An open-label Phase Ib clinical trial is expected to enroll up to 42 patients with locally advanced or metastatic solid tumors in order to evaluate the safety and efficacy of multiple doses of PTC299 alone and in combination with Taxotere chemotherapy. The primary objective of the study is to determine the maximum tolerated dose of PTC299.
This trial is also expected to assess the overall safety profile of the drug when administered alone and in combination with chemotherapy, evaluate its effect on the production of vascular endothelial growth factor (VEGF), its antitumor activity, and its pharmacokinetics. The study will be conducted at Memorial Sloan-Kettering Cancer Center in New York.
In addition, a second open-label Phase I/II clinical trial is expected to enroll up to 45 patients with Kaposi’s sarcoma associated with human immunodeficiency virus infection. The primary objectives of the study are to determine the maximum tolerated dose of PTC299 and to evaluate the overall safety and efficacy profile of the drug as therapy for Kaposi’s sarcoma.
PTC299 is an orally delivered, investigational new drug that offers an innovative approach to anti-angiogenesis, limiting the formation of new blood vessels for tumors.
Langdon Miller, chief medical officer of PTC Therapeutics, said: PTC299 offers a new approach to anti-VEGF therapy in cancer because it appears selective for pathological VEGF production and acts early in the VEGF pathway, upstream of currently available anti-angiogenic agents. These new trials broaden our existing efforts to characterize PTC299 activity and safety in multiple treatment settings.