Alnylam Pharmaceuticals, Inc. has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a human clinical trial of ALN-RSV01, its proprietary RNAi therapeutic for the treatment of patients with respiratory syncytial virus (RSV) infection.
Following clearance of the IND by the FDA, Alnylam plans to initiate a Phase I safety study in healthy adult volunteers, representing the first program the company will have advanced to human clinical trials. Alnylam is also preparing to initiate a Phase I trial in Europe.
"Submission of our IND for ALN-RSV01 is a major milestone towards Alnylam's goal of developing and commercializing RNAi therapeutics to treat important diseases with significant unmet medical need. Pending IND clearance, we expect to initiate human clinical studies by year-end," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam.
"ALN-RSV01, our first Direct RNAi program to enter the clinical phase, represents a novel approach for the treatment of RSV. This program is a clear example of the potential for RNAi therapeutics both to target previously 'non-druggable' targets and to treat major infectious diseases."
ALN-RSV01 is believed to be the most advanced RNAi therapeutic for the treatment of infectious diseases caused by a virus. It is also the only RNAi therapeutic known to be in development for the treatment of RSV infection, a major cause of respiratory illness in young children, the elderly and in immune-deficient patient populations.
"Effective antiviral drugs to treat RSV infection, particularly in pediatric populations, are greatly needed as RSV infection is the leading cause of infant hospitalizations in the United States," said John P. DeVincenzo, M.D., Associate Professor of Pediatrics at the University of Tennessee Health Science Center.
"Based on the encouraging pre-clinical data for ALN-RSV01, and a novel mechanism of action demonstrating impressive antiviral target specificity, I'm excited about the potential of advancing this promising new therapy into clinical trials."
ALN-RSV01 selectively and potently silences the RSV nucleocapsid 'N' gene. This gene is among the most highly conserved genes in the virus and is essential for RSV replication. Data presented to date show both in vitro and in vivo efficacy of ALN-RSV01 in pre-clinical animal studies.
Intranasally delivered ALN-RSV01 specifically inhibits RSV replication in animals and is active in the prevention and treatment of RSV infection. Additionally, Alnylam scientists have reported completion of GLP toxicology studies for ALN-RSV01 without any significant toxicities being observed.