This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency patients for 24 months.
September 2025 Update
Applied Therapeutics has completed a meeting with the U.S. Food and Drug Administration to discuss a potential new drug application for govorestat in CMT-SORD. The company is awaiting official meeting minutes to determine next steps. CMTA continues to follow these developments.
April 2026 Update
On April 17, Cycle Pharmaceuticals announced it is halting all clinical studies of govorestat. This includes the ongoing Open Label Extension of the INSPIRE Phase III trial for CMT-SORD as well as all Early Access Programs. The company said it is stopping current studies to determine what other data is needed for regulatory approval.
Cycle Group Holdings bought Applied Therapeutics in early 2026.
