Actio Biosciences Doses First Participant in Phase 1 Trial of ABS-0871 for CMT2C
Actio Biosciences, a clinical-stage biotechnology company focused on using its “one to many” paradigm in genetics and precision medicine, has announced that the first participant has been dosed in the Phase 1 healthy volunteer clinical trial for its lead program, ABS-0871. ABS-0871 is a novel TRPV4 inhibitor being developed for the treatment of TRPV4-positive Charcot-Marie-Tooth Disease subtype 2C (CMT2C), a severe peripheral nerve disorder. This milestone marks a significant step in Actio Biosciences’ efforts to develop targeted therapeutics that address shared underlying biology in both rare and common diseases.
People living with subtype 2C of Charcot-Marie-Tooth Disease (CMT), a genetically distinct form of hereditary neuropathy, face a variety of unique and debilitating symptoms. These symptoms can include muscle weakness, difficulty with mobility, vocal cord paralysis, respiratory issues, and bladder urgency. Actio Biosciences has developed ABS-0871, a potential treatment that targets the genetic cause of the disease. By addressing this underlying genetic factor, ABS-0871 could offer a solution that eliminates many of the symptoms of CMT2C, significantly improving the quality of life for affected patients.
David Goldstein, CEO of Actio Biosciences, emphasized the importance of this breakthrough. “We are thrilled to initiate this trial — the first clinical trial for Actio,” he said. “CMT2C is a genetically burdened disease that lacks any approved therapeutic treatments, and ABS-0871 could provide patients with much-needed relief by targeting the root cause of the disease.”
The initiation of the Phase 1 clinical trial represents a significant milestone for Actio and the Charcot-Marie-Tooth community. Sue Bruhn, Ph.D., CEO of the Charcot-Marie-Tooth Association, highlighted how critical this step is for CMT2C patients and their families. “We envision a future where effective treatments do not only alleviate symptoms but also alter the course of the disease, delivering long-term therapeutic effects that extend throughout a patient’s lifetime.”
The Phase 1 clinical trial is designed as a randomized, double-blind, placebo-controlled study. It will assess both single and multiple-ascending doses of ABS-0871 in healthy volunteers. The trial aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of the drug, with a focus on understanding how the medication works and whether it can be safely administered.
ABS-0871 is a small-molecule TRPV4 inhibitor that could potentially be taken once daily in pill form. It is being developed for the treatment of CMT2C as well as TRPV4-related bone diseases. TRPV4 is an ion channel that plays a crucial role in regulating calcium levels inside cells, a vital process for a variety of cellular functions.
Mutations in the TRPV4 gene can cause the channel to become overactive, which disrupts the blood-neural barrier and results in CMT2C. This condition can appear as early as infancy. ABS-0871 works by inhibiting the overactive TRPV4 channel, aiming to restore proper function in key areas such as the vocal cords, respiratory system, bladder, and other affected areas.
The preclinical studies of ABS-0871 in CMT2C disease models have shown encouraging results. In novel, construct-valid CMT2C models, ABS-0871 was associated with significant improvements in motor function and mobility when compared to untreated controls. These results provide a solid foundation for the ongoing clinical trial and further demonstrate the potential of ABS-0871 as a therapeutic option for CMT2C.
In addition, ABS-0871 received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) in August 2024, further underscoring the significance of the drug and its potential to address unmet medical needs in rare diseases.
Actio Biosciences is a biotechnology company focused on using advances in precision medicine to develop innovative therapeutics. The company’s approach leverages shared genetic pathways in both rare and common diseases, allowing for a more targeted treatment approach. By applying its expertise in genetics, drug discovery, and data sciences, Actio aims to minimize biological and technical risks in the drug development process. This enables them to bring forward potent, precisely targeted therapeutics with the potential to benefit a wide range of patients.
Actio is currently advancing two lead programs: ABS-0871 for CMT2C and ABS-1230 for KCNT1-related epilepsy. Both programs have the potential to be first-in-class therapies for these conditions, and Actio is committed to their development. Founded in 2021 and based in San Diego, Actio Biosciences is guided by a team of leaders with extensive experience in genetics and drug development. The company is also backed by top healthcare investors who share its vision for bringing meaningful therapies to patients with unmet needs.
Actio’s focus on precision medicine and innovative drug development holds promise for patients suffering from both rare and common diseases. By targeting shared genetic mechanisms, Actio hopes to improve the lives of many, bringing forward therapies that address the root causes of diseases rather than just their symptoms. Through its continued work on ABS-0871 and ABS-1230, Actio is paving the way for future breakthroughs in rare disease treatment.
