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 on Tuesday that the US 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), a rare genetic disease driven by deficient frataxin production.

According to the company, this new formulation of GV101 was specifically optimised 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 FA. Following the FDA's 30-day review period, Graviton received IND clearance on 7 August 2026, enabling the initiation of a Phase 2 clinical study in individuals living with FA.

Graviton plans to initiate a 12-week, randomised, 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 FA. The primary endpoint is an increase from baseline in frataxin levels measured in patients' cells.

The Phase 2 study will be followed by an open-label extension, offering active drug to all participants who complete the 12-week trial. Participants will be able to continue receiving their existing FDA-approved standard-of-care treatment throughout the study while taking GV101. Enrolment is expected to begin in the first quarter of 2027.