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RSV Infection
ALN-RSV: Respiratory Syncytial Virus
We are developing a novel therapeutic that has the potential to significantly improve outcomes in patients infected with RSV. Respiratory syncytial virus infection, or RSV, is a serious respiratory infectious disease and the leading cause of pediatric hospitalizations in the U.S. (125,000 hospitalizations per year in the U.S.) RSV is also a major infectious disease in the elderly and in other adults with compromised immune systems (175,000 hospitalizations per year in the U.S.).
Alnylam's lead program, ALN-RSV01, is being developed for the treatment of RSV infection. Since initiating this program in 2005, we have made rapid progress. Our RNAi therapeutic was designed to target the nucleocapsid "N" gene of the RSV genome, a gene that is required for the replication of the RSV. ALN-RSV01 silences the N gene, thereby reducing the virus' ability to reproduce. Our extensive pre-clinical work demonstrated potent and highly specific anti-viral efficacy with molecular proof of an RNAi mechanism of action. With these promising data in hand, we progressed toward human studies, and our Phase I studies carried out in 2006 and 2007 established the safety and tolerability of ALN-RSV01 in adult human volunteers.
In early 2008, we presented data from our Phase II GEMINI study which showed a statistically significant decrease in infection rate in adults experimentally infected with RSV, demonstrating that ALN-RSV01 has anti-viral activity in a disease setting. With these results in a randomized, double-blind, placebo-controlled study, we believe we have demonstrated human proof of concept with an RNAi therapeutic — a first for the field.
In July 2009, along with our partner Cubist, we reported the complete data from our Phase II study in adult lung transplant patients naturally infected with RSV. These results document for the first time the safety and tolerability of inhaled ALN-RSV01 in naturally infected patients, which we consider an important step forward in the advancement of our overall ALN-RSV program. In addition, while the study was not powered for efficacy and is too small to make firm conclusions, we are encouraged by the 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
Patient enrollment for our Phase IIb trial of ALN-RSV in adult lung transplant patients for the treatment RSV infection was recently completed. This study is aimed to repeat and extend the results we saw in this patient population with our recently reported Phase II study. We expect to report results in mid-2012
We are excited about the progress we have made to date, and believe the results we have demonstrated to date underscore not only the potential to treat RSV, but the broader potential for RNAi therapeutics in human disease.
The ALN-RSV program is partnered with Kyowa Hakko Kirin in Asia and Cubist Pharmaceuticals worldwide except Asia.
ALN-RSV01 Clinical Timeline

