PTC has initiated 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).
Reportedly, 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 phase 2 clinical trial of PTC299 is supported by a grant from the US Department of Defense Neurofibromatosis Research Program.
Langdon Miller, CMO of PTC, said: We are excited to rapidly advance the clinical development of PTC299 in NF2. 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.