Emmanus Announces Positive Top-Line Results of it’s Phase 3 Clinical Trial for Sickle Cell


Sickle cell patients have had very few treatment options to help them cope with the pain crises brought on by this debilitating disease.  A new drug for sickle cell, developed by Emmaus Life Sciences, today announced positive results for Phase 3 of its clinical trial (see press release below).

The study, which enrolled 230 adults and children with sickle cell disease, showed a statistically significant 25 percent reduction in the median frequency of sickle cell crises and a statistically significant 33 percent reductionin the median frequency of hospitalizations for the treatment group, with a well tolerated safety profile.

The company, founded by Dr. Yutaka Niihara, a hematologist and oncologist at Harbor UCLA Medical Center, intends to submit a New Drug Application to the FDA for marketing approval in mid 2014.

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 (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 percent 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.

“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, M.D., M.P.H., 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 Dr. Niihara at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. The therapy has Orphan Drug designation in the U.S. 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.

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