PTC Therapeutics Initiates Phase 2 Clinical Trial of PTC299 in Patients with Neurofibromato...( SOUTH PLAINFIELD N....)

SOUTH PLAINFIELD, N.J., July 27 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of a Phase 2 clinical trial of PTC299 in adult patients with neurofibromatosis type 2 (NF2), a rare genetic disorder that causes the development of non-malignant brain tumors. PTC299 is a novel, orally administered investigational new drug that is designed to selectively block tumor-related vascular endothelial growth factor (VEGF). With its novel mechanism of action, PTC299 has the potential to meet significant unmet medical need in NF2 and other conditions resulting from the overexpression of VEGF.

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VEGF plays a critical role in the formation of new blood vessels or angiogenesis and its suppression has been shown to decrease tumor size and improve hearing in some patients with NF2. PTC299 selectively blocks the tumor-related production of VEGF, while sparing the physiological VEGF expression necessary to maintain the growth and function of normal blood vessels. Patients with NF2 typically develop tumors that compress the auditory nerves, often leading to deafness and problems with balance. If unchecked, NF2-related tumors may eventually damage other cranial nerves and the brainstem, and become life-threatening. Current standard treatments for NF2-related tumors include surgery and radiation therapy. However, a study conducted at Massachusetts General Hospital and recently published in the New England Journal of Medicine validates the potential of anti-angiogenesis therapies for NF2 patients.

"The development of PTC299 for the treatment of NF2 is noteworthy for patients, especially following our recent findings that an angiogenesis inhibitor improved hearing and led to tumor shrinkage in some patients," said Sco
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