Initial Data from Ongoing Phase 1 MAD Trial of DT-216 for FA Expected in the Third Quarter of 2023: Design is evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 MAD clinical trial designed to evaluate the safety, tolerability, pharmacokinetic, biodistribution, and pharmacodynamic effects of three weekly doses of DT-216 in adults with FA. FA is a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the frataxin (FXN) gene that impairs transcription and reduces FXN mRNA. DT-216 is designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression. An unanticipated vendor issue related to the study drug vial stopper caused a short delay in product supply, which has been resolved. Design now anticipates presenting initial results from the MAD trial in the third quarter of 2023. The company plans to initiate a Phase 2 trial in the second half of 2023.
Wednesday, May 10, 2023
Design Therapeutics Provides Pipeline Updates and Reports First Quarter 2023 Financial Results
CARLSBAD, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.
