ALN-RSV01 is being evaluated for the treatment of respiratory syncytial virus (RSV) infection and is the first RNAi therapeutic in human clinical development for an infectious disease.
Alnylam conducted two phase I trials with ALN-RSV01 to evaluate the safety, tolerability, and pharmacokinetics of the drug candidate in healthy adult volunteers. Both trials were double-blind, placebo-controlled, and randomized.
In total, 101 subjects were enrolled in the trials and 65 were exposed to ALN-RSV01. The subjects received single or multiple daily doses of ALN-RSV01 or saline placebo in a nasal spray. Results showed that ALN-RSV01 was safe when administered intranasally in relevant doses to human volunteers, and comparable to placebo with respect to any reported adverse events.
All reported adverse events for both ALN-RSV01 and placebo subjects were mild, with no reported serious adverse events. Furthermore, there was no evidence of laboratory or electrocardiographic abnormalities in subjects exposed to drug.
Finally, as expected, there was no significant systemic exposure of ALN-RSV01 when administered intranasally.
“These phase I study results are an important step forward for Alnylam and for the entire field of RNAi therapeutics,” said Dr John Maraganore, president and CEO of Alnylam. “Treatment of RSV infection, the leading cause of pediatric hospitalization in the US today and a prevalent infection in certain adult populations, represents a major unmet medical need in a large number of patients. We believe that ALN-RSV01 is a promising treatment option for these patients and, as an unpartnered clinical program, an important component of Alnylam’s balanced pipeline of wholly owned and partnered RNAi therapeutics.”
The data from the phase I trials were presented at the 2006 Pediatric Academic Societies’ annual meeting in San Francisco.