Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has published results from its Phase II GEMINI study in the Proceedings of the National Academy of Sciences (PNAS) (DOI: 10.1073/pnas.0912186107). These data, previously presented at the International Symposium on Respiratory Viral Infections in Singapore in 2008, showed human proof of concept with an RNAi therapeutic in a randomized, double blind, placebo-controlled study. In this study, treatment with ALN-RSV01 showed statistically significant anti-viral activity, including a decreased RSV infection rate and an increase in the number of subjects who remained free of infection.
“a major scientific breakthrough that happens once every decade or so”
"The positive results generated from GEMINI established encouraging human proof of concept with an RNAi therapeutic," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. "GEMINI also set the stage for us to advance ALN-RSV01 into the naturally RSV-infected adult lung transplant population where we are currently conducting a Phase IIb clinical study. We are encouraged by the cumulative data to date in this program and look forward to the program's continued advancement."
GEMINI was a double blind study, where 88 adult subjects experimentally infected with RSV were randomized to receive ALN-RSV01 or placebo. Administration of ALN-RSV01 resulted in statistically significant anti-viral activity with a 38% relative reduction in RSV infection rate (p<0.01) resulting in a 95% increase in the number of infection-free subjects as compared with placebo (p<0.01). ALN-RSV01 was also found to be safe and well tolerated.
"Results from the GEMINI study are notable, as they established anti-viral activity for an RNAi therapeutic in a major respiratory disease and provide a solid foundation for ongoing development of ALN-RSV01," said John P. DeVincenzo, M.D., Professor of Pediatrics and Infectious Diseases and Professor of Molecular Sciences at the University of Tennessee Health Science Center and Principal Investigator for the GEMINI study. "I am enthusiastic about the continued development of RNAi therapeutics for the treatment of RSV infection, a major infectious disease in both adult and pediatric populations where there are no effective treatments used widely today."
Earlier this year, Alnylam initiated a randomized, double blind, placebo-controlled Phase IIb trial of ALN-RSV01 in RSV-infected adult lung transplant patients. The primary endpoint of this study is the incidence of new or progressive bronchiolitis obliterans syndrome (BOS), a life-threatening complication of RSV infection in the transplanted lung resulting in approximately 50% mortality within three to five years of onset. The Phase IIb study is expected to enroll up to 76 patients.
The ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia, and Cubist Pharmaceuticals, Inc. worldwide except Asia. In parallel with Alnylam's development of ALN-RSV01 for the treatment of RSV in lung transplant patients, Alnylam and Cubist are developing a second-generation compound, ALN-RSV02, which will be focused on the pediatric patient population. Cubist will take the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam, and Cubist retains an opt-in right for ALN-RSV01 in the adult transplant indication.