Benzinga | Oct 26, 2021 08:28AM EDT

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

* 4D Molecular Therapeutics Inc (NASDAQ: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.

* Also Read: 4D Molecular To Test Its Wet AMD Gene Therapy In Patients.

* Price Action: FDMT shares closed at $32.94 on Monday.