Graviton BioScience 的 GV101 获 FDA 快速通道资格，用于治疗 Friedreich 共济失调

• Fast Track Designation recognizes the serious unmet medical need in Friedreich's ataxia and is intended to facilitate the development and expedite the review of GV101.
• GV101 is a potent, highly selective oral ROCK2 inhibitor that has demonstrated the ability to increase frataxin protein levels, offering a potentially disease-modifying approach to Friedreich's ataxia.
• The designation follows the FDA's recent clearance of Graviton's Investigational New Drug application for GV101 and provides further momentum as Graviton BioScience initiates a Phase 2 study in patients with Friedreich's ataxia. Enrollment is expected to begin at U.S. sites in Q4 of 2026 and globally shortly thereafter.

, /PRNewswire/ -- Graviton BioScience today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to GV101, the Company's clinical-stage, potent and selective oral Rho/Rho-associated coiled-coil containing protein kinase 2 (ROCK2) inhibitor, for the treatment of Friedreich's ataxia (FA).

Friedreich's ataxia is a rare, inherited, multisystem disorder caused by decreased expression of the protein frataxin. Low frataxin levels trigger mitochondrial dysfunction. Such dysfunction heavily impacts cardiovascular, nervous, and musculoskeletal systems. People living with Friedreich's ataxia experience a progressive loss of coordination (ataxia), weakness, wheelchair dependence, slurred speech (dysarthria), difficulty swallowing, and diminished reflexes. These progressive symptoms heavily impact daily mobility, education, employment, independence, and overall quality of life and cause premature mortality, usually before age 50. FA patients also commonly experience hypertrophic cardiomyopathy, and cardiac complications account for more than half of FA-related deaths.

GV101 is one of Graviton's proprietary, investigational oral selective ROCK2 inhibitors. ROCK2 inhibition is a novel mechanism for FA therapy that targets the root genetic cause of the disease by increasing both frataxin transcription and translation. This genetic restoration alleviates mitochondrial dysfunction and cellular stress in cardiovascular and neurological tissues. Furthermore, ROCK2 inhibition has been shown to offer neuroprotection, promoting axonal regeneration and increasing neuronal survival. Promising preclinical studies have demonstrated that GV101 increases frataxin mRNA and protein levels, providing a potentially disease-modifying therapeutic approach to Friedreich's ataxia.

"Receiving Fast Track Designation from the FDA is an important milestone for GV101 and underscores the significant unmet need for therapies that address the underlying biology of Friedreich's ataxia," said Samuel Waksal, Ph.D., Founder and Chief Executive Officer of Graviton BioScience. "We believe GV101 has the potential to provide a differentiated treatment approach by increasing frataxin, the protein that is deficient in individuals living with Friedreich's ataxia. Together with our recent IND clearance, Fast Track Designation provides important momentum as we move forward with preparations for our Phase 2 study and work closely with the FDA to efficiently advance the development of GV101 for patients."

The FDA's Fast Track program is designed to facilitate the development and expedite the review of investigational therapies intended to treat serious conditions and address unmet medical needs.

Planned Phase 2 Study in Friedreich's Ataxia

Graviton is beginning a 12-week, randomized, placebo-controlled, dose-finding Phase 2 clinical trial evaluating GV101 in individuals with Friedreich's ataxia. First enrollment is expected in Q4 2026.

GV101 Clinical Safety

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.

About Friedreich's Ataxia

Friedreich's ataxia is a rare, inherited, progressive neurodegenerative disease affecting approximately 5,000 people in the United States and an estimated 15,000 people worldwide. The disease is caused by GAA trinucleotide repeat expansions in the frataxin 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, 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 therapeutic objective 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 diseases. Graviton has developed a portfolio of selective ROCK2 inhibitors among other innovative and novel therapies for patients with fibrotic, autoimmune, and CNS diseases. GV101 is in clinical development, with additional assets advancing through the pipeline.

Leading the Company is Dr. Samuel Waksal, the founder and former Chairman and CEO of Kadmon Pharmaceuticals, which was acquired by Sanofi. Dr. Waksal is also the founder and former CEO of ImClone Systems, which was acquired by Eli Lilly, and a founder of MeiraGTx.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the potential therapeutic benefits and development of GV101, the timing and design of planned clinical trials, the potential effects of Fast Track Designation, and Graviton BioScience's development plans. These statements are based on current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially from those expressed or implied. GV101 is an investigational product and has not been approved by the FDA or any other regulatory authority.

Media Contact
Graviton BioScience Corporation
Melanie Glickman
Commercial Strategy
[email protected]

SOURCE Graviton BioScience Corporation