Alnylam and Cubist have provided complete data from their phase II study of ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection.
The phase II study was a randomized, double-blind study of inhaled ALN-RSV01 or placebo, in adult lung transplant patients naturally infected with RSV. Clinical evaluations at 90 days confirmed that the primary objective of safety and tolerability was achieved.
The phase II study, conducted at 11 sites in 4 countries with 24 lung transplant patients with confirmed RSV infection, were randomized to receive inhaled ALN-RSV01 or placebo once daily for three consecutive days. The study achieved its primary objective of demonstrating safety and tolerability of ALN-RSV01.
In the study, ALN-RSV01 treatment was associated with a statistically significant decrease in the total incidence of new or progressive bronchiolitis obliterans syndrome (BOS) at 90 days, compared to placebo. Over 50% of placebo patients showed new or progressive BOS, as compared to only 7.1% of ALN-RSV01-treated patients, said the company.
Akshay Vaishnaw, senior vice president of Clinical Research of Alnylam, said: We are very pleased with the outcome of this study, which demonstrated for the first time the safety and tolerability of inhaled ALN-RSV01 in naturally infected patients.
As such, these data provide important de-risking for the advancement of our overall ALN-RSV program. While the study was not powered for efficacy and is too small to make firm conclusions, we are encouraged by the 90 day clinical endpoint data, including improvement in lung function and a statistically significant reduction in new or progressive BOS in patients receiving ALN-RSV01 as compared with placebo, he added.