Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Alnylam Pharmaceuticals has achieved human proof of concept with an RNAi therapeutic, a first for the industry. Results from the company's Gemini trial with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus or RSV, showed statistically significant anti-viral efficacy.
Subscribe to our email newsletter
In the Phase II experimental infection study, treatment with ALN-RSV01 led to a decreased RSV infection rate, an increase in the number of subjects who remained free of infection, and was safe and well tolerated.
The goal of the Gemini study was to establish the anti-viral activity, safety, and tolerability of ALN-RSV01 in an experimental RSV infection model. Gemini was a double-blind, placebo-controlled study, where a total of 88 adult subjects experimentally infected with RSV was randomized to receive ALN-RSV01 or placebo. ALN-RSV01 or placebo was administered intranasally for five consecutive days – two days prior and three days after viral inoculation. Efficacy measures included infection rate and the effects of treatment on viral dynamics and clinical symptoms. As designed, Gemini had sufficient power to detect a statistically significant difference in infection rate between ALN-RSV01 and placebo.
Akshay Vaishnaw, vice president, clinical research at Alnylam, said: “We believe these findings point to the potential for clinical benefit with ALN-RSV01 in a naturally infected patient population, and we look forward to initiating a Phase II study in naturally infected adult patients in the first half of 2008.”