Alnylam’s Phase I study for ALN-RSV01 was a randomized, double-blind, placebo-controlled trial in healthy adult volunteers to assess the safety, tolerability, and pharmacokinetics of ALN-RSV01 administrated by inhalation via nebulizer. All major objectives of the trial were met, including definition of a safe and well-tolerated dose and regimen for advancement of ALN-RSV01 into further Phase II development. Importantly, data showed that the efficiency of delivery of ALN-RSV01 delivered via inhalation, as measured by plasma levels, was significantly greater in humans than observed pre-clinically.
ALN-RSV01 is an RNAi therapeutic being developed as a treatment for respiratory syncytial virus (RSV) infection, the leading cause of pediatric hospitalization in the US and a prevalent infection in immune-compromised adults. The Phase I study represents the first-ever clinical study of an RNAi therapeutic administered via inhalation.