Graviton now plans to initiate a 12-week, randomized, placebo-controlled, dose-finding Phase 2 trial enrolling up to 48 participants at multiple sites in the United States and internationally. The study is designed to evaluate the efficacy, safety, and tolerability of a range of GV101 capsule doses in individuals living with Friedreich's ataxia. The primary endpoint is an increase from baseline in frataxin levels measured in patients' cells.
Friedreich Ataxia and close related scientific news. Topics related to rare diseases.
Tuesday, August 11, 2026
Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of Friedreich's Ataxia
NEW YORK, Aug. 11, 2026 /PRNewswire/ -- Graviton BioScience Corporation, a privately held, clinical-stage biotechnology company developing a diverse pipeline of therapeutics, including selective ROCK2 inhibitors for metabolic, inflammatory, fibrotic, and central nervous system disorders, announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a novel capsule formulation of GV101, a proprietary oral ROCK2 inhibitor being developed for Friedreich's ataxia (FA). This new formulation of GV101 was specifically optimized for development in FA and other potential orphan indications. Preclinical and clinical studies have demonstrated that GV101 increases frataxin protein levels, which is a potentially disease modifying therapeutic approach that addresses the root cause of Friedreich's ataxia (FA). Following the FDA's 30-day review period, Graviton received IND clearance on August 7, 2026, enabling the initiation of a Phase 2 clinical study in individuals living with FA.