4D Molecular's Fabry Disease Candidate Shows Early Proof-Of-Concept

4D Molecular Therapeutics Inc FDMT has announced interim data from the Phase 1/2 trial of intravenous 4D-310 in Fabry disease patients.
The Company said that 4D-310 demonstrated a manageable safety profile with the initial evidence of clinical activity.
The Company said that all three patients had mean serum AGA enzyme activity within or significantly above the normal range following the infusion.
While no serious adverse events were observed in patients 1 and 3, the second patient had transient, self-limited atypical hemolytic uremic syndrome classified as a serious adverse event.
“These interim data demonstrate clinical proof-of-concept for tolerability and clinical activity,” CEO David Kirn remarked.
4D Molecular also shared updates on its ongoing Phase 1/2 clinical trial for 4D-310 in choroideremia.
In the dose-escalation study, six patients with the advanced form of the disease have been enrolled, and 4D-310 has led to no dose-limiting toxicities or serious adverse events.
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Price Action: FDMT shares closed at $32.94 on Monday.