US-based clinical-stage biotechnology company Actio Biosciences has closed a $66m Series B financing round, co-led by Regeneron Ventures and Deerfield Management.
Other participants include existing investors Canaan, Droia Ventures, and Euclidean Capital.
The funding aims to advance Actio’s genetics-driven pipeline of small-molecule therapeutics targeting both rare and common diseases.
Deerfield Management partner Jason Fuller said: “Actio has done an exceptional job of identifying serious unmet needs in rare disease and advancing solutions that may also be applicable to patients with more common diseases.
“We’re pleased to support the company, encouraged by its progress, and excited by the team’s potential to make a difference for patients.”
Actio will use the funds to develop its lead programmes, ABS-1230 and ABS-0871, which received the US Food and Drug Administration (FDA) rare paediatric and orphan drug designations.
ABS-1230, a KCNT1 inhibitor, is designed to treat KCNT1-related epilepsy, a rare and often fatal paediatric epileptic encephalopathy affecting approximately 2,500 individuals in the US.
The drug has shown promise in preclinical studies by inhibiting pathogenic mutations in the KCNT1 gene.
Actio plans to initiate a Phase 1 clinical trial for ABS-1230 in the second half of 2025, with a Phase 1b study in early 2026.
ABS-0871, a TRPV4 inhibitor that has been granted FDA fast-track designation, is being evaluated for Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder.
CMT2C is a rare inherited disorder affecting motor and sensory functions, with a US prevalence of approximately 2,500 individuals.
Preclinical evaluations have shown significant improvements in motor function. ABS-0871 is currently in a Phase 1 trial, with plans for a Phase 1b study in 2026.
Actio co-founder and CEO David Goldstein said: “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.”
