Design Therapeutics, Inc. announced that it has completed dosing in the first single ascending dose (SAD) cohort of its Phase 1 clinical trial of DT-216 in patients with Friedreich ataxia (FA). DT-216 is a novel GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation, the underlying cause of FA, and restore frataxin (FXN) gene expression. Additionally, Design announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to DT-216 for the treatment of patients with FA.

Design's Phase 1 clinical trial of DT-216 is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics and FXN levels from single ascending doses of IV-administered DT-216 in adult patients with FA. FA patients are expected to receive a single dose of either DT-216 or placebo across five ascending dose cohorts. Additional information may be found at ClinicalTrials.gov [1], using Identifier NCT: NCT05285540.

The company anticipates reporting topline data from the Phase 1 trial in the second half of 2022. The Fast Track process is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions. With Fast Track designation, DT-216 is eligible for early and frequent communication with the FDA throughout the entire drug development and review process.

In addition, it is now eligible for Accelerated Approval and Priority Review, if relevant criteria are met, and a Rolling Review for its New Drug Application (NDA).