AC Immune SA reported positive phase 1 safety data for ACI-19764, an oral NLRP3 inflammasome inhibitor, in a study conducted in Europe.
The clinical-stage biopharmaceutical company said the drug was safe and well-tolerated across single and multiple ascending dose cohorts in healthy volunteers. Doses up to 20 mg per day were evaluated, with no serious adverse events or treatment withdrawals reported. The expected therapeutic dose is 10 mg or less once daily, based on pharmacokinetic data.
Pharmacokinetic analysis showed a serum half-life exceeding 30 hours and demonstrated cerebrospinal fluid exposure, indicating brain penetration. A blinded review of whole blood assays confirmed dose-dependent inhibition of IL-1beta release. In vitro potency ranged from 2 to 20.5 nM for IL-1beta production inhibition.
ACI-19764 achieved pharmacokinetic concentrations above the IC90 at daily doses of 10 mg or less. The company has initiated a phase 1b cohort evaluating the drug in patients with cardiovascular disease risk, defined by elevated high-sensitivity C-reactive protein levels and the presence of type 2 diabetes or obesity. Interim results from this cohort are expected before year-end, with full phase 1/1b trial results anticipated in the first half of 2027.
