Emmaus Life Sciences Presents Positive Transfusion Data from a Post-Hoc Analysis of its Phase 3 Clinical Study of Endari® in Patients With Sickle Cell Disease at the 63rd American Society of Hematology Annual Meeting
December 14, 2021 at 08:23 am
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Emmaus Life Sciences, Inc. announced positive transfusion data from a post-hoc analysis of its phase 3 clinical study of Endari®, the company's prescription L-glutamine oral powder, in patients with sickle cell disease (SCD). The data was introduced in a poster presentation on December 13, 2021 at the 63rd American Society of Hematology (ASH) Annual Meeting and Exhibition at the Georgia World Congress Center in Atlanta, Georgia, and virtually. The multicenter phase 3 trial enrolled a total of 230 patients, randomized 2:1, to receive Endari (152 patients) or placebo (78 patients). Following 48 weeks of therapy, patients in the Endari group had significantly fewer pain crises and fewer hospitalizations than those in the placebo group. Two thirds of the patients in both trial groups received concomitant hydroxyurea. The multicenter study of hydroxyurea showed that the treatment group differed from the placebo group both in the number of units of packed red blood cells (RBC) transfused and in the number of patients receiving transfusions. Since an evaluation of transfusions was not pre-specified in the Endari study, a post-hoc analysis was performed on the number of RBCs transfused and on the number of transfusions that took place during the study. Results showed that there was a significant difference in the number of units of RBCs transfused in the Endari treatment arm than in the placebo arm; 2.86 units per patient-year in the treatment group vs. 5.38 units per patient-year in the placebo group. There was a lower trend in the mean cumulative number of RBC transfusion episodes in the treatment arm than the placebo arm during the 48-week treatment period; 1.702 RBC transfusion episodes per patient-year in the Endari arm compared to 2.659 RBC transfusion episodes per patient-year in the placebo arm.
Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company. The Company is engaged in the discovery, development, marketing, and sale of treatments and therapies, primarily for rare and orphan diseases. The Companyâs lead product, Endari (prescription-grade L-glutamine oral powder), helps to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients aged five years and older. Its product pipeline includes ELS001/ELS007, ELS004, ELS005, ELS003, and ELS002. The Company, through Kainos Medicine, Inc. (Kainos), has a license in the territory encompassing the United States, the United Kingdom, and the European Union to patent rights, know-how, and other intellectual property relating to Kainosâs IRAK4 inhibitor, referred to as KM10544, for the treatment of cancers, including leukemia, lymphoma, and solid tumor cancers. It has also developed chondrocyte and osteoblast cell sheets using human mesenchymal stem cells.
Emmaus Life Sciences Presents Positive Transfusion Data from a Post-Hoc Analysis of its Phase 3 Clinical Study of Endari® in Patients With Sickle Cell Disease at the 63rd American Society of Hematology Annual Meeting