Emmaus completes all patient visits for Phase 3 trial of L-glutamine for treatment of sickle cell disease

Jan 8 2014

Emmaus Medical, Inc. (the “Company,” or “Emmaus”), a biopharmaceutical company engaged in the discovery, development and commercialization of innovative and cost-effective treatments and therapies primarily for rare and orphan diseases, today announced it has completed all patient visits for its Phase 3 clinical trial for the treatment of sickle cell disease. Emmaus expects top-line data from the Phase 3 clinical trial to be available in the first quarter of 2014.

The Phase 3 trial, which completed enrollment in December 2012, was a prospective, randomized, double-blind, placebo-controlled, parallel-group, multi-center study designed to evaluate the safety and efficacy of L-glutamine as a therapy for sickle cell anemia and sickle β⁰-thalassemia. Study enrollment totaled 230 patients across 31 sites in the United States.

“We are very proud to have completed the last patient visit of our Phase 3 clinical trial,” said Dr. Yutaka Niihara, M.D., M.P.H., founder and CEO of Emmaus Medical. “As a physician and researcher I have dedicated much of my life to treating patients with sickle cell disease and to finding a widely available, safe and effective treatment to help them. With the completion of our patient visits for this trial, we are now potentially one step closer to bringing the first new treatment to patients in more than 20 years.”

L-glutamine treatment for sickle cell anemia and sickle β⁰-thalassemia is a patent protected treatment, for which research has been led by Dr. Niihara and investigators at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. L-glutamine for the treatment of sickle cell disease has received Fast Track designation from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from both the FDA and the European Commission.