Emmaus Life Sciences Inc. announced that preliminary top-line results of its Phase 3 clinical trial evaluating the safety and efficacy of its treatment for sickle cell anemia and sickle beta-0 thalassemia met both the primary and secondary endpoints of the clinical trial.
The prospective, randomized, double-blind, placebo-controlled, parallel-group, multi-center clinical trial enrolled 230 adult and pediatric patients as young as five years of age, across 31 U.S. sites. For the primary endpoint, top-line data based on an initial analysis revealed a statistically significant 25 percent reduction in the median frequency of sickle cell crises over a 48-week time period. For the secondary endpoint, top-line data based on an initial analysis also showed a statistically significant 33% reduction in the median frequency of hospitalizations over a 48-week time period. Both adult and pediatric patients receiving treatment demonstrated improvement. Furthermore, the therapy demonstrated a well-tolerated safety profile.
“We are very pleased with the strength of our Phase 3 data with respect to the primary and secondary endpoints. We intend to submit a New Drug Application to the FDA in mid-2014 for marketing approval of this treatment for sickle cell disease patients,” said Dr. Yutaka Niihara, founder and CEO of Emmaus Life Sciences. “I particularly want to acknowledge the patients and medical centers whose participation made this clinical trial possible and the support of our investors who helped us achieve this significant milestone.”
The company’s research on sickle cell disease and sickle beta-0 thalassemia was initiated by Niihara at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. The therapy has Orphan Drug designation in the United States and Europe, and Fast Track designation from the FDA. Further results from the clinical trial will be released when available and are expected to be presented at a scientific meeting later this year.
Date: March 19, 2014
Source: Emmaus Life Sciences