Phase 2 trial of experimental FA treatment to start recruiting soon
FDA has also granted fast-track designation to the oral therapy GV-101
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Graviton Bioscience has launched a Phase 2 clinical trial to test its experimental therapy GV101 in people with Friedreich’s ataxia (FA).
The trial (NCT07870590) is planned to enroll 48 people with FA, ages 16 to 40, who are able to walk (with or without assistance) and who meet certain other criteria related to lab tests and clinical scores. Participants will be given GV101 at one of three doses (200 mg, 300 mg, or 400 mg) or a placebo, with the main goal of assessing the treatment’s effects on levels of a form of the frataxin protein measured in blood after 12 weeks.
The Phase 2 study is currently active at a site in New Jersey, but recruitment has not yet begun. According to a press release from Graviton, the study is expected to begin enrolling patients before the end of the year.
The company also announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation to GV101 as a potential treatment for FA. The FDA gives this designation to experimental therapies that have the potential to address unmet needs in the care of serious medical conditions, with the goal of speeding up the review process. The designation gives Graviton access to perks such as more frequent feedback from the FDA during the development process.
“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, PhD, founder and CEO of Graviton. “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.”
GV101 aimed at boosting frataxin protein levels
FA is a genetic disease caused by mutations that lead to low levels of frataxin, a protein that’s vital for the activity of energy-producing mitochondria in cells. Deficiency of the frataxin protein leads to problems in cells throughout the body, ultimately giving rise to FA symptoms.
GV101 is an experimental oral medication designed to block the activity of a protein called ROCK2 (Rho/Rho-associated coiled-coil containing protein kinase 2). This protein plays a role in regulating cellular contraction, migration, and adhesion. According to Graviton, preclinical tests have indicated that blocking ROCK2 can help to boost frataxin protein levels in FA.
“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,” Waksal said.
Graviton is also developing GV101 for other conditions, and in clinical tests it has been given to more than 500 people. The company said that safety data have been favorable thus far.
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