PTC Therapeutics Receives $1 Million Challenge Grant Award from the National Institutes of Health

Wednesday, December 2, 2009 General News
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- Funding supports continued focus on rare disorders -

SOUTH PLAINFIELD, N.J., Dec. 2 -- PTC Therapeutics,

Inc. (PTC) today announced the receipt of a $1 million Challenge Grant award from the National Heart, Lung, and Blood Institute (NHLBI) as part of the National Institutes of Health (NIH).  According to the NIH, 840 grants were awarded out of the approximately 20,000 applications they received for the Challenge Grants, a new program under the American Recovery and Reinvestment Act designed to stimulate new areas of research.  This was the largest response to a grant in NIH's history.  The two-year grant will support an ongoing Phase 2a clinical trial of ataluren in hemophilia A and B due to a nonsense mutation.

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"We are honored to receive this grant from the NIH," said Stuart W. Peltz, Ph.D., president and Chief Executive Officer of PTC Therapeutics.  "A majority of the grants were awarded to academic institutions, non-profits and hospitals and we are proud to be one of the few corporations to receive funding.  This grant supports our continued commitment to developing innovative therapies for patients with serious and life threatening conditions."

Patients with hemophilia, a rare and debilitating genetic disorder, have a loss of blood clotting proteins, which can lead to serious, recurrent bleeding episodes.  Current therapies require repeated, frequent intravenous infusions to maintain their protein levels.  PTC recently announced the initiation of the Phase 2a clinical trial of ataluren, an oral investigational new drug, in adult male patients with nonsense mutation hemophilia A and nonsense mutation hemophilia B.  Approximately 10 to 30 percent of patients with hemophilia have the disease due to a nonsense mutation.  More information regarding hemophilia is available through the National Hemophilia Foundation ( and through the NHLBI (  Additional information on the clinical trial can be found on (NCT00947193 or keywords: ataluren and hemophilia).

About Challenge Grants

The American Recovery and Reinvestment Act (ARRA) of 2009 makes supplemental appropriations for job preservation and creation, infrastructure investment, energy efficiency and science, assistance to the unemployed and State and local fiscal stabilization.  Through the ARRA, NIH awarded $389,161,204 to 840 projects for the Challenge Grants Program in Health and Science Research.  This new program is designed to spur new areas of research and supports research on Challenge Topics, which address specific scientific and health research challenges in biomedical and behavioral research that will benefit from significant two-year jumpstart funds.  Challenge Areas, defined by the NIH, focus on specific knowledge gaps, scientific opportunities, new technologies, data generation, or research methods that would benefit from an influx of funds.  The research in these areas should have a high impact in biomedical or behavioral science and/or public health.  The grant is supported by Award Number RC1HL100159 from the NIH's National Heart, Lung, and Blood Institute.  The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Heart, Lung, and Blood Institute.

About PTC Therapeutics

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of oral drugs that target post-transcriptional control processes.  Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function.  PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases.  PTC has developed proprietary technologies that it applies in its drug discovery activities and are the basis for collaborations with leading biopharmaceutical companies such as Celgene, Genzyme, Gilead, Pfizer, Merck and Roche.  For more information, visit the company's web site at

SOURCE PTC Therapeutics, Inc.

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