Emmaus Life Sciences

Emmaus Life Sciences, Inc. (OTC: EMMA) is a commercial-stage biopharmaceutical company focused on developing and commercializing treatments for rare and orphan diseases, with its principal marketed product being Endari (L-glutamine oral powder) — the first FDA-approved treatment specifically indicated to reduce the acute complications of sickle cell disease in adults and pediatric patients five years of age and older. According to its website at emmausmedical.com, Endari received FDA approval in 2017 following Phase 3 clinical trial results demonstrating that L-glutamine therapy significantly reduced the frequency of sickle cell crises and hospitalizations compared to placebo in patients with sickle cell disease. Sickle cell disease is a serious, lifelong blood disorder affecting millions of patients worldwide, particularly those of African, Mediterranean, Middle Eastern, and Indian descent, and the development of effective treatments that reduce disease burden represents a significant medical need.

Endari's mechanism of action, described at emmausmedical.com, involves increasing the ratio of nicotinamide adenine dinucleotide (NAD) redox potential in sickle red blood cells — improving the oxidative stress profile of sickle erythrocytes and reducing their adherence to vascular endothelium, which in turn reduces the vaso-occlusive events that cause painful sickle cell crises, acute chest syndrome, and other acute complications. L-glutamine is a naturally occurring amino acid that serves as a precursor to NAD biosynthesis, and Emmaus Life Sciences has developed a pharmaceutical-grade oral powder formulation that delivers therapeutic amounts of L-glutamine in a convenient, easily consumed dosage form that has demonstrated safety and tolerability in both adult and pediatric sickle cell patients. Endari is taken twice daily with food and can be prescribed as an adjunct to hydroxyurea, the most widely used pharmacological treatment for sickle cell disease.

Emmaus Life Sciences markets Endari through a specialty pharmaceutical commercial organization focused on hematologists, pediatric hematologists, and sickle cell disease specialists at major academic medical centers and community practices that manage significant populations of sickle cell disease patients. According to emmausmedical.com, the company also provides patient support services that help eligible patients access Endari through specialty pharmacy channels, including reimbursement assistance programs for patients whose insurance coverage may require prior authorization or other documentation. Emmaus Life Sciences' combination of its FDA-approved Endari product, rare disease market focus, specialty commercial organization, and growing awareness of L-glutamine therapy among sickle cell disease specialists positions it as a committed participant in improving the lives of patients with this serious and historically undertreated hematologic disorder.