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

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  • 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.
  • Also Read: 4D Molecular To Test Its Wet AMD Gene Therapy In Patients.
  • Price Action: FDMT shares closed at $32.94 on Monday.
Posted In: BiotechNewsHealth CareSmall CapGeneralBriefsFabry diseasePhase 1 TrialPhase 2 Trial
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