Acceleron Pharma, Inc.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) announced on Tuesday the approval of a new drug developed by Merck for the treatment of a rare lung disease. This approval further solidifies the pharmaceutical company’s success in the potential blockbuster product sector. The drug, named sotaterept, was previously approved in the US in March 2024 and is marketed under the brand name Winrevair. It has also received approval in the EU and over 30 other markets.The MHRA's approval is based on research data involving 323 patients with pulmonary arterial hypertension. In this study, sotaterept demonstrated significantly better effects on improving patients' exercise capacity compared to a placebo. The development of sotaterept is part of Merck's acquisition of Acceleron Pharma for $11.5 billion in 2021. Through this acquisition, Merck obtained sotaterept and has been expanding its cardiovascular drug portfolio to address potential sales impacts from the expiration of patents on its bestselling cancer treatment Keytruda by the end of the century. Regulators noted that sotaterept can be used to treat adult patients with moderate to significant physical activity limitations to help improve their exercise capacity. The recommended dosing schedule is a self-injection every three weeks. According to the National Health Service (NHS) data, there were 9,263 active pulmonary arterial hypertension patients in the UK as of March 2024. Pulmonary arterial hypertension refers to elevated blood pressure in the vessels supplying blood to the lungs, making it more difficult for the heart to pump blood and potentially leading to organ failure. Sotaterept works by targeting the activation protein, which plays a key role in pulmonary artery narrowing. Since its launch in March, Winrevair has achieved $149 million in sales. According to data compiled by LSEG, analysts expect its sales to exceed $1 billion by 2025. In the US, the therapy is priced at $14,000 per vial. Merck has not yet responded to inquiries from reporters regarding the pricing and availability of the therapy in the UK.

MSD acquired the therapy from Acceleron Pharma. Credit: Copyright © 2024 Merck & Co., Inc., Rahway, NJ, USA and its affiliates. All rights reserved. MSD has received the UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for sotatercept, marketed as Winrevair, to treat pulmonary arterial hypertension (PAH) in the adult population. The therapy is used along with other PAH medications to improve exercise capacity in patients with moderate to severe limitations in physical activity. Designed to address the underlying causes of PAH, which involves high blood pressure in the pulmonary arteries that can lead to right heart damage, sotatercept operates by easing the narrowing of these arteries, helping the heart pump blood more efficiently to the lungs and improving physical activity capabilities. A pivotal study involving 323 PAH subjects demonstrated the therapy’s effectiveness compared to placebo in enhancing exercise capacity. The main measure of this was the change in the six-minute walk distance (6MWD) after 24 weeks of treatment. Subjects who received the therapy, in addition to their standard PAH therapy, could walk approximately 34 metres farther than before treatment, while those on placebo improved by only one metre. The median treatment difference between the therapy and placebo groups stood at 40.8 metres. The UK regulator has recommended a dosing schedule of one injection every three weeks, which patients can self-administer. Common adverse events reported in more than one in 10 subjects include low blood platelet count, elevated haemoglobin levels and skin rash. The safety and effectiveness of the therapy will continue to be monitored closely by the agency. The approval of sotatercept came through the International Recognition Procedure (IRP). MSD’s acquisition of Acceleron Pharma in September 2021 for $11.5bn, at $180 per share, led to the addition of sotatercept to its portfolio. MHRA healthcare quality and access interim executive director Julian Beach stated: “Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us. We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep its safety under close review.”

Former CEO of NPS Pharma brings strong biotech expertise to further accelerate GEn1E’s development and commercialization of dual signal modulators as novel precision therapies PALO ALTO, Calif., Dec. 11, 2024 (GLOBE NEWSWIRE) -- GEn1E Lifesciences (GEn1E), a clinical-stage, Phase 2 company, focused on accelerating novel therapies for inflammatory and rare diseases using machine learning, is pleased to announce the appointment of François Nader, MD, MBA as chair of its Board of Directors, effective immediately. "We are thrilled to welcome François to GEn1E’s Board of Directors as Chair," said Ritu Lal, PhD, CEO and Co-founder of GEn1E Lifesciences. "François’ deep expertise in biopharmaceutical innovation, strategic leadership, and rare diseases aligns perfectly with our mission to develop novel therapies for patients suffering from inflammatory and rare diseases. His transformational guidance will be invaluable as we continue to accelerate our pipeline to bring precision therapies to patients in need." "We are entering a pivotal time for treating inflammatory and rare diseases, and I'm excited to support GEn1E as it pioneers innovative therapies that could redefine patient care," said Dr. Nader. "I look forward to collaborating with Ritu and the GEn1E team to fully harness the potential of precision signal modulators and to accelerate the company's pipeline in addressing devastating diseases for patients." Dr. Nader currently serves as an Independent Board Director at Moderna and Ring Therapeutics, and Senior Advisor at Blackstone Life Sciences. Dr. Nader is the Chairman of BioLink.org and serves on the Board of Trustees for the Lebanese American University. Dr. Nader brings 30+ years of experience in the life sciences industry, including a highly successful tenure at NPS Pharmaceuticals as President and CEO where he led the company through a period of remarkable growth and achievement, culminating in its acquisition by Shire Pharmaceuticals for $5.2 billion. Formerly, François served as a chair or director of the Board of several public and private companies including chairman for Acceleron Pharma (acquired by Merck for $11.5B), and member of the Board for Alexion Pharmaceuticals (acquired by AstraZeneca for $39B) and Baxalta (acquired by Shire for $32B). Dr. Nader earned his French Doctorate in Medicine from St. Joseph University (Lebanon) and his Physician Executive MBA from the University of Tennessee. About GEn1E Lifesciences: GEn1E Lifesciences is a clinical-stage, Phase 2 company accelerating novel, dual signal modulator precision therapies for rare and inflammatory diseases. GEn1E Lifesciences is based in Palo Alto, California, with a laboratory in Mountain View, California. GEn1E has developed a well-differentiated "Platform-in-a-Mechanism" drug development model that uses AI which spans the entire end-to-end drug development cycle. The company has accelerated several milestones to build a portfolio of 21+ novel Dual Signal Modulators across two targets, which are actively being developed as first-in-class therapies for rare and inflammatory diseases with lead compound in Phase 2 currently enrolling patients in the US. For more information on GEn1E, visit www.gen1e.com and follow GEn1E on LinkedIn. Contacts:PR: info@gen1elifesci.comPartnering: partnering@gen1elifesci.comWeb: http://www.gen1e.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/30ec883f-2e8f-4aae-833c-40edef90287a