CMTA-STAR Alliance Partner Actio Biosciences has announced that the first participant has been dosed in its Phase I clinical trial of ABS-0871, an investigational TRPV4 inhibitor being developed for CMT2C.
CMT2C is an often-severe form of CMT caused by mutations in the TRPV4 gene. It leads to progressive muscle weakness, vocal cord paralysis, respiratory muscle weakness, and other debilitating symptoms.
“This Phase I clinical trial is an important step forward for the CMT2C community and our CMTA-STAR Alliance Partner, Actio Biosciences,” said CMTA CEO Sue Bruhn, PhD. “CMT2C is a complex form of CMT that presents significant challenges for patients and their families. We proudly support Actio Biosciences in advancing this potential treatment.”
The Phase I study is a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, and pharmacokinetics of ABS-0871 in healthy volunteers. Pharmacokinetics refers to how a drug is absorbed, distributed, broken down, and eliminated from the body. The company says data from this study will inform future clinical trials in CMT2C.
ABS-0871 has been granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the FDA, underscoring the urgent need for treatment options for this rare disease.
“People living with CMT2C face unique and debilitating challenges, including muscle weakness, ambulatory difficulties, vocal cord paralysis, respiratory complications, and bladder urgency,” said Actio Biosciences CEO David Goldstein. “We designed ABS-0871 to address the underlying genetic cause of the disease and thereby improve quality of life.”
CMTA remains committed to working with our CMTA-STAR Alliance Partners to advance research that brings us closer to treatment options for the CMT community. We look forward to following Actio Biosciences’ progress as they move this program forward for people living with CMT2C.
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Published on: March 3, 2025