LA BioMed Spinoff Announces Publishing of Phase 3 Trial Results from Sickle Cell Disease Drug

Investigator(s)

LA BioMed spinoff, Emmaus Life Sciences has announced that The New England Journal of Medicine has published the results of its 48-week phase 3 clinical trial of Endari™ (L-glutamine oral powder) which supported the FDA approval in July 2017 to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.

"Dr. Niihara and his team have demonstrated that innovative breakthrough therapies are possible for those most in need," said LA BioMed CEO David Meyer, PhD. "We are optimistic about the impact this drug will have on the lives of countless individuals who suffer with this disease"

An estimate 25 million worldwide – including approximately 100,000 people in the US –suffer from sickle cell disease. Patients spend much of their lives in the hospital fighting extreme pain and infections with the only cure being a costly and risky bone marrow transplant.

"Our hope in sharing the results of this data from the New England Journal of Medicine, a publication with worldwide reach and significance, is to aid in increasing the awareness of sickle cell disease, a lifelong hereditary blood disorder which commonly affects those of African descent, as well as those from Central and South America and people of Middle Eastern, Asian, Indian and Mediterranean descent," said co-author Yutaka Niihara, MD, CEO and founder of Emmaus.

About LA BioMed
LA BioMed is an incubator of innovation with a global reach and a 66-year reputation of improving and saving lives. Driven by the positive social impacts of breakthrough therapies, the Southern California-based research institute and incubator – with over 100 principal investigators (PhDs, MDs and MD/PhDs) working on over 600 research studies, including therapies for multiple orphan diseases – is an essential element to one of the nation’s top-ten biotech clusters. Find out more at https://labiomed.org.

About Emmaus Life Sciences, Inc.
Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan disease.  Its lead product, Endari, demonstrated positive clinical results in the completed Phase 3 clinical trial for sickle cell anemia and sickle ß0-thalassemia and has received FDA approval.  Visit: http://www.emmausmedical.com.

About Sickle Cell Disease
Sickle cell disease is an inherited blood disorder characterized by the production of an altered form of hemoglobin which polymerizes and becomes fibrous, causing red blood cells to become rigid and change form so they appear sickle shaped instead of soft and rounded.  Patients with sickle cell disease suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells occlude blood vessels.  Sickle cell crises cause excruciating pain as a result of insufficient oxygen being delivered to tissue, referred to as tissue ischemia, and inflammation.  These events may lead to a variety of other adverse outcomes such as acute chest syndrome that requires hospitalization.  Sickle cell disease is an orphan disease, affecting approximately 100,000 patients in the U.S. and millions worldwide, with significant unmet medical needs.

About Endari™

Indication
Endari (L-glutamine oral powder) is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.

Important Safety Information
The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain and chest pain.

Adverse reactions leading to treatment discontinuation included one case each of hypersplenism, abdominal pain, dyspepsia, burning sensation and hot flash.

The safety and efficacy of Endari in pediatric patients with sickle cell disease younger than five years of age has not been established.

 

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