Emmaus Life Sciences Inc (OTCQX:EMMA) announced positive transfusion data from a post-hoc analysis of its Phase 3 study of Endari in patients with sickle cell disease.
Endari is a prescription L-glutamine oral powder that is approved by the US Food and Drug Administration to reduce complications of sickle cell disease.
Analysis of the Phase 3 data indicated that patients who got blood transfusions required 43% fewer units of red blood cells when treated with Endari.
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According to Emmaus, patients in the Endari treatment arm required 2.86 units per patient-year in the treatment group, versus 5.38 units per patient-year in the placebo group.
There was also a lower trend in the average cumulative number of red blood cell transfusion episodes in the treatment arm than the placebo arm during the 48-week treatment period: 1.702 red blood cell transfusion episodes per patient-year in the Endari arm compared to 2.659 red blood cell transfusion episodes per patient-year in the placebo arm, the company said.
Phase 3 involved 230 patients randomized 2:1, with 152 patients receiving Endari and 78 patients receiving a placebo. Following 48 weeks of therapy, patients in the Endari group had significantly fewer pain crises and fewer hospitalizations than those in the placebo group, according to a release.
The post-hoc analysis confirms that Endari meaningfully reduces both pain crises and hospitalizations for patients with sickle cell disease, Emmaus CEO Yutaka Niihara told investors.
"Importantly, for patients requiring blood transfusions, those treated with Endari required approximately 43% fewer units of red blood cells, which is important considering the fact that two-thirds of participants in both arms of this study were already on hydroxyurea therapy,” Niihara said in a statement.
“This serves to illustrate that Endari can safely be used in combination with other therapeutics to provide additional benefits to sickle cell disease patients in need."
The data was introduced in a poster presentation on December 13, 2021, at the 63rd American Society of Hematology Annual Meeting and Exhibition in Atlanta, Georgia.
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