Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of Friedreich's Ataxia

• Graviton received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for a novel capsule formulation of GV101 on August 7, 2026.
• FDA clearance marks a critical regulatory milestone in advancing this formulation of GV101, a capsule with improved PK and oral availability, into human trials for Friedreich's ataxia (FA).
• GV101 is a best-in-class selective ROCK2 inhibitor that increases frataxin protein levels, a therapeutic approach with potential disease-modifying effects in 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.

"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." Rui Wu, Head of Research and Preclinical

Planned Phase 2 Study in Friedreich's Ataxia

GV101 is one of Graviton's proprietary investigational oral ROCK2 inhibitors advancing within the Company's broader development pipeline. The planned Phase 2 study will utilize a novel capsule formulation of GV101 that demonstrated improved pharmacokinetic (PK) properties and oral bioavailability in healthy volunteers in a Phase 1 study. This formulation of GV101 is being developed as a potential treatment for Friedreich's ataxia, a rare genetic disease driven by deficient frataxin production.

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.

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. Enrollment is expected to begin in the first quarter of 2027.

GV101 Development Highlights

GV101 has demonstrated a favorable safety and tolerability profile in preclinical and clinical studies. More than 500 participants have received GV101 in clinical studies for durations of up to 24 weeks, with extension studies exceeding one year. In a Phase 1 multiple ascending dose study in healthy volunteers, GV101 achieved clinically relevant exposure and demonstrated a favorable safety and tolerability profile.

Preclinical and clinical studies have also demonstrated that GV101 increases both frataxin mRNA and protein levels in patient-derived B-lymphocytes and fibroblasts. Consistent with these findings, oral ROCK2 inhibition with GV101 increased frataxin levels in vivo in an obesity study that evaluated patient-derived peripheral blood mononuclear cells (PBMCs).

Collectively, these results provide evidence that ROCK2 inhibition may increase frataxin expression across multiple experimental systems. Increasing frataxin protein levels has the potential to move patients toward levels observed in asymptomatic carriers, supporting GV101's potential as a disease-modifying therapy aimed at addressing the underlying cause of Friedreich's ataxia.

About Friedreich's Ataxia

Friedreich's ataxia (FA) is a rare, inherited, progressive neurodegenerative disease affecting approximately 5,000 people in the United States and an estimated 15,000 people worldwide, qualifying it as an orphan disease. Friedreich's ataxia is caused by GAA trinucleotide repeat expansions in the FXN gene that result in reduced production of frataxin, a protein essential for mitochondrial function and cellular energy production. Symptoms typically begin in childhood or adolescence and worsen over time, affecting coordination, balance, speech, muscle strength, and mobility. Many individuals eventually become wheelchair-dependent and may develop serious complications, including cardiomyopathy, cardiac arrhythmias, diabetes, and difficulties with swallowing and speech. Significant unmet need remains for treatments that address the underlying frataxin deficiency that drives the disease.

About GV101

GV101 is a clinical-stage, best-in-class, selective inhibitor of Rho/Rho-associated coiled-coil containing protein kinase 2 (ROCK2). ROCK2 is a key regulator of multiple cellular pathways involved in inflammation, fibrosis, metabolism, and gene expression. Through selective inhibition of ROCK2, GV101 has demonstrated the potential to modulate disease-relevant biological processes, including increasing frataxin production, a key factor in Friedreich's ataxia.

About Graviton BioScience

Graviton BioScience Corporation is a clinical-stage drug discovery and development company dedicated to engineering and developing best-in-class therapeutics for treating metabolic, CNS, inflammatory, fibrotic, and other disease indications. Graviton has developed a portfolio of selective ROCK2 inhibitors among other safe, innovative, and novel therapies for patients with fibrotic, autoimmune, and CNS diseases. GV101 is in clinical studies, with additional assets advancing through the pipeline. Leading the Company is Dr. Samuel Waksal, the founder and former Chairman and CEO of Kadmon Pharmaceuticals (acquired by Sanofi). Dr. Waksal is also the founder and former CEO of ImClone Systems (acquired by Eli Lilly) and a founder of MeiraGTx (NASDAQ: MGTX).

Forward-Looking Statements

This press release contains forward-looking statements regarding Graviton's development plans, regulatory strategy, and potential future clinical applications for its selective ROCK2 inhibitors. Such statements are subject to risks and uncertainties, including, but not limited to, biological, clinical, regulatory, financial, and operational risks. Actual results may differ materially.

Graviton BioScience Contact
Melanie Glickman
Senior Associate, Operations Strategy
[email protected]

SOURCE Graviton Bioscience Corporation