Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of 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. 

 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.

Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow

Smith BK, Coker MA, Liberati C, Meyer BP, Norman S, Ehrbar J, Leon-Astudillo C, Subramony S, Corti M. Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow. J Neuromuscul Dis. 2026 Jun 8:22143602261452334. doi: 10.1177/22143602261452334. Epub ahead of print. PMID: 42253100; PMCID: PMC13437907. 

The study demonstrates respiratory dysfunction in patients with FRDA, and shows that disease severity and muscle weakness affect airway clearance. PCF is a more direct and clinically meaningful indicator of cough effectiveness than FVCpp. Comprehensive respiratory evaluations, including muscle-strength testing, can identify individuals who may benefit from targeted interventions to prevent complications.

Impaired Glur2 palmitoylation in cerebellar Purkinje cells of a Friedreich ataxia mouse model

Elizabeth Mercado-Ayón, Jennifer Coulman, Jia-Ying Lee, Eunjoo Lancaster, Elliot Goga, Mohammad Asad, Eric Witze, David R. Lynch, Impaired Glur2 palmitoylation in cerebellar Purkinje cells of a Friedreich ataxia mouse model, Experimental Neurology, 2026, 115955, ISSN 0014-4886, doi:10.1016/j.expneurol.2026.115955.

Mechanistically, reduced GluR2 palmitoylation associates with decreased expression and palmitoylation of the palmitoyl acyltransferase DHHC3, while levels of depalmitoylating enzymes remain unchanged. In vitro, DHHC3 enhances GluR2 palmitoylation, supporting a direct enzymatic relationship. Partial restoration of frataxin expression rescues GluR2 and DHHC3 protein levels and partially restores GluR2 palmitoylation. These findings identify impaired GluR2 palmitoylation as an early, selective synaptic alteration in FRDA and implicate dysregulated lipid-dependent post-translational modification as a mechanism linking frataxin deficiency to cerebellar synaptic vulnerability.