NEWTON, MA, DECEMBER 6, 2011. AESRX, LLC announced today it has
commenced a clinical trial of Aes-103, the company’s novel anti-sickling agent. Aes-103,
designated as an orphan drug by the U.S. Food and Drug Administration, is a first-in-
class, orally bioavailable small molecule therapeutic under investigation for the treatment
of sickle cell disease (SCD). This trial will examine the safety and tolerability of Aes-103
in healthy volunteers as well as the drug’s pharmacokinetic and pharmacodynamic
Aes-103’s proposed mechanism of action is targeted to reduce cell sickling. AesRx
believes it is the only anti-sickling drug currently in human trials. The present trial is part
of an ongoing collaboration between AesRx and two separate components of the National
Institutes of Health (NIH) -- the National Heart, Lung, and Blood Institute (NHLBI) and
the Therapeutics for Rare and Neglected Diseases (TRND) program. The collaboration is
planned to develop Aes-103 through completion of initial proof of principle trials.
“We are obviously delighted with today’s announcement,” commented Stephen R Seiler,
AesRx’s Chief Executive Officer. “Aes-103 represents an attractive development
opportunity in our view because the proposed mechanism of action has been widely
studied and X-ray crystallography indicates it binds a target of interest on sickle
hemoglobin. This trial marks an important milestone in the development of Aes-103. We
are extremely gratified to have had support from the NIH to advance the research on this
“We are very excited to see Aes-103 being studied in a clinical trial,” added Lanetta B.
Jordan, M.D., M.P.H., M.S.P.H., Chief Medical Officer of the Sickle Cell Disease
Association of American and a member of AesRx’s Strategic Advisory Board. “If
successful, it would represent a breakthrough in the treatment of sickle cell disease.
Although SCD was first discovered over 100 years ago, there has never been a drug
developed specifically to treat SCD and there is currently no known drug that directly
blocks the sickling which causes the morbidity and mortality associated with the disease.
We look forward to the further development of this important therapeutic.”
About NIH, NHLBI, and TRND
The National Institutes of Health (NIH) is the nation's medical research agency. It
includes 27 Institutes and Centers and is a component of the U.S. Department of Health
and Human Services. NIH is the primary federal agency conducting and supporting basic,
clinical, and translational medical research, and is investigating the causes, treatments,
and cures for both common and rare diseases.
The NHLBI plans, conducts, and supports research related to the causes, prevention,
diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep
TRND is overseen by the NIH Center for Translational Therapeutics (NCTT), which is
administered by the National Human Genome Research Institute, another component of
the NIH. NCTT’s Bridging Interventional Development Gaps (BrIDGs) program also
contributed research material to develop Aes-103.
AesRx is a biopharmaceutical company dedicated to the development of treatments for
two orphan diseases. The Company’s lead program (Aes-103) is targeted to the treatment
of sickle cell disease. 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 13 million
individuals world-wide are afflicted with sickle cell disease. Aes-103 works by increasing
the affinity of sickle hemoglobin for oxygen. Because only red blood cells with no bound
oxygen will sickle, increasing the ability of the sickle red blood cells to bind oxygen
reduces the number of cells that can sickle. AesRx is developing Aes-103 in collaboration
with the National Institutes of Health. AesRx’s second development program, Aes-210, is
targeted to treat certain inflammatory diseases of the lower intestine, including distal
ulcerative colitis, pouchitis and radiation induced proctitis.
This press release contains certain statements that may be forward-looking within the
meaning of Section 27a of the Securities Act of 1933, as amended, including statements
relating to the product portfolio, pipeline and clinical programs (collectively the
“Products”) of AesRx LLC (the “Company”), the market opportunities for the Products,
the potential effectiveness of the Products based on the interpretation of past and/or
planned pre-clinical or clinical data and the Company’s goals and objectives. These
statements are subject to numerous risks and uncertainties.