NHLBI awards stimulus funds for hemophilia drug research

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.

"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 (www.hemophilia.org) and through the NHLBI (http://www.nhlbi.nih.gov/health/dci/Diseases/hemophilia/hemophilia_what.html). Additional information on the clinical trial can be found on clinicaltrials.gov (NCT00947193 or keywords: ataluren and hemophilia).


PTC Therapeutics, Inc.


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