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.
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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.