PureTech Presents New Data from Phase 2b Open-Label Extension Study of Deupirfenidone (LYT-100), Further Supporting Strong and Durable Efficacy and Potential to Serve as New Standard of Care in IPF




PureTech Presents New Data from Phase 2b Open-Label Extension Study of Deupirfenidone (LYT-100), Further Supporting Strong and Durable Efficacy and Potential to Serve as New Standard of Care in IPF

Patients who switched from placebo or pirfenidone to deupirfenidone in the open-label extension study achieved stabilization of lung function with favorable tolerability

Findings further substantiate safety and efficacy results from the randomized, placebo- and active-controlled 26-week trial and highlight opportunity to address patients inadequately served by current therapies

Regulatory engagement underway; update on Phase 3 trial design expected in Q4 2025

BOSTON–(BUSINESS WIRE)–PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced new data from the open-label extension (OLE) of its Phase 2b ELEVATE IPF trial of deupirfenidone (LYT-100) in people living with idiopathic pulmonary fibrosis (IPF). These new results showed that participants who completed 26 weeks of placebo or pirfenidone treatment in the randomized portion of the trial and then switched to deupirfenidone for an additional 26 weeks in the OLE achieved stabilization of lung function. These findings, delivered in a late-breaking oral presentation at the 2025 European Respiratory Society (ERS) Congress in Amsterdam, Netherlands, highlight the potential for deupirfenidone to become a new standard of care for the treatment of IPF.

“The blinded portion of the ELEVATE trial challenged the perspective that the biggest opportunity for new therapies in IPF is improved safety, by showing that treatment with deupirfenidone 825 mg three times a day can achieve lung function stabilization with favorable tolerability. The initial 52-week extension data then raised the bar by demonstrating that this effect with deupirfenidone was durable,” said Argyrios E. Tzouvelekis, M.D., Ph.D., University of Patras, Greece, and presenting investigator at ERS 2025. “Now we are seeing that two additional patient cohorts who experienced lung function decline in Part A of the trial achieved stabilization once switched to deupirfenidone. These findings reinforce that the blinded results with deupirfenidone are reproducible and support the potential for benefit in patients transitioning from standard of care.”

ELEVATE IPF, a global, randomized, double-blind, active- and placebo-controlled Phase 2b trial, achieved its primary endpoint and demonstrated a statistically significant and clinically meaningful reduction in lung function decline at 26 weeks with deupirfenidone 825 mg three times a day (TID) compared to placebo (Part A). As previously announced, participants treated with deupirfenidone 825 mg TID experienced a slower rate of lung function decline, as measured by change from baseline of Forced Vital Capacity (FVC), at 26 weeks versus those who were treated with pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks. Following the completion of the blinded portion of the trial, 170 participants (more than 90%) enrolled in the OLE (Part B). Those who remained on deupirfenidone 825 mg TID maintained a robust treatment effect and experienced an overall FVC decline of -32.8 mL over the 52-week period,¹ which is similar to the expected natural decline in lung function in healthy older adults over that time (approximately -30.0 mL to -50.0 mL).²

The new results presented at ERS provide additional evidence from participants who initially received placebo or pirfenidone for 26 weeks during Part A and then switched to deupirfenidone for 26 weeks in Part B. Those who switched from placebo to deupirfenidone 825 mg TID (n=17) had a mean change in FVC of +20.0 mL (placebo switch cohort), while those who switched from pirfenidone to deupirfenidone 825 mg TID (n=16) had a mean change in FVC of -23.1 mL (pirfenidone switch cohort).³ These results provide further evidence from two additional patient cohorts that deupirfenidone may stabilize the decline in lung function to that expected of healthy older adults and suggest a potential benefit for patients transitioning from standard of care to deupirfenidone.

Deupirfenidone continued to be well tolerated at both doses studied in Part B after six months of treatment, and the safety profile remained consistent with Part A. Additional analyses shared at ERS included a summary of the most common treatment-emergent adverse events (TEAEs) in the OLE, defined as occurring in at least 10% of participants in either treatment group. As of May 9, 2025, the most common TEAEs were nausea (8.4% vs. 11.5%), dyspepsia (14.5% vs. 12.6%), upper respiratory infections (16.9% vs. 17.2%), and cough (10.8% vs. 4.6%) for deupirfenidone 550 mg TID (n=83) and deupirfenidone 825 mg TID (n=87), respectively.

“The ELEVATE trial has been designed and executed to provide one of the most rigorous Phase 2 evaluations of a potential therapy in IPF,” said Sven Dethlefs, Ph.D., Chief Executive Officer of Celea Therapeutics, the PureTech Founded Entity created to advance deupirfenidone. “The reproducibility we’re seeing across blinded and extension data, and now in switch cohorts, gives us strong confidence in the robustness of the findings. We believe deupirfenidone has the potential to become a new standard of care in IPF, and we are actively engaging with regulators to finalize the Phase 3 trial design and expect to share an update in the fourth quarter.”

About Deupirfenidone (LYT-100)

Deupirfenidone (LYT-100) is in development as a potential new standard of care for the treatment of idiopathic pulmonary fibrosis (IPF). It is a deuterated form of pirfenidone, which – along with nintedanib – is one of the two FDA-approved treatments for IPF. Both approved therapies offer only modest efficacy in slowing lung function decline, largely due to tolerability challenges that limit the ability to achieve higher doses that could significantly improve patient outcomes. These limitations have contributed to low treatment uptake and poor adherence, with approximately 25% of people with IPF in the U.S. ever receiving either drug.⁴ Despite this, combined peak global sales exceed $5 billion, representing a significant market opportunity in IPF and other fibrotic lung diseases. ⁵

Deupirfenidone may overcome these limitations. In the global Phase 2b ELEVATE IPF trial, deupirfenidone demonstrated the potential to stabilize lung function decline over at least 26 weeks as a monotherapy while maintaining a favorable safety and tolerability profile. Initial data from an ongoing open-label extension study suggest that this effect may be sustained through at least 52 weeks. These findings support the potential for deupirfenidone to offer a meaningful advance for people living with this progressive and deadly disease. Beyond IPF, deupirfenidone may also address multiple underserved fibrotic conditions, including progressive fibrosing interstitial lung diseases.

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue that leads to a steady decline in lung function. Median survival following diagnosis is estimated to be two to five years, and currently there is no cure.⁶

About Celea Therapeutics

Celea Therapeutics is dedicated to advancing transformative treatments for people with serious respiratory diseases. Drawn from the Latin word for “sky,” the name reflects the company’s mission to rise above the status quo and deliver therapies that change lives. The company’s lead program, deupirfenidone (LYT-100), is a Phase 3-ready therapeutic candidate with the potential to set a new standard of care for idiopathic pulmonary fibrosis (IPF) and other fibrotic lung diseases.

Celea was founded by PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a biotherapeutics company dedicated to giving life to science. PureTech’s innovative R&D model drives the creation of Founded Entities like Celea, enabling the advancement of highly promising medicines to patients in a capital-efficient manner. For more information, please visit www.celeatx.com and www.puretechhealth.com.

About PureTech Health

PureTech Health is a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value. We do this through a proven, capital-efficient R&D model focused on opportunities with validated pharmacology and untapped potential to address significant patient needs. This strategy has produced dozens of therapeutic candidates, including three that have received U.S. FDA approval. By identifying, shaping, and de-risking these high-conviction assets, and scaling them through dedicated structures backed by external capital, we accelerate their path to patients while creating sustainable value for shareholders.

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

Cautionary Note Regarding Forward-Looking Statements

This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements that relate to continued development of and regulatory interactions related to deupirfenidone, the potential of deupirfenidone in IPF and other indications, our expectations around our therapeutic candidates and approach towards addressing major diseases, our plans to advance our programs and deliver on our milestones, our future plans, prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption “Risk Factors” in our Annual Report on Form 20-F for the year ended December 31, 2024 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

PureTech
Public Relations

publicrelations@puretechhealth.com
Investor Relations

IR@puretechhealth.com

UK/EU Media
Ben Atwell, Rob Winder

+44 (0) 20 3727 1000

puretech@fticonsulting.com

US Media
Justin Chen

jchen@tenbridgecommunications.com

Savara Presents New Data From the Phase 3 IMPALA-2 Clinical Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at the European Respiratory Society (ERS) Congress 2025




Savara Presents New Data From the Phase 3 IMPALA-2 Clinical Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at the European Respiratory Society (ERS) Congress 2025

– The Beneficial Clinical Effects of Molgramostim Were Similar in Patients with aPAP Regardless of Disease Severity Defined by DLco% –

– Changes in DLco%, the Primary Endpoint in IMPALA-2, Were Significantly Correlated with Changes in Measures of Quality of Life, Patient Functionality, and Surfactant Burden in Patients with aPAP –

– Savara’s Partner, TrilliumBiO, Presented Data on the Development of a Dried Blood Spot Test to Aid in the Diagnosis of aPAP –

LANGHORNE, Pa.–(BUSINESS WIRE)–Savara Inc. (the “Company”) (Nasdaq: SVRA), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced additional analyses from its pivotal Phase 3 IMPALA-2 clinical trial of molgramostim in aPAP were presented as poster presentations at the ERS Congress 2025 in Amsterdam, The Netherlands.

ERS 2025 Posters

Savara

Title: Efficacy of Inhaled Molgramostim According to Severity of Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Presenter: Cormac McCarthy, M.D., Ph.D., FRCPI, Associate Professor of Medicine at the University College Dublin (UCD) and Consultant Respiratory Physician, St. Vincent’s University Hospital, Dublin, Ireland

Poster Number: PA3026

Poster Summary:

• Prespecified analyses were conducted to determine if the beneficial clinical effects of molgramostim in IMPALA-2, a Phase 3 clinical trial, were similar in patients with aPAP based on their disease severity as measured by hemoglobin-adjusted percent predicted diffusing capacity of the lungs for carbon monoxide, or DLco% (≤50% or >50%) at randomization
• Molgramostim demonstrated improvement compared with placebo on the primary endpoint, change in DLco% from baseline to Week 24, in patients with aPAP regardless of disease severity
• Molgramostim significantly improved measures of pulmonary gas transfer (DLco%), respiratory health-related quality of life (St. George’s Respiratory Questionnaire [SGRQ] Total and Activity scores), and patient functionality (exercise capacity expressed as peak metabolic equivalents [METs]) compared with placebo in both subgroups of patients with DLco% values of ≤50% or >50% at randomization

Title: Relationship Between Pulmonary Gas Transfer, Respiratory Health-related Quality of Life (HRQoL), Exercise Capacity, and Surfactant Burden in Autoimmune Pulmonary Alveolar Proteinosis (PAP)

Presenter: Francesco Bonella, M.D., Ph.D., Head of the Center for Interstitial and Rare Lung Diseases and Assistant Professor, Ruhrlandklinik University Hospital, Essen, Germany

Poster Number: PA3027

Poster Summary:

• DLco% (a measure of pulmonary gas transfer) was chosen as the primary endpoint in the IMPALA-2 Phase 3 clinical trial because it is a standardized measure of pulmonary gas transfer widely used in clinical practice
• Previous research has shown that changes in DLco% correlate with changes in aPAP disease severity (i.e., surfactant accumulation/burden) and may predict the need for whole-lung lavage
• Post-hoc correlation analyses of IMPALA-2 data were conducted to evaluate the relationship between DLco% and measures of HRQoL, patient functionality, and surfactant burden in patients with aPAP
• Significant negative correlations were observed between DLco% and measures of respiratory HRQoL (SGRQ Total and Activity scores; higher scores indicate worse quality of life), as well as DLco% and surfactant accumulation (ground-glass opacity scores; lower scores indicate less surfactant) at Week 24. Significant positive correlations were observed between DLco% and patient functionality (exercise capacity expressed as peak METs; higher scores indicate improved exercise capacity) at Weeks 24 and 48
• Results further support the clinical relevance of changes in DLco% in aPAP; changes in DLco% are associated with changes in clinical outcomes and the extent of pulmonary pathology, making it a clinically meaningful measure in this rare lung disease

TrilliumBiO

Title: Development of a Dried Blood Spot Assay for the Detection of GM-CSF Autoantibodies to Aid in the Diagnosis of Autoimmune Pulmonary Alveolar Proteinosis

Presenter: Eagappanath Thiruppathi, Ph.D., Director of Test and Method Development, TrilliumBiO and Joannah Kim, Vice President of Development and Operations, TrilliumBiO

Poster Number: PA3025

Poster Summary:

• The study was conducted to develop and validate a novel particle-based flow cytometry assay using dried blood spot (DBS) samples for detection of quantitative GM-CSF autoantibody levels to aid in the accurate and timely diagnosis of aPAP
• A strong linear correlation was observed between GM-CSF autoantibody levels measured in serum from venipuncture and those measured using serum-derived dried blood spot samples
• The novel assay demonstrated high precision and sensitivity for the detection of GM-CSF autoantibodies in dried blood spot samples. This approach offers a practical and convenient diagnostic tool to help confirm or rule-out aPAP, representing a significant advancement in the detection of this rare and serious lung disease

For more details about the ERS Congress please visit their website.

The posters are available on the Congresses & Publications page of the Company’s corporate website.

About the IMPALA-2 Trial

IMPALA-2 is a global, pivotal, Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg self-administered once daily by inhalation with matching placebo in patients with aPAP. The trial is being conducted at 43 clinical trial sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and countries in Europe, including Turkey. The primary efficacy assessment was diffusing capacity of the lungs for carbon monoxide (DLco%), a gas transfer measure, and the primary endpoint was change from baseline to Week 24 in percent predicted DLco%, with a secondary endpoint of change from baseline to Week 48 in percent predicted DLco%. Three additional secondary efficacy variables evaluated clinical measures of direct patient benefit: St. George’s Respiratory Questionnaire (SGRQ) Total score, SGRQ Activity score, and exercise capacity using a treadmill test, with each endpoint measured at Weeks 24 and 48. The primary time point for efficacy assessments was at Week 24; however, efficacy was assessed through Week 48 to evaluate durability of effect. Safety was assessed through Week 48. All patients who completed the 48-week double-blind treatment period continued into a 96-week open-label period during which molgramostim 300 mcg is administered once daily.

About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas transfer, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant.

About Savara

Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via a proprietary investigational eFlow^® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of molgramostim. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com and LinkedIn.

Contacts

Media and Investor Relations Contact

Savara Inc.

Temre Johnson, Executive Director, Corporate Affairs

ir@savarapharma.com

Genmab to Acquire Merus, Expanding Late-Stage Pipeline and Accelerating into a Wholly Owned Model




Genmab to Acquire Merus, Expanding Late-Stage Pipeline and Accelerating into a Wholly Owned Model

Company Announcement

• Genmab to acquire Merus for USD 97.00 per share in an all-cash transaction representing a transaction value of approximately USD 8.0 billion
• Proposed acquisition adds petosemtamab, a late-stage asset with two Breakthrough Therapy Designations, to Genmab’s portfolio
• Transaction anticipated to be accretive to EBITDA by end of 2029
• Genmab to host a conference call today at 1:00 PM CEST / 12:00 PM BST / 7:00 AM EDT

COPENHAGEN, Denmark & UTRECHT, Netherlands–(BUSINESS WIRE)–Genmab A/S (Nasdaq: GMAB) and Merus N.V. (Nasdaq: MRUS) announced today that they have entered into a transaction agreement pursuant to which Genmab intends to acquire all the shares of Merus, a clinical-stage biotechnology company with its late-stage breakthrough therapy asset petosemtamab, which is in Phase 3 development, for USD 97.00 per share in an all-cash transaction representing a transaction value of approximately USD 8.0 billion. The transaction has been unanimously approved by the Boards of Directors of both companies. A wholly owned subsidiary of Genmab (“Purchaser”) will commence a tender offer for 100% of Merus’ common shares, which is anticipated to close by early in the first quarter of 2026.

The proposed acquisition of Merus is expected to meaningfully accelerate Genmab’s shift to a wholly owned model, expanding and diversifying the company’s revenue, driving sustained growth into the next decade and contributing to Genmab’s evolution into a biotechnology leader. The addition of petosemtamab, Merus’ lead asset, to Genmab’s promising late-stage pipeline is a compelling strategic fit with Genmab’s portfolio and aligns with Genmab’s expertise in antibody therapy development and commercialization in oncology. Following the closing of the transaction, Genmab will have four proprietary programs expected to drive multiple new drug launches by 2027.

“The proposed acquisition of Merus clearly aligns with our long-term strategy. It has the potential to significantly accelerate our evolution into a global biotechnology leader by providing durable growth for the company well into the next decade,” said Jan van de Winkel, Ph.D., President and Chief Executive Officer of Genmab. “Petosemtamab has the potential to be a transformational therapy for patients living with head and neck cancer. With our proven track record of success, both in clinical development and in commercialization, we are confident that we will be able to unlock the promise of petosemtamab.”

“We are excited for the opportunity to join Genmab, a leader in antibody therapeutics, to further develop and bring petosemtamab to patients. Our two companies have a rich history of innovation with multiple approvals in the field of multispecific antibodies. We believe Genmab has the right vision and experience to advance petosemtamab in recurrent/metastatic head and neck cancer and beyond,” said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. “I’m immensely proud of the Merus team who have pioneered our foundational platform technologies to make better medicines and who have demonstrated – with an approved product and a product candidate, petosemtamab, in registrational studies – an ability to deliver on our promise to close in on cancer.”

Petosemtamab is an EGFRxLGR5 bispecific antibody with the potential to be both first- and best-in-class in head and neck cancer. It has been granted two Breakthrough Therapy Designations (BTD) by the U.S. Food and Drug Administration (FDA) for first- and second-line plus head and neck cancer indications. Of note, compelling Phase 2 data was presented at the American Society for Clinical Oncology (ASCO) 2025 Annual Meeting showing both an overall response rate and median progression free survival that were substantially higher than standard of care.

Merus is currently running two Phase 3 trials in first- and second/third line head and neck cancer, with topline interim readout of one or both trials anticipated in 2026. Based on Genmab’s experience in late-stage development and excellence in commercial execution, Genmab anticipates the potential for the initial launch of petosemtamab in 2027, subject to clinical results and regulatory approvals. Genmab also intends to broaden and accelerate petosemtamab’s development with potential expansion into earlier lines of therapy. Following its initial anticipated approval, Genmab believes that petosemtamab will be accretive to EBITDA with at least one-billion-dollar annual sales potential by 2029, with multi-billion-dollar annual revenue potential thereafter.

Details of the Transaction and Financing

Under the transaction agreement, Purchaser, a wholly owned subsidiary of Genmab, will commence a tender offer for all the outstanding common shares of Merus. Following the closing of the tender offer, Merus and Genmab will effect a series of transactions resulting in Genmab owning 100% of the common shares of Merus (or a successor entity). Depending on the structure of the back end transactions, Merus shareholders that do not tender their shares into the tender offer will either receive the same consideration for their common shares as the common shares tendered into the tender offer (subject to applicable withholding taxes) or a fair price for their common shares determined by a Dutch court in statutory buy-out proceedings. The closing of the tender offer is subject to the satisfaction of customary closing conditions for similar transactions, including a minimum acceptance condition of at least 80% of Merus’ common shares (which threshold may be reduced to 75% unilaterally by Genmab if all other closing conditions are satisfied), approval by Merus’ shareholders of resolutions relating to Merus’ post-closing governance and the back end transactions at Merus’ extraordinary shareholders meeting to be held for that purpose, and completion of the relevant works councils consultation processes.

The USD 97.00 per common share purchase price payable in the tender offer represents a premium of approximately 41% over Merus’ closing stock price on September 26, 2025, of USD 68.89 and approximately 44% over Merus’ 30-day volume weighted average price of USD 67.42.

The transaction is not subject to a financing condition. Consideration is expected to be funded through a combination of cash on hand and approximately $5.5 billion of non-convertible debt financing. Genmab has obtained a funding commitment from Morgan Stanley Senior Funding, Inc. for this amount.

The financing package includes a meaningful portion of prepayable debt, in line with Genmab’s commitment to deleveraging with a target of gross leverage <3x within two years after the closing of the proposed transaction. Today’s news does not impact Genmab’s financial guidance for the full year 2025, last issued on August 7, 2025. Genmab will provide its financial outlook for the full year 2026 in conjunction with its full year 2025 earnings report in February 2026.

PJT Partners and Morgan Stanley & Co. International plc are acting as joint financial advisors to Genmab and A&O Shearman and Kromann Reumert as its legal advisors.

Jefferies LLC is acting as financial advisor to Merus and Latham & Watkins and NautaDutilh as its legal advisors.

Conference Call Details

Genmab will hold a conference call to discuss the transaction today, September 29, 2025 at 1:00 PM CEST / 12:00 PM BST / 7:00 AM EDT. To join the call please use the following registration link https://register-conf.media-server.com/register/BI65be2c038b9b42dbb064dfc843b6a478. Registered participants will receive an email with a link to access dial-in information as well as a unique personal PIN. A live and archived webcast of the calls and relevant slides will be available at https://www.genmab.com/investor-relations.

About Genmab

Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab’s vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines^®.

Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X.

About Merus

Merus is an oncology company developing innovative full-length human bispecific and trispecific antibody therapeutics, referred to as Multiclonics^®. Multiclonics^® are manufactured using industry standard processes and have been observed in preclinical and clinical studies to have several of the same features of conventional human monoclonal antibodies, such as long half-life and low immunogenicity. For additional information, please visit Merus’ website, LinkedIn and Bluesky.

Additional Information

The tender offer for the common shares (“Common Shares”) of Merus referenced in this announcement has not yet commenced. This announcement is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell Common Shares or any other securities, nor is it a substitute for the tender offer materials that Genmab and Purchaser will file or cause to be filed with the Securities and Exchange Commission (the “SEC”) upon the commencement of the tender offer. This communication may be deemed to be solicitation material in respect of the EGM Proposals (defined below). At the time the tender offer is commenced, Genmab and Purchaser will file or cause to be filed with the SEC a tender offer statement on Schedule TO (the “Tender Offer Statement”), and Merus will file with the SEC a solicitation/recommendation statement on Schedule 14D-9 (the “Solicitation/Recommendation Statement”), in each case, with respect to the tender offer. Merus also intends to file with the SEC a proxy statement on Schedule 14A in connection with an extraordinary general meeting of Merus’ shareholders, at which Merus’ shareholders will vote on certain proposed resolutions (the “EGM Proposals”) in connection with the transactions referenced herein, and will mail the definitive proxy statement and a proxy card to each shareholder of Merus entitled to vote at the extraordinary general meeting. THE TENDER OFFER STATEMENT (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS), THE SOLICITATION/RECOMMENDATION STATEMENT AND THE PROXY STATEMENT WILL CONTAIN IMPORTANT INFORMATION. SHAREHOLDERS OF MERUS ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS OF COMMON SHARES SHOULD CONSIDER BEFORE MAKING ANY DECISION WITH RESPECT TO THE TENDER OFFER OR MAKING ANY VOTING DECISION. The tender offer for Common Shares will be made only pursuant to the Offer to Purchase, the Letter of Transmittal and related documents filed as a part of the Tender Offer Statement. The Tender Offer Statement (including the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents), as well as the Solicitation/Recommendation Statement, will be made available to all holders of Common Shares at no expense to them. The Tender Offer Statement and the Solicitation/Recommendation Statement will be made available for free at the SEC’s website at www.sec.gov. Copies of the documents filed by Genmab or Purchaser with the SEC will also be available free of charge on Genmab’s website at https://www.genmab.com/investor-relations or by contacting Genmab’s investor relations department at ir@genmab.com. Copies of the documents filed by Merus with the SEC will also be available free of charge on Merus’ website at https://ir.merus.nl/ or by contacting Merus’ investor relations department at s.spear@merus.nl. In addition, shareholders of Merus may obtain free copies of the tender offer materials by contacting the information agent for the tender offer that will be named in the Tender Offer Statement.

Participants in the Solicitation

Merus and certain of its directors, executive officers and other members of management and employees may be deemed to be participants in soliciting proxies from its shareholders in connection with the proposed back end transactions. Information regarding the persons who may, under the rules of the SEC, be considered to be participants in the solicitation of Merus’ shareholders in connection with the proposed back end transactions will be set forth in Merus’ definitive proxy statement for its extraordinary general meeting at which the EGM Proposals will be submitted for approval by Merus’ shareholders. You may also find additional information about Merus’ directors and executive officers in Merus’ Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the SEC on February 27, 2025 (as amended) and Merus’ Definitive Proxy Statement for its 2025 annual general meeting of shareholders, which was filed with the SEC on April 24, 2025.

This Company Announcement contains forward looking statements. The words “believe,” “expect,” “anticipate,” “intend” and “plan” and similar expressions identify forward looking statements. Statements in this Company Announcement that are forward looking may include, but are not limited to, statements regarding: the benefits and potential effects of the proposed transaction; the development plan, regulatory approval, data release timing, commercial launch timing and revenue potential of petosemtamab; the expected timing of the closing of the proposed transaction; and Genmab’s expectations regarding financing the proposed transaction, de-levering and timing of new drug launches. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, the occurrence of any event, change or other circumstance that could give rise to the right of Genmab or Merus or both of them to terminate the transaction agreement, including circumstances requiring a party to pay the other party a termination fee pursuant to the transaction agreement; the failure to obtain applicable regulatory approvals or clearances or Merus shareholder approval in a timely manner or otherwise; the risk that the proposed transaction may not close in the anticipated timeframe or at all due to one or more of the other closing conditions to the proposed transaction not being satisfied or waived; the risk that there may be unexpected costs, charges or expenses resulting from the proposed transaction; risks related to the ability of Genmab to successfully integrate Merus’ business with Genmab’s existing businesses and achieve the expected benefits of the proposed transaction within the expected timeframes or at all and the possibility that such integration may be more difficult, time consuming or costly than expected; risks that the proposed transaction disrupts Genmab’s or Merus’ current plans and operations; risks related to disruption of each company’s management’s time and attention from ongoing business operations due to the proposed transaction; continued availability of capital and financing and rating agency actions; the risk that any announcements relating to the proposed transaction could have adverse effects on the market price of Genmab’s and/or Merus’ securities or operating results; the risk that the proposed transaction and its announcement could have an adverse effect on the ability of Genmab and Merus to retain and hire key personnel, and to maintain relationships with their respective business partners and on their respective operating results and businesses generally; risks typically associated with conducting clinical trials, including the risk that additional clinical trials testing Merus’ products may not be successful; the risk that Merus’ products may not be approved on expected timelines or at all; the risk of litigation that could be instituted against Genmab or its directors, managers or officers and/or regulatory actions related to the proposed transaction, including the effects of any outcomes related thereto; risks related to unpredictable and severe or catastrophic events, including but not limited to acts of terrorism, war or hostilities, cyber-attacks, or the impact of any pandemic, epidemic or outbreak of an infectious disease in the United States or worldwide on Genmab’s and/or Merus’ business, financial condition and results of operations, as well the response thereto by each company’s management; and other business effects, including the effects of industry, market, economic, political or regulatory conditions. Also, actual results or performance of Genmab and Merus may differ materially from any future results or performance expressed or implied by such statements for a number of additional reasons as described in Genmab’s and Merus’ respective filings with the SEC, including those included in Genmab’s most recent Annual Report on Form 20-F, which is available at www.genmab.com and www.sec.gov, and those included in Merus’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which is available at https://merus.nl/ and www.sec.gov. Neither Genmab nor Merus undertakes any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab^®; the Y-shaped Genmab logo^®; Genmab in combination with the Y-shaped Genmab logo^®; HuMax^®; DuoBody^®; HexaBody^®; DuoHexaBody^®, HexElect^® and KYSO^®.

Multiclonics^®, Biclonics^®, Triclonics^® and ADClonics^® are registered trademarks of Merus N.V.

Contacts

Genmab Contacts:

Marisol Peron, Senior Vice President, Global Communications & Corporate Affairs

T: +1 609 524 0065; E: mmp@genmab.com

Andrew Carlsen, Vice President, Head of Investor Relations

T: +45 3377 9558; E: acn@genmab.com

Merus Contacts:
Sherri Spear, Senior Vice President, Investor Relations and Strategic Communications

T: 617-821-3246; E: s.spear@merus.nl

Kathleen Farren, Director Investor Relations and Corporate Communications

T: 617-230-4165; E: k.farren@merus.nl

Virometix Announces Completion of Enrollment in Phase I Trial of V-212 — a Fully Synthetic, Serotype-Independent Vaccine Candidate Against Streptococcus Pneumoniae




Virometix Announces Completion of Enrollment in Phase I Trial of V-212 — a Fully Synthetic, Serotype-Independent Vaccine Candidate Against Streptococcus Pneumoniae

SCHLIEREN, Switzerland–(BUSINESS WIRE)–Virometix AG, a Swiss clinical-stage biotechnology company pioneering fully synthetic vaccines, today announced that it has successfully completed enrollment in the Phase I clinical trial of V-212, a peptide-based, serotype-independent vaccine candidate targeting Streptococcus pneumoniae infections.

“Completing enrollment in this Phase I trial marks a significant milestone for V-212,” said Anna Sumeray, CEO of Virometix. “This fully synthetic, serotype-independent vaccine candidate is designed to advance our mission of delivering scalable, safe, and broad-spectrum protection against pneumococcal disease, while addressing the current limitations of existing PCV approaches. Through our collaboration with CEVAC, we are well-positioned to deliver high-quality Phase I data, with topline results anticipated in the first quarter of 2026.”

Prof. Isabel Leroux-Roels, Principal Investigator at CEVAC, added, “We are proud to collaborate with Virometix on this first-in-human study of V-212. Pneumococcal infections remain a major global health challenge, underscoring the urgent need for next-generation vaccines with broader and more durable protection. V-212’s fully synthetic, serotype-independent approach is highly innovative, and we look forward to advancing the clinical evaluation of this important candidate.”

About Virometix and the V-212 Program

Virometix develops structure-based, fully synthetic nanoparticle vaccines designed to elicit targeted, robust, and durable immune responses. Its proprietary Synthetic Virus-Like Particle (SVLP) platform employs conformational synthetic peptide mimetics displayed on self-assembling lipopeptidic nanoparticles that include built-in adjuvant elements, including T-helper epitopes and Toll-like receptor (TLR) ligands—eliminating dependence on biological components and simplifying manufacturing.

V-212, the lead pneumococcal vaccine candidate, is specifically engineered as a serotype-independent, peptide-based immunogen. Multiple conserved antigenic epitopes from key Streptococcus pneumoniae surface proteins are synthesized and conjugated to SVLP nanoparticles, aiming to induce broad immunity across diverse serotypes—addressing the limitations of current conjugate vaccines.

Preclinical studies have demonstrated robust, long-lasting immunogenicity in mouse and rabbit models. V-212 prevented lethal sepsis in a serotype 3 challenge, inhibited bacterial dissemination into blood, and reduced pulmonary burden. It also conferred protection against serotype 8 infections. Moreover, antisera elicited by V-212 recognized multiple pneumococcal serotypes, including non-PCV-13 types, underscoring its serotype-independent potential.

Phase I Trial Design and Enrollment Highlights

• Study ID: NCT06975319 (VMX-SPN-212-001)
• Design: A randomized, double-blind, placebo-controlled, first-in-human, Phase I trial in healthy adult volunteers.
• Participants: A total of 60 healthy subjects aged 18–45 years have been enrolled.
• Collaboration: The trial is being conducted in collaboration with CEVAC (Centre for Vaccinology) at Ghent University Hospital, a leading European clinical trial unit with extensive expertise in vaccine development.
• Dosing Regimen: Subjects receive three intramuscular injections of either V-212 or placebo, across low, medium, and high dose groups to assess safety, tolerability, and immunogenicity.
• Primary Objective: Evaluate safety and tolerability across dose levels.
• Secondary Objective: Assess immunogenicity to identify an optimal dose for subsequent studies.
• Next Milestone: Topline safety and immunogenicity data are expected in Q1 2026. 

About Virometix

Virometix AG is a privately held Swiss biotechnology company developing a new generation of fully synthetic vaccines to generate targeted and protective immune responses against infectious diseases and cancer. There is a considerable medical need for vaccines to combat infectious as well as a number of chronic human diseases, including cancer. Rational molecular design, chemical synthesis and Virometix’ proprietary “Synthetic Virus-Like Particle” platform technology allow for the rapid production and optimization of vaccine candidates with the potential to demonstrate superior properties in terms of safety, efficacy, ease and cost of manufacturing and stability. Learn more at www.virometix.com

Forward-Looking Statements

This release contains forward-looking statements regarding the clinical development of V-212. Trial outcomes, timelines, and future steps involve inherent risks and uncertainties.

Contacts

Media & Investor Contact
For further inquiries, please contact:

Virometix AG

Wagistrasse 14

8952 Schlieren, Switzerland

+41 43 433 86 60

Email: press@virometix.com

Veracyte Announces that Decipher-Enabled Biomarker Predicts Hormone Therapy Benefit in Men with Recurrent Prostate Cancer




Veracyte Announces that Decipher-Enabled Biomarker Predicts Hormone Therapy Benefit in Men with Recurrent Prostate Cancer

Findings from the first prospective validation trial for biomarker presented at ASTRO 2025

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–$VYCT #ASTRO25—Veracyte, Inc. (Nasdaq: VCYT), a leading genomic diagnostics company, announced that new data from the prospective, randomized integral biomarker BALANCE trial (NCT03371719) finds that the PAM50 molecular signature predicts which patients with recurrent prostate cancer benefit from hormone therapy with apalutamide in addition to salvage radiation therapy. The prostate PAM50 biomarker is currently available for Research Use Only on the Decipher GRID (Genomic Resource for Intelligent Discovery) research tool.

The new findings were shared today by Daniel Spratt, M.D., University Hospitals Seidman Cancer Center, Case Western Reserve University, in a podium presentation at ASTRO 2025, the annual meeting of the American Society for Radiation Oncology, being held in San Francisco.

“Our findings mark the first time, to my knowledge, that a predictive biomarker has been validated in a prospective, biomarker-driven, randomized trial in non-metastatic prostate cancer,” said Dr. Spratt. “Thus, this is an unprecedented advancement for patients who can be more-precisely selected to receive hormone therapy or forego the treatment and the potential side effects.”

For the study, 295 men with recurrent, non-metastatic prostate cancer following prostate-removal surgery were randomly assigned to salvage radiation therapy with a placebo or apalutamide for 6 months. The PAM50 biomarker was a key stratification variable to ensure each arm had a similar proportion of luminal B and non-luminal B subtypes. They were followed for a median of 5 years during which they were evaluated for biochemical failure, which is a rise in levels of prostate-specific antigen (PSA) post treatment—an early sign of salvage therapy failure. Among the 127 men with luminal B molecular subtype tumors (as determined by the PAM50 signature), 72% of those taking apalutamide did not experience biochemical failure, as compared to the 54% rate in the placebo group [HR 0.45 (80% CI 0.29-0.68), p=0.0062]. In the non-luminal B subset, there was no difference between those taking apalutamide versus placebo (70% vs 71%) [HR 0.95 (80% CI 0.65-1.41), p=0.44].

“These results from NRG GU006 represent the highest level of evidence to support routine biomarker testing in recurrent prostate cancer patients planned to receive secondary radiotherapy,” Dr. Spratt added. “With such a strong difference in the metastasis-free survival response to hormone therapy between luminal B and non-luminal-B tumors, the use of the predictive PAM50 biomarker is a game changer to help personalize treatment for men with recurrent prostate cancer beyond merely prognostic tools.”

The PAM50 signature is the third biomarker—assessed through the whole-transcriptome-based Decipher platform—that a major study has shown predicts benefit from hormone therapy, radiation therapy or chemotherapy. Another trial—PREDICT-RT—recently completed enrollment two years early and is evaluating the Decipher Prostate test’s ability to predict benefit of combined hormone therapy (ADT and apalutamide) concurrent with radiation in patients with high-risk prostate cancer at initial diagnosis.

“Prostate cancer, like all cancers, is a disease of the genome,” said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. “Our Decipher GRID tool uniquely enables researchers to better pinpoint adverse molecular features that are associated with poor outcomes. This can ultimately lead to more-personalized care for each patient based on their tumor’s unique molecular make-up. We are proud to partner with the world’s leading prostate cancer researchers to help uncover insights that can change the trajectory of care for each individual patient and also help deliver the next generation of prostate cancer diagnostics.”

The BALANCE trial results are among 9 Decipher-focused abstracts being presented at the ASTRO 2025 conference. More information can be found here.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients across the full spectrum of prostate cancer. The test is performed on biopsy or surgically resected samples and conveys the aggressiveness of the cancer. For patients with localized or regional prostate cancer, the Decipher score indicates a patient’s risk of metastasis, helping to determine treatment timing and intensity. For patients with metastatic prostate cancer, the Decipher score indicates the likelihood of cancer progression and survival benefit with treatment intensification. Armed with this information, physicians can better personalize their patients’ care. The Decipher Prostate test’s performance and clinical utility has been demonstrated in over 90 studies involving more than 200,000 patients. It is the only gene expression test to achieve “Level I” evidence status and inclusion in the risk-stratification table in the most recent NCCN^® Guidelines* for prostate cancer. More information about the Decipher Prostate test can be found here.

About Veracyte

Veracyte (Nasdaq: VCYT) is a global diagnostics company whose vision is to transform cancer care for patients all over the world. We empower clinicians with the high-value insights they need to guide and assure patients at pivotal moments in the race to diagnose and treat cancer. Our Veracyte Diagnostics Platform delivers high-performing cancer tests that are fueled by broad genomic and clinical data, deep bioinformatic and AI capabilities, and a powerful evidence-generation engine, which ultimately drives durable reimbursement and guideline inclusion for our tests, along with new insights to support continued innovation and pipeline development. For more information, please visit www.veracyte.com or follow us on LinkedIn or X (Twitter).

About Decipher GRID

The Decipher GRID database includes more than 250,000 whole-transcriptome profiles from patients with urologic cancers and is used by Veracyte and its partners to contribute to continued research and help advance understanding of prostate and other urologic cancers. GRID-derived information is available on a Research Use Only basis. More information about Decipher GRID can be found here.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to our statements regarding the use of the predictive PAM50 biomarker to help personalize treatment for men with recurrent prostate cancer beyond merely prognostic tools and the belief that this is a game changer, and expectations that our Decipher GRID tool uniquely enables researchers to better pinpoint adverse molecular features that are associated with poor outcomes; that this can ultimately lead to more-personalized care for each patient based on their tumor’s unique molecular make-up; and that this can help uncover insights that can change the trajectory of care for each individual patient and also help deliver the next generation of prostate cancer diagnostics. Forward-looking statements can be identified by words such as: “appears,” “anticipate,” “intend,” “plan,” “expect,” “believe,” “should,” “may,” “will,” “enable,” “positioned,” “offers,” “designed,” “ultimately,” and similar references to future periods. Actual results may differ materially from those projected or suggested in any forward-looking statements. These statements involve risks and uncertainties, which could cause actual results to differ materially from our predictions, and include, but are not limited to the potential impact the Veracyte Diagnostics Platform can have on scientific advancements in cancer and, in turn, patient care. Additional factors that may impact these forward-looking statements can be found under the caption “Risk Factors” in our Annual Report on Form 10-K filed on February 28, 2025. Copies of these documents, when available, may be found in the Investors section of our website at https://investor.veracyte.com. These forward-looking statements speak only as of the date hereof and, except as required by law, we specifically disclaim any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise.

Veracyte, the Veracyte logo, and Decipher are registered trademarks of Veracyte, Inc., and its subsidiaries in the U.S. and selected countries.

* National Comprehensive Cancer Network. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

Contacts

Investors:
Shayla Gorman

investors@veracyte.com
619-393-1545

Media:
Karen Possemato

media@veracyte.com

New Data from HELIOS-B Phase 3 Study Demonstrate Lower Rates of Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran




New Data from HELIOS-B Phase 3 Study Demonstrate Lower Rates of Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran

− Treatment with Vutrisiran Led to 37- 49% Lower Rates of Gastrointestinal Events, a Multisystem Manifestation of ATTR-CM, Across Multiple Treatment Groups, Compared to Placebo –

− Additional Analyses Reinforce Vutrisiran’s Safety and Efficacy Profile as a Monotherapy and Illustrate Consistent Benefit from Treatment with Vutrisiran Across a Range of Patients’ Baseline Health Status and Quality of Life –

− Findings Presented at the Heart Failure Society of America Annual Scientific Meeting 2025 Highlight the Impact of Vutrisiran which Delivers Rapid Knockdown of Transthyretin –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results from new analyses of the HELIOS-B Phase 3 study of AMVUTTRA^® (vutrisiran), an RNAi therapeutic approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Data from a post hoc analysis of the HELIOS-B study, which assessed whether treatment with vutrisiran was associated with a reduction in gastrointestinal (GI) adverse events in patients with ATTR-CM, compared to placebo, were presented during a late-breaking session at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2025 in Minneapolis, Minnesota. The analysis showed that treatment with vutrisiran was associated with a lower rate of GI events across the overall, vutrisiran monotherapy, and baseline tafamidis treatment groups, compared to placebo, a trend that was consistent in patients living with both the wild-type and hereditary forms of the disease.

Patients with ATTR-CM often experience disease manifestations beyond the heart including GI events such as diarrhea, abdominal pain and discomfort, constipation, nausea, and vomiting. In the analysis, a 42% lower rate of GI events was observed in patients treated with vutrisiran in the overall population, compared to placebo. Consistent results were also observed across the vutrisiran monotherapy group and in patients treated with tafamidis at baseline. In the vutrisiran monotherapy group, a 37% lower rate of GI events was observed, compared to placebo. In the baseline tafamidis group, a 49% lower rate of GI events was observed, compared to placebo. When looking specifically at individual GI symptoms known to significantly impact quality of life (QOL), including diarrhea, nausea, and vomiting, reductions of greater than 50% were observed across all three study populations: the overall population, the vutrisiran monotherapy population, and the population of patients treated with tafamidis at baseline. This corresponded to rate ratios (RR) for diarrhea of 0.46, 0.48, and 0.44; for nausea of 0.35, 0.17, and 0.48; and for vomiting of 0.16, 0.25, and 0.00, respectively. The lower rate of GI events in patients treated with vutrisiran, compared to placebo, was observed as early as three months and was consistent across hereditary and wild-type patients throughout the double-blind period. These findings suggest a potential treatment effect in all study populations assessed.

“As a multisystem disease, it is well-known that ATTR-CM impacts more than just the heart,” said Marcus Urey, M.D., Associate Professor, University of California San Diego Health. “For patients living with both hereditary and wild-type forms of the disease, gastrointestinal complications such as diarrhea, abdominal pain, constipation, nausea, and vomiting are a part of their journey with ATTR-CM, with some of these symptoms often significantly impacting their quality of life. As a physician who sees the multisystem impact of this disease every day, I am encouraged by these findings which underscore vutrisiran’s differentiated clinical profile and its potential to address the multisystem nature of this disease.”

A second post hoc analysis of the HELIOS-B study presented at the HFSA Annual Scientific Meeting assessed the efficacy and safety of vutrisiran as a monotherapy by censoring patients who initiated tafamidis during the double-blind period at investigator discretion. This analysis was designed to isolate the effect of vutrisiran treatment alone, without the potential confounding influence of additional therapy. Tafamidis initiation occurred in 21.5% of monotherapy patients, with a median time to initiation of approximately 12 months. In this censored monotherapy population, patients treated with vutrisiran demonstrated a statistically significant 32% reduction in the risk of the primary composite endpoint of all-cause mortality and recurrent cardiovascular events through 36 months, compared to patients who received placebo (hazard ratio [HR] 0.68; 95% confidence interval [CI]: 0.49–0.95; p=0.022). These results were consistent with the primary monotherapy analysis of the study which included patients who later initiated tafamidis (HR 0.67; 95% CI: 0.49–0.93; p=0.016). Moreover, within the censored population, outcomes for the secondary endpoints and safety findings were consistent with the results of the primary analysis. These results reinforce the findings from the powered monotherapy subgroup and provide further evidence of vutrisiran’s efficacy and safety as a standalone first-line therapy, without the confounding effects of additional treatments.

“The new HELIOS-B analyses presented at the HFSA Annual Scientific Meeting build on AMVUTTRA’s differentiated first-line profile,” said John Vest, M.D., Senior Vice President, TTR Global Clinical Lead, Alnylam. “We understand that gastrointestinal symptoms place a significant burden on patients, thus I’m encouraged to observe a reduction in these events in as early as three months after initiation of treatment. These findings, taken together with further evidence of AMVUTTRA’s strong monotherapy profile, underscore its potential to deliver meaningful impact as a first-line treatment for patients living with this rapidly progressive multisystem disease.”

A third post hoc analysis of the HELIOS-B study evaluated outcomes by baseline Kansas City Cardiomyopathy Questionnaire overall summary (KCCQ-OS) score, and demonstrated that treatment with vutrisiran resulted in consistent benefits in survival, cardiovascular outcomes, functional capacity, quality of life, cardiac biomarkers, and reduced GI adverse events, regardless of baseline health status.

Data from the HELIOS-B study supported the recent approvals of AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM in adults in the United States (US), Brazil, European Union (EU), Japan, United Arab Emirates (UAE) and United Kingdom (UK). Collectively, AMVUTTRA has more than 8,000 patient-years of experience worldwide and is the first RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

For additional information on Alnylam’s presentations at the HFSA Annual Scientific Meeting 2025, please visit Capella.

Indications and Important Safety Information

Indications Approved by the U.S. FDA

AMVUTTRA^® (vutrisiran) is indicated for the treatment of the:

• cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
• polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA treatment leads to a decrease in serum vitamin A levels.

Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

In a study of patients with ATTR-CM, no new safety issues were identified.

For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025)

About AMVUTTRA

AMVUTTRA^® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, EU, UK, Brazil, Japan, and UAE. In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR).

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.^1-4

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.⁵ Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.⁶ By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made.⁵ This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran as a treatment for ATTR-CM, including vutrisiran’s potential to deliver meaningful impact for ATTR-CM patients; vutrisiran’s potential as a standalone first-line treatment for ATTR-CM; the consistent benefit of treatment with vutrisiran across a range of patients’ baseline health status and quality of life; vutrisiran’s potential to address the multisystem nature of ATTR-CM; and Alnylam’s ability to execute on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P⁵x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

References

¹ Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.

² Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.

³ Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.

⁴ Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

⁵ Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.

⁶ Zamore P. Cell. 2006;127(5):1083-1086.

Contacts

Alnylam Pharmaceuticals, Inc.

Christine Akinc  

(Investors and Media)

+1-617-682-4340

Josh Brodsky

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Pfizer Highlights Momentum in Redefining Standards of Care in Cancer at ESMO 2025




Pfizer Highlights Momentum in Redefining Standards of Care in Cancer at ESMO 2025

More than 45 abstracts, including five late-breaking presentations and recognition in Presidential Symposium, showcase impact of approved medicines and potential of next-generation pipeline

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) will highlight data across its extensive Oncology portfolio at the European Society for Medical Oncology (ESMO) Congress 2025, being held October 17-21 in Berlin, Germany. Data from more than 45 company-sponsored, investigator-sponsored, and collaborative research abstracts, including 11 oral/mini oral presentations and five late-breaking sessions, will be presented across Pfizer’s core scientific modalities and key tumor areas.

“At ESMO, Pfizer is demonstrating how earlier interventions with our innovative medicines have the potential to deliver greater impact to even more patients,” said Jeff Legos, Chief Oncology Officer, Pfizer. “The survival benefits we’re seeing across certain cancer types reinforce our commitment to accelerating innovative medicines that bring new hope to patients everywhere, while pipeline data highlight the next wave of potential breakthroughs that could transform care for even more people living with cancer.”

Pfizer will share highlights from its leading Oncology portfolio at ESMO, including:

• In a Presidential Symposium, unprecedented survival results from the Phase 3 EV-303 trial (KEYNOTE-905) evaluating PADCEV^® (enfortumab vedotin-ejfv), a Nectin-4 directed antibody-drug conjugate (ADC), plus KEYTRUDA^® (pembrolizumab)* in patients with muscle-invasive bladder cancer who are ineligible for or declined cisplatin-based chemotherapy, showing potential to redefine standard of care in these patients (Presentation #LBA2)
• Final overall survival results from the Phase 3 EMBARK trial evaluating XTANDI^® (enzalutamide)** in combination with leuprolide and as monotherapy in non-metastatic hormone-sensitive prostate cancer with high-risk biochemical recurrence, highlighting the benefit of XTANDI in this earlier line of treatment (Presentation #LBA87)
• Updated overall survival data from the Phase 2 PHAROS study of BRAFTOVI^® (encorafenib) plus MEKTOVI^® (binimetinib)*** in patients with BRAF V600E-mutant metastatic non-small cell lung cancer (NSCLC), reinforcing this combination as a potential key treatment option for these patients (Presentation #1849MO)

Information on significant Pfizer and partner-sponsored abstracts, including date and time of presentation, follows in the chart below. A complete list of Pfizer and partner-sponsored abstracts and presentations is available here.

* Pfizer and Astellas have a clinical collaboration agreement with Merck to evaluate the combination of PADCEV^® and KEYTRUDA^® in patients with previously untreated metastatic urothelial cancer.

** XTANDI^® is jointly developed and commercialized by Pfizer and Astellas in the United States.

*** The PHAROS trial is conducted with support from Pierre Fabre.

**** Led by the Australian and New Zealand Urogenital and Prostate Cancer Trials Group Limited (ANZUP) with Astellas funding

***** Pfizer and Arvinas have a global collaboration for the co-development and co-commercialization of vepdegestrant.

****** The BREAKWATER trial was conducted with support from ONO Pharmaceutical, Merck KGaA, Darmstadt, Germany and Eli Lilly and Company.

Prescribing Information for Pfizer Medicines

Please see full Prescribing Information, including BOXED WARNING, for PADCEV^® (enfortumab vedotin).

Please see full Prescribing Information for XTANDI^® (enzalutamide).

Please see full Prescribing Information for BRAFTOVI^® (encorafenib).

Please see full Prescribing Information for MEKTOVI^® (binimetinib).

Please see full Prescribing Information for IBRANCE^® (palbociclib) tablets and IBRANCE^® (palbociclib) capsules.

About Pfizer Oncology

At Pfizer Oncology, we are at the forefront of a new era in cancer care. Our industry-leading portfolio and extensive pipeline includes three core mechanisms of action to attack cancer from multiple angles, including small molecules, antibody-drug conjugates (ADCs), and multispecific antibodies, including other immune-oncology biologics. We are focused on delivering transformative therapies in some of the world’s most common cancers, including breast cancer, genitourinary cancer, hematology-oncology, and thoracic cancers, which includes lung cancer. Driven by science, we are committed to accelerating breakthroughs to help people with cancer live better and longer lives.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on X at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice

The information contained in this release is as of September 25, 2025. The Company assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about Pfizer Oncology, Pfizer’s Oncology portfolio of marketed and investigational therapies, including combinations, and an investigational therapy for a cancer-related condition; expectations for our product pipeline, in-line products and product candidates, including their potential benefits, clinical trial results and other developing data; potential breakthrough, best- or first-in-class or blockbuster status or expected market entry of our medicines; and other statements about our business, operations and financial results that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of Pfizer’s oncology portfolio; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizer’s product candidates; whether and when any such applications that may be pending or filed for any of Pfizer’s product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizer’s products or product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws or regulations; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

Contacts

Media Contact: PfizerMediaRelations@Pfizer.com

Investor Contact: IR@Pfizer.com

Marginum HIVEN® for Neurosurgery Is Cleared for CE Mark at Record Speed




Marginum HIVEN® for Neurosurgery Is Cleared for CE Mark at Record Speed

KUOPIO, Finland–(BUSINESS WIRE)–#hiven–Marginum announces a significant milestone as its flagship device, HIVEN^®, is cleared for the CE mark. The underlying MDR certification signifies an important regulatory milestone.

Marginum, a Finnish medical technology company, made rapid strides in validating the aspirate tissue monitoring (ATM) technique and received the MDR certificate at a record-breaking speed, just over 4.5 years. Marginum’s team successfully developed a breakthrough class IIb medical device that addresses an unmet clinical need to accurately detect tumorous tissue during surgery, without compromising existing workflows.

New era of precision in cancer surgery

HIVEN^® is a novel device for assisting in intraoperative margin assessment that provides near real-time feedback to support surgeons in achieving a safer and more complete tumour resection. By providing surgical teams with immediate insight into the tumour margins while resecting, the device aims to improve patient outcomes and reduce the likelihood of reoperations. HIVEN^® is designed to detect fluorescent cancer tissue from aspirated tissue during surgery without disrupting standard workflow.

“Achieving clearance for the CE mark is a pivotal step in bringing the HIVEN^® into clinical practice and improving outcomes for patients in Europe. This certificate reflects the hard work of our team and the strength of our scientific and clinical foundations,” says Juho Leskinen, CTO and co-founder of Marginum.

Clinical Benefits & Indications

The aspirate tissue monitoring technology aims to overcome surgical challenges that may damage healthy tissues and leave tumour cells undetected. Incomplete removal, damage to healthy tissues, and reoperations exacerbate patient suffering, compromised standard of care, and long-term complications – all directly escalating healthcare costs.

Critical structures like blood vessels often create blind spots – behind corners and tissue ridges – where cancerous tissue can be difficult to detect. The HIVEN^® aspirate tissue monitoring device addresses this challenge by allowing resected tissue to be transported directly for fluorescence analysis. This provides surgeons with more comprehensive information about the surgical site.

“In glioma surgery, our ability to distinguish tumour from healthier tissues is limited by anatomical constraints, blood and compromised visibility, particularly in deep-seated areas. We wanted HIVEN^® to provide critical feedback beyond sensory limitations; you can consider it a sixth sense for tumour detection,” comments docent Antti-Pekka Elomaa MD PhD, a consultant neurosurgeon and one of Marginum co-founders.

The HIVEN^® is approved for fluorescence-guided neurosurgery of high-grade gliomas, where precise margin identification is critical. HIVEN^® enhances surgical accuracy by enabling objective tissue detection in hard-to-reach areas and simplifying the procedure workflow.

About Marginum:

Marginum, a leading innovator in fluorescence-guided oncological surgery, is a medical technology company developing fluorescence-based tissue detection systems. HIVEN^® by Marginum enables safe and efficient monitoring of tumour tissues during cancer surgery. www.marginum.com

Contacts

Media contact:
Samu Lehtonen, CEO & Co-Founder

Email: samu.lehtonen@marginum.com
Mobile: +358405797890

https://www.marginum.com

Corstasis Therapeutics and U.S. Heart and Vascular® Collaboration Will Seek to Improve Heart Failure Care with ENBUMYST™ (Bumetanide Nasal Spray)




Corstasis Therapeutics and U.S. Heart and Vascular® Collaboration Will Seek to Improve Heart Failure Care with ENBUMYST™ (Bumetanide Nasal Spray)

The collaboration will seek to foster the adoption of appropriate protocols and pathways for the integration of ENBUMYST™ (bumetanide nasal spray) into clinical care with the goal of reducing readmissions, improving fluid management and enhancing outcomes through the novel delivery of diuretic therapy.

HENDERSON, Nev.–(BUSINESS WIRE)–#chf–Corstasis Therapeutics Inc., an innovative biopharmaceutical company providing enhanced outpatient therapeutic options for patients with cardiovascular and renal disease, today announced a strategic collaboration with U.S. Heart and Vascular® (USHV), the nation’s premier cardiovascular management services organization, seeking to enable earlier intervention and enhance outcomes for heart failure patients. Corstasis and USHV will co-develop, and seek to optimize, value-based care pathways for the outpatient treatment of fluid overload in patients with congestive heart failure, liver disease and chronic kidney disease with ENBUMYST™ (bumetanide nasal spray).

“ENBUMYST was developed with the goal of giving providers a new, self-administered outpatient option,” said Brian Kolski M.D., Chief Medical Officer of Corstasis Therapeutics. “Our collaboration with USHV reflects a shared commitment to health system innovation, value-based care delivery and patient-centered outcomes.”

The collaboration between Corstasis and USHV will seek to:

• Develop and validate protocols and clinical workflows that are consistent with approved labeling and enable adoption of early intervention via nasal spray diuresis when clinically indicated

• Develop associated provider and staff training to ensure seamless integration of this important new intervention into clinical care

• Capture outcomes and healthcare economic data to support payer engagement and future reimbursement strategies

“At USHV, we are continuously seeking new tools that empower our affiliated physicians and deliver measurable value to patients and payers,” said Emily Rash, Chief Value-Based Care Officer, USHV. “We believe an outpatient-friendly alternative represents a meaningful step forward in ambulatory heart failure care and we’re proud to work with Corstasis to bring this innovation into practice.”

The U.S. Food and Drug Administration (FDA) recently approved ENBUMYST for the treatment of edema associated with congestive heart failure (CHF), and hepatic and renal disease, including nephrotic syndrome in adults.

This effort is intended to put Corstasis and USHV at the forefront of improving outpatient heart failure management, with the goal of enabling the option of earlier intervention, reduced escalation of care and a more cost-effective path to euvolemia. Fluid overload associated with CHF, liver disease and chronic kidney disease is responsible for driving millions of hospitalizations and readmissions annually.

About ENBUMYST

ENBUMYST is a nasal spray loop diuretic indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR ENBUMYST™ (BUMETANIDE NASAL SPRAY).

INDICATION

ENBUMYST is indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

IMPORTANT SAFETY INFORMATION

ENBUMYST is contraindicated in patients with anuria, who are in hepatic coma and have a history of hypersensitivity to bumetanide.

ENBUMYST is a diuretic that may cause fluid, electrolyte, and metabolic abnormalities. Excessive fluid loss can lead to dehydration, decreased blood volume, and increased risk of blood clots. Abnormalities may include changes in blood electrolytes, nitrogen, glucose, and uric acid. The chance of getting these abnormalities is higher in people who are elderly, use higher doses or who do not get enough electrolytes by mouth.

If increasing azotemia and oliguria occur during treatment of severe progressive renal disease, discontinue bumetanide.

Although unlikely at the recommended doses, the potential for ototoxicity must be considered a risk of intravenous therapy, at high doses, repeated frequently in the face of renal excretory function impairment.

Avoid use in patients with significant nasal mucosal or structural abnormalities, such as acute episodes of rhinitis or congestion due to any cause.

Advise lactating women treated with ENBUMYST to monitor their infants for excessive urine output, dehydration, and lethargy.

Most common adverse reactions are hypovolemia, headache, muscle cramps, dizziness, hypotension, nausea and encephalopathy (in patients with pre-existing liver disease).

These are not all of the possible side effects of ENBUMYST. To report suspected adverse reactions, contact Corstasis Therapeutics at 1-877-300-5339 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for ENBUMYST.

About Corstasis Therapeutics

Corstasis Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on transforming the treatment of fluid overload in patients with heart failure, liver disease, and kidney disease. Its lead asset, ENBUMYST™ (bumetanide nasal spray), was approved by the FDA on September 12, 2025 and is designed to offer rapid, reliable diuresis outside the hospital setting. For more information, please visit www.corstasis.com.

About U.S. Heart and Vascular (USHV)

Formed in 2021 in Nashville, TN, US Heart and Vascular is a physician-led management platform enabling independent cardiologists to expand patient access, improve outcomes, and reduce costs to the healthcare system. USHV builds collaborative partnerships with best-in-class cardiovascular practices, providing non-clinical management solutions and resources that allow practices to expand access to compassionate and comprehensive care, improve patient outcomes, and strategically reduce costs to the healthcare system. Currently investing in practices across five states, USHV is actively seeking new partnerships with quality cardiovascular practices and entrepreneurial physicians across the U.S. Visit https://usheartandvascular.com/.

Contacts

Media Contacts:
Ben Esque, CEO

Corstasis Therapeutics Inc.

Phone: 702-541-9222

Email: Info@corstasis.com

Carrie Moore, VP Communications

U.S. Heart & Vascular

Email: carrie.moore@usheartandvascular.com

ClearNote Health Receives In Vitro Diagnostic Approval in United Kingdom for Avantect® Multi-Cancer Detection Test and Avantect® Ovarian Cancer Test




ClearNote Health Receives In Vitro Diagnostic Approval in United Kingdom for Avantect® Multi-Cancer Detection Test and Avantect® Ovarian Cancer Test

SAN DIEGO–(BUSINESS WIRE)–#5hmC—ClearNote Health, a company focused on improving early detection for some of the deadliest cancers, today announced that it has received a United Kingdom Conformity Assessed (UKCA) marking for its Avantect® Multi-Cancer Detection Test and Avantect® Ovarian Cancer Test. Part of the UK’s independent product safety framework following its departure from the European Union, this designation confirms compliance with UK medical device regulations and is a prerequisite for selling products in the UK. The company’s Avantect Pancreatic Cancer Test received the same certification in July 2025.

The Avantect Multi-Cancer Detection Test is a simple blood test designed to screen for several types of cancer simultaneously in asymptomatic, generally healthy persons. It targets some of the deadliest cancers by analyzing both the epigenomic biomarker 5-hydroxymethylcytosine (5hmC) and genomic features in circulating cell-free DNA. Unlike conventional methods, ClearNote Health’s 5hmC-based approach measures changes in active biology, offering a highly specific signal of early cancer development and identifying the likely tissue of tumor origin.

This test was one of only two assays recently selected for the critical Vanguard Study funded by the National Cancer Institute, part of the National Institutes of Health, following a thorough evaluation of 23 emerging multi-cancer detection technologies. The Vanguard Study implemented a stringent, multi-stage selection process to evaluate multi-cancer detection assays based on sensitivity, specificity, tissue of origin prediction accuracy, and assay failure rates. Key selection criteria included early-stage detection performance for at least three cancer types. The study includes nine geographically diverse clinical trial hubs and will enroll up to 24,000 total participants to assess the implementation of multi-cancer detection testing.

The Avantect Ovarian Cancer Test was designed to aid in earlier diagnosis of cancer in women at elevated risk, particularly those with inherited genetic mutations, such as BRCA1 and BRCA2, Lynch syndrome, or a strong family history of ovarian, breast, uterine, or colorectal cancer, as well as other significant risk factors. The test uses ClearNote Health’s underlying Virtuoso™ epigenomics platform to measure the presence or absence of an abnormal signal associated with ovarian cancer in cell-free DNA.

“By achieving UKCA markings for all three of our Avantect cancer tests, ClearNote Health is well poised to deliver on our mission of helping to eradicate the deadliest forms of cancer through early detection,” said Dave Mullarkey, CEO at ClearNote Health. “These regulatory milestones are a testament to the dedication of our cross-functional team as we expand into new international markets and bring our innovative, life-saving technology to more patients worldwide.”

For more information on the Avantect cancer tests, please visit www.avantect.com.

About ClearNote Health

ClearNote Health is a privately held company dedicated to improving early detection and monitoring for some of the deadliest forms of cancer. Developed by scientists in the Stephen Quake laboratory at Stanford University, the company’s patented core Virtuoso™ epigenomics platform builds on the latest advances in artificial intelligence and bioinformatics to measure active biological differences between cancer and healthy cells in a blood sample. Its highly sensitive, noninvasive Avantect® pancreatic and ovarian diagnostic tests may identify cancers in high-risk patient populations earlier than conventional approaches, when patients may be more likely to benefit from treatment. ClearNote Health’s headquarters and CLIA-certified, CAP-accredited laboratory are located in San Diego. For more information, visit www.clearnotehealth.com or follow the company on LinkedIn.

ClearNote Health, the ClearNote Health logo, and Avantect are registered trademarks of ClearNote Health.

Contacts

Media Contact
Andrew Noble

415-722-2129

andrew@bioscribe.com