XFOR

Sarepta Therapeutics (SRPT) came out with quarterly earnings of $0.64 per share, beating the Zacks Consensus Estimate of $0.58 per share. This compares to earnings of $2.02 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of +10.35%. A quarter ago, it was expected that this biopharmaceutical company would post earnings of $0.9 per share when it actually produced earnings of $3.16, delivering a surprise of +251.11%. Over the last four quarters, the company has surpassed consensus EPS estimates two times. Sarepta Therapeutics, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $401.25 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 12.85%. This compares to year-ago revenues of $611.09 million. The company has topped consensus revenue estimates four times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Sarepta Therapeutics shares have lost about 23.8% since the beginning of the year versus the S&P 500's gain of 13%. While Sarepta Therapeutics has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Sarepta Therapeutics was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $0.55 on $357.8 million in revenues for the coming quarter and $4.77 on $1.83 billion in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 44% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. X4 Pharmaceuticals (XFOR), another stock in the same industry, has yet to report results for the quarter ended June 2026. This company is expected to post quarterly loss of $0.19 per share in its upcoming report, which represents a year-over-year change of +94.5%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. X4 Pharmaceuticals' revenues are expected to be $2 million, up 1.5% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Sarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis Report X4 Pharmaceuticals, Inc. (XFOR) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

BOSTON, Aug. 06, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today reported financial results for the second quarter ended June 30, 2026 and provided a corporate update. “Since we began leading X4 last August, we have taken meaningful actions to strengthen the execution of the mavorixafor Phase 3 4WARD trial in chronic neutropenia, positioning 4WARD for the successful completion of enrollment,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “Following our complete overhaul of the clinical operating infrastructure and engagement of a top oncology Clinical Research Organization (CRO) to manage the Phase 3 trial, we are now in a position to revisit the sample size of the study with the FDA. We expect to provide an update on our meeting with the FDA and the completion of enrollment by the end of the third quarter.” “Alongside these operational improvements, we continue to actively engage with the medical community to educate physicians on the potential benefits of mavorixafor as an improved treatment option for patients, including a strong presence at this year’s EHA Congress. During the quarter, we also achieved an important milestone with the European Commission’s marketing authorization of XOLREMDI® for WHIM syndrome, supporting the therapeutic potential of mavorixafor across rare hematologic conditions,” concluded, Dr. Craig. Recent Accomplishments and Updates Transitioned management of the Phase 3 4WARD trial to a premier Clinical Research Organization (CRO) to further strengthen the study execution and support completion of enrollment. Advanced enrollment in the global 4WARD Phase 3 trial of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, supported by several targeted initiatives including activation of over 110 active clinical trial sites worldwide, ongoing Medical Affairs engagement, and utilizing data-driven approaches to identify potential participants. Continued to expand awareness in the medical community with a strong presence at the European Hematology Association (EHA) Congress including a publication entitled, “A Pivotal Phase 3 Study To Investigate Efficacy, Safety, and Tolerability of Mavorixafor in Participants with Primary Chronic Neutropenia”. The Medical Affairs team also hosted a booth and held an investigator meeting to engage with physicians regarding the 4WARD trial and the potential benefits of mavorixafor in chronic neutropenia. Received marketing authorization for XOLREMDI® (mavorixafor) by the European Commission for the treatment of patients with WHIM syndrome in the European Union (EU). The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). European commercialization will be led by X4 Pharmaceuticals’ partner, Norgine. In July 2026, the EU marketing authorization was transferred to Norgine. Under the terms of the license and supply agreement, X4 could receive up to an additional €221 million contingent upon the achievement of certain regulatory and commercial milestones, in addition to escalating double-digit royalties of up to the mid-twenties on any future net sales in the licensed territories. First Quarter Financial Results Cash Position: As of June 30, 2026, the Company’s cash position was $208.0 million. Revenue: Revenue was $8.8 million and $11.5 million for the three and six months ended June 30, 2026, respectively. Revenue included net product sales of $2.4 million and $4.8 million for the three and six months ended June 30, 2026, respectively, attributable to XOLREMDI® product sales in the United States. License and other revenue for the three and six month period ended June 30, 2026 included $6.4 million and $6.7 million, respectively, attributable to the Company’s Norgine license and supply agreement. Revenue was $2.0 million and $30.8 million for the three and six months ended June 30, 2025, respectively. Revenue for the six months ended June 30, 2025 included $27.9 million of license revenue attributable to the Norgine license and supply agreement. Research and Development (R&D) expenses: R&D expenses were $15.1 million and $30.5 million for the three and six months ended June 30, 2026 as compared to $18.4 million and $36.9 million for the same periods in prior year. The decrease in both periods is primarily attributable to decreased headcount following the 2025 strategic restructuring, partially offset by higher clinical costs, primarily CRO costs related to our 4WARD trial. General and Administrative (G&A) expenses: G&A expenses were $8.5 million and $15.5 million for the three and six months ended June 30, 2026 as compared to $9.5 million and $24.6 million for the same periods in the prior year. The decrease in both periods is primarily attributable to a reduction in sales and marketing expenses, a reduction in outside legal expenses, and reductions in compensation costs due to lower head count in general and administrative functions. Net Loss: Net loss for the three months ended June 30, 2026 was $16.2 million, or $(0.13) per share, as compared to net loss for the three months ended June 30, 2025 of $25.7 million, or $(3.47) per share. Net loss for the six months ended June 30, 2026 was $36.4 million, or $(0.29) per share, as compared to net loss of $25.5 million, or $(3.59) per share, for the same period in 2025. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a primary, rare blood condition characterized by abnormally low levels of circulating neutrophils in the blood lasting more than three months, persistently or intermittently. As a result, people with chronic neutropenia are at an increased risk of serious and life-threatening infections and reduced quality of life. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Mavorixafor is a small molecule delivered in a capsule for oral dosing as a selective antagonist of the chemokine receptor, CXCR4. Down-regulation of the CXCR4 receptor by mavorixafor has been shown to mobilize functional neutrophils from the bone marrow into the peripheral bloodstream across multiple disease states. The level of circulating neutrophils is typically determined by the absolute neutrophil count (ANC) obtained from a blood draw. About the 4WARD Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 176 patients aged 12 years and older with confirmed trough absolute neutrophil count (ANC) levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoints of the study are the reduction in annualized infection rate and positive ANC response. For more information, visit clinicaltrials.gov (NCT06056297). About X4 Pharmaceuticals X4 Pharmaceuticals is a company focused on improving the lives of people with rare hematology diseases by developing and commercializing innovative therapies in areas with significant unmet needs. Leveraging expertise in diseases of the immune system and CXCR4 biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is commercially available in the U.S. as XOLREMDI® in its first indication. The Company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in chronic neutropenic disorders. The U.S. FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the Company’s ability to obtain and maintain regulatory approval for its product candidates, plans for the commercialization of XOLREMDI in the European Union by Norgine, the potential achievement of milestones and receipt of royalties under the Company’s licensing and supply agreement with Norgine, the expected design and enrollment of the Company’s clinical trials, including expected timing for full enrollment in 4WARD, the sufficiency of the Company’s cash resources and its expected cash runway, and future plans for the Company. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that even if approved, mavorixafor may not ultimately be commercially successful; the Company is unable to initiate and complete its clinical trials, including the 4WARD trial; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its Quarterly Reports on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. Source: X4 Pharmaceuticals, Inc. Balance Sheet Data (unaudited) CONTACT: X4 Investor Contact: Remy Bernarda Jenny Kobin IR Advisory Solutions [email protected]

- Global 4WARD Phase 3 Chronic Neutropenia Trial On Track to Complete Enrollment by the end of Q3 2026 - - European Commission Approval of XOLREMDI® (Mavorixafor) Provides the First and Only Authorized Treatment for Patients with WHIM Syndrome in the European Union - - Balance Sheet Provides Cash Runway through 2028 - BOSTON, May 06, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today reported financial results for the first quarter ended March 31, 2026 and provided a corporate update. “Our top priority remains the execution and full enrollment of the pivotal 4WARD Phase 3 trial of mavorixafor in chronic neutropenia,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “We remain on track to complete enrollment by the end of the third quarter of this year. In addition, we are pleased that the European Commission granted marketing authorization for XOLREMDI® (mavorixafor) for the treatment of patients with WHIM syndrome in the European Union, reinforcing the broader potential of our program to advance therapies for individuals affected by rare hematologic disorders. We look forward to a smooth transfer of the marketing authorization application to our European partner, Norgine, later this year as they proceed with commercialization plans throughout Europe.” Recent Accomplishments and Updates Continued to advance enrollment in the global 4WARD Phase 3 trial of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, supported by several targeted initiatives: Expanded to over 110 active clinical trial sites worldwide, including more than 20 in the U.S., enhancing enrollment and regulatory alignment; Increased global Medical Affairs engagement including the implementation of a dedicated patient referral pathway for physicians; and, Leveraging data-driven approaches including database mining to identify potential participants. The European Commission granted marketing authorization for XOLREMDI® (mavorixafor) for the treatment of patients with WHIM syndrome in the European Union (EU). The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). European commercialization will be led by X4 Pharmaceuticals’ partner, Norgine. Under the terms of the license and supply agreement, X4 will receive up to €226 million contingent upon the achievement of certain regulatory and commercial milestones, in addition to escalating double-digit royalties of up to the mid-twenties on any future net sales in the licensed territories. First Quarter Financial Results Cash Position: As of March 31, 2026, the Company’s cash position was $233.7 million. Revenue: Revenue was $2.7 million and $28.8 million for the three months ended March 31, 2026 and 2025, respectively. Net product sales in both periods were primarily attributable to XOLREMDI product sales in the United States. Revenue for the three months ended March 31, 2025 included $27.9 million of license revenue attributable to the Norgine license and supply agreement. Research and Development (R&D) expenses: R&D Expenses were $15.5 million for the three months ended March 31, 2026 as compared to $18.5 million for the three months ended March 31, 2025. The decrease is primarily attributable to decreased headcount following the 2025 strategic restructuring. General and Administrative (G&A) expenses: G&A Expenses were $7.0 million for the three months ended March 31, 2026 as compared to $15.0 million for the three months ended March 31, 2025. The decrease is primarily attributable to decreased headcount following the 2025 strategic restructuring. Net (Loss) Income: Net loss for the three months ended March 31, 2026 was $(20.2) million, or $(0.16) per share, as compared to net income for the three months ended March 31, 2025 of $0.3 million or $0.04 per share. The difference was due to the Norgine license revenue recognized in the first quarter of 2025, partially offset by a significant reduction in operating expenses. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a primary, rare blood condition characterized by abnormally low levels of circulating neutrophils in the blood lasting more than three months, persistently or intermittently. As a result, people with chronic neutropenia are at an increased risk of serious and life-threatening infections and reduced quality of life. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Mavorixafor is a small molecule delivered in a capsule for oral dosing as a selective antagonist of the chemokine receptor, CXCR4. Down-regulation of the CXCR4 receptor by mavorixafor has been shown to mobilize functional neutrophils from the bone marrow into the peripheral bloodstream across multiple disease states. The level of circulating neutrophils is typically determined by the absolute neutrophil count (ANC) obtained from a blood draw. About the 4WARD Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 176 patients aged 12 years and older with confirmed trough absolute neutrophil count (ANC) levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoints of the study are the reduction in annualized infection rate and positive ANC response. For more information, visit clinicaltrials.gov (NCT06056297). About X4 Pharmaceuticals X4 Pharmaceuticals is a company focused on improving the lives of people with rare hematology diseases by developing and commercializing innovative therapies in areas with significant unmet needs. Leveraging expertise in diseases of the immune system and CXCR4 biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is commercially available in the U.S. as XOLREMDI® in its first indication. The Company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in chronic neutropenic disorders. The U.S. FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the Company’s ability to obtain and maintain regulatory approval for its product candidates, plans for the commercialization of XOLREMDI in the European Union by Norgine, the potential achievement of milestones and receipt of royalties under the Company’s licensing and supply agreement with Norgine, the expected design and enrollment of the Company’s clinical trials, including expected timing for full enrollment in 4WARD, the sufficiency of the Company’s cash resources and its expected cash runway, and future plans for the Company. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that even if approved, mavorixafor may not ultimately be commercially successful; the Company is unable to initiate and complete its clinical trials, including the 4WARD trial; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its Quarterly Reports on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. X4 Investor Contact: Remy Bernarda Jenny Kobin IR Advisory Solutions [email protected] Source: X4 Pharmaceuticals, Inc. (Tables Follow)

- Global 4WARD Phase 3 Chronic Neutropenia Trial On Track to Complete Enrollment in Q3 2026 - - Positive Opinion from European Medicines Agency Recommending Marketing Authorization of Mavorixafor for the Treatment of WHIM Syndrome in the European Union - - Balance Sheet Provides Cash Runway through 2028 - BOSTON, March 17, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today reported financial results for the fourth quarter and year ended December 31, 2025 and provided a corporate update. “Our focus remains on our pivotal 4WARD trial in chronic neutropenia as we execute our strategy to deliver life-changing therapies to people living with rare blood diseases,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “We have sharpened our operational focus and implemented targeted initiatives to drive continued progress in 4WARD with enrollment on track for completion in the third quarter of this year. Additionally, we were pleased to receive the positive opinion from the European Medicines Agency recommending the potential European approval of mavorixafor in WHIM syndrome, which would be the first and only therapy in Europe to treat this devastating condition.” Recent Accomplishments and Updates Deployed a series of proactive measures to increase enrollment in the global 4WARD Phase 3 trial of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, including: Expanding the number of current active clinical trial sites to over 100, including approximately 20 in the U.S.; Enhancing our global Medical Affairs activities to increase Medical Science Liaison (MSL) field engagement and site interaction to educate physicians on our 4WARD trial and mavorixafor’s potential in treating chronic neutropenia; Establishing a patient referral pathway for physicians to assist them in finding trial sites for their chronic neutropenia patients; and Investing in database mining to identify potential participants. European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization of mavorixafor the treatment of WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome in the European Union (EU). The positive opinion will now be reviewed by the European Commission (EC) with a final approval decision anticipated in the second quarter of 2026. Cash runway through 2028 due to recent equity financings and operating expense reductions. Fourth Quarter and Full Year Financial Results Net product sales of $2.3 million and $6.5 million for the three and twelve months ended December 31, 2025, respectively, were entirely attributable to XOLREMDI product sales in the United States. License and other revenue of $0.3 million and $28.6 million for the three and twelve months ended December 31, 2025, respectively, were entirely attributable to the Company's Norgine out-licensing agreement. Operating loss was $23.7 million and $86.9 million for the three and twelve months ended December 31, 2025, respectively. Net loss for the three and twelve months ended December 31, 2025 was $23.9 million, or $(0.22) per share, and $79.2 million, or $(1.87) per share, respectively. Cash, cash equivalents and short-term investments totaled $253.0 million as of December 31, 2025, which management believes will enable the Company to fund its operations through 2028. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a primary, rare blood condition characterized by abnormally low levels of circulating neutrophils in the blood lasting more than three months, persistently or intermittently. As a result, people with chronic neutropenia are at an increased risk of serious and life-threatening infections and reduced quality of life. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Mavorixafor is a small molecule delivered in a capsule for oral dosing as a selective antagonist of the chemokine receptor, CXCR4. Down-regulation of the CXCR4 receptor by mavorixafor has been shown to mobilize functional neutrophils from the bone marrow into the peripheral bloodstream across multiple disease states. The level of circulating neutrophils is typically determined by the absolute neutrophil count (ANC) obtained from a blood draw. About the 4WARD Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 176 patients aged 12 years and older with confirmed trough absolute neutrophil count (ANC) levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoints of the study are the reduction in annualized infection rate and positive ANC response. For more information, visit clinicaltrials.gov (NCT06056297). About X4 Pharmaceuticals X4 Pharmaceuticals is a company focused on improving the lives of people with rare hematology diseases by developing and commercializing innovative therapies in areas with significant unmet needs. Leveraging expertise in diseases of the immune system and CXCR4 biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is commercially available in the U.S. as XOLREMDI® in its first indication. The Company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in chronic neutropenic disorders. The U.S. FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the Company’s ability to obtain and maintain regulatory approval for its product candidates, including expected timing of the EC’s approval decision regarding mavorixafor for the treatment of WHIM in the EU, the expected design and enrollment of the Company’s clinical trials, including expected timing for full enrollment in 4WARD, the sufficiency of the Company’s cash resources and its expected cash runway, and future plans for the Company. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: the Company is unable to obtain or maintain regulatory approval for its product candidates, including for mavorixafor for the treatment of WHIM in the European Union; the Company is unable to initiate and complete its clinical trials, including the 4WARD trial; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its quarterly reports on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. X4 Investor Contact: Remy Bernarda Jenny Kobin IR Advisory Solutions [email protected] Source: X4 Pharmaceuticals, Inc. (Tables Follow) Balance Sheet Data (unaudited)

Vericel Corporation (VCEL) came out with quarterly earnings of $0.45 per share, in line with the Zacks Consensus Estimate . This compares to earnings of $0.38 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of +0.74%. A quarter ago, it was expected that this company would post a loss of $0.02 per share when it actually produced earnings of $0.1, delivering a surprise of +600%. Over the last four quarters, the company has surpassed consensus EPS estimates three times. Vericel, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $92.92 million for the quarter ended December 2025, surpassing the Zacks Consensus Estimate by 1.83%. This compares to year-ago revenues of $75.38 million. The company has topped consensus revenue estimates two times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Vericel shares have added about 3% since the beginning of the year versus the S&P 500's gain of 1.5%. While Vericel has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Vericel was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.02 on $65.35 million in revenues for the coming quarter and $0.60 on $326.28 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 36% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. One other stock from the same industry, X4 Pharmaceuticals (XFOR), is yet to report results for the quarter ended December 2025. This company is expected to post quarterly loss of $0.36 per share in its upcoming report, which represents a year-over-year change of +94%. The consensus EPS estimate for the quarter has been revised 2.8% higher over the last 30 days to the current level. X4 Pharmaceuticals' revenues are expected to be $1.68 million, up 17.2% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Vericel Corporation (VCEL) : Free Stock Analysis Report X4 Pharmaceuticals, Inc. (XFOR) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Company’s strategic focus and highest priority is now the advancement of the 4WARD Phase 3 chronic neutropenia trial Previously announced workforce reductions resulted in $13M annualized cost savings Successful completion of two financial transactions totaling $240.3M; cash runway now extends to the end of 2028 BOSTON, Nov. 05, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare hematology diseases, today reported financial results for the third quarter ended September 30, 2025 and provided a corporate update. “The third quarter of 2025 was a time of corporate restructuring at X4 with the start of a new leadership team and a renewed focus on chronic neutropenia,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “With a strengthened financial position through two successful financings totaling $240.3 million, our primary focus is now on the completion of the 4WARD Phase 3 pivotal trial of mavorixafor in patients with moderate and severe chronic neutropenia, which has a potential addressable market of 15,000 patients in the US. With a cash runway to the end of 2028, we are now positioned to unlock mavorixafor’s full potential and to establish X4 as a world-class rare hematology company.” Recent Accomplishments and Updates Since early August, the Company has initiated a number of measures to restructure its operations: A shift in the primary focus of the Company to the successful completion of the 4WARD Phase 3 pivotal trial of mavorixafor in patients with moderate and severe chronic neutropenia. A deprioritization of the commercialization of mavorixafor (XOLREMDI) for patients with WHIM syndrome, while maintaining patient access. A 50% reduction in the workforce (expected to generate approximately $13 million in annualized cost savings) with continued cost cutting measures. An increase in the enrollment target for the pivotal Phase 3 4WARD study to 176 patients with enrollment now expected to be completed in third quarter of 2026. The promotion of John Volpone to the role of Chief Operating Officer, in addition to his responsibilities as President. Dr. Adam Craig expanded his role to include oversight of clinical development activities. Since August, the Company raised $240.3 million in gross proceeds from two successful financings: the closing of a $155.3 million underwritten public offering and an $85 million upsized private placement. With a strengthened cash runway to the end of 2028, X4 is now expected to be able to complete the 4WARD trial, file a potential sNDA for the chronic neutropenia indication, and, if successful, launch mavorixafor in this new indication by the end of 2028. Third-Quarter and Recent 2025 Financial Results Net product sales of $1.6 million and $4.3 million for the three and nine months ended September 30, 2025, respectively, were entirely attributable to XOLREMDI product sales in the United States. Net product sales were $0.6 million and $1.1 million for the three and nine months ended September 30, 2024, respectively. License and other revenue of $0.2 million and $28.3 million for the three and nine months ended September 30, 2025, respectively, were entirely attributable to the Company’s Norgine out-licensing agreement. Operating loss was $27.5 million and $34.5 million for the three months ended September 30, 2025, and 2024, respectively, and $63.2 million and $0.7 million for the nine months ended September 30, 2025, and 2024, respectively. The decrease in operating loss between the three-month periods ended September 30, 2025, and 2024 was primarily attributable to the impact of our 2025 Strategic Restructuring activities. Operating expenses for the nine months ended September 30, 2024, are net of a gain of $105.0 million realized on the sale of a priority review voucher. Exclusive of this gain, the decrease in operating loss between the nine-month periods ended September 30, 2025, and 2024 was primarily attributable to the impact of the Company’s strategic restructuring activities undertaken in 2025. Net loss for the three months ended September 30, 2025 was $29.8 million, or $0.69 for basic and diluted loss per share, compared to net loss of $36.7 million, or $5.48 for basic and diluted loss per share, for the same period in 2024. Net loss for the nine months ended September 30, 2025 was $55.3 million, or $2.87 for basic and diluted loss per share, compared to net income of $2.4 million, or $0.35 for basic and diluted income per share, for the same period in 2024. Cash, cash equivalents and short-term investments totaled $122.2 million for the period ended September 30, 2025. On October 27, 2025, the Company completed a public offering with net proceeds of $145.6 million, which management believes will enable the Company to fund its operations into the end of 2028. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a primary, rare blood condition lasting more than three months, persistently or intermittently, and characterized by low levels of circulating neutrophils and increased risk of serious and life-threatening infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize functional neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC). About the 4WARD Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 176 patients with confirmed trough ANC levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoints of the study are the reduction in annualized infection rate and positive ANC response. About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare hematology diseases and significant unmet needs. Leveraging expertise in CXCR4, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial (4WARD) in patients with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the expected cost savings from the workforce reduction, the expected results of the restructuring of business operations, the sufficiency of the Company’s cash resources and its expected cash runway, the potential addressable market for moderate and severe chronic neutropenia, the timing for launch of mavorixafor and future plans for the Company. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: the workforce reduction does not result in the anticipated cost savings described herein; the results of the restructuring of business operations are not as anticipated; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its quarterly report on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. X4 Investor Contact: [email protected] Balance Sheet Data (unaudited)