ALNYLAM BEGINS STUDY OF INHALED RNAI THERAPEUTIC

Alnylam Pharmaceuticals announced that it has initiated a Phase I human clinical trial of an inhaled formulation of ALN-RSV01 in the U.S. to evaluate its safety, tolerability and pharmacokinetics in healthy adult volunteers. The inhaled formulation of ALN-RSV01 used in this Phase I study is a nebulized formulation, which advances previous clinical using ALN-RSV01 as an intranasal spray. ALN-RSV01, an un-partnered Alnylam program, is an RNAi therapeutic in clinical development for the treatment of respiratory syncytial virus infection.

Alnylam previously completed two Phase I human clinical trials of ALN-RSV01 using an intranasal formulation. The studies demonstrated that ALN-RSV01 was safe and well-tolerated when administered intranasally. This newly initiated Phase I trial with inhaled ALN-RSV01 uses a formulation that is designed to deliver the RNAi therapeutic to the lungs. Models of this aerosolized formulation of ALN-RSV01 predict that it can be delivered to the alveoli and the small airways of the infant and adult human lung.

The study will be a randomized, double-blind, placebo-controlled trial in healthy adult volunteers to assess the safety, tolerability and pharmacokinetics of aerosolized ALN-RSV01 when administrated by inhalation via nebulizer. It is anticipated that a total of 80 healthy adults will be enrolled into one of eight cohorts. Single ascending doses of aerosolized ALN-RSV01 or placebo will be administered by inhalation to four cohorts of eight subjects each with five subjects receiving ALN-RSV01 and three subjects receiving placebo in each cohort for a total of 32 subjects. Subsequently, multiple ascending doses of aerosolized ALN-RSV01 or placebo will be administered once daily by inhalation for three consecutive days to four cohorts of 12 subjects each with eight subjects receiving ALN-RSV01 and four subjects receiving placebo in each cohort for a total of 48 subjects.