Graviton BioScience Cleared to Test ROCK2 Inhibitor for Friedreich's Ataxia

The upcoming 12-week, randomized, placebo-controlled trial will enroll up to 48 participants across multiple international sites. Researchers intend to evaluate the safety and efficacy of the drug, with a primary focus on measuring increases in frataxin protein levels from baseline. The study design includes an open-label extension, allowing patients to continue treatment after the initial trial period while maintaining their existing standard-of-care therapies.

GV101 functions as a selective ROCK2 inhibitor, a mechanism that has shown promise in preclinical and early clinical settings by boosting frataxin production. According to Rui Wu, Head of Research and Preclinical, the drug's ability to induce frataxin in patient-derived cells suggests it could serve as a disease-modifying agent. The company plans to begin patient enrollment in the first quarter of 2027, leveraging a capsule formulation optimized for better pharmacokinetic properties and oral bioavailability.