Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the pivotal Phase 3 LIBERTY ASTHMA QUEST study of dupilumab in a broad population of patients with uncontrolled, persistent asthma met its two primary endpoints. Dupilumab, when added to standard therapies, reduced severe asthma attacks (exacerbations) and improved lung function. At 52 weeks, in the 300 mg dose group, dupilumab reduced severe asthma attacks by 46 percent in the overall population, 60 percent in patients with 150 eosinophilic cells/microliter or greater, and 67 percent in patients with 300 eosinophilic cells/microliter or greater (p less than 0.001 for all groups). At 12 weeks, in the 300 mg dupilumab dose group, mean improvement in lung function over placebo as assessed by forced expiratory volume over one second (FEV1) was 130 mL (9 percent) in the overall population, 210 mL (11 percent) in patients with 150 eosinophilic cells/microliter or greater, and 240 mL (18 percent) in patients with 300 eosinophilic cells/microliter or greater (p less than 0.001 for all groups). The companies plan to submit a Supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) by the end of this year.

"Approximately one million U.S. adults and adolescents live with uncontrolled, persistent asthma, and continue to experience serious asthma attacks, despite taking an intensive regimen of standard therapies," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "Dupilumab has now demonstrated positive late-stage results in two serious allergic diseases -- asthma and atopic dermatitis -- with robust efficacy and an extensive safety database. These results continue to support our hypothesis that the IL-4/IL-13 pathway is a critical driver of allergic disease, and we remain committed to further investigating the IL-4/IL-13 pathway in other allergic diseases."

The results for the 200 mg and 300 mg dupilumab dose groups were generally comparable on both exacerbations and FEV1. The extent of patient response correlated with allergic or atopic status as reflected by blood eosinophils and other markers. Less activity was observed in patients with less than 150 eosinophilic cells/microliter. The overall rates of adverse events, deaths, infections, conjunctivitis, herpes and discontinuations were comparable between the dupilumab and placebo groups. Injection site reactions were more common in the dupilumab groups occurring in 17 percent of dupilumab patients compared to 8 percent of placebo patients.

Detailed results from this study will be submitted for presentation at a future medical congress. QUEST is the second pivotal trial in uncontrolled persistent asthma following a positive Phase 2b pivotal study of dupilumab. Data from another Phase 3 study known as VENTURE examining the ability of dupilumab to reduce oral corticosteroid use in patients with severe steroid-dependent asthma are expected later this year. Also included in the LIBERTY ASTHMA clinical development program is the TRAVERSE trial, a long-term safety extension study. The potential use of dupilumab in asthma is currently under clinical development and the safety and efficacy have not been fully evaluated by any regulatory authority.