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Shares of BioMarin Pharmaceutical BMRN were down 4% on Monday after it reported results from the phase III ENERGY 3 study, which evaluated BMN 401, an investigational enzyme replacement therapy (ERT), for a rare genetic disorder called ENPP1 deficiency in children aged 1 to 12. The study did not meet one of its two main goals. The mixed results were a setback for BioMarin, which added BMN 401 through its $270 million acquisition of Inozyme Pharma last year. This ERT was the lead asset in Inozyme’s pipeline, and the ENERGY 3 study was already underway at the time of the acquisition. Year to date, the stock has lost 16% compared with the industry’s 3% fall. Image Source: Zacks Investment Research The study assessed two primary endpoints — changes in plasma inorganic pyrophosphate (PPi) and Radiographic Global Impression of Change (RGI-C) scores after 52 weeks of treatment. While treatment with BMN 401 after 52 weeks resulted in statistically significant increases in plasma PPi, there was no corresponding improvement in RGI-C scores. An increase in plasma PPi is considered an important biomarker response because ENPP1 deficiency leads to low PPi levels, which can result in progressive damage to blood vessels, soft tissues and bones. However, BMN 401 failed to demonstrate improvement in RGI-C scores, a key measure used to assess treatment impact in children with rickets. The lack of radiographic improvement suggests that the biomarker gains did not translate into measurable skeletal benefits during the study period. The therapy also did not show positive trends across secondary endpoints, including Rickets Severity Score (RSS), a measure of rickets severity, and growth Z-scores evaluating height/body length and weight. BioMarin stated that it will continue analyzing the complete ENERGY 3 dataset and engage with global regulatory authorities to determine the next steps for BMN 401’s development program. The company also plans to present detailed findings from the study at a future medical meeting. The stock currently carries a Zacks Rank #3 (Hold). BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote Some better-ranked stocks from the sector are Immunocore IMCR and Indivior Pharmaceuticals INDV, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 30 days, estima...

CorMedix Therapeutics CRMD delivered first-quarter 2026 diluted earnings of 43 cents per share, up 43.3% year over year, beating the Zacks Consensus Estimate of 35 cents. Net revenue was $127.4 million, up significantly from the year-ago sales of $39.08 billion. The reported figure beat the Zacks Consensus Estimate of $110 million. Results reflected stronger DefenCath execution and underlying demand trends, with DefenCath net revenues of $97.5 million in the quarter. Management also lifted its full-year outlook following the better-than-expected start to 2026. The stock gained 5.7% on Thursday following the earnings release. DefenCath remained the key operating lever in the period, supported by higher utilization among outpatient dialysis customers. Its sales increased, primarily boosted by the onboarding of a large dialysis organization in mid-2025, along with strong positive demand trends. Quarterly DefenCath performance also benefited from a favorable change in estimate tied to certain sales allowances, including items such as Medicaid rebates and product returns. While that impact provided a lift, management pointed to underlying utilization momentum as the more important signal on demand durability. The Melinta portfolio contributed $29.9 million in the first quarter. Its acquisition in the last year added a meaningful second revenue stream and broadened CorMedix’s commercial footprint. The Melinta contribution also changed the year-over-year comparison framework for CorMedix, given that the acquisition occurred in August 2025. As a result, the year-ago period reflected revenue from only DefenCath, making the current quarter’s mix and scale structurally different. Shares of CorMedix have plunged 31.9% so far this year against the industry’s 1.1% growth. Image Source: Zacks Investment Research Operating expenses increased sharply year over year as the company absorbed a larger cost base following the Melinta acquisition. Total operating expenses were $41.5 million, up 138.5% from the prior-year quarter, which management attributed primarily to expenses related to the acquired portfolio and the broader combined-company footprint. R&D expenses climbed to $7.2 million, up 125% year over year, due to higher personnel spending and clinical trial services tied to ongoing programs, including pediatric studies for certain brands and continued DefenCath developme...

Catalyst Pharmaceuticals CPRX reported adjusted earnings of 79 cents per share for the first quarter of 2026, beating the Zacks Consensus Estimate of 64 cents. The company had recorded adjusted earnings of 68 cents in the year-ago quarter. Total revenues, the majority of which comprised product revenues, amounted to $149.4 million in the reported quarter, representing growth of 6% year over year. The recorded figure also surpassed the Zacks Consensus Estimate of $147 million. Catalyst Pharmaceuticals’ top line primarily comprised revenues from the sale of Firdapse, the first approved drug for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) and the newer muscle disease drug, Agamree (vamorolone). Revenues generated from the sale of CPRX’s epilepsy drug Fycompa (perampanel) CIII also contributed to the top line. Firdapse generated sales worth $98.86 million in the reported quarter, up 18% year over year, driven by organic sales growth. The reported figure marginally missed the Zacks Consensus Estimate of $98.9 million. The drug has been witnessing strong demand, increasing prescription rates from LEMS patients and continued diagnosis of new LEMS patients. In 2023, Catalyst Pharmaceuticals acquired exclusive rights to manufacture and supply Agamree from Santhera Pharmaceuticals through a licensing agreement. In late 2023, the FDA approved Agamree for treating Duchenne Muscular Dystrophy in patients aged two years and older, which gave the company a third approved product. The drug was commercially launched in the United States in the middle of March 2024. In the reported quarter, Agamree generated revenues worth $36.7 million, up 67% year over year. The reported figure beat the Zacks Consensus Estimate of $35 million. Year to date, Catalyst Pharmaceuticals shares have gained 33.5% against the industry’s 4.7% decline. Image Source: Zacks Investment Research In 2023, Catalyst Pharmaceuticals acquired the U.S. rights for Fycompa (perampanel) CIII from Eisai Co., Ltd. This acquisition diversified the company’s portfolio by adding a commercial-stage epilepsy asset. Catalyst Pharmaceuticals started recording sales of Fycompa in 2023. Fycompa generated net product revenues of $13.8 million, down 61% year over year, as tablet generics began hitting the market in May 2025 following the expiration of its first U.S. patent, with another slated to expire in July...

Intellia Therapeutics NTLA incurred first-quarter 2026 loss of 81 cents per share, narrower than the Zacks Consensus Estimate of a loss of 92 cents. In the year-ago quarter, the company had incurred a loss of $1.10 per share. Intellia’s total revenues currently comprise only collaboration revenues. The company reported revenues of $15 million for the first quarter of 2026, which missed the Zacks Consensus Estimate of $16 million. Total revenues declined 9.5% year over year. Year to date, shares of NTLA have surged 60.4% against the industry’s 2.7% decline. Image Source: Zacks Investment Research Research and development expenses totaled $80.7 million, down 25.5% from the year-ago quarter’s figure. The decrease was due to lower employee-related expenses, stock-based compensation and reduced spending on research materials and contracted services. General and administrative expenses in the first quarter were $34.8 million, up 20.1% year over year, primarily due to continued investments in building the company’s commercial infrastructure and higher legal expenses, partially offset by lower stock-based compensation. As of March 31, 2026, Intellia had cash, cash equivalents and marketable securities worth $517.2 million compared with $605.1 million as of Dec. 31, 2025. Following an underwritten public offering of common stock, the company expects its cash runway to support operations into 2028. Intellia has collaborated with Regeneron Pharmaceuticals REGN to develop its investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z), which is being studied for two indications — ATTR amyloidosis with polyneuropathy (ATTRv-PN) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). In March, the FDA lifted the clinical hold on the investigational new drug application (IND) for the phase III MAGNITUDE study evaluating nex-z in patients with ATTR-CM. Earlier this year, the FDA lifted the clinical hold on the IND application for the phase III study, MAGNITUDE-2, evaluating nex-z in patients with ATTRv-PN. Enrollment in this study is expected to be completed in the second half of 2026. With the removal of the clinical hold, Intellia is now focusing on completing patient enrollment in both late-stage studies as promptly as possible. In April, Intellia announced top-line data from the global phase III HAELO study evaluating lonvo-z, an in vivo CRISPR gene editi...

ANI Pharmaceuticals ANIP delivered first-quarter 2026 adjusted EPS of $2.05, up nearly 21% year over year and well ahead of the Zacks Consensus Estimate of $1.28. Quarterly revenues totaled $237.5 million, up 20.5% from the year-ago period. The metric also beat the Zacks Consensus Estimate of $205.4 million. The quarter reflected solid execution across the portfolio, led by continued momentum for Purified Cortrophin Gel and contributions from a newly monetized intellectual property licensing arrangement. Rare Disease and Brands' total net revenues were $128.2 million, up 36% year over year, supported by contributions from both Cortrophin Gel and Iluvien. Within that bucket, Cortrophin Gel net revenues rose 42% to $75.1 million, while Iluvien sales increased 19.5% to $19.3 million. The reported Cortrophin sales marginally missed the Zacks Consensus Estimate of about $76 million. Per management, the drug’s sales were impacted primarily by seasonality tied to insurance re-verifications, which took longer to clear early in the quarter due to higher patient volume at physicians’ offices and weather-related physician office closures in some regions. ANIP's shares fell more than 2% on Friday, suggesting some investors focused on the slightly softer-than-expected Cortrophin print despite the broader earnings and revenue beat. Year to date, the stock has gained about 4% against the industry’s nearly 3% decline. Image Source: Zacks Investment Research The company also reported $21.5 million in brand royalties and other revenues in the quarter, reflecting the up-front payment and early royalty income tied to its Harmony Biosciences HRMY licensing agreement. By contrast, Brands' revenues declined 51% year over year to $12.3 million as demand normalized for certain products. In January, the company’s Novitium subsidiary entered into an agreement with Harmony Biosciences, under which ANIP out-licensed intellectual property related to pitolisant, marketed under the brand name Wakix. The agreement generated a $15 million upfront license fee and includes low single-digit royalties on sales of pitolisant-based products. It provides for an additional $10 million in development milestones that management expects to be achieved in the second and third quarters of 2026. Generics and Other net revenues were $109.2 million in the first quarter, up 6% year over year. Growth was driv...

Theravance Biopharma TBPH reported first-quarter 2026 adjusted earnings of 1 cent per share, beating the Zacks Consensus Estimate of breakeven earnings. In the year-ago quarter, the company had incurred an adjusted loss of 17 cents per share. Total revenues in the quarter were $17.7 million, slightly short of the Zacks Consensus Estimate of $18 million. Revenues surged 15% year over year, driven by growth in collaboration revenues for Yupelri sales and improved operating leverage. Year to date, shares of Theravance have declined 9.1% against the industry’s 0.9% growth. Image Source: Zacks Investment Research Theravance’s top line consisted solely of collaboration revenues from partner Viatris VTRS tied to Yupelri (revefenacin) sales in the United States. Theravance and VTRS have collaborated on the development and commercialization of Yupelri, which is approved in the United States for the maintenance treatment of patients with chronic obstructive pulmonary disease. Viatris and Theravance share U.S. profits and losses associated with the commercialization of Yupelri. While Viatris gets 65% of the profits, Theravance receives 35%. Viatris' collaboration revenues include Theravance’s 35% share of Yupelri net sales, as well as its proportionate amount of the total shared costs incurred by the two companies. In March, Theravance and Viatris reached a settlement agreement with Mankind Pharma, granting the company a license to launch a generic version of Yupelri beginning April 23, 2039. Research and development expenses (excluding share-based compensation) totaled $5.2 million, down 49.8% from the year-ago quarter’s level, driven by cost savings from the restructuring announced in March and the ongoing wind-down of the CYPRESS study on its lead candidate, ampreloxetine. Selling, general and administrative expenses (excluding share-based compensation) increased 2.1% year over year to $14.9 million. As of March 31, 2026, Theravance had cash, cash equivalents and marketable securities worth $394.7 million compared with $326.5 million as of Dec. 31, 2025. In early March, Theravance announced disappointing top-line data from the pivotal phase III CYPRESS study, which evaluated its lead pipeline candidate, ampreloxetine, a norepinephrine reuptake inhibitor for the treatment of symptomatic neurogenic orthostatic hypotension in patients with multiple system atrophy, a pr...

Insmed INSM reported a first-quarter 2026 loss of 76 cents per share, which was narrower than the Zacks Consensus Estimate of a loss of 90 cents. In the year-ago quarter, the company posted a loss of $1.42. Quarterly revenues soared 230% year over year to $306 million, entirely from the sales of its two marketed products. Yet, this figure missed the Zacks Consensus Estimate of $308.1 million. Insmed currently has two marketed drugs in its portfolio, Arikayce and Brinsupri. While Arikayce is approved to treat refractory mycobacterium avium complex (MAC) lung disease in adults with limited or no treatment options, Brinsupri is approved for non-cystic fibrosis bronchiectasis (NCFB). Sales of Arikayce rose 6% year over year to $98.1 million, driven by strong growth across ex-U.S. markets. This was the second full quarter in which Insmed generated revenues from Brinsupri sales since its approval in August 2025. The drug contributed $207.9 million to the top line during the quarter, up from $144.6 million in the previous quarter, driven by strong patient uptake. Shares of Insmed declined 23% post the earnings announcement. Though the company’s top line registered significant year-over-year growth, investors were disappointed by the slight miss in consensus sales estimates. Sentiment was further impacted after management disclosed that part of Brinsupri’s strong initial launch demand came from “ready and waiting” (R&W) patients — those who were already aware of the drug before approval and began treatment quickly once it became available. Management estimated that around 3,500 of the 9,000 patient additions in the fourth quarter and about 1,500 of the 7,800 starts in the first quarter came from the R&W group. This raised concerns among a few investors that some early demand may have been pulled forward from future quarters. At the conference call, however, Insmed stated that beginning in the second quarter of 2026, Brinsupri’s growth is expected to be driven primarily by organic demand rather than contributions from the R&W patient pool. Year to date, the stock has lost 40% against the industry’s nil growth. Image Source: Zacks Investment Research During the reported quarter, research and development expenses rose 37% year over year to $209.5 million. This uptick was driven by a rise in employee headcount, resulting in increased compensation and benefit-related exp...

United Therapeutics UTHR reported first-quarter 2026 earnings per share (EPS) of $5.82, which missed the Zacks Consensus Estimate of $6.73. Earnings decreased 12% year over year. United Therapeutics markets four products for pulmonary arterial hypertension (PAH): Tyvaso, Orenitram, Adcirca and Remodulin. It also markets Unituxin for treating pediatric patients with high-risk neuroblastoma. Revenues in the first quarter totaled $781.5 million, which missed the Zacks Consensus Estimate of $800 million. Revenues decreased 2% year over year. A key driver of the company’s top line is Tyvaso products. United Therapeutics markets two versions of Tyvaso: Tyvaso dry powder inhalation (DPI) and nebulized Tyvaso. Both versions are approved for the treatment of PAH and pulmonary hypertension associated with interstitial lung disease (PH-ILD) indications. Combined Tyvaso sales totaled $457.5 million, down 2% year over year due to lower revenues from nebulized Tyvaso. Tyvaso sales fell short of the Zacks Consensus Estimate of $469 million. Tyvaso DPI generated revenues of $330.3 million, climbing 9% year over year due to an increase in patient demand and some pricing benefits. Revenues from nebulized Tyvaso (treprostinil) were $127.2 million, down 22%, largely due to reduced U.S. demand and weaker international sales, despite modest price increases. Sales of Orenitram rose 12% year over year to $135.6 million, primarily driven by higher volumes sold. Remodulin (including Remunity Pump) sales declined 8% year over year to $126.6 million. Unituxin sales were down 8% year over year to $53.6 million. Adcirca sales were $2.9 million, down 52% year over year. Research and development expenses were $138.2 million in the quarter, down 7% year over year, mainly due to lower milestone payments for drug delivery device technologies, partly offset by higher personnel costs. Selling, general and administrative expenses increased 8% to $184.1 million in the quarter. As of March 31, 2026, UTHR had cash, cash equivalents and investments of $3.8 billion compared with $4.6 billion as of Dec. 31, 2025. The company had no debt. United Therapeutics Corporation price-consensus-eps-surprise-chart | United Therapeutics Corporation Quote United Therapeutics’ key phase III programs include Tyvaso in patients with various forms of chronic fibrosing interstitial lung disease (TETON studies) and oral...

Iovance Biotherapeutics IOVA incurred a first-quarter 2026 loss of 19 cents per share, in line with the Zacks Consensus Estimate. In the year-ago quarter, the company reported a loss of 36 cents. Total revenues for the quarter rose 45% year over year to $71.4 million, generated entirely from the sales of the company’s two marketed drugs. The top line missed the Zacks Consensus Estimate of $77.1 million. Iovance currently has two marketed drugs in its portfolio — the IL-2 product Proleukin and the TIL therapy Amtagvi. While Proleukin is approved to treat metastatic renal cell carcinoma and metastatic melanoma in adults, Amtagvi is approved for the advanced melanoma indication. The company recorded approximately $60 million from Amtagvi sales during the quarter, up 38% from the year-ago period. Demand trends improved through the quarter, with management pointing to accelerating referrals and earlier use in the treatment pathway as awareness builds across treatment centers. Yet, the drug’s sales missed the Zacks Consensus Estimate and our model estimate, each pegged at $70 million. Proleukin sales rose 91% to about $11 million during the quarter, benefiting from its use alongside Amtagvi. The figure also missed the Zacks Consensus Estimate and our model estimate, both pegged at $23 million. Shares of Iovance plunged 13% yesterday, likely due to the soft sales performance of both therapies. Still, the stock has rallied 30% so far this year against the industry’s 2% decline. Image Source: Zacks Investment Research Research & development expenses totaled $62.5 million in the quarter, down 12% from the year-ago period, reflecting ongoing operational efficiencies alongside pipeline expansion efforts. Selling, general and administrative expenses declined 11% to about $39 million. Management positioned the cost structure as improving alongside manufacturing centralization and internal efficiency initiatives, aimed at supporting a clearer path to profitability as revenues scale. As of March 31, 2026, Iovance had cash, cash equivalents and investments of $319 million compared with $303 million in the previous quarter. Management now expects its existing cash balance to fund operations into 2028 (previously: third-quarter 2027), driven by ongoing cost discipline alongside revenue growth and improving manufacturing leverage. Iovance discussed its approach to financing on...

Krystal Biotech KRYS reported first-quarter 2026 earnings per share (EPS) of $1.83, which surpassed the Zacks Consensus Estimate of $1.45. The reported EPS was up from $1.20 in the year-ago quarter. Revenues of $116.4 million rose 32% year over year in the reported quarter, beating the Zacks Consensus Estimate of $112 million. Revenues came in solely from Vyjuvek sales. The FDA approved Krystal’s lead drug, Vyjuvek, the first-ever revocable gene therapy, in 2023 for the treatment of patients aged six months or older with dystrophic epidermolysis bullosa (DEB), a rare and severe monogenic disease that affects the skin and mucosal tissues. The drug has also been approved by the FDA for the treatment of DEB patients from birth, with authorization for at-home administration by patients or their caregivers. The company secured more than 695 reimbursement approvals for Vyjuvek in the United States, supporting nationwide access. Internationally, robust patient demand continues to drive steady uptake following the launches in Germany, France and Japan, with more than 140 patients being prescribed the therapy across these markets. Shares of KRYS rose nearly 8% on Monday, likely driven by the better-than-expected earnings results. Year to date, shares of KRYS have risen 16.4% against the industry’s 3.2% decline. Image Source: Zacks Investment Research The top line comprises product revenues from Krystal’s only marketed drug, Vyjuvek. Krystalgenerated $116.4 million in product revenues from Vyjuvek, up from $88.2 million in the year-ago quarter, driven by strong patient uptake. The gross margin in the reported quarter was 95%. Research and development (R&D) expenses were approximately $15.3 million, including stock-based compensation, up 7.5% year over year. Selling, general and administrative (SG&A) expenses totaled approximately $41 million, including stock-based compensation, up 25.6% from the year-ago level. This increase was primarily due to increased headcount, legal and consulting services, and marketing costs to support the global launches of Vyjuvek. As of March 31, 2026, cash, cash equivalents and investments totaled approximately $1 billion compared with $955.9 million as of Dec. 31, 2025. Krystal Biotech reiterated its non-GAAP combined R&D and SG&A expense guidance of $175 million to $195 million for full-year 2026. For Vyjuvek, pricing negotiations with r...

Agios Pharmaceuticals AGIO reported a loss of $1.69 per share for the first quarter of 2026, narrower than the Zacks Consensus Estimate of a loss of $1.81. In the year-ago quarter, the company had incurred a loss of $1.55 per share. Total revenues for the first quarter of 2026 came in at $20.7 million, beating the Zacks Consensus Estimate of $13.8 million. Revenues surged 138% year over year, primarily driven by the U.S. commercial launch of Aqvesme and continued strong growth in Pyrukynd sales. The company’s lead drug, mitapivat, is marketed under two brand names in the United States — Pyrukynd and Aqvesme. While Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency, Aqvesme is approved to treat anemia in adults with alpha- or beta-thalassemia. Aqvesme received approval in the United States in December 2025 and was subsequently launched in January following the implementation of its Risk Evaluation and Mitigation Strategy program. Agios reported a strong initial uptake, with 242 prescriptions written as of March 2026. Shares of AGIO rose 13% on Wednesday, likely due to the better-than-expected sales performance of its marketed products. Year to date, the stock has risen 3.1% against the industry’s 1.4% decline. Image Source: Zacks Investment Research Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications. In March 2026, Pyrukynd received approval for thalassemia in the United Arab Emirates. Meanwhile, a marketing authorization application seeking label expansion for Pyrukynd in the thalassemia indication is currently under review in the EU. The top line entirely comprises product revenues from Pyrukynd and Aqvesme. However, the company did not disclose separate sales figures for the two drugs. Agios generated $18.8 million of product revenues from the sales of both drugs in the United States. The reported figure was up 116% year over year, driven by strong early momentum of the U.S. commercial launch of Aqvesme in thalassemia. The company added $1.9 million from ex-U.S. territories, reflecting demand for Pyrukynd in the thalassemia indication across the Gulf Council Countries. Research & development expenses increased by approximately 11.6% year over year to $81.1 million in the first quarter due to higher costs related to pipeli...

Viking Therapeutics VKTX posted a first-quarter 2026 loss of $1.37 per share, wider than the Zacks Consensus Estimate of a loss of 95 cents. The company had incurred a loss of 41 cents per share in the year-ago quarter. Currently, Viking Therapeutics does not have any approved products in its portfolio. It has yet to generate revenues. Year to date, the stock has lost 11% compared with the industry’s nearly 3% decline. Image Source: Zacks Investment Research Research and development expenses surged to $150.2 million in the first quarter of 2026 from $41.4 million a year ago. Management attributed the increase primarily to higher spending tied to clinical studies, manufacturing for drug candidates, consultants, salaries and benefits, and preclinical work. The stepped-up R&D cadence aligns with the company’s effort to run multiple large studies in parallel, including its ongoing phase III program for subcutaneous (SC) formulation of its investigational obesity drug, VK2735. Viking Therapeutics is also planning to move the oral version of the drug into late-stage development later this year. General and administrative expenses were $14.0 million, compared with $14.1 million in the year-ago quarter. Management cited lower costs related to legal and patent services and stock-based compensation, partially offset by higher spending on consulting, salaries and benefits, and scientific and disease education. Even with G&A largely stable, Viking continues to build operational capability around its obesity opportunity. During the quarter, the company announced the appointment of Neil Aubuchon as its first chief commercial officer, adding commercial leadership as VK2735 advances toward potential registration milestones. Viking ended the quarter with $603.0 million in cash, cash equivalents and short-term investments, down from $706.0 million at the end of 2025. The decline reflects the elevated cost of sustaining late-stage studies and supporting broader development activity across the pipeline. Management addressed the cadence of spending and cash usage. On the earnings call, the company indicated that next quarter’s expense and cash usage could be around the first-quarter level, potentially modestly lower. Expectations are for spending to taper somewhat in the second half of the year as the company continues to manage its balance sheet alongside clinical and operation...