4D Molecular: Updated Data From Currently Paused Fabry Disease Cardiomyopathy Study
- 4D Molecular Therapeutics Inc 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.
- 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.
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