SOUTH PLAINFIELD, N.J., Sept. 26 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post- transcriptional control processes, today announced that it has received a three-year, $2.2 million grant from the Department of Defense (DoD) to fund preclinical and clinical development of the company's product candidate PTC299 as a potential treatment for breast cancer.
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PTC299 was discovered through PTC's proprietary Gene Expression Modulation by Small-molecules (GEMS) technology. PTC299 is an orally-administered small molecule designed to inhibit the production of vascular endothelial growth factor, or VEGF. PTC299 functions by targeting the post-transcriptional control processes that regulate VEGF formation. PTC299 inhibits VEGF production through a mechanism that is distinct from other VEGF inhibitors. VEGF plays a major role in the growth of most tumors, therefore PTC299 may have applicability in many cancer types, including breast cancer. PTC299 is currently in Phase 1 clinical trials in healthy volunteers.
"We are very pleased to receive this award from the DoD to assist us in the Phase 1-2 development of PTC299 patients with breast cancer," stated Langdon Miller, M.D., Chief Medical Officer, PTC Therapeutics, Inc. and principal investigator for the grant. "The grant provides important validation to PTC's innovative scientific approach of modulating post- transcriptional control elements with a small molecule to treat human diseases."
PTC is collaborating with researchers at the New York University (NYU) Medical Center in New York City. Dr. Robert Schneider, Director of Translational Cancer Research, Co-Director of the Breast Cancer Research Program and an Associate Director of the NYU Cancer Institute, who serves as a co-principal investigator for the DoD award, commented, "This collaboration combines the preclinical and clinical development expertise of an experienced biopharmaceutical company and a DoD Breast Cancer Center of Excellence. This funding will support translation of preclinical pharmacological and biomarker data into the clinic while offering the potential to address the high unmet medical need of patients with breast cancer."
About Department of Defense Clinical Translational Research Award
The DoD Clinical Translational Research Award sponsors innovative preclinical and clinical/translational research that may result in substantial improvements over current approaches to breast cancer chemoprevention and therapy. As part of the Congressionally Directed Medical Research Programs, it administers funds for peer-reviewed research toward specific diseases and supports research that positively affects the health and well-being of Americans. For this grant (grant number W81XWH-06-1-0629), the U.S. Army Medical Research Acquisition Activity, 820 Chandler Street, Fort Detrick, MD 21702-5014 is the awarding and administering acquisitions office. The content of this press release does not necessarily reflect the position or the policy of the Government, and no official endorsement should be inferred.
About NYU Medical Center Breast Cancer Research
Located in the heart of New York City, NYU Medical Center is one of the nation's premier centers of excellence in health care, biomedical research, and medical education. The mission of the NYU Cancer Institute is to decrease and eliminate cancer as a significant health problem throughout New York, the nation, and the world, by developing and maintaining excellent programs in patient care, research, education, and prevention.
About GEMS
Gene Expression Modulation by Small-molecules (GEMS) is PTC's novel and proprietary screening technology for the identification of small-molecules that modulate post-transcriptional control mechanisms. Compounds identified through the GEMS technology modulate gene expression by targeting the post- transcriptional control processes that act through the untranslated regions (UTRs) of messenger RNA (mRNA) molecules. GEMS was the basis for the discovery of PTC299 and is being used in multiple ongoing drug discovery programs.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary 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 has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.
SOURCE PTC Therapeutics, Inc.