025. Excluding US$7.8 million of non-recurring Abliva acquisition-related expenses, the first quarter of 2025 would have reflected adjusted operating profit of US$0.8 million. The adjusted operating profit declined due to the decrease in revenues, increased R&D spending mainly related to the addition of napazimone (KL1333) to our pipeline, and unfavorable currency translation effects, partially offset by other expense savings. The finance result (net) and share of result in associates amounted to a gain of US$0.2 million compared to a loss of US$4.8 million in the first quarter of 2025. This improvement was primarily driven by favorable EUR/USD exchange rate movements, resulting in a foreign currency gain of US$2.4 million, compared to a loss of US$2.6 million in the first quarter of 2025. In the first quarter of 2026, a net loss of US$5.2 million was realized, compared to a net loss of US$14.9 million in the first quarter of 2025. In addition to the aforementioned drivers, the net result was positively impacted by a lower tax expense of US$0.5 million compared to a tax expense of US$3.1 million in the first quarter of 2025. Cash generated from operations amounted to US$2.0 million, compared to US$0.2 million in the first quarter of 2025. Cash and cash equivalents, including restricted cash and marketable securities, decreased from US$181.1 million at the end of fourth quarter of 2025 to US$171.8 million at the end of the first quarter of 2026. This decrease was primarily driven by a US$12.3 million settlement of the lease liability following the early termination of the DSP facility lease at Pivot Park in Oss, the Netherlands. Outlook/Summary For 2026, the Company anticipates: Total revenues between US$405 million and US$425 million (8% to 13% growth), with quarterly fluctuations expected. Total operating expenses between US$330 million and US$335 million (6% to 8% growth), including US$60 million incremental R&D expenses to advance the pipeline and US$9 million structural G&A cost reductions based on the plan announced in October 2025. Continued RUCONEST® growth, and significant and accelerating Joenja® U.S. and ex-U.S. growth. Progress towards additional regulatory approvals and commercial launches for leniolisib for APDS patients 12 years of age or older and for pediatric label expansion in key global markets. Top-line data readouts for the two ongoing leniolisib Phase II clinical trials in PIDs with immune dysregulation to expand the asset’s addressable patient population. Completion of enrollment in the pivotal FALCON clinical study for napazimone (KL1333) in mitochondrial DNA-driven primary mitochondrial diseases. Enhancing capital allocation to drive growth and build a leading global rare disease company. Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases. Financing, if required, would come via a combination of our strong balance sheet and access to capital markets. No further specific financial guidance for 2026 is provided. Additional information Presentation The conference call presentation is available on the Pharming.com website from 07:30 CEST today. Conference Call The conference call will begin at 13:30 CEST / 07:30 EDT on Thursday, May 7. A transcript will be made available on the Pharming.com website in the days following the call. Please note, the Company will only take questions from dial-in attendees. Webcast Link: https://edge.media-server.com/mmc/p/topu6hc2/ Conference call dial-in details: https://register-conf.media-server.com/register/BI1dba0cdf00fd47289729b51369140831 Additional information on how to register for the conference call/webcast can be found on the Pharming.com website. Financial Calendar 2026 Annual General Meeting of Shareholders May 28 2Q/1H 2026 financial results July 30 3Q 2026 financial results November 5 For further public information, contact: Investor Relations Michael Levitan, VP Investor Relations and Capital Markets T: +1 (908) 705 1696 E: [email protected] Media Relations Global: Saskia Mehring, Head of Corporate Communications T: +31 6 28 32 60 41 E: [email protected] U.S.: Ethan Metelenis (Precision AQ on behalf of Pharming) T: +1 (917) 882-9038 Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming) T: +31 6 53 81 64 27 About Pharming Group N.V. Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations. For more information, visit www.pharming.com and find us on LinkedIn. Forward-looking Statements This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information. Inside Information This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. Pharming Group N.V. Condensed Consolidated Interim Financial Statements in US Dollars (unaudited) For the period ended March 31, 2026 Condensed consolidated statement of income Condensed consolidated statement of comprehensive income Condensed consolidated balance sheet Condensed consolidated statement of changes in equity Condensed consolidated statement of cash flow Attachment Pharming Group reports 1Q26 results_EN_07MAY26
TranscriptFY2026 Q12026-05-07FY2026 Q1 earnings call transcript
Earnings source - 89 paragraphs
FY2026 Q1 earnings call transcript
Good day, and thank you for standing by. Welcome to the Pharming Group N.V. first quarter 2026 results webcast and conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there'll be a question-and-answer session. To ask a question during the session, you will need to press star one and one on your telephone. You will hear an automated message advising your hand is raised. To withdraw your question, please press star one and one again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Fabrice Chouraqui, CEO. Please go ahead.
Thank you, operator, good morning and good afternoon, everyone, and welcome to our Q1 2026 earning call. I'll be joined on this call today by Leverne Marsh, our Chief Commercial Officer, Anurag Relan, our Chief Medical Officer, and Kenneth Lynard, our Chief Financial Officer. Next slide. In this call, we will be making forward-looking statements that are based upon our current insight and plan. As you know, this may differ from future results. Next slide. As you saw in our press release, we made important progress across the business in this first quarter, despite a drop in quarterly revenue driven by RUCONEST. The RUCONEST revenue decline was largely expected due to inventory drawdown at specialty pharmacy, which we discussed on our Q4 2025 call in March. The commercial exit from non-U.S. markets also contributed to the year-on-year decline.
We announced that decision last year as part of our renewed financial discipline since the commercialization of RUCONEST in this market was not financially sustainable. Now, if we look at the underlying fundamentals, we see limited interest from patient on RUCONEST to try alternative therapies. Nine months after the launch of a new oral therapy, we have retained the overwhelming majority of RUCONEST patients, and we continue to see new patients starting RUCONEST. Leverne will elaborate on this market dynamic in a few moments. Coming now to Joenja. This product is an important growth driver, still early in its life cycle. Joenja revenues grew by 34%, reflecting strong momentum both in the U.S., where the number of patients increased by 25% year-on-year, but also in international markets.
We've also made meaningful regulatory progress this quarter, positioning us well to launch Joenja in Japan and in Europe later this year. To extend Joenja's label to the pediatric population in the U.S. After the disappointing CRL, we had a constructive dialogue with the FDA, we've already resubmitted the sNDA for the two highest dose, covering a meaningful proportion of children from four to 11. We are also planning to submit an sNDA this summer for the lowest doses. Finally, our disciplined cost management helped us to maintain positive cash flow from operations in this quarter, despite the variability in revenues. We are maintaining our revenue guidance of $405 million-$425 million for 2026, representing growth between 8%-13% year-on-year. Next slide.
As you can see, the durability of the RUCONEST franchise and the strong momentum and growth potential of Joenja underpin the transformation of Pharming into a profitable high-growth biotech with two late-stage pipeline programs offering billion-dollar sales potential. RUCONEST is the foundation of our portfolio and a reliable cash engine for the future, even in the ever more crowded HAE market, given its differentiated value proposition for the difficult to treat patient subpopulation and its highly specific manufacturing process. Joenja is just at the beginning of its life cycle with multiple growth catalysts in APDS through pediatric and geographic expansion and the potential expansion into higher prevalent PIDs with two phase II readout later this year. Anurag will discuss an exciting presentation at CIS conference taking place today that summarizes clinician experience treating patients with CVID, with immune dysregulation enrolled in our access program.
Last but not the least, napazimone, previously known as KL1333, for primary mitochondrial disease, is another billion-dollar-plus opportunity with the registrational study expected to complete enrollment this year and read out next year. These commercial assets and high-value pipeline, combined with durable source of cash flow, provide a solid foundation for Pharming to become a leading global rare and ultra-rare disease company with substantial near and long-term value creation potential. Let me now turn it over to Leverne, who will provide deeper insights into the performance of our commercial products.
Good morning. Good afternoon, everybody. Let me start with RUCONEST performance in the first quarter. Revenue was down 15% year-on-year. Importantly, as Fabrice mentioned, this was anticipated and largely driven by three distinct factors. First, inventory dynamics, which reduced quarterly revenue by 8%. This reflects what we previously stated on our March Q4 call and accounts for the majority of the impact. Second, our planned exit from ex-U.S. markets contributed approximately 3%. This is consistent with our strategy to focus our resources where we can generate the highest return. Third, with new treatment options entering the U.S. HAE market, we've seen measured impact from competition, specifically limited patient interest in trialing or switching to other therapies, with many returning. This has been in line with our expectations. Additionally, what's important is what's happening underneath these headline numbers.
We added approximately 50 new patient enrollments in the first quarter this year, and we brought on 23 new prescribers onto RUCONEST. This is a meaningful signal that clinicians continue to see the value of RUCONEST, in specifically in the high attack, high severity segment, and are initiating new patients even as the treatment landscape expands. Next slide, please. On this slide, this really gets to the heart of why RUCONEST continues to play a critical role in HAE management. We know HAE is not a uniform disease. For patients on the more severe end of the spectrum, meaning those with frequent attacks, rapid onset symptoms, or high anxiety around unpredictability or the attack location, the need is very clear. They require a treatment that works quickly, consistently, and durably, and that's exactly where RUCONEST fits, and it's reflected in what we're seeing in the market today.
After nine months into the launch of a new oral competitor in HAE in the U.S., the overwhelming majority of RUCONEST patients have remained on therapy. Among those who had explored alternatives, many high-burden patients are returning to RUCONEST, in particular when response to new treatments have not been adequate. This reinforces the importance of having a dependable on-demand therapy like RUCONEST and underpins our confidence in the long-term role of RUCONEST in this evolving HAE market. Next slide, please. Turning to Joenja, we delivered another strong quarter building on the momentum from last year. Revenue grew 34% compared to the first quarter of 2025, reaching $14.1 million globally. In the United States, patient growth is the central driver of performance.
By the end of the quarter, we had 127 patients on paid therapy in the U.S. alone, which represents a 25% increase over the first quarter of 2025. We accelerated the rate of new patient starts to seven during the quarter, an improvement over the additions seen in the previous two quarters. The U.S. fill rate remained high at 85%, reflecting our highly effective reimbursement support and patient services process. Equally important, we continue to broaden the pool of APDS patients. We've identified 187 APDS patients older than 12 years old in the U.S. and an additional 57 eligible patients in the four to 11 years old group, this represents the next frontier for growth in the U.S.
In international markets, we continue to see strong patient uptake in the U.K. and significant growth in the number of patients on government-supported access programs in other countries. This momentum sets us well to drive growth in APDS and other indications to come. Next slide, please. Now, stepping beyond the quarter, I want to put Joenja into its broader strategic context. We are building more than a single rare disease product. We are building indeed a scalable immunology franchise with multiple clearly defined growth levers. The first growth lever is continued expansion within APDS itself. We are still early in identifying APDS patients, and there is significant proportion of patients yet to be identified. The second growth lever is further U.S. APDS expansion, which includes the pediatric launch in the United States and an upside U.S. reclassification opportunity across all ages. Thirdly is international expansion.
We are in the early stages outside the United States, and upcoming launches in Europe and Japan will open meaningful new markets for us. Finally, the fourth growth lever is life cycle and label expansion beyond APDS, specifically exponentially larger patient pools in genetic PIDs and CVID with immune dysregulation. Taken together, these four levers create sequential growth engines over the coming years. APDS drives the initial growing foundation, pediatric expansion deepens penetration, geographic expansion broadens reach, and new indications continue to give us access to significantly larger patient segments, which extends our platform. Now to share more about our pediatric submission and life cycle efforts on Joenja, I will now hand it over to Dr. Anurag Relan, our Chief Medical Officer.
Thank you, Leverne.
In addition to the important regulatory milestones in Japan and Europe, we made significant progress in the U.S. in our efforts to expand the Joenja label to pediatrics for children ages four to 11 with APDS following the receipt of a CRL from FDA in January. As we previously explained, we believe the clinical pharmacology and analytical batch testing methodology issues outlined in the FDA letter were addressable. We held a Type A meeting with FDA at the end of March, which included two APDS expert physicians, and we were pleased with the constructive dialogue and understanding of the issues raised by FDA in the CRL. The FDA also appreciated the unmet need, including the serious and progressive nature of APDS, as well as challenges with clinical trial recruitment in young children with an ultra-rare disease.
We worked collaboratively with FDA to define the most expedient path forward. We have that now, with the first step being the resubmission of the sNDA for the highest doses, specifically 40 and 50 mg. This took place in April, in fact, on the same day that we received the FDA's meeting minutes. As is typical, we plan to issue a press release upon FDA acceptance of the resubmission. These doses, as Fabrice mentioned, cover a meaningful proportion of four to 11-year-old children. An FDA decision on this is expected in six months or sooner. The second step will be a new sNDA for the doses covering the lowest weight patients, which is planned for this summer. For this sNDA, we also expect a six-month review. Next slide.
At the Clinical Immunology Society annual meeting this week, Pharming and our collaborators are presenting seven abstracts, five expanding the evidence base in APDS, and two that begin to provide data on the much larger opportunity in other PIDs with immune dysregulation. These include the clinical expanded access experience with leniolisib to treat immune dysregulation in patients with common variable immune deficiency, or CVID, and CVID-like disorders, which I will cover in more detail in a few slides. As you see, APDS is just the beginning for leniolisib. Next slide. In addition to APDS, we continue to make progress in other PIDs with immune dysregulation, which is based on the observation of the key role of PI3K delta as an important regulator of immune cells, an imbalance in the pathway which underlies the immune dysfunction across several primary immune deficiencies.
This mechanistic understanding forms the scientific rationale for our Joenja development program. Joenja, as you know, is currently approved for APDS, where gain-of-function mutations drive a hyperactive pathway leading to immune deficiency alongside broad immune dysregulation. APDS, in fact, serves as proof of concept for the ongoing two phase II studies evaluating leniolisib in other PIDs. These have significantly greater prevalence in APDS, but share unmet medical needs, underlying mechanisms, and disease pathology. The programs target two similar populations. The first is genetically identified PIDs with immune dysregulation, which represent a prevalence that's five times greater than APDS or more than 2,500 patients in the U.S. alone.
The second is Common Variable Immune Deficiency with immune dysregulation, which is identified independently of genetics, and this is even a larger group of patients, which is approximately 26 times the size of APDS or more than 13,000 patients in the U.S. alone. I'll now talk to you about the studies in the next slide. Both proof-of-concept studies share a common design architecture. Single-arm, open-label dose range finding allowing cross-study comparability. The CVID study is a multicenter study enrolling 20 patients, and the genetic PID study is a single center study conducted at the NIH with 12 patients. Both studies are now fully enrolled with trial readouts expected later this year. Both also employ a three-dose escalation design to characterize dose response and confirm the optimal dosing strategy. The studies address two core objectives.
First, of course, to address safety, tolerability, and the pharmacokinetics and pharmacodynamics to confirm dosing. Second, and most clinically meaningful, to estimate the efficacy against immune dysregulation, specifically looking at the lymphoproliferation and autoimmune aspects. These efficacy endpoints are aligned with the key disease manifestations which are focused on these aspects. In addition, we'll also be collecting patient-recorded outcome measures which were developed through a custom process involving expert input and formal interview studies with CVID patients. Next slide, please. Ahead of these study readouts, we can see some important early clinical evidence supporting leniolisib's potential in CVID with immune dysregulation being presented today at the CIS meeting.
Six CVID or CVID-like patients with immune dysregulation amongst the sickest patients refractory to other therapies received leniolisib through an expanded access program for a median of 1.4 years, with individual exposure ranging from half a year to two and a half years, providing meaningful duration of observation for a small cohort. The clinical signal is encouraging and consistent across disease manifestations. Clinicians reported improvement with no patient showing progression, spanning cytopenias, splenomegaly, lymphadenopathy, liver disease, and lung disease. Immune profile showed reduced transitional and CD21 low B cells, confirming the meaningful PI3K delta pathway modulation consistent with the APDS experience. This biomarker data is also being collected in the phase II studies. Regarding safety, adverse events were generally manageable and consistent with the disease severity.
While this is clinician-reported data and not a prospective clinical study, the breadth and consistency of improvement across these various endpoints is a compelling early signal ahead of the formal study readouts in the second half of this year. Quite a bit to look forward later this year. With that, I'll turn it over to Kenneth to walk through our financials.
Thank you, Anurag. I will now briefly cover our Q1 2026 results and our full year outlook. Q1 revenues were EUR 72.4 million, down 8% year-on-year. RUCONEST revenue declined 15%, reflecting the expected U.S. inventory normalization contributing 8% decline, consistent with our expectation for 7%-9% headwind that we communicated on the March Q4 call, as well as also our planned strategic exit from U.S. markets, which contributed 3% to the decline. Q1 is also typically the lowest seasonal quarter for RUCONEST due to ordering patterns and inventory dynamics. Joenja revenues were strong and increased 34% year-on-year, driven by strong U.S. momentum, continued patient growth, and expanding international demand. Revenue was modestly affected by inventory timing, and excluding this, growth would have been $1 million-$2 million higher.
Total operating expenses were down by 9% year-on-year. Adjusted for non-recurring Abliva-related acquisition costs in Q1 2025, overall expenses were flat. This demonstrates our ability to increase pipeline investments without increasing costs overall. Adjusted operating profit declined slightly year-over-year, noting that $7.8 million of the non-recurring Abliva acquisition-related costs are excluded from the adjusted Q1 2025 figure shown on the slide. In 2026, we have incremental R&D investments for napazimone of EUR 2.7 million included. We generated positive operating cash flow in Q1 of EUR 2 million, reflecting continued strong cash, cost management and financial discipline. Total cash and marketable securities decreased by EUR 9.3 million to EUR 171.8 million, primarily due to a EUR 12.3 million payment related to early term-termination of the DSP facility lease.
For the full year 2026, we are pleased to reaffirm our expectation for total revenues of $405 million-$425 million, representing full year growth of approximately 8%-13% versus 2025. This growth is expected to be driven by continued expansion of RUCONEST in the U.S., partially offset by the exit from ex-U.S. markets and significant and accelerating growth for Joenja. We delivered a strong exit to Q1, and the low % of HAE patients switching to competing oral therapies gives us confidence in our guidance range. Overall, we assume low single-digit annual RUCONEST growth at the midpoint of our guidance range, with some pressure expected on RUCONEST revenue in Q2 and growth in the second half of the year. For Joenja, we are well-positioned for launches in Japan and Europe this year.
We also now include expected U.S. pediatric label revenues later this year, previously excluded from our guidance in our outlook. We expect Joenja growth to accelerate, with annual growth over 10 percentage points higher than in 2025. The pediatric APDS indication remains an important long-term driver, and for planning purposes, we conservatively assume a six-month FDA review period following resubmission with a launch right thereafter. We continue to expect operating expenses between $330 million-$335 million, including $60 million in incremental R&D investment to advance our pipeline. This includes up to $30 million additional for the development of napazimone. This also reflects the $9 million benefit from the 20% G&A structural headcount reduction announced in October 2025, alongside stable marketing and sales spending.
We remain very committed to strong cost management and financial discipline, prioritizing investments that support both near and long-term value creation. There are no changes made to any other guidance assumptions, including milestone payments or gross margins. As a reminder, for Joenja, we do not assume the EUR 10 million commercial milestone or additional milestone payments this year. Gross margin is expected to be approximately 90%. Finally, as previously stated, our available cash and future operating cash flows are expected to fully support all pipeline investments, including all pre-launch activities. With that, I'll now hand over to Fabrice for his closing remarks.
Thank you, Kenneth. In summary, this first quarter demonstrated important progress across the business while reflecting viability in RUCONEST revenues. We are encouraged by the opportunity we see for Joenja in the short and long term, and the potential for RUCONEST to remain a significant cash engine as an important on-demand treatment for the difficult-to-treat patient subpopulation. We have significant pipeline catalysts later this year. First, the readout of the two phase II trials for leniolisib in higher prevalent PIDs. Second, the completion of the enrollment of the napazimone registrational study in primary mitochondrial disease. As you've seen, the decisive steps that we've taken to improve financial discipline, including optimizing G&A accounts, are starting to deliver tangible results.
With our strong commercial and development capabilities, a growth-oriented leadership team, and a scalable organization, we are committed to driving sustainable revenue growth and value creation to achieve our vision of being a leading global rare disease company. Let me now open the line for questions.
Thank you. To ask a question you will need to press star one and one on your telephone and wait for your name to be announced. To withdraw your question, please press star one and one again. We will now go to the first question. One moment, please. Your first question today comes from the line of Benjamin Jackson from Jefferies. Please go ahead.
Brilliant. Thank you for the question, guys. I've got two, if I may. The first, just on RUCONEST. Could you talk a little bit more about why you think you'll see further pressure in the second quarter on that sales line? Then why you think that you or what gives you the confidence of returning to growth into the second half of the year beyond what you've already described? Then within that also, are you expecting any reversal of this inventory drawdown at all that may help as a bit of a tailwind in context of that? Then secondly, on Joenja, perhaps if you could just help paint the picture about how meaningful you think the Europe will be this year and how quickly we should anticipate this ramping.
Perhaps you could touch on which countries will likely come online in Europe when and how quickly you think you can secure reimbursement there. Anything to build out that picture a little bit more for me would be, would be super useful. Thanks so much.
Thank you, Ben. Leverne?
Indeed. Ben, thank you so much for the question. I think your first one on further pressure in Q2 that we may be anticipating. We're in the early stages of competitive entry, right? 9 months into the sebetralstat launch, followed quickly by prophylactic treatments. What we're seeing is it takes a few reorder cycles, so three to four reorder cycles for us to see the full impact of trialing behavior and switching behavior. As we get into essentially the fourth quarter of a launch post sebetralstat, we'll start to see further impact normalize in the second quarter. The second piece that you asked around growth in the second half of the year. Today, we continue to add both new prescribers and new patient enrollments to RUCONEST.
What that tells us is there is a clearly defined subpopulation of HAE patients, who are high burden patients, so high frequency of attack patients, high attack location patients, where RUCONEST continues to have a place. Despite competitive entries, we continue to see new patient generation and new prescriber dynamics in that segment. I'll let Kenneth speak to the inventory drawdown, and I'll talk about Joenja, the question that you had on European launches. As you know, we had a positive CHMP opinion earlier this year. We're waiting for final approval. Our first launch in Europe will be in Germany this year. We're really excited about that launch coming in at the end of toward the second quarter of this year.
That will be meaningful for us because we are anticipating commercial patients, so paid funded commercial patients, into the second quarter. Additionally, our Japan approval that we received also earlier this year, we're anticipating that launch in August of this year. Some key growth drivers for us in the second year for Joenja, in addition to the pediatric approval that we are anticipating for the higher doses in the U.S. Kenneth, do you want to respond to the inventory question?
Yeah, absolutely. Thanks for the question, Ben. In 2026, we have seen the inventory drawdown, which follows the normal cycle of the year. Compared to last year, where in 2025, the inventory drawdown was lower as the previous year's build was lower as well. We do anticipate that we are in a year that again is more reflective of the normal cycle, where there will be inventory build during the second half of the year to basically reflect the demand. That's how we are looking into the rest of the year.
Great. Thank you so much.
Thank you. Your next question today comes from the line of Jeff Jones from Oppenheimer. Please go ahead.
Good morning or good afternoon, guys, and thanks for taking the question. Maybe one follow-up on RUCONEST and then on leniolisib. Can you help us maybe link the 4% drop in revenue not associated with the inventory drawdowns in the planned U.S. or the ex-U.S. exit with the offset of the 50 new patients on therapy that you mentioned during 1Q? For leniolisib, you talked a little bit about the readouts from the phase I/IIs that you're running currently for PIDS and CVID. Can you help us link those efficacy-related readouts to expectations around endpoints in phase III and how we can think about expectations and endpoints moving ahead into more pivotal aligned studies? Thank you.
Thank you, Jeff. I'll take the first part of your questions on the enrollment. Then I'll let Anurag cover the leniolisib part. When it comes to the enrollments that Leverne mentioned, these are 50 new patients which have been enrolled, will receive a script. These are not yet 50 new patients on the drug. There is always actually a delay between enrollment and a patient on therapy. Obviously, we'll be working actively on that. I think you should look at enrollment as patient in the pipes that ultimately will, for a large proportion, be treated by RUCONEST.
Again, seeing a significant number of new enrollment, new scripts for RUCONEST, and a significant number of new prescribers, I think reinforce the recognition of RUCONEST as a distinctive treatment, in the HAE on-demand, category. I hope I was able to bring color.
Thank you for that.
All these new patients are expected to offset the small number of patients that may adopt, actually. As Leverne said today, you know, we've seen only a very limited interest from RUCONEST patients to try actually, and we've seen a very small number of these patients adopting the drug. This is obviously linked to the nature of these patients, which are for vast majority of them, have a high burden disease and often have already failed a number of treatments. Anurag, would you like to elaborate on the leniolisib data that are being presented today?
Sure. Jeff, I think we have to zoom out of here a bit and look at what the unmet need really here is in this group of patients. The unmet need is all centered around immune dysregulation. The immune dysregulation we're talking about is these aspects such as lymphoproliferation and autoimmune disease that isn't being managed adequately by immune globulin replacement therapy that these patients currently receive. Those are the disease manifestations that we're looking at. Those are, in fact, what we see in the expanded access program. These six patients that are being presented today at the CIS meeting, you can see these same disease manifestations, whether it's improvements in their cytopenias, improvements in lymphoproliferation, or improvements in some of the other aspects of the autoimmune disease.
Those are the things that we're also going to be measuring in both of the phase II studies. We're looking at lymph node size, spleen size. We're looking at the blood counts. We're looking at some of these other markers of end organ disease activity. Those will then form the basis for the phase III study. It's exactly, you know, the endpoints are, I think, very well aligned with the disease manifestation. Again, what we see early from these six patients is improvement or stabilization in all of these aspects.
Thank you very much.
Thank you. Your next question comes from the line of Suzanne Van Voorthuizen from Van Lanschot Kempen. Please go ahead.
Yes. Thank you for taking my questions. On your revenue guidance, what could be key drivers that could make the difference between hitting the top end and the bottom end of your range? What are your assumptions here? Could you share more color on the compassionate use experience in CVID? How much do these patients resemble the patients in your phase II study? Thank you.
Kenneth Lynard.
Yeah. Thank you. This is Kenneth. Thanks, Suzanne. I think the way to think about it is that we are anticipating 6 months For approval and launch right thereafter for the U.S. pediatric population following the submission. Obviously, an accelerated timing of the approval and launch will provide an upside compared to what we are kind of looking into now and therefore would put us higher up in the guidance range. That would be the primary driver.
Okay, that's clear. Could you share more color on the data that was presented at CIS on the compassionate use experience in CVID? How much do these six patients resemble the patients in your phase II study? Thank you.
Sure.
Hi, Susila. It's actually a great question and something I didn't cover. These CVID patients and CVID-like patients in the compassionate use experience very much resemble the types of patients that are being enrolled in the CVID study, so the 20-patient multicenter study. The reason for that is that these patients, all of them have those aspects of immune dysregulation. Now, I would say the only difference here is that this is a much sicker group than the general CVID immune dysregulation population, which is already quite ill to begin with, but this is a group that has been even more refractory to other types of therapies. The fact that we can see improvements here is, I think, quite meaningful and quite encouraging for us as we look ahead to the results later this year.
Okay. That's clear. Thank you.
Thank you. Your next question comes from the line of Joseph Pantginis from H.C. Wainwright. Please go ahead.
Hi, this is Josh on for Joe. Thanks for taking our questions. For the first one, could you guys provide more color around the proportion of the identified four to 11-year-old APDS patients in the U.S.? Specifically, how many could be covered by the initial 40 and 50-mgm resubmission? For the Type A meeting, did the FDA feedback change how you're thinking about pediatric dosing more broadly, or has your overall strategy remained largely unchanged?
On the first part of the question, Leverne.
Sure. Thanks, Josh. On the first one, on the four to 11 age group, you can assume approximately half, so roughly 50% of that population would be eligible for the high dose leniolisib, and half would be on the lower end.
Anurag?
Josh, on the Type A meeting and the feedback that we got, and actually all of the discussions that we've had, I think it really has not changed our dosing strategy. In fact, what I think, you know, based on this constructive dialogue that we had with FDA, they were, we shared with them the efficacy that we'd observed across the doses, and that has allowed us to maintain the same doses in our resubmission strategy. The lower weight patients would be maintained on or we're proposing to maintain them on the same doses that were used in the clinical trial. That really is tied to the efficacy that was observed in the lower weight patients, which was very similar to the efficacy that was observed in the higher weight patients.
I think on that basis, we have not changed the dosing strategy, and I think we've come to an agreement with FDA on what the contents of these two resubmissions would be.
Great. Thank you. Very helpful.
Thank you. Your next question comes from the line of Whitney Ijem from Canaccord. Please go ahead.
Hey, good morning, guys. Thanks for taking the question. Just to follow up to clarify for the phase II leniolisib readouts in the second half, just wanted to confirm, will those be read out at the same time, so it is one readout for both, or is it two? Could they come at different times?
Anurag?
The study is completed enrollment around the same time. One of the studies is one month shorter in duration, so it is possible that that one we have all of the data available slightly earlier than the other study. And once we have the data cleaned and available to evaluate, we'll have more specifics on the exact timing of that readout.
Okay. Got it. Headed into those, can you help set investor expectations, I guess, in terms of what would be good data, or what you're looking for, in both of those studies?
Sure, Whitney.
Either quantitatively or more qualitatively.
A lot of the things that we're looking for, first of all, they're aligned with what we observed already in APDS. We saw lymph nodes shrink. We saw spleens get smaller. We saw improvements in immune profiles. We saw improvements in blood counts, so the cytopenias, autoimmune cytopenias that occur in these patients. We've seen that already in APDS. That's why, again, I really think it is a very nice proof of concept for what we've already done. What we know is this is also the unmet need in these other primary immune deficiencies, so really looking for the same things. We're looking to see if lymph nodes get smaller. We're looking to see if the spleens get smaller.
We're looking to see platelet counts or other blood cell counts increase. We're looking for other end organ disease manifestations to see how they also improve. I think this is also, again, lines up very nicely with the data.
We presented today that I shared in the slide is that we see already in these in this early experience with these six patients, we see those same types of improvements. I think that is again a very encouraging early sign. It's not a clinical trial, but it's an early sign that both based on the APDS experience as well as this six-patient expanded access experience, the kinds of things that we can expect to see in the readouts of these two phase II studies.
Got it. Thank you.
Thank you. As a reminder, if you would like to ask a question, please press star one and one on your telephone. That is star one and one to ask a question. We'll now go to the next question. The next question today comes from the line of Natalia Webster from RBC. Please go ahead.
Hi there. Thanks for taking my questions. I have a couple of follow-ups, please. Firstly, on RUCONEST, just on the around 50 new enrollments that you've seen and 23 new prescribers in Q1, do you see this as a sustainable run rate going forward? Secondly, appreciate it can take sort of three to four reorder cycles to see the full impact. Are you able to provide any quantification on what sort of percentage of your patient base has tried EKTERLY and what sort of return rate you're seeing to date? Finally on Joenja, you added seven net U.S. patients on paid therapy in Q1 and believe you previously guided to accelerating enrollment this year. Is this acceleration dependent on the pediatric approval, or are you also expecting an acceleration in adult patients in the coming quarters? Thank you.
Thank you, Natalia, for your question. I'll take the first part on RUCONEST and let Libby elaborate on the second part on Joenja. Clearly, we've seen over the past quarter really our ability to see a sustainable stream of new enrollments. Despite the launch of new therapies, whether these are prophylactic therapies or on-demand therapy, we've seen that because of the differentiated profile of RUCONEST, we've been able to gain quarter after quarter a significant number of new patients. We don't see that changing. Specifically as the launch of EKTERLY is making the on-demand market more dynamic.
We see a significant increase of switchers, and doctors are more prone to engage with their patients on whether they are well controlled. We see an opportunity for RUCONEST to capture a higher number of patients that would not be controlled correctly on their current treatment. To complement what Kenneth said earlier, this is also a significant element to reach the upper end of our guidance. Clearly, reaching the upper end of our guidance is about the timing for the U.S. approval of the pediatric extension, but also our ability to grow RUCONEST and leverage this market dynamic with more switchers.
I will let now, Libby comment on the Joenja, on your questions related to Joenja.
Thanks, Fabrice, and thank you, Natalia. On the Joenja acceleration, we still have significant room for growth in the 12 and above patient population, right? As we mentioned, we added seven new patients on therapy in Q1, and we're seeing some good sustainable momentum into Q2 already. As you think about the adult 12 and above opportunity, we continue to identify new patients. We continue to convert those new patients, and that is a sustainable source of growth for us in the future because there's a lot of room for us to grow. I think the point that you mentioned on the pediatric indication in the second half of the year will be an additional growth lever for us in the U.S., right?
Ex-U.S., as we mentioned, will be the launch in Germany and launch in Japan later this year. I would think about the year in these sort of phased steps of acceleration in the current population, the pediatric population, and the international expansion in the second half of the year.
Great. That is very helpful. Thank you.
Thank you. The final question comes from the line of Simon Scholes from First Berlin. Please go ahead.
Yes. Hello. Thanks for taking my question. I've got a question on leniolisib and the Type A meeting. My impression in March was that you would be able to deliver the additional information that the FDA required and that probably you'd be able to make resubmissions, immediate resubmissions in both the high dose and the low dose. Could you just outline what extra work you're going to need to do on the low dose patients until you resubmit in the summer?
Sure, I can answer that, Simon. I think what we really, you know, when we met with FDA, and I think what we tried to define was the fastest way to bring Joenja to this youngest group of patients as and, to try to do it in a way that allowed us to leverage the data we already had. That's why we went with this two-submission approach that allows this 40 and 50 mg submission to already occur. We submitted it, as I said, on the same day that we received the FDA meeting minutes. I think that was a very important outcome.
For the second group, really, this was just making sure that we had all of the efficacy data, and as I said earlier, the efficacy data that lined up very nicely with the in both the high-dose and the low-dose groups or the high-weight and lower weight patients. It's really just putting that data package together. I think the key piece or the key point to note is that we have an agreement with FDA on what the contents of that submission will be. Importantly, it doesn't require an additional clinical trial, this submission. I think that's where we are, and that's why we went with this two-submission approach. Then we expect to make the second submission in this summer.
Okay, thanks very much.
Thank you. I will now hand the call back to Fabrice Chouraqui for closing remarks. Please go ahead.
Thank you so much, operator. I hope we were able to provide clarity on the on our performance in the first quarter. As we said, we've seen meaningful improvements across the business, despite some revenue viability. I personally believe as the rest of the leadership team that we are those progress are really positioning Pharming Group extremely well for long-term value creation. We look forward to updating you on our plan for the short and midterm and long-term as well. Thank you so much.
Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

