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4D Molecular Therapeutics Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials & Development Plans for Fabry Disease Cardiomyopathy at WORLDSymposium™
All three patients with 12 months of follow-up demonstrated improvement in multiple FDA-recommended cardiac endpoints at relatively low dose of 1E13
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"All three patients with 12 months of follow-up demonstrated improvement in multiple FDA-recommended cardiac endpoints at relatively low dose of 1E13 vg/kgCardiac biopsy demonstrated selective and widespread transgene expression within ~50% of cardiomyocytes Engaging with FDA to lift clinical hold and resume enrollment with updated exclusion criteria and highly effective rituximab/sirolimus immunosuppressive regimen to reduce risk of atypical hemolytic uremic syndrome (“aHUS”)Otherwise, generally well-tolerated with no liver, heart, or dorsal root ganglia (“DRG”) toxicity observedCompany to host live webcast today at 4:30 p.m. EST EMERYVILLE, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, ‘4DMT’), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today announced updated interim safety and efficacy data from the two 4D-310 INGLAXA Phase 1/2 clinical trials for Fabry disease cardiomyopathy. All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints. Treatment was generally well tolerated, with transient acute aHUS being the only significant adverse event. Following a detailed investigation into the etiology of aHUS, 4DMT is engaging with the FDA to resume enrollment based on updated exclusion criteria and the highly effective rituximab/sirolimus immunosuppressive regimen. Data will also be presented at the WORLDSymposium™ 2023 in Orlando, Florida on February 25th, 2023. “As demonstrated by the positive cardiac outcomes and biopsy biomarker data, these clinical proof-of-concept results with 4D-310 mark another important milestone for 4DMT,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “This is our third proprietary vector that has been validated in clinical trials across three different therapeutic areas, which further validates our Therapeutic Vector Evolution platform. We are developing 4D-310 for the treatment of Fabry disease cardiomyopathy, which is the primary cause of death and not addressed by current therapies. In addition, our focus on patient safety led us to voluntarily pause enrollment on our two INGLAXA trials in January 2023 after observing an aHUS dose-limiting toxicity, and FDA subsequently put the progra...