Emmaus Life Sciences reports results of Phase III trial for sickle cell disease

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 (p=0.008) 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 (p=0.018) over a 48-week time period. Both adult and pediatric patients receiving treatment demonstrated improvement. Furthermore, the therapy demonstrated a well-tolerated safety profile.

Emmaus Life Sciences founder and CEO Dr Yutaka Niihara noted the company is very pleased with the strength of its Phase III 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. 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," Dr Niihara added.

The company’s research on sickle cell disease and sickle beta-0 thalassemia was initiated by Dr Niihara at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center.

The therapy has Orphan Drug designation in the US 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 2014.