MedKoo product information:

  PTC299

PTC299, a VEGF inhibitor,  is an orally bioavailable, small molecule inhibitor of vascular endothelial growth factor (VEGF) synthesis with potential antiangiogenesis and antineoplastic activities. VEGF inhibitor PTC299 targets post-transcriptionally by selectively binding the 5'- and 3'-untranslated regions (UTR) of VEGF messenger RNA (mRNA), thereby preventing translation of VEGF. This inhibits VEGF protein production and decreases its levels in the tumor and bloodstream. In turn, this may result in the inhibition of migration, proliferation and survival of endothelial cells, microvessel formation, the inhibition of tumor cell proliferation, and eventually the induction of tumor cell death. VEGFs are upregulated in a variety of tumor cell types and play key roles during angiogenesis. In addition, PTC299 may enhance the antitumor activity of other chemotherapeutic agents. PTC299, is currently in Phase II trials as a potential agent to treat vestibular schwannoma.  Check for active clinical trials or closed clinical trials using this agent. (NCI Thesaurus).

Current developer:    PTC Therapeutics

PTC299 is currently being developed for the treatment of cancer. It has completed Phase 1a trials in healthy volunteers, and is now being studied in a Phase 1-2 trials in patients with solid tumors, metastatic breast cancer and neurofibromatosis type 2. PTC299 was designed to inhibit VEGF production in tumors. Because PTC299 inhibits VEGF production, its action occurs at a different point in the VEGF pathway than other therapies. PTC299 may be active both as a single agent or when used in combination with other anti-angiogenic agents or with chemotherapy agents for the treatment of cancers. (source: http://www.ptcbio.com/ptc299).

D. Knight, A. Tiersten, H. Miao, M. Llorente, G. Elfring, Y. Novik, J. Speyer, M. Volm and L. Miller PTC299, a Novel Regulator of Tumor VEGF Expression, Is Well Tolerated and Achieves Target Plasma Concentrations: Dose-Ranging Results of a Phase 1b Study in Women with Metastatic Breast Cancer. Cancer Research: December 15, 2009; Volume 69, Issue 24, Supplement 3. doi: 10.1158/0008-5472.SABCS-09-6092