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The FDA has granted Toca 511 & Toca FC Breakthrough Therapy designation for the treatment of patients with recurrent high-grade glioma (HGG). The Toca 511 & Toca FC combination is designed to directly kill cancer cells and immune-suppressive myeloid cells resulting in activation of the immune system against cancer.

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Toca 511 & Toca FC is currently under evaluation in an international, randomized phase II/III clinical trial, which is designed to serve as a potential registrational study. The trial is in patients with first or second recurrence of glioblastoma or anaplastic astrocytoma who are undergoing resection. Enrollment in the phase II portion of the trial has been completed and top-line results are anticipated in the first half of 2018.


The Breakthrough Therapy designation application was based on data from three phase I, ascending-dose clinical trials involving 126 patients with recurrent brain cancer. The clinical data includes safety data; patient survival data; and durable, complete or partial tumor shrinkage determined by independent radiology review (Science Translational Medicine 2016; doi: 10.1126/scitranslmed.aad9784). In addition, preclinical information was provided supporting a novel immunological mechanism of action involving the depletion of immune-suppressive myeloid cells in the tumor microenvironment. The FDA previously granted Fast Track designation to Toca 511 & Toca FC for the treatment of recurrent HGG, and Orphan Drug designation for the treatment of glioblastoma multiforme.


Breakthrough Therapy designation from the FDA indicates preliminary clinical evidence demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy designation intensifies FDA involvement to ensure an efficient drug development program.