Graviton BioScience Corporation, a clinical-stage biotechnology company developing therapies for metabolic, inflammatory, fibrotic and central nervous system disorders, has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for a novel capsule formulation of GV101, an oral ROCK2 inhibitor being developed for Friedreich’s Ataxia (FA).

The FDA cleared the IND on August 7, 2026, following its 30-day review period. The regulatory milestone enables Graviton to advance GV101 into Phase 2 clinical development for FA, a rare inherited disorder associated with insufficient production of the frataxin protein.

The new GV101 capsule formulation has been specifically optimised for development in Friedreich’s ataxia and other potential orphan indications. According to the company, preclinical and clinical research indicates that GV101 may increase frataxin mRNA and protein levels, supporting its potential as a disease-modifying treatment that targets an underlying mechanism of FA.

Rui Wu, Head of Research and Preclinical at Graviton BioScience, said, “Our preclinical work has suggested that ROCK2 inhibition in cells derived from FA patients increased frataxin significantly. ROCK2 inhibition also increased frataxin in PBMCs in patients receiving GV101 in an obesity study. We believe, therefore, that this induction of frataxin by GV101 may become an orally available disease modifying agent.” 

Graviton plans to initiate a 12-week, randomised, placebo-controlled, dose-finding Phase 2 trial involving up to 48 participants across multiple sites in the US and internationally. The study will evaluate different doses of the GV101 capsule for efficacy, safety and tolerability in people living with Friedreich’s ataxia.

The primary endpoint will assess the change from baseline in frataxin levels in patients’ cells, providing an early measure of GV101’s potential biological activity.

Participants completing the 12-week study will have the opportunity to enter an open-label extension in which all participants receive active treatment. Patients will also be permitted to continue their existing FDA-approved standard-of-care treatment while participating in the study. Enrollment is expected to begin in Q1 2027.

GV101 is one of Graviton’s proprietary oral ROCK2 inhibitors and has already been evaluated in preclinical and clinical studies. More than 500 participants have received GV101 in clinical studies for periods of up to 24 weeks, while extension studies have followed participants for more than one year.

In a Phase 1 multiple-ascending-dose study in healthy volunteers, GV101 demonstrated clinically relevant drug exposure along with a favourable safety and tolerability profile. The newly developed capsule formulation also demonstrated improved pharmacokinetic characteristics and oral bioavailability in healthy volunteers.

Preclinical studies have shown that GV101 can increase frataxin mRNA and protein levels in patient-derived B-lymphocytes and fibroblasts. Similar findings were observed in an obesity clinical study, where oral ROCK2 inhibition with GV101 increased frataxin levels in patient-derived peripheral blood mononuclear cells (PBMCs).

Graviton said these findings collectively support the potential of ROCK2 inhibition to increase frataxin expression across multiple experimental models. Raising frataxin protein levels toward those observed in asymptomatic carriers could potentially address an underlying biological driver of Friedreich’s ataxia and support the development of GV101 as a disease-modifying therapy.