Design Therapeutics Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2

Core Insights - Design Therapeutics, Inc. has initiated the RESTORE-FA trial for DT-216P2, targeting Friedreich ataxia (FA) with the first patient dosed via intravenous infusion [1][2] - The trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DT-216P2, with initial results showing no adverse events [2][4] - The company submitted an IND application to the FDA to expand the trial to U.S. sites but received a clinical hold notice due to nonclinical deficiencies [3][5] Group 1: Trial Details - The RESTORE-FA trial is designed to evaluate both intravenous and subcutaneous administration of DT-216P2 in FA patients [2] - Initial data from the ongoing Phase 1 single-ascending dose trial in healthy volunteers indicated that DT-216P2 was well-tolerated, with no cases of injection site thrombophlebitis reported [4] - The company expects to report data from the multiple-ascending dose trial, including frataxin expression levels, in 2026 [2] Group 2: Regulatory and Development Updates - The FDA's clinical hold on the IND application is expected to be addressed by the company once further details are received [3][5] - The company plans to work closely with the FDA to expedite the development process in the U.S. [5] - DT-216P2 is an improved formulation designed to target the GAA repeat expansion mutation causing FA and aims to restore endogenous frataxin production [6] Group 3: Company Overview - Design Therapeutics is focused on developing a new class of therapies using GeneTAC technology, which targets disease-causing genes [7] - The company is advancing multiple programs, including DT-216P2 for FA and DT-168 for Fuchs endothelial corneal dystrophy, along with research in myotonic dystrophy type-1 and Huntington's disease [7]