Release Details

SOUTH PLAINFIELD, NJ – October 22, 2012 – PTC Therapeutics, Inc. (PTC) today announced the receipt of a $1 million grant award from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH) to discover antiviral agents for the treatment of dengue fever, a mosquito-borne disease that is a leading cause of illness and death in tropical and subtropical regions around the world. The one-year grant will support research into promising compounds identified by PTC's proprietary GEMS™ (Gene Expression Modulation by Small-molecules) technology that demonstrate activity against the dengue virus (DENV).

"Dengue is a major public health problem worldwide and continues to spread uncontrollably," said Eva Harris, Ph.D., Professor, Division of Infectious Diseases and Vaccinology and Director, Center for Global Public Health, University of California Berkeley School of Public Health, and a collaborator on the grant with PTC. "In the absence of effective vaccines or mosquito control programs, development of antivirals that can reduce both the burden of classic dengue fever and the risk of developing more severe, life-threatening dengue disease is critical."

Through its GEMS technology, PTC has established a cell-based DENV assay to screen a series of small molecules aimed at inhibiting key components of the virus. The NIH grant enables the company to continue investigating these compounds with the goal of developing the first oral therapy specifically designed for the treatment of dengue.

"We are honored to receive this grant from the NIH, which is an important validation of the applicability of PTC's science," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "There are currently no treatments specifically targeting dengue and the disease represents a growing global health concern."

ABOUT DENGUE
According to the NIH, worldwide, about 50 million cases of dengue infection occur each year, with 22,000 deaths, mostly in children. This includes 100 to 200 cases in the United States, mostly in people who have recently traveled abroad. Many more cases likely go unreported because some healthcare providers do not recognize the disease. In the Western hemisphere, the estimated economic burden of dengue is about $2.1 billion per year. During the last part of the 20th century, many tropical regions of the world saw an increase in dengue cases. Epidemics also occurred more frequently and with more severity. In addition to typical dengue, dengue hemorrhagic fever (DHF) and dengue shock syndrome also have increased in many parts of the world. Globally, there are an estimated several hundred thousand cases of DHF per year. More information about Dengue can be found at http://www.niaid.nih.gov/topics/DengueFever/Understanding/Pages/overview.aspx

ABOUT THE NIH GRANT
This research is supported by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health under Award Number R56AI098837. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

ABOUT PTC THERAPEUTICS, INC.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered small-molecule 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 rare genetic disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and that have served as the basis for collaborations with leading biopharmaceutical companies. For more information, visit the company's website at www.ptcbio.com.

FOR MORE INFORMATION:

Jane Baj                                            Sheryl Seapy
PTC Therapeutics, Inc.                     Pure Communications
(908) 912-9167                                 (949) 608-0841
jbaj@ptcbio.com                               sheryl@purecommunicationsinc.com