"We are very pleased with the
PTC596 is an orally bioavailable small molecule tubulin binding agent that arrests tumor cells in G2/M phase, including cancer stem cells, through the action of inhibiting tubulin polymerization. It is currently in a Phase 1b study for LMS, which accounts for approximately 10 to 28% of all soft tissue sarcomas.1 Approximately 4000 patients are diagnosed with LMS annually in the US, the risk of developing metastases is approximately 40%, and the 5-year survival rate is estimated to be 13.6% for metastatic LMS.2 PTC596 is also currently in a clinical study for DIPG, an ultra-rare glioma arising in the brainstem that makes up 10 to 15% of all brain tumors in children.3 Approximately 300 patients are diagnosed with DIPG annually in the US, and the median overall survival with the current standard of care of radiation therapy, is approximately 9 months with a two-year overall survival rate of less than 10%.4
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
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As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory or prove to be commercially successful.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
1 Bessen, T., Caughey, G. E., Shakib, S., Potter, J. A., Reid, J., Farshid, G., Roder, D., & Neuhaus, S. J. (2019). A population-based study of soft tissue sarcoma incidence and survival in
2 Ries LAG, Young JL, Keel GE, Eisner MP, Lin YD, Horner M-J (editors). SEER Survival Monograph: Cancer Survival Among Adults:
3 Raabe, E. H., & Cohen, K. (n.d.). What is DIPG? Retrieved
4 Jansen, M. H., Veldhuijzen van Zanten, S. E.,