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National Brain Tumor Society Statement on Tocagen Designation

Published on February 23, 2017 in Press Release

Today, Tocagen, a company developing a gene therapy to treat cancer, announced that it has received “Breakthrough Therapy Designation*” for its lead product candidate Toca 511 & Toca FC** from the U.S. Food and Drug Administration (FDA) for the treatment of recurrent high-grade glioma (glioblastomas and anaplastic astrocytomas).

Tocagen was granted Breakthrough Therapy Designation based on data from three Phase 1 clinical trials reported in 2016, which included favorable, early patient safety and survival data. Tocagen had previously been granted “Fast Track” designation for Toca 511 & Toca FC. NBTS is an early funder of Tocagen’s development of Toca 511 & Toca FC.

National Brain Tumor Society (NBTS) Chief Executive Officer David Arons released the following statement:

“National Brain Tumor Society is encouraged by the news that the FDA has granted Breakthrough Therapy status for Toca 511 & Toca FC, as it positions this potential treatment option for the most intensive level of regulatory review. NBTS supported, and advocated for, the establishment of the Breakthrough Therapy Designation because of its intended purpose – to provide an ‘all-hand-on-deck’ review by the FDA for potential new treatments such as those for aggressive brain tumors. We offer our congratulations to Tocagen on this important step forward.”

*The Breakthrough Therapy Designation was created through legislation in 2012 that NBTS advocated for along with a coalition of other disease groups led by Friends of Cancer Research, to help promising treatments get through the FDA evaluation and approval process faster. Specifically, the designation, “is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.”

A breakthrough therapy designation allows a drug’s sponsor to have access to all of the FDA’s “fast track program” benefits, including, “more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review.”

This does not guarantee that Toca 511 & Toca FC will approved for all recurrent GBM patients, that still has to be determined through further clinical trial evaluation and final review and approval by the FDA.

**Toca 511 & Toca FC are investigational agents designed to be used together to selectively kill cancer cells and activate the immune system against tumors. Toca 511 & Toca FC are currently being evaluated in multiple clinical trials. The trial his designation relates to is the Toca 5 trial, an international, randomized Phase 2/3 clinical trial involving high-grade glioma patients with first or second recurrence. Enrollment in the Phase 2 aspect of the trial has completed and initial results are expected to be announced in the first half of 2018. 


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