Concert Pharmaceuticals Announces NIH Research Collaboration for Rare and Neglected Diseases

Concert Pharmaceuticals, Inc.

Concert Pharmaceuticals Announces NIH Research Collaboration for Rare and Neglected Diseases

November 15, 2011

Lexington, MA – Concert Pharmaceuticals, Inc. today announced that it has entered into a research collaboration with The National Institutes of Health (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program.  The collaboration was established to advance Concert’s deuterium-modified praziquantel program as a potential treatment for schistosomiasis, a disease caused by parasitic worms that affects more than 200 million people worldwide.  TRND will support the collaborative evaluation of Concert’s deuterated compounds in a series of in vitro and in vivo preclinical studies and potentially through Phase 1 clinical testing.  The research program will be directed by a joint Concert-TRND steering committee.

“Our early research demonstrates that deuterium modification may address current limitations of praziquantel treatment,” said Roger Tung, Ph.D., President and CEO of Concert Pharmaceuticals.  “We look forward to collaborating with TRND to advance a treatment for this important, underserved need.”

According to the Centers for Disease Control and Prevention, schistosomiasis is second only to malaria as the most devastating parasitic disease worldwide.  Current standard of care for schistosomiasis is treatment with oral praziquantel (PZQ). Because it is highly metabolized through a pronounced first-pass effect, PZQ has low effective bioavailability.  As a result, in order to achieve therapeutic blood levels patients are treated repeatedly with large doses of the drug.  Research carried out at Concert through its deuterated chemical entity platform (DCE Platform™) suggests that deuterium-modified analogs of PZQ may require a substantially reduced effective dose.  These analogs are also single enantiomers which may address the notably bitter taste and vomiting associated with PZQ, potentially resulting in improved tolerability.

About TRND

Therapeutics for Rare and Neglected Diseases (TRND) program (currently administered by the NIH Center for Translational Therapeutics, an intramural program of the National Human Genome Research Institute) is part of a congressionally mandated program to encourage and speed the development of new drugs for rare and neglected diseases. This program is specifically intended to stimulate drug discovery and development research collaborations between NIH and academic scientists, non-profit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. The TRND program provides an opportunity to partner with and gain access to rare and neglected disease drug development capabilities, expertise, and clinical/regulatory resources in a collaborative environment with the goal of moving promising therapeutics into human clinical trials. TRND uses an application and evaluation process to select collaborators. Selected investigators provide the drug project starting points and ongoing biological/disease expertise throughout the project.

About Concert

Concert Pharmaceuticals is a clinical stage biotechnology company focused on applying the company’s DCE Platform™ (deuterated chemical entity platform) to create novel and differentiated small molecule drugs. Concert’s approach leverages decades of pharmaceutical and clinical experience to reduce the time, risk and expense needed to create important new medicines.  The company has a broad research pipeline encompassing many therapeutic areas including antiviral disease, renal disease, and CNS disorders, among others.  Founded in 2006, Concert has raised more than $110 million of venture and institutional capital.  For more information on Concert Pharmaceuticals, please visit www.concertpharma.com.