New York, New York, August,11, 2026
Graviton Bioscience Corporation, a clinical-stage biotechnology company developing selective ROCK2 inhibitors, has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for GV101, a proprietary oral therapy candidate being developed for Friedreich’s ataxia (FA). The FDA clearance, received on August 7, 2026, marks an important regulatory milestone for the company and enables Graviton to advance a novel capsule formulation of GV101 toward a planned Phase 2 clinical study in people living with this rare genetic neurodegenerative disease.
FDA Clearance Advances GV101 Toward Phase 2
The newly cleared IND covers a novel capsule formulation of GV101 that has been specifically optimized for development in Friedreich’s ataxia and other potential orphan indications. According to Graviton, the formulation demonstrated improved pharmacokinetic (PK) properties and oral bioavailability in healthy volunteers during a Phase 1 study. The company now plans to evaluate the formulation in a controlled clinical study involving patients with FA. Graviton expects 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 different GV101 capsule doses in individuals with Friedreich’s ataxia.
The primary endpoint will measure the change from baseline in frataxin levels in patients’ cells, providing a biomarker-based assessment of the investigational treatment. Following the 12-week treatment period, participants who complete the trial will have the opportunity to enter an open-label extension study in which all participants receive active GV101. Graviton expects enrollment in the Phase 2 study to begin in the first quarter of 2027. Participants will also be permitted to continue their existing FDA-approved standard-of-care treatment while receiving GV101 during the study.
GV101 Targets Frataxin Deficiency in FA
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GV101 is a selective inhibitor of Rho/Rho-associated coiled-coil containing protein kinase 2 (ROCK2). Graviton is developing the oral candidate across several therapeutic areas, including metabolic, inflammatory, fibrotic and central nervous system disorders. In Friedreich’s ataxia, the company is investigating whether selective ROCK2 inhibition can increase production of frataxin, a protein that is deficient in people affected by the disease. Friedreich’s ataxia is caused by GAA trinucleotide repeat expansions in the FXN gene, resulting in reduced frataxin production. Frataxin plays an important role in mitochondrial function and cellular energy production.
The resulting deficiency can contribute to progressive neurological symptoms affecting coordination, balance, speech, muscle strength and mobility, while some patients can also experience complications such as cardiomyopathy, cardiac arrhythmias and diabetes. Graviton reports that preclinical and clinical studies have shown GV101 increased frataxin mRNA and protein levels in patient-derived B-lymphocytes and fibroblasts. The company also observed increased frataxin levels in patient-derived peripheral blood mononuclear cells in an obesity study involving oral ROCK2 inhibition. These findings provide the scientific basis for evaluating GV101 as a potential disease-modifying approach for FA.
Clinical Development Builds on GV101 Data
GV101 has already been evaluated in clinical studies involving more than 500 participants, with treatment durations of up to 24 weeks and extension studies exceeding one year, according to Graviton. The company reports that the candidate has demonstrated a favorable safety and tolerability profile in preclinical and clinical development. The upcoming Phase 2 study represents a significant next step because it will evaluate GV101 specifically in patients with Friedreich’s ataxia and assess whether increasing frataxin levels can provide a meaningful therapeutic effect.
The company believes that raising frataxin toward levels observed in asymptomatic carriers could potentially address an underlying biological driver of FA. The FDA IND clearance therefore moves GV101 into a new stage of clinical development and provides Graviton with an opportunity to test its ROCK2 inhibition and frataxin-restoration strategy in a controlled patient study. However, GV101 remains investigational, and its safety and efficacy as a treatment for Friedreich’s ataxia have not been established.
Source: Graviton Bioscience press release
