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Diseases

Actio Biosciences Secures $66 Million in Series B Financing to Advance Rare Disease Programs

Strategic Insights

— 2 min read

Actio Biosciences, a clinical-stage biotechnology company, has closed a $66 million Series B financing led by Regeneron Ventures and Deerfield Management. This new funding will support the advancement of the company's lead programs, ABS-1230 and ABS-0871, which have the potential to be transformative disease-modifying therapies in their respective rare indications. The funding is a significant milestone for Actio, allowing the company to continue its precision medicine approach to develop new therapeutics that target shared underlying biology in both rare and common diseases.

Key Takeaways:

  • Actio Biosciences has secured $66 million in Series B financing to advance its lead programs, ABS-1230 and ABS-0871.
  • ABS-1230 is a potential first-in-class oral, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy, a rare and often fatal pediatric epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals.
  • ABS-0871 is a potential first-in-class oral, small molecule TRPV4 inhibitor for the treatment of Charcot-Marie-Tooth disease type 2C and overactive bladder, with a U.S. prevalence of approximately 2,500 individuals.
  • ABS-1230 has been granted rare pediatric and orphan drug designations by the FDA, while ABS-0871 has been granted rare pediatric, orphan, and fast track designations.
  • Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in the second half of 2025, with plans to expand into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026.
  • Enrollment is ongoing in the Phase 1 trial for ABS-0871, targeting the treatment of Charcot-Marie-Tooth disease type 2C.
  • The funding will also support the advancement of Actio's pipeline, which is led by its precision medicine approach to genetics and drug development.

Statistics:

  • U.S. prevalence of KCNT1-related epilepsy: approximately 2,500 individuals
  • U.S. prevalence of Charcot-Marie-Tooth disease type 2C: approximately 2,500 individuals
  • Series B financing amount: $66 million
  • Period expected for Phase 1 clinical trial of ABS-1230: second half of 2025
  • Planned expansion into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients: early 2026

Sources:

  • BusinessWire: Actio Biosciences Announces Closing of $66 Million Series B Financing (https://www.businesswire.com/news/home/20250618424417/en/)
  • Actio Biosciences: Actio Biosciences Secures $66 Million in Series B Financing to Advance Rare Disease Programs (https://www.actiobiosciences.com/)
  • Food and Drug Administration (FDA): Rare Pediatric Disease Priority Review Voucher Program (https://www.fda.gov/patients/drug-development-process/priority-review-voucher-program/rare-pediatric-disease-priority-review-voucher-program)
  • Food and Drug Administration (FDA): Orphan Drug Designation (https://www.fda.gov/patients/directories-drug-product-information-catalog/orphan-drug-designation)

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