Actio Biosciences Secures $66M in Series B Funding for Rare Disease Drug Development

Actio Biosciences, a San Diego-based biotechnology startup, has successfully raised $65 million in a Series B financing round to advance its drug research programs targeting rare genetic diseases. The funding, announced on Wednesday, will support the company's pursuit of what it calls "inclusive precision medicine," with potential applications extending beyond rare conditions.

Funding and Investor Support

The Series B round was co-led by new investor Regeneron Ventures and existing backer Deerfield Management. Additional participants included Canaan, Droia Ventures, and Euclidean Capital. This latest funding follows Actio's emergence from stealth in late 2023, when it secured $55 million in Series A funding.

David Goldstein, Actio's CEO and former co-founder of Praxis Precision Medicines, attributed the successful fundraising to the company's focus on programs with "very high biological plausibility." He noted the current challenging climate for biotech funding, stating, "You really need to have programs that have a pretty predictable path."

Pipeline and Development Programs

Actio's drug development pipeline currently features two small molecule candidates:

1. ABS-1230: Targeting KCNT1-related epilepsies, this drug is expected to enter clinical trials by the end of the year. Actio believes its approach can inhibit all repeatedly observed, disease-causing mutations, potentially benefiting a broad range of patients with this condition.

2. ABS-0871: Currently in Phase 1 trials with healthy volunteers, this drug blocks the TRPV4 ion channel protein. Actio plans to initiate a Phase 1b study in patients with Type 2C Charcot-Marie-Tooth disease by year-end. The company is also exploring its potential in treating overactive bladder.

Strategic Approach to Drug Development

Actio's strategy centers on developing drugs that can address a wide range of disease-causing genetic mutations. This approach, which the company terms "inclusive precision medicine," aims to create therapies with broader applicability within rare disease categories and potential extensions to more common disorders.

Goldstein emphasized the promise of small molecules in targeting conditions like KCNT1-related epilepsies, despite some researchers' shift towards biologics. He stated, "This kind of inclusive precision medicine is a key priority for the company."

As Actio Biosciences advances its programs, the biotech industry will be watching closely to see if this approach to rare disease drug development can deliver on its promise of broader therapeutic impact.