4D Molecular Discloses Updated Data From Currently Paused Fabry Disease Cardiomyopathy Study

  • 4D Molecular Therapeutics Inc FDMT announced updated interim safety and efficacy data from the two 4D-310 INGLAXA Phase 1/2 trials for Fabry disease cardiomyopathy. 
  • All three patients with 12 months of follow-up demonstrated improved cardiac contractility, exercise capacity, and quality of life endpoints. 
  • Treatment was generally well tolerated, with transient acute aHUS being the only significant adverse event. 
  • Also Read: BMO Capital Markets Says 4D Molecular Could Be 'Attractive Acquisition Target.'
  • 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. 
  • aHUS (atypical hemolytic uremic syndrome) is a rare disease that causes tiny blood clots in the small blood vessels.
  • The company voluntarily paused enrollment in two INGLAXA trials in January 2023 after observing an aHUS dose-limiting toxicity, and FDA subsequently put the program on clinical hold. 
  • 4DMT anticipated that any future clinical development of 4D-310 would be with rituximab-sirolimus under a new clinical protocol(s) and amended or new INDs.
  • The company is confident that the rituximab and sirolimus immunosuppressive regimen and updated exclusion criteria will mitigate safety risks and improve patient benefits.
  • Price Action: FDMT shares are up 0.94% at $20.44 on the last check Thursday.
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