TORRANCE, Calif.--(BUSINESS WIRE)--VIEW E-MEDIA KIT – Emmaus Life Sciences, Inc. (Emmaus) announced today that the New England Journal of Medicine (NEJM) 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.
The article reports results that showed significantly fewer sickle cell crises in those receiving Endari compared to placebo by 25 percent; p=0.005 (median 3 vs. median 4) and significantly fewer hospitalizations by 33 percent; p=0.005 (median 2 vs. median 3). Additional findings showed lower cumulative days in hospital of 41 percent; p=0.02 (median 6.5 days vs. median 11 days) and a lower incidence of acute chest syndrome (ACS) by more than 60 percent; p=0.003 (13 of 152 patients [8.6%] had at least 1 ACS compared with 18 of 78 in the placebo group [23.1%]). The most common adverse reactions, occurring in greater than 10 percent, of the clinical study were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain.
“Endari is the first approved treatment for sickle cell disease in pediatric patients 5 years of age and older and the first in nearly 20 years for adults. 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.
“Sickle cell disease affects thousands of people in the United States,” said Beverley Francis-Gibson, President and CEO of The Sickle Cell Disease Association of America, Inc. “While there is no universal cure for this life-threatening disease, patient awareness and education on treatment options remain important factors for the sickle cell community.”
Additional Information About the Phase 3 Clinical Study
The
randomized, double-blind, placebo-controlled, multicenter Phase 3 trial
evaluated the efficacy and safety of Endari (0.3 gram per kilogram of
body weight per dose) administered twice daily by mouth, as compared
with placebo. The study included patients at least 5 years of age with
sickle cell anemia or sickle β0-thalassemia, with a history
of two or more pain crises during the previous year. Patients who were
receiving hydroxyurea at a dose that had been stable for at least 3
months before screening and who continued such therapy during the
48-week treatment period were eligible. A total of 230 patients (age
range, 5 to 58 years; 53.9% female) were randomly assigned, in a 2:1
ratio, to receive L-glutamine (152 patients) or placebo (78 patients).
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
Endari (L-glutamine oral powder)
is a prescription oral treatment approved by the U.S. Food and Drug
Administration (FDA) to reduce the acute complications of sickle cell
disease in adult and pediatric patients 5 years of age and older.
Endari was approved in July 2017 and was the first treatment in nearly
20 years shown to reduce the acute complications of sickle cell disease
in adults when used as directed. Endari is also the first treatment
approved to reduce the acute complications of sickle cell disease in
children five years and older when used as directed. Endari has received
Orphan Drug designation in the U.S., and Orphan Medicinal Product
designation in the EU.
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.
For more information, please see full Prescribing Information of Endari at: www.ENDARIrx.com/PI
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/.
Forward-Looking Statements
This press release contains
forward-looking statements as that term is defined in the Private
Securities Litigation Reform Act of 1995, regarding the research,
development and potential commercialization of pharmaceutical products.
Such forward-looking statements are based on current expectations and
involve inherent risks and uncertainties, including factors that could
delay, divert or change any of them, and could cause actual outcomes and
results to differ materially from current expectations. Additional risks
and uncertainties are described in reports filed by Emmaus Life
Sciences, Inc., with the U.S. Securities and Exchange Commission,
including its Annual Report on Form 10-K and Quarterly Reports on Form
10-Q. Emmaus is providing this information as of the date of this press
release and does not undertake any obligation to update any
forward-looking statements as a result of new information, future events
or otherwise.
Article Reference
Niihara, Y, et al. A Phase 3 Trial of L-Glutamine in Sickle Cell Disease. NEJM to be published 19 July 2018
