Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that treatment with VX-787 in a Phase 2 influenza challenge study resulted in statistically significant improvements in viral and clinical measurements of infection. VX-787 is an investigational new class of medicine for the treatment of influenza and is designed to directly inhibit replication of the virus. The study met its primary endpoint, and showed a statistically significant decrease in the amount of virus in nasal secretions (viral shedding) over the seven-day study period. In addition, at the highest dosing regimen evaluated in the study, there was a statistically significant reduction in the severity and duration of influenza-like symptoms. People in this dose group experienced influenza-like symptoms for a median of 1.9 days, compared to 3.7 days in the placebo group. In addition, 93 percent of people in this dose group showed no clinical symptoms of influenza after three days of treatment, compared to 41 percent of people in the placebo group. In this study, VX-787 was generally well-tolerated, with no adverse events leading to discontinuation. Based on these data, Vertex will explore collaborative opportunities to support further development of VX-787.
“There is an urgent need for new medicines targeting influenza that work more quickly, address resistant and pandemic strains, and are effective when taken more than two days after symptoms appear,” said Chris Wright, M.D., Ph.D., Vertex's Senior Vice President, Global Medicines Development and Medical Affairs. “Further development of VX-787 may offer an opportunity to address these needs. The data from this proof-of-concept study validate VX-787’s new mechanism of action, and underscore its potential to significantly reduce the severity and duration of influenza.”
About the Phase 2a Study
This double-blind, randomized, placebo-controlled Phase 2a study of VX-787 enrolled and dosed 104 healthy people (72 in the VX-787 arms; 32 in the placebo arm) ages 18 to 45 who volunteered to be experimentally exposed to an attenuated form of live H3N2 influenza A virus. H3N2 is a common type of influenza virus, and was the most common type observed in the 2012/2013 influenza season in the United States. The study evaluated four dosing regimens of VX-787 given once-daily (QD) for five days beginning 24 hours after infection with flu virus. This was followed by an additional two days of observation. The study was conducted in a controlled quarantine facility with participants under close observation.