Nephrosis

Amicus licenses exclusive U.S. rights to commercialize DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS) DMX-200 is in a pivotal Phase 3 trial for FSGS, a rare and fatal kidney disease with no FDA-approved therapies Dimerix successfully completed Type C meeting with the FDA in March 2025, aligning on proteinuria as the primary endpoint for approval Dimerix to receive US$30 million (~AU$48 million) upfront payment, up to US$560 million (~AU$892 million) for success-based milestone payments, in addition to tiered royalties on DMX-200 net U.S. sales MELBOURNE, Australia and PRINCETON, NJ, USA I April 30, 2025 I Dimerix Limited (ASX: DXB, “Dimerix”) and Amicus Therapeutics (Nasdaq: FOLD, “Amicus”) today announced that the two companies have entered into an exclusive license agreement for the commercialization of Dimerix’ Phase 3 drug candidate DMX-200 for all indications, including FSGS, in the United States (U.S.). Dimerix retains all rights to commercialize DMX-200 in all territories other than those already exclusively licensed. DMX-200 is a small molecule inhibitor of the chemokine receptor 2 (CCR2) under development in a pivotal Phase 3 study, ACTION3, for the treatment of Focal Segmental Glomerulosclerosis (FSGS) kidney disease. In early 2024, Dimerix reported positive interim results from the ACTION3 trial in FSGS showing DMX-200 was performing better than placebo in reducing proteinuria with no safety concerns to date. Full enrollment of ACTION3 is expected by year-end 2025. An additional blinded interim analysis is planned once the revised primary and secondary endpoints have been pre-specified in the protocol and agreed with the FDA. In a March 2025 Type C meeting, Dimerix successfully aligned with the FDA on proteinuria as an appropriate primary endpoint for traditional marketing approval for DMX-200. “Amicus is thrilled to enter into this collaboration with Dimerix to bring DMX-200 to patients in the U.S., and we are incredibly impressed by their achievements to date. We look forward to leveraging our regulatory, commercial, medical, and advocacy capabilities to bring this potentially transformative treatment to people living with FSGS in the U.S.,” said Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics. “This licensing agreement represents a major step forward in our strategy to strengthen our portfolio and fully aligns with our mission to develop and deliver transformative medicines for people living with rare diseases.” “We are delighted to partner with Amicus in the United States. The Amicus team has a remarkable history of successfully delivering rare disease medicines to those in need. Their expertise and resources will be crucial to help achieve our mutual objective of commercializing this innovative treatment,” said Dr. Nina Webster, CEO and Managing Director of Dimerix. “I’m grateful to the dedicated Dimerix team, trial participants, and investigators for their continued commitment to developing a new therapy for patients with FSGS who currently have a poor prognosis and very limited treatment options.” Dimerix will continue to fund and execute the ACTION3 study, and Amicus will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, Amicus will have the exclusive rights to develop DMX-200 in other future indications in the United States. Amicus and Dimerix will form a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S. The agreement otherwise contains terms common for an arrangement of this kind. In exchange for these rights, Dimerix will receive a US$30 million (~AU$48 million) upfront payment. The next potential milestone payment is based on positive data from the Phase 3 trial in FSGS. In total, Dimerix is eligible to receive potential success-based development and regulatory milestone payments of up to US$75 million (~AU$119 million) until FDA approval of DMX-200 in FSGS, US$35 million (~AU$56 million) on first sale, commercial sales milestone payments of up to US$410 million (AU$653 million), and tiered royalties from the low-teens to low-twenties percentages of DMX-200 net sales in the U.S. In addition, Dimerix is eligible to receive up to US$40 million (~AU$64 million) in milestone payments for potential future indications. The upfront payment from Amicus will be funded with cash on hand. All contracted financial terms are denominated in U.S. dollars. Evercore Partners International LLP is acting as exclusive financial advisor to Dimerix, and Cooleys LLP is serving as Dimerix legal advisor. Wilson Sonsini Goodrich & Rosati is serving as Amicus legal advisor. Authorized for lodgment with ASX by the Board of Dimerix. About ACTION3 Phase 3 Study The Phase 3 study, which is titled “Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis”, or ACTION3 for short, is a pivotal (Phase 3), multi-center, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients will be randomized to receive either DMX-200 (120 mg capsule twice daily) or placebo. The single Phase 3 trial in FSGS patients has two interim analysis points built in that are designed to capture evidence of proteinuria and kidney function (eGFR slope) during the trial, aimed at generating sufficient evidence to support marketing approval. Further information about the study can be found on ClinicalTrials.gov (Study Identifier: NCT05183646) or Australian New Zealand Clinical Trials Registry (ANZCTR) (Study Identifier ACTRN12622000066785). About DMX-200 DMX-200 is a chemokine receptor (CCR2) antagonist administered to patients already receiving an angiotensin II type I receptor (AT1R) blocker, the standard of care treatment for hypertension and kidney disease. DMX-200 is protected by granted patents in various territories until 2032, with patent applications submitted globally that may extend patent protection to 2042, in addition to Orphan Drug Designation granted by the FDA in the United States. About FSGS FSGS is a rare, serious kidney disorder characterized by progressive scarring (sclerosis) in parts of the glomeruli—the kidney’s filtering units. This scarring leads to proteinuria, progressive loss of kidney function, and often end-stage renal disease. FSGS is increasingly understood to have an inflammatory component, with monocyte and macrophage activation contributing to glomerular injury. In the United States, more than 40,000 people are estimated to be living with FSGS, including both adults and children. There are no therapies specifically approved for FSGS in the U.S., and management relies on non-specific immunosuppressive and supportive therapies. In patients with progressive or treatment-resistant FSGS, the average time from diagnosis to end-stage kidney disease can be as short as five years. Even among those who undergo kidney transplantation, disease recurrence occurs in up to 60% of cases, underscoring the urgent need for new, disease-modifying treatments. About Dimerix Limited Dimerix (ASX: DXB) is a clinical-stage biopharmaceutical company working to improve the lives of patients with inflammatory diseases, including kidney diseases. Dimerix is currently focused on developing its proprietary Phase 3 product candidate DMX-200, for Focal Segmental Glomerulosclerosis (FSGS) kidney disease, and is also developing DMX-700 for respiratory disease. DMX-200 and DMX-700 were both identified using Dimerix’ proprietary assay, Receptor Heteromer Investigation Technology (Receptor-HIT), which is a scalable and globally applicable technology platform enabling the understanding of receptor interactions to rapidly screen and identify new drug opportunities. For more information, please visit the company’s website at www.dimerix.com and follow on X and LinkedIn . About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com , and follow on X and LinkedIn . SOURCE: Amicus Therapeutics

- 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

MISSION, Kan., April 21, 2025 /PRNewswire/ -- (Family Features) Senior year of high school is a time for big changes for most people, but for Emani McConnell-Brent, she did not expect it would include changes to her health. After being rushed to the emergency room with severe stomach pain, McConnell-Brent learned the problem was her kidneys and she was diagnosed with a kidney disease called focal segmental glomerulosclerosis (FSGS). Continue Reading Photo courtesy of American Kidney Fund Photo courtesy of American Kidney Fund After her diagnosis, McConnell-Brent struggled to get the right medicines to treat her disease. She was encouraged to undergo a genetic test and discovered her FSGS was the result of APOL1-mediated kidney disease (AMKD), a rapidly progressive genetic kidney disease that can cause kidney failure. The diagnosis brought McConnell-Brent some relief, both physically and emotionally. "Knowing it's genetic and knowing I'm taking care of myself makes a big difference in my mental state," said McConnell-Brent, now 21 and an ambassador for the American Kidney Fund. Everyone has two copies of the apolipoprotein L1 (APOL1) gene – one from each parent – but Black Americans of West and Central African ancestry are more likely to have changes (variants or mutations) in their APOL1 gene that cause AMKD. This genetic form of kidney disease can develop even at a young age, in otherwise healthy people, and can advance faster than other kidney diseases. An estimated 13% of Black Americans have the two APOL1 gene variants that are associated with AMKD. Those who have variants in both copies of the APOL1 gene have a 1 in 5 chance of developing kidney disease. "A lot of people don't even know about how prevalent the APOL1 gene is in the African American community," McConnell-Brent said. Early diagnosis of AMKD can keep your kidneys working longer, delaying the need for dialysis or a kidney transplant. If you have kidney damage, symptoms may not occur until your kidneys are close to failing. As kidney damage worsens, one or more of these symptoms may occur: Protein in urine Swelling in legs or weight gain Feeling weak or tired High blood pressure The only way to get an official diagnosis for AMKD is through a genetic test. You can find more information about the risks and benefits of genetic testing online. "If you got genetic testing, you would have the facts of what your genes are telling you," McConnell-Brent said. "Your genes are telling you a story of what did happen, is happening and could happen." This AMKD Awareness Day, on April 29, become APOL1 Aware by learning how your genes impact your kidney health and help build awareness in your community by visiting KidneyFund.org/APOL1Aware. Michael French [email protected] 1-888-824-3337 editors.familyfeatures.com About Family Features Editorial Syndicate A leading source for high-quality food, lifestyle and home and garden content, Family Features provides readers with topically and seasonally relevant tips, takeaways, information, recipes, videos, infographics and more. Find additional articles and information at Culinary.net and eLivingToday.com. SOURCE Family Features Editorial Syndicate WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED