US-based Actio Biosciences has closed a Series B financing round, raising $66m to advance the genetics-driven small molecule therapeutics pipeline aimed at rare and common diseases.
The funding will primarily be used to propel the development of the company's lead oral programmes, ABS-1230 and ABS-0871.
The round was jointly led by new investor Regeneron Ventures and current investor Deerfield Management.
Other participants were current investors Canaan, Euclidean Capital and Droia Ventures.
ABS-1230 is a selective potassium channel subfamily T member 1 (KCNT1) inhibitor designed to address KCNT1-related epilepsy, a severe paediatric epileptic encephalopathy.
The company is preparing to launch the healthy volunteer segment of a Phase I trial of the therapy in the second half of 2025.
The company then plans to expand into a Phase Ib proof-of-concept trial in patients with KCNT1-related epilepsy in early 2026.
The US Food and Drug Administration (FDA) has awarded ABS-1230 both orphan drug and rare paediatric designations.
ABS-0871, another small molecule in Actio's pipeline, inhibits transient receptor potential vanilloid 4 (TRPV4) and is being developed for the treatment of the rare inherited neurological disorder, Charcot-Marie-Tooth disease type 2C (CMT2C).
Actio is progressing this therapy through the healthy volunteer phase of a Phase I trial and aims to move into a Phase Ib trial in individuals with TRPV4+ CMT2C in 2026.
The US regulator has granted orphan drug, fast track and rare paediatric drug designations to the therapy.
Actio Biosciences CEO and co-founder David Goldstein stated: “We have made tremendous progress across our pipeline – executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development.
“ABS-1230 and ABS-0871 have the potential to be transformative disease-modifying therapies in their respective rare indications, and growing evidence supports expansion into broader indications. This new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programmes.”
