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AesRx, LLC, announced it has received regulatory and ethics committee approval for a Phase 2 clinical trial of its novel Aes-103 anti-sickling agent in patients with sickle cell disease (SCD). The Company plans for patient enrollment to start this month.
This is the first Phase 2 trial of Aes-103 for SCD and as such marks an important milestone in the drug's development. This double-blind, placebo controlled study will evaluate the safety and pharmacokinetics of 28-day dosing of Aes-103. In addition, the impact of Aes-103 on several sickle cell clinical endpoints will be evaluated. The trial is part of an ongoing collaboration between AesRx and the National Center for Advancing Translational Sciences (NCATS) through its Therapeutics for Rare and Neglected Diseases (TRND) program. NCATS is one of the National Institutes of Health (NIH).
"Aes-103 is now in Phase 2. This is a major step forward for the program," commented Stephen R. Seiler, AesRx's Chief Executive Officer. "In 2012 we completed a Phase 1 trial in healthy volunteers and recently successfully completed a Phase 1/2a trial in patients with stable sickle cell disease. Aes-103 doses from 300mg to 4000mg per day were observed to be safe and well tolerated in both healthy volunteers and sickle cell patients. We have reported the results of the Phase 1 trial (American Society of Hematology, Dec. 2012) and anticipate presenting the results of the Phase 1/2a trial at a major scientific conference later this year. The current Phase 2 trial will allow us for the first time to begin examining not only safety over a longer dosing period, but also the potential impact of Aes-103 on some of the important clinical symptoms associated with SCD."
"TRND researchers have worked collaboratively with AesRx to provide preclinical drug development expertise and clinical and regulatory resources necessary to move Aes-103 into human clinical trials," said John McKew, Ph.D., acting director of NCATS Division of Preclinical Innovation and director of TRND. "Through TRND, NCATS supports innovative methods and collaborative approaches to accelerate the discovery process while developing promising treatments for the patients who need them."
Aes-103 has been designated as an orphan drug by the U.S. Food and Drug Administration. It is a first-in-class, orally bioavailable small molecule for the treatment of SCD. AesRx believes it is the only drug currently in human trials that directly blocks cell sickling.
Sickle cell disease is a recessive disorder of the hemoglobin which can lead to a wide range of serious, sometimes life-threatening, conditions including: chronic hemolytic anemia, chronic pain and acute painful crisis, stroke, acute chest syndrome, and cumulative damage to tissues and organs. More than 100,000 people in the United States are afflicted with sickle cell disease.
Note: References to the National Institutes of Health, its programs or its staff, should not be viewed as an endorsement or implied endorsement of AesRx, its products or services.
Additional information about sickle cell disease is available at:
Sickle Cell Anemia, http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_WhatIs.html
Sickle Cell Disease, http://www.cdc.gov/NCBDDD/sicklecell/index.html
Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
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