Glomerulonephritis, Membranous

- 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

Welcome back to Endpoints Weekly! This week’s recap kicks off with Andrew Dunn’s examination of the biotech playbook for surviving market turmoil. As former Alnylam CEO John Maraganore put it, recent events have “put biotech CEOs in a very difficult set of shoes compared to what we’ve had to deal with in the past.” See below for more details on how companies might handle the uncertainty. Meanwhile, our reporters tracked news on potential pharma-specific tariffs, which President Donald Trump said would be arriving “very shortly.” And a congressional commission is pushing for a big investment in the biotechnology sector to keep up with Chinese competition. If you’ve got a bit of downtime this weekend, grab a cup of tea and peruse our handful of Q&As out this week, including conversations with GLP-1 co-discoverer Jens Juul Holst, gene editing pioneer David Liu and Leerink Partners senior managing director David Risinger. See you on Monday! — Nicole DeFeudis It’s a tough market for biotech companies, but the sector has been here before. Endpoints News’ Andrew Dunn reports that the playbook remains largely the same. Take a look here at the 10 moves Dunn said biotech CEOs are likely considering, including: 🪓 Buckle down and extend the cash runway: Drug programs on the chopping block can be out-licensed or spun into new subsidiaries. Or they may be ultimately shut down. The goal is to extend cash to make it through to catalysts for the most promising drug programs, Dunn reports. 💰 Selling royalty rights: For biopharmas with a potential approved drug in sight, royalty deals can raise capital without diluting shareholders. But they give away a chunk of future upside. 🤝 Pharma partnerships: They bring in cash, keep research going, and provide a credibility boost. But as former Vertex Pharmaceuticals CEO Joshua Boger told Dunn over a year ago: “Realize they’re going to be painful, it’s going to be horrible, and you need to do it.” Read the whole playbook here. And click here for more insight from Drew Armstrong and Max Bayer’s Q&A this week with Leerink Partners senior managing director David Risinger. “In a typical financial crisis, corporations have to think about their cash flows and balance sheets,” Risinger said. “But then the FDA-specific turmoil that is happening really makes it difficult for corporations to plan for the future.” 🌎 Much of the world got a reprieve from President Donald Trump’s tariffs this week. But the president said earlier in the week that pharma-specific tariffs would be arriving “very shortly.” Analysts weighed in on what a potential rollout could look like here. “We’re going to tariff our pharmaceuticals and once we do that, they’re going to come rushing back into our country,” Trump said at a National Republican Congressional Committee event. Trump has previously praised companies like Eli Lilly , Johnson & Johnson and Merck for boosting their US footprint. All three companies have announced multibillion-dollar investments to build or expand US factories. On Thursday, Swiss-based Novartis said it would invest $23 billion in American manufacturing and R&D over the next five years. A Novartis spokesperson declined to clarify how much of the $23 billion investment had already been planned or disclosed. Meanwhile, the European Federation of Pharmaceutical Industries and Associations warned of a potential “exodus” to the US. The group said in a statement on Tuesday that “unless Europe delivers rapid, radical policy change then pharmaceutical research, development and manufacturing is increasingly likely to be directed towards the US.” It called for “immediate action” on several fronts, including strengthening intellectual property provisions and “adopting a world leading regulatory framework conducive to innovation.” 💸 Amid the burgeoning trade war with China, a Congressional commission is looking to find a way for American biotechs to remain competitive. This week, the National Security Commission on Emerging Biotechnology — which is made up of members from both chambers of Congress as well as civilian experts — urged a $15 billion investment in the sector to keep up with Chinese competition. The timing comes amid cuts across the federal government, including at HHS, which laid out plans last month to cut thousands of employees. Sen. Todd Young (R-IN) recognized “the fiscal challenges associated with persuading colleagues to add new priorities to the list.” But he said the focus should be on setting the right priorities without needing to “fund everything.” This week’s recommendation echoes efforts of the Biosecure Act, a bill introduced last year. The legislation sought to ban US reliance on suppliers like WuXi AppTec, but didn’t eventually become law. Unlike the Biosecure Act, the commission’s report does not name specific Chinese companies. 💰 Merida Biosciences emerged from stealth this week with $121 million in its pockets . Their goal? Create and develop new medications for autoimmune diseases and allergies, with an initial focus in experimental drugs for a thyroid condition called Graves disease, food allergy and primary membranous nephropathy, an autoimmune kidney disease. Beating Xolair will be a challenge and may take years. Last year, the Roche and Novartis drug racked up $4.4 billion in sales, and other companies have tried to crack food allergy before. But CEO Adam Townsend thinks food allergy can be Merida’s “gateway into showing our science and how it works there.” 💬 Q&As with industry and academic thought leaders filled Endpoints’ newsletters this week, and what better way to start your weekend than by reading what they had to say? Elizabeth Cairns sat down with GLP-1 co-discoverer Jens Juul Holst to discuss the future of incretin-based drugs, and Ryan Cross had a conversation with gene editing pioneer David Liu about the economic and political turmoil in science. Both came from the sidelines of the Breakthrough Prizes in Life Sciences, also known as the “Oscars of Science.” We hope you enjoy.

The funding was co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock Ventures. Credit: Dalin Ou via Getty Images. US biotech Merida Biosciences has launched with $121m in Series A financing to develop therapeutics targeting disease-causing antibodies in autoimmune diseases and allergies. The financing round was co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock Ventures, with participation from GV and Perceptive Xontogeny Venture (PXV) Funds. The biotech is advancing a proprietary protein engineering platform to develop fragment crystallisable (Fc)-based biologics that selectively bind and eliminate pathogenic antibodies while sparing healthy components of the immune system. Merida’s therapeutics also aim to deplete the B cells responsible for producing the harmful antibodies. Merida’s lead programme will focus on Graves’ disease, an autoimmune condition that causes the thyroid to overproduce hormones, resulting in hyperthyroidism. The company is positioning its candidate as a more targeted alternative to antithyroid drugs (ATDs), which while being standard of care (SOC), often fail to provide adequate disease control. The company claims its approach could offer a more convenient dosing schedule and an easier route of administration compared to current and emerging treatments. Merida is not the only biotech setting its sights on the Graves’ disease market. Immunovant is developing batoclimab, a monoclonal antibody currently in a Phase IIa trial (NCT05907668) for Graves’ disease patients who remain uncontrolled on ATDs. Interim data released in September 2024 showed a 76% response rate at 12 weeks. If batoclimab makes it to market, GlobalData projects that it will generate $872m in global sales by 2030. GlobalData is the parent company of Pharmaceutical Technology. In addition to Merida and Immunovant, Biohaven is developing BHV-1300, a small molecule therapy that targets and degrades immunoglobulin G1 (IgG1) autoantibodies that cause Graves’ disease. BioHaven previously announced plans to launch a Phase II study of the drug in mid-2025. Following its lead Graves’ disease programme, Merida plans to pursue candidates for food allergies and primary membranous nephropathy, a rare autoimmune kidney disorder. Merida will be led by CEO Adam Townsend, former COO of Apellis Pharmaceuticals and ex-Biogen executive. He will work alongside chief scientific officer Dario Gutierrez, a former executive director at MSD and current consultant at Third Rock Ventures. “For the first time, we have the potential to precisely target the clear pathogenic drivers of an intractable set of diseases with absolute selectivity and a degree of completeness and durability that has not been achievable through any approach to date,” said Townsend in the 8 April announcement.