Following clearance, 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.
The company believes ALN-RSV01 is the most advanced RNAi therapeutic for the treatment of infectious diseases caused by a virus. It is the only RNAi therapeutic known to be in development for the treatment of respiratory syncytial virus (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 US,” said John DeVincenzo, associate professor of pediatrics at the University of Tennessee Health Science Center. “Based on the encouraging preclinical 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 so far show both in vitro and in vivo efficacy of ALN-RSV01 in preclinical animal studies. Intranasally delivered ALN-RSV01 specifically inhibits RSV replication in animals and is active in the prevention and treatment of RSV infection.