Chronic lung disease

PITTSBURGH and BOSTON, May 1, 2025 /PRNewswire/ -- Synhale Therapeutics Inc. (Synhale), a virtual, clinical-stage biotech company committed to transforming Pulmonary Hypertension (PH), announced the acquisition of Telaglenastat (CB-839), the first-in-class glutaminase inhibitor. Synhale is advancing into a Phase 2 clinical program across PH Groups 1-4. "This acquisition represents a unique opportunity to rapidly deliver clinical data addressing a disease with high mortality and substantial market potential," said Chad D. Holland, President & CEO of Synhale. "Leveraging Telaglenastat's prior human clinical experience, our capital-efficient model aims to accelerate transformative therapy to patients while maximizing shareholder value." Stephen Chan, MD, PhD, Founder of Synhale, added, "The unmet patient need in PH is immense, particularly in Groups 2 and 3, where heart failure with preserved ejection fraction (HFpEF) and chronic lung disease remain deadly and underserved. PH with HFpEF carriers a five-year mortality approaching 50%, with similarly grim outcomes in COPD and other lung diseases. By targeting glutaminase activity, we address a fundamental metabolic pathway across all PH groups, offering hope to patients with few options." PH, a severe condition, is characterized by remodeling and stiffening in the pulmonary vasculature and often dramatically increases mortality risk when combined with other conditions. Current classifications fail to reflect the molecular drivers of PH, leaving treatment gaps. Synhale's approach with Telaglenastat targets elevated glutaminase activity, a key driver of vascular, cardiac, and pulmonary pathology, potentially benefiting not only rare Pulmonary Arterial Hypertension (Group 1) and chronic thromboembolic disease (Group 4) but also more prevalent PH linked to heart failure (Group 2) and chronic lung disease (Group 3). Telaglenastat, previously dosed in over 800 patients during oncology development, is Phase 2-ready with extensive preclinical data for PH. Synhale's clinical advancement strategy addresses an estimated $12B-$24B market opportunity across PH Groups 1-4. "We are embarking on our next round of fundraising to advance Telaglenastat through critical clinical milestones," added Holland. "Our virtual operating model enables us to be capital-efficient while maintaining world-class expertise focused on delivering breakthrough therapies for PH patients." About Synhale Therapeutics Synhale Therapeutics Inc. is a virtual biotech company dedicated to transforming Pulmonary Hypertension treatment by targeting elevated glutaminase activity—a key driver of vascular stiffness, fibrosis, and proliferation. Synhale's mission is to redefine PH treatment through mechanism-driven therapies that address the root cause of this disease. For Media & Investor Inquiries: Chad D. Holland [email protected] 617-758-8643 SOURCE Synhale Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor) recommended for people with CF ages 6 years and older with at least one non-class I mutation in the CFTR gene - - In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and superior compared to KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) at reducing sweat chloride - LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor)for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Our goal has always been to serially innovate to help people with cystic fibrosis live healthier and longer lives. If approved, this new medicine would be indicated for people with CF ages 6 years and older with at least one non-class I mutation, meaning more patients would be eligible for a medicine that gets them closer to normal levels of sweat chloride,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “CFTR modulators have already revolutionized the way we treat CF and I am encouraged that, if approved, this medicine could advance CF treatment even further,” said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin. “The results we saw from the two deutivacaftor/tezacaftor/vanzacaftor Phase 3 clinical trials were motivating as they showed non-inferiority in ppFEV1 and superior improvement of sweat chloride levels compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.” ALYFTREK® is currently licensed in the U.S. and UK and is under regulatory review in Canada, Switzerland, Australia and New Zealand. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease. About ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic, M.D., and Professor Marcus A. Mall, M.D., in this press release, statements regarding the expected eligible patient population for ALYFTREK, if approved, and statements regarding the potential benefits of ALYFTREK. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities in the EU may not approve ALYFTREK on a timely basis or at all, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Vertex Pharmaceuticals IncorporatedInvestors:InvestorInfo@vrtx.com Media:mediainfo@vrtx.comorInternational: +44 20 3204 5275

BRIDGEWATER, N.J., April 24, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that it has called all $569.5 million aggregate principal amount of its remaining outstanding 0.75% Convertible Senior Notes Due 2028 (the "Notes") (CUSIP No. 457669AB5) for redemption on June 6, 2025 (the "Redemption Date"). Insmed is redeeming the Notes as permitted under Section 11.03 of the indenture governing the Notes (the "Indenture"). Redemption Process The redemption price will be payable on the Redemption Date in cash and equal to 100% of the principal amount of the Notes outstanding on the Redemption Date, plus accrued and unpaid interest on such Notes to, but excluding, the Redemption Date (the "Redemption Price"). For each $1,000 principal amount of Notes, the Redemption Price will be equal to approximately $1,000.10. Unless Insmed defaults in making the payment of the Redemption Price, interest on the Notes will cease to accrue on and after the Redemption Date. For all Notes surrendered in book-entry form, payment of the Redemption Price will be made through the facilities of the Depository Trust Company ("DTC"), and all redeemed Notes in book-entry form will be surrendered for payment of the Redemption Price in accordance with the applicable rules and procedures of DTC. The paying agent is Computershare Trust Company, N.A. and the address of the paying agent for delivery of any Notes in certificated form is Corporate Trust Operations, 1505 Energy Park Drive, St. Paul, MN 55108. Right to Convert the Notes Holders of the Notes may surrender their Notes (or any portion thereof having a principal amount that is an integral multiple of $1,000) for conversion at any time prior to 5:00 p.m. (New York City time) on June 4, 2025 or, if the Company fails to pay the Redemption Price on the Redemption Date, such later date on which the Redemption Price is paid. To convert any Note, the holder must comply with the applicable rules and procedures of DTC. The Company has elected to settle any conversions of Notes in shares of common stock in accordance with the Indenture. As of April 24, 2025, the conversion rate of the Notes is 30.7692 shares of common stock of the Company per $1,000 principal amount of Notes, which is equivalent to a conversion price of approximately $32.50 per share of common stock. Based on this conversion rate, an aggregate of up to 17,523,336 shares of common stock will be issued if all of the Notes are converted. This press release shall not constitute a notice of redemption or convertibility of the Notes. This press release is neither an offer to sell nor a solicitation of an offer to buy the Notes or any other securities and shall not constitute an offer to sell or a solicitation of an offer to buy, or a sale of, the Notes or any other securities in any jurisdiction in which such offer, solicitation or sale is unlawful. No representation is made as to the correctness or accuracy of the CUSIP number either as printed on the Notes or as contained in this press release. About Insmed Insmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inflammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue. Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending four consecutive years as the No. 1 Science Top Employer. Visit to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements. The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any forward-looking statements. The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 and any subsequent Company filings with the Securities and Exchange Commission (SEC). The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Contact: Investors: Bryan Dunn Vice President, Investor Relations (646) 812-4030 [email protected] Media: Claire Mulhearn Vice President, Corporate Communications (862) 842-6819 [email protected] SOURCE Insmed Incorporated WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED