[Ip-health] First drug candidate from NIH program acquired by biopharmaceutical company

Claire Cassedy claire.cassedy at keionline.org
Thu Jul 10 09:20:35 PDT 2014


First drug candidate from NIH program acquired by biopharmaceutical company

Embargoed for Release: Wednesday, July 9, 2014 9 a.m. EDT

Potential treatment targets sickle cell disease

A drug candidate developed by researchers at the NIH’s National Center for
Advancing Translational Sciences (NCATS) and its collaborators to treat
sickle cell disease has been acquired by Baxter International’s BioScience
business. The drug candidate, Aes-103, is the first specifically developed
to target the underlying molecular mechanism of sickle cell disease. Baxter
now will advance the clinical development activities required for
regulatory approval and commercialization.

Sickle cell disease is a genetic blood disorder that affects millions
worldwide, including approximately 100,000 people in the United States —
among them, 1 in 500 African-Americans.

This is the first time a company has acquired a drug candidate developed
with NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program
resources. Baxter International recently acquired AesRx, LLC, Newton,
Massachusetts — the TRND program collaborator — including Aes-103. TRND and
AesRx researchers worked together to develop Aes-103 through a Phase II
clinical trial to evaluate safety and effectiveness. The trial data
indicated that Aes-103 significantly reduced patients’ pain.

“This is a wonderful example of why NCATS was created,” said NIH Director
Francis S. Collins, M.D., Ph.D. “The progress made thus far in the
development of Aes-103 demonstrates NCATS’ catalytic role in bringing
together the necessary players, whether academic, nonprofit or industry, to
overcome obstacles to translation and advance badly needed treatments to

Individuals living with sickle cell disease have defective hemoglobin, the
protein in red blood cells that carries oxygen. This defect causes their
cells to become rigid and crescent-shaped, blocking small blood vessels and
causing inflammation, pain and strokes, and decreased blood flow.

Aes-103 works by binding directly to hemoglobin and changing its structure,
thereby reducing the sickling of red blood cells. This structural change
may lessen sickling-related complications in patients.

Sickle cell disease disproportionately affects African-Americans and is
considered both rare and neglected in the United States. African-Americans
with sickle cell often face significant health disparities in clinical
care. Life expectancy for people with sickle cell disease is only to mid-
to late 40s.

Prior to AesRx’s collaboration with TRND researchers, and despite promising
data on Aes-103, the company had difficulty securing private financing
because potential investors lacked interest in funding an early-stage
project that was considered too risky. AesRx did not have the resources to
complete preclinical and early clinical development.

Currently, the only drug approved by the U.S. Food and Drug Administration
(FDA) to treat sickle cell disease is hydroxyurea, a drug initially
developed to treat cancer. However, the clinical utility of hydroxyurea is
limited. Many individuals with sickle cell disease either do not respond to
the drug, or they may experience undesirable side effects.

“Sickle cell was the first disease to ever have its molecular cause
discovered — more than 65 years ago — and now a potential treatment based
on that discovery has at last been developed,” said NCATS Director
Christopher P. Austin, M.D. “This success validates the NCATS model, which
is based on a novel collaborative approach that de-risks intervention
development programs to enable private-sector investment. We look forward
to applying this model to the thousands of rare diseases that are currently
untreatable so that we realize the NCATS mission of getting more treatments
to more patients more quickly.”

TRND researchers signed a collaborative agreement with AesRx in 2010 and
established a project team made up of NCATS and AesRx scientists as well as
a leading sickle cell disease clinical researcher at the National Heart,
Lung, and Blood Institute (NHLBI). Other key project collaborators received
support through NHLBI grants, the NIH Clinical Center and its pharmacy, and
NCATS’ Bridging Interventional Development Gaps program. Aes-103 was
licensed by AesRx from Virginia Commonwealth University, Richmond, where
the compound was discovered.

“This is an important milestone for the development of this potential
sickle cell disease therapeutic, and we are pleased that NHLBI researchers
were able to play a role in advancing this project from the beginning,”
said NHLBI Director Gary H. Gibbons, M.D. “NHLBI is dedicated to advancing
sickle cell disease research as a strategic priority in an effort to
improve the quality of care received by patients.”

In less than one year, the team completed the preclinical toxicology,
chemistry, manufacturing, controls and regulatory studies necessary to
support an investigational new drug (IND) application, which AesRx filed
with the FDA. After IND clearance, Aes-103 moved into Phase I clinical
trials in healthy volunteers and sickle cell disease patients in 2011 and
into a Phase II trial in patients in 2013. The project results also helped
AesRx obtain a Massachusetts Life Science Accelerator loan to support
development of Aes-103.

“This project may never have reached clinical trials if not for the TRND
program and its preclinical drug development expertise and novel
approaches,” said Stephen Seiler, AesRx’s founder and former CEO. “We
believe Aes-103 has the potential to be a breakthrough in the treatment of
sickle cell disease. TRND’s support for AesRx has enabled us to bring that
potential closer to realization.”

“Acquiring AesRx and this clinical development program is an important
opportunity, as it complements Baxter’s established relationships and
expertise in treating rare and challenging blood disorders,” said Ludwig
Hantson, Ph.D., president of Baxter BioScience. “This investment reflects
our continued focus on addressing high unmet clinical needs for patients
with inadequate treatment options and no recent major clinical

To read more about NCATS and its TRND program, visit

The National Center for Advancing Translational Sciences (NCATS) is a
distinctly different entity in the research ecosystem. Rather than
targeting a particular disease or fundamental science, NCATS focuses on
what is common across diseases and the translational process. The Center
emphasizes innovation and deliverables, relying on the power of data and
new technologies to develop, demonstrate and disseminate improvements in
translational science that bring about tangible improvements in human
health. For more information, visit http://www.ncats.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation's medical
research agency, 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. For more information about NIH and its
programs, visit www.nih.gov.

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