Acceleron Pharma, Inc.

With new, positive data in hand, Merck & Co. plans to move its cardiovascular drug Winrevair into late-stage testing as a treatment for a condition often caused, at least in part, by high blood pressure.This condition known as heart failure with preserved ejection fraction, or HFpEF is common, affecting more than half of the roughly 6.7 million people in the U.S. believed to be living with heart failure. In these people, the hearts main pumping chamber stiffens over time due to a variety of possible factors, including age, obesity and hypertension. While the chamber still pumps a normal amount of blood, patients with HFpEF can experience fatigue, shortness of breath, as well as life-threatening health complications.The Food and Drug Administration has already approved several medicines for HFpEF, among them AstraZenecas Farxiga, Novartis Entresto, and Eli Lilly and Boehringer Ingelheims Jardiance. Merck has been trying to expand that list with Winrevair, a first-of-its-kind therapy that works by regulating signals from activin proteins that spur cell growth and division. In heart failure, the over-production of certain cells, molecules and proteins can harden the organ and impair its function.On Tuesday, Merck announced Winrevair had hit the main goal of a mid-stage clinical trial that enrolled 164 adults with high blood pressure in their pulmonary arteries caused by HFpEF. The study pitted two different doses of Winrevair against a placebo and, according to Merck, showed that about six months into treatment, the drug provided a significant and clinically meaningful improvement to blood flow through the lungs to the heart.In a statement, Mahesh Patel, vice president of global clinical development at Merck Research Laboratories, said these kinds of blood flow improvements could translate to better outcomes for this patient population.Merck didnt release any detailed data, but said it intends to present results at a future scientific meeting. In the meantime, its forging ahead into Phase 3 development.For Merck, which stands to lose patent protection on the worlds top-selling drug, Keytruda, in the not-too-distant future, the success of products like Winrevair is crucial. In 2024, Keytruda accounted for a little over half of the companys $57.4 billion in pharmaceutical sales.Merck picked up Winrevair through its $11.5 billion acquisition of Acceleron Pharma back in 2021. The drug is one of 20 growth drivers that, by Mercks estimates, could eventually make up for the expected declines to the Keytruda franchise. The company argues that almost all of these drivers have the potential to generate at least $1 billion in annual revenue and, collectively, their commercial opportunity exceeds $50 billion.Winrevair first received FDA approval in the spring of 2024, for a separate blood pressure disorder called pulmonary arterial hypertension. Sales of the drug totaled $976 million over the first nine months of this year.Carter Gould, an analyst at the investment firm Cantor Fitzgerald, wrote in a note to clients that this latest trial win should help Mercks stock price, especially since investors were ascribing value to Winrevair beyond PAH.Gould added that, among analysts, the average forecast has annual Winrevair sales peaking at $8 billion to $8.5 billion, with around $1 billion to $2 billion coming from outside of PAH, namely HFpEF.Merck shares were up more than 3%, to trade above $96 apiece, by late Tuesday morning. '

Bengaluru: Merck & Co posted higher third-quarter revenue on Thursday, as growth from its blockbuster cancer drug Keytruda offset falling sales from human papillomavirus vaccine Gardasil in China. However, shares fell nearly 2% after Merck lowered the high end of its revenue forecast for the full year. It now expects revenue of $64.5 billion to $65.0 billion compared with its prior view of $64.3 billion to $65.3 billion. J.P.Morgan analysts called the quarterly beat "modest," adding the annual forecasts were "largely reiterated." Merck shares have fallen about 5% so far in 2025, as investors weigh the company's efforts to grow beyond Keytruda against broader industry headwinds, including potential U.S. drug pricing reforms. "We do expect continued growth in Keytruda, albeit at a slightly slower pace than we've seen as we are getting to peak penetration in some of the indications, and we do expect some headwind from price in our ex-U.S. markets," said CFO Caroline Litchfield. Merck is working to add new drugs to its pipeline before Keytruda faces competition from cheaper biosimilars later this decade. The company is focusing on newer drugs, including lung disease treatments Winrevair, from its 2021 acquisition of Acceleron Pharma, and Ohtuvayre, which was recently acquired in a $10 billion takeover of UK-based Verona Pharma. Sales of Winrevair rose 141% to $360 million in the quarter. However, Bernstein analyst Courtney Breen said the drug's sales were materially below expectations, calling it "not a good sign for a key growth driver for the company." Merck posted revenue of $17.28 billion in the quarter, compared with analyst estimates of $16.96 billion, according to LSEG data. On an adjusted basis, the company earned $2.58 per share, topping Wall Street estimates by 23 cents. Sales of immunotherapy Keytruda rose 10% to $8.1 billion in the quarter. Gardasil sales fell to $1.75 billion, but came in ahead of the Wall Street forecast of $1.66 billion. The company has not been making new shipments of the vaccine for China. It has said distributors there are working through their inventories after a fall in demand due to pressure on consumer spending in the country. Excluding China, Gardasil sales fell 3%, mainly due to lower sales in Japan following the end of reimbursement for the catch-up vaccination program. Merck said it expects full-year earnings of $8.93 to $8.98 a share, compared with its prior forecast of $8.87 to $8.97. (Reporting by Michael Erman and Christy Santhosh in Bengaluru; Editing by Stephen Coates and Bill Berkrot)

RAHWAY, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved an update to the U.S. product label based on the Phase 3 ZENITH trial for WINREVAIR™ (sotatercept-csrk) for injection, 45mg, 60mg. WINREVAIR, an activin signaling inhibitor, is now FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1 pulmonary hypertension) to improve exercise capacity and WHO functional class (FC), and reduce the risk of clinical worsening events, including hospitalization for PAH, lung transplantation and death. WINREVAIR was initially approved based on the pivotal STELLAR study in March 2024. Today’s approval expanded the indication of WINREVAIR to include components of the clinical worsening events: hospitalization for PAH, lung transplantation and death. In ZENITH (N=172; 86 WINREVAIR, 86 placebo), adding WINREVAIR to background therapy demonstrated a statistically significant and clinically meaningful 76% reduction in the risk of major morbidity and mortality outcomes in adults with PAH WHO functional class III or IV compared to placebo (HR: 0.24; 95% CI: 0.13, 0.43; p<0.0001). The trial’s composite primary efficacy endpoint events — time to first occurrence of all-cause death, lung transplantation or PAH-worsening hospitalization of ≥24 hours — occurred in 15 WINREVAIR-treated participants (17%) versus 47 placebo-treated participants (55%). Due to overwhelming efficacy based on the primary endpoint result, the ZENITH trial was stopped early at the interim analysis and patients were offered the opportunity to receive WINREVAIR through an open-label long-term follow-up study. “For patients with PAH, the risk of serious events such as hospitalization, transplantation or death remains unacceptably high despite being maximally treated with traditional therapies,” said Dr. Vallerie McLaughlin**, Kim A Eagle MD Endowed Professor of Cardiovascular Medicine and Director, Pulmonary Hypertension Program, University of Michigan in Ann Arbor. “Results from the pivotal ZENITH trial add to the growing body of data and support the potential for WINREVAIR as standard of care.” Healthcare providers should monitor hemoglobin and platelets before each dose of WINREVAIR for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required. WINREVAIR may increase hemoglobin and may lead to erythrocytosis, which if severe may increase the risk of thromboembolic events or hyperviscosity syndrome. WINREVAIR also may decrease platelet count and lead to severe thrombocytopenia, which may increase the risk of bleeding; thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Treatment should not be initiated if platelet count is <50,000/mm3. See additional Selected Safety Information below. The most common adverse reactions (≥10% for WINREVAIR and at least 5% more than placebo) in ZENITH were infections (67.4% vs 44.2%), epistaxis (45.3% vs 9.3%), diarrhea (25.6% vs 17.4%), telangiectasia (25.6% vs 3.5%), increased hemoglobin (15.1% vs 1.2%), rash (10.5% vs 4.7%), erythema (10.5% vs 3.5%) and gingival bleeding (10.5% vs 2.3%).The median duration of exposure was longer in the WINREVAIR group (435 days) than in the placebo group (268 days). In the WINREVAIR group, 1 patient (1%) discontinued study intervention due to an adverse event, compared with 4 patients (5%) in the placebo group. “Merck’s leadership in PAH research is anchored in a comprehensive clinical program that continues to advance science and deliver meaningful evidence for physicians and patients,” said Dr. Joerg Koglin, senior vice president, global clinical development, Merck Research Laboratories. “This approval represents another step forward in our mission to deliver on the promise of WINREVAIR, an activin signaling inhibitor with an indication recognizing its impact to adult patients with PAH on the risk of clinical worsening events, including death, lung transplantation and PAH hospitalization." *World Health Organization **Dr. McLaughlin is a member of the adult sotatercept steering committee, an investigator in the ZENITH study and a paid consultant to Merck. About ZENITH The ZENITH study (NCT04896008) was a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which 172 adult participants with PAH (WHO FC III or IV) at high risk of mortality were randomized in a 1:1 ratio to either WINREVAIR (target dose 0.7 mg/kg) (n=86) plus background PAH therapy or placebo (n=86) plus background PAH therapy administered subcutaneously once every 3 weeks. The most common PAH etiologies were idiopathic PAH (50%), PAH associated with connective tissue diseases (CTD) (28%), and heritable PAH (11%). The mean time since PAH diagnosis to screening was 8 years. The study excluded patients diagnosed with human immunodeficiency virus (HIV)-associated PAH, PAH associated with portal hypertension, pulmonary veno-occlusive disease, or pulmonary capillary hemangiomatosis or overt signs of capillary and/or venous involvement. Participants were on background PAH treatment, 72% on triple therapy, 28% on double therapy and 59% on prostacyclin infusion therapy. There were more participants in WHO FC III (74%) compared to WHO FC IV (26%). The REVEAL Lite 2 risk score was <9 for 2% of participants, 9 to 10 for 67% of participants and ≥11% for 30% of participants. The primary efficacy endpoint was time to first confirmed major morbidity or mortality event. Events were defined as all-cause death, lung transplantation or PAH worsening-related hospitalization of ≥24 hours. Secondary endpoints included overall survival and several additional measures. About WINREVAIR™ (sotatercept-csrk) for injection, for subcutaneous use, 45 mg, 60 mg WINREVAIR is FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1 pulmonary hypertension) to improve exercise capacity and World Health Organization (WHO) functional class (FC), and reduce the risk of clinical worsening events, including hospitalization for PAH, lung transplantation and death. WINREVAIR is the first activin signaling inhibitor therapy approved to treat PAH. WINREVAIR improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, WINREVAIR induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodeling and improved hemodynamics. WINREVAIR is the subject of a licensing agreement with Bristol Myers Squibb. Selected Safety Information WINREVAIR may increase hemoglobin (Hgb). Severe erythrocytosis may increase the risk of thromboembolic events or hyperviscosity syndrome. Monitor Hgb before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter, to determine if dose adjustments are required. WINREVAIR may decrease platelet count. Severe thrombocytopenia may increase the risk of bleeding. Thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Do not initiate treatment if platelet count is <50,000/mm3. Monitor platelets before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine whether dose adjustments are required. In clinical studies, serious bleeding (e.g., gastrointestinal, intracranial hemorrhage) was reported in 4% vs 1% (STELLAR) and 7% vs 5% (ZENITH) of patients taking WINREVAIR vs placebo, respectively. Patients with serious bleeding were more likely to be on prostacyclin background therapy and/or antithrombotic agents, or have low platelet counts. Advise patients about signs and symptoms of blood loss. Evaluate and treat bleeding accordingly. Do not administer WINREVAIR if the patient is experiencing serious bleeding. WINREVAIR may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment with WINREVAIR and for at least 4 months after the final dose. Pregnancy testing is recommended for females of reproductive potential before starting WINREVAIR treatment. Based on findings in animals, WINREVAIR may impair female and male fertility. Advise patients on the potential effects on fertility. The most common adverse reactions (≥10% for WINREVAIR and at least 5% more than placebo) occurring in the STELLAR Phase 3 clinical trial were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%), rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea (15.3% vs 10.0%), dizziness (14.7% vs 6.3%) and erythema (13.5% vs 3.1%). The most common adverse reactions in the ZENITH trial were infections (67.4% vs 44.2%), epistaxis (45.3% vs 9.3%), diarrhea (25.6 % vs 17.4%), telangiectasia (25.6 % vs 3.5%), increased hemoglobin (15.1% vs 1.2%), rash (10.5% vs 4.7%), erythema (10.5% vs 3.5%) and gingival bleeding (10.5% vs 2.3%). Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with WINREVAIR, and for 4 months after the final dose. About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research- intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov). Please see Prescribing Information for WINREVAIR (sotatercept-csrk) at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_pi.pdf, Patient Information for WINREVAIR at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ppi.pdf, and Instructions for Use for WINREVAIR (1-vial kit, 2-vial kit) at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf.