The new PTC299 clinical study is an open-label Phase 2 trial that will evaluate the efficacy, safety, and pharmacodynamics of PTC299 in up to 25 patients with NF2 for up to 48 weeks. The primary objective of the study is to assess how PTC299 affects tumor volume and changes in hearing and word recognition among patients with NF2. The trial will also assess the effects of PTC299 on tumor blood flow and VEGF-related proteins. The study will be conducted at the Massachusetts General Hospital in Boston, Massachusetts.
"We are excited to rapidly advance the clinical development of PTC299 in NF2," said Langdon Miller, M.D., Chief Medical Officer of PTC Therapeutics. "We are hopeful that the novel mechanism of PTC299 will address unmet medical need for patients with NF2, building upon ongoing evaluations of the drug in patients with metastatic breast cancer, Kaposi's sarcoma, and other solid tumors."
In previous Phase 1 clinical studies, PTC299 was generally well tolerated by healthy volunteers who received single or multiple doses, with no serious, dose-limiting or definitively drug-related adverse events reported. All clinical adverse events observed were mild or moderate in severity, did not require intervention, and were not clearly related to PTC299. In addition, adverse events such as bleeding, hypertension and proteinuria were not observed.
The Phase 2 clinical trial of PTC299 is supported by a grant from the U.S. Department of Defense Neurofibromatosis Research Program.
|SOURCE PTC Therapeutics, Inc.|
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