Emmaus Life Sciences releases top Phase III results for sickle cell anemia treatment

US biotech company Emmaus Life Sciences, which focuses on orphan diseases, has announced preliminary top-line results of its Phase III 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.

Study results

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 US sites. For the primary endpoint, top-line data based on an initial analysis revealed a statistically significant 25% 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.