KYORIN Pharmaceutical Co., Ltd.

Study enrolled 268 patients, exceeding target enrollment. Largest interventional study ever to be conducted in pulmonary sarcoidosis. Topline data are expected in the third quarter of 2025. July 22, 2024 -- aTyr Pharma, Inc. (Nasdaq: ATYR), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that it has completed enrollment in its global pivotal Phase 3 EFZO-FIT™ study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease with limited treatment options. The study enrolled 268 patients at 85 centers in 9 countries, exceeding target enrollment. Topline data from the study are expected in the third quarter of 2025. “Completing enrollment in this landmark study is an important milestone that brings us one step closer to delivering a potentially groundbreaking treatment to address the significant unmet need for pulmonary sarcoidosis patients,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “We are grateful to all of the patients and their caregivers, our principal investigators and their teams, our many advocacy partners and our partner Kyorin Pharmaceutical Co., Ltd., who helped make this accomplishment possible. The historic number of patients enrolled in this study signifies the strong patient demand for a new treatment option such as efzofitimod.” “This is a monumental achievement for the sarcoidosis community. It is by far the largest interventional study ever to be conducted in sarcoidosis. We expect the results of this trial to yield valuable insights that will inform sarcoidosis research and treatment in the years to come,” said Daniel A. Culver, D.O., Chair of the Division of Pulmonary Medicine at The Cleveland Clinic and Lead Primary Investigator of the study. “We are optimistic based on the positive Phase 1b/2a results that efzofitimod could be a potentially transformative therapy for sarcoidosis patients, which is greatly needed. We look forward to the readout from this study in 2025.” Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod has received orphan drug designation in the U.S., E.U. and Japan for sarcoidosis and Fast Track designation in the U.S. for pulmonary sarcoidosis. The EFZO-FIT™ study is a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously once a month for a total of 12 doses. The study enrolled 268 subjects with pulmonary sarcoidosis at multiple centers in the United States, Europe, Japan and Brazil. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of lung function and sarcoidosis symptoms. More information on the EFZO-FIT™ study is available at www.clinicaltrials.gov (NCT05415137) and www.efzofit.com. Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT™ study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes. aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. 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The European Commission approved Pierre Fabre Laboratories’ Obgemsa (vibegron) for the symptomatic treatment of overactive bladder (OAB) syndrome in adults, more than three years after being cleared by the FDA. The decision follows a positive stance adopted by the European Medicines Agency's (EMA) drug advisory body in April. Eric Ducournau, CEO of Pierre Fabre said the approval “will allow European patients to benefit from a new therapeutic option for OAB,” which is estimated to affect more than 70 million people in the region. The approval was backed by findings from the Phase III EMPOWUR trial and a 52-week extension study, which showed that the 75-mg daily dose of Gemtesa significantly improved urgency, increased urination frequency, and urge urinary incontinence in patients with OAB compared with placebo.In 2022, Pierre Fabre gained an exclusive license to commercialise vibegron in the EU from Sumitomo Pharma’s subsidiary Urovant Sciences, which markets it as Gemtesa in the US. The once-daily oral drug is also approved to treat symptoms of OAB in Japan and South Korea under the name Beova, where it is marketed by Kyorin Pharmaceutical and JEIL Pharmaceutical, respectively.

SAN DIEGO--(BUSINESS WIRE)-- Actio Biosciences, Inc., a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Ben Cravatt, Ph.D., has joined the company’s board of directors. Dr. Cravatt is professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute. His research group is interested in developing chemical proteomic technologies that enable protein and drug discovery on a global scale and applying these methods to characterize biochemical pathways that play important roles in human physiology and disease. “By combining genuine expertise across biology, chemistry and genetics, Ben has pioneered an approach to drug discovery and development that sits at the heart of Actio’s mission,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “We are thrilled to welcome Ben to our board and look forward to working together to discover and develop new life-changing medicines.” “Years of research has taught us that the best targets are those supported by human genetics – and it is becoming clear that specific kinds of genetic information are especially informative,” said Dr. Cravatt. “Actio’s laser-focused approach targeting genes responsible for rare devastating diseases promises new treatments for those diseases and a powerful framework for rational expansion to more common indications sharing mechanistic biology.” In addition to his role at Scripps, Dr. Cravatt is a co-founder of Activx Biosciences (acquired by Kyorin Pharmaceuticals), Abide Therapeutics (acquired by Lundbeck Pharmaceuticals) and Vividion Therapeutics (acquired by Bayer AG). His honors include a Wolf Prize in Chemistry, a Searle Scholar Award, the Eli Lilly Award in Biological Chemistry, a Cope Scholar Award, the ASBMB Merck Award, the Royal Society of Chemistry Jeremy Knowles Award, and memberships in the National Academy of Sciences, National Academy of Medicine, and American Academy of Arts and Sciences. Dr. Cravatt obtained his undergraduate education at Stanford University, receiving a B.S. in biological sciences and a B.A. in history. He received a Ph.D. from The Scripps Research Institute (TSRI) in 1996 and joined the faculty at TSRI in 1997. About Actio Biosciences Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com.