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First Patient Dosed in Phase 1 Trial of ABS-0871 for Charcot-Marie-Tooth Disease 2C Treatment

• Actio Biosciences has initiated Phase 1 clinical trials for ABS-0871, a novel TRPV4 inhibitor designed to treat Charcot-Marie-Tooth Disease 2C (CMT2C), marking the company's first clinical program.

• ABS-0871, granted both orphan drug and rare pediatric disease designations, is an oral small-molecule treatment targeting the underlying genetic cause of CMT2C by inhibiting overactivated ion channel activity.

• The randomized, double-blind, placebo-controlled trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers, potentially offering hope for patients currently lacking approved treatment options.

Actio Biosciences has launched its first clinical trial, dosing the initial participant in a Phase 1 study of ABS-0871, a novel TRPV4 inhibitor developed for treating Charcot-Marie-Tooth Disease subtype 2C (CMT2C). The drug candidate, which recently received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD), represents a potential breakthrough for patients suffering from this severe peripheral nerve disorder.

Disease Impact and Unmet Need

CMT2C is a genetically defined form of hereditary neuropathy that can manifest as early as infancy. Patients face severe challenges including muscle weakness, ambulatory difficulties, vocal cord paralysis, respiratory complications, and bladder urgency. Currently, there are no approved therapeutic options for this debilitating condition.
"People living with the subtype 2C of CMT face a number of unique and debilitating symptoms," stated David Goldstein, CEO of Actio Biosciences. "We have designed ABS-0871 to address the underlying genetic cause of disease, therefore potentially offering patients an option that could eliminate symptoms and significantly improve daily quality of life."

Innovative Mechanism of Action

ABS-0871 is designed as a once-daily oral small-molecule TRPV4 inhibitor. The drug targets TRPV4, an ion channel that regulates cellular calcium concentration crucial for various cellular processes. In CMT2C, mutations in the TRPV4 gene lead to increased channel activity and disruption of the blood-neural barrier. By inhibiting this overactivated ion channel activity, ABS-0871 aims to restore vocal cord, respiratory, bladder, and other functions.

Clinical Trial Design and Preliminary Evidence

The Phase 1 trial employs a randomized, double-blind, placebo-controlled design, incorporating both single- and multiple-ascending dose components. The study will evaluate safety, tolerability, pharmacokinetics, and measures of pharmacodynamic activity in healthy volunteers.
Preclinical studies using novel construct-valid CMT2C disease models have shown promising results, with marked improvements in motor function and mobility compared to untreated controls.

Expert Perspective

Sue Bruhn, Ph.D., CEO of the Charcot-Marie-Tooth Association, emphasized the significance of this milestone: "The initiation of this Phase 1 clinical trial is an incredibly important step for our STAR Alliance Partner, Actio Biosciences, and CMT2C patients and their families. We envision a world where effective treatments provide symptomatic relief to patients along with altering the course of their disease, demonstrating a sustained therapeutic effect over a patient's lifetime."
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