SAN DIEGO – Feb. 23, 2017 – Tocagen Inc., a clinical-stage, cancer-selective gene therapy company, today announced the U.S. Food and Drug Administration (FDA) has granted Toca 511 & Toca FC Breakthrough Therapy Designation for the treatment of patients with recurrent high grade glioma (HGG). Toca 511 & Toca FC is currently under evaluation in an international, randomized Phase 2/3 clinical trial, which is designed to serve as a potential registrational study. The trial involves patients with first or second recurrence of glioblastoma or anaplastic astrocytoma who are undergoing resection. Enrollment in the Phase 2 portion of the trial has 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 1, ascending-dose clinical trials involving 126 patients with recurrent brain cancer. The clinical data includes results published in Science Translational Medicine, including safety data, patient survival data and durable, complete or partial tumor shrinkage determined by independent radiology review. 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.
“Receiving Breakthrough Therapy Designation from the FDA for recurrent HGG, the first time it has been granted for this type of brain cancer, highlights the urgent need for new treatment options for this deadly disease,” said Marty Duvall, chief executive officer of Tocagen. “Based on the encouraging safety and efficacy data we have seen to date, advancing the development of Toca 511 & Toca FC continues to be our team’s top priority. We look forward to working closely with the FDA to expedite development and review of the product candidate and related activities.”
Breakthrough Therapy Designation from the FDA indicates preliminary clinical evidence demonstrates the drug may have substantial improvement on one or more clinically significant endpoints over available therapy. Breakthrough Therapy Designation intensifies FDA involvement to ensure an efficient drug development program and is an organizational commitment from the FDA to involve their senior managers.
HGGs are among the most common and aggressive primary brain cancers. The total number of patients with HGG expected to be diagnosed in 2017 is about 160,000 worldwide and about 14,000 in the United States. The two most common forms of HGG are glioblastoma and anaplastic astrocytoma. Standard treatment for newly diagnosed HGG includes safe surgical removal of as much of the tumor as possible followed by radiation therapy and chemotherapy. However, HGG recurs in most patients even after maximal treatment. After recurrence, median survival is typically seven to nine months.
About Toca 511 & Toca FC
Tocagen’s lead product candidate is a combination of an investigational biologic, Toca 511, and an investigational small molecule, Toca FC, designed to be used together. 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. Toca 511 & Toca FC is currently being evaluated in multiple clinical trials. The ongoing Toca 5 trial is an international, randomized, Phase 2/3 clinical trial for the treatment of patients with recurrent HGG. The ongoing Toca 6 study is a Phase 1 trial for the treatment of patients with metastatic cancer. Tocagen plans to expand the Toca 6 trial to include patients with recurrent HGG and to initiate Toca 7, a Phase 1 trial in patients with newly diagnosed HGG. The FDA has granted Toca 511 & Toca FC Breakthrough Therapy Designation and Fast Track designation for the treatment of patients with recurrent HGG, and Orphan-Drug designation for the treatment of glioblastoma multiforme (GBM).
Tocagen is a clinical-stage, cancer-selective gene therapy company developing first-in-class, broadly applicable product candidates designed to activate a patient’s immune system against their own cancer. The company is developing its lead investigational product candidate, Toca 511 & Toca FC, initially for the treatment of recurrent HGG, a disease with significant unmet medical need. Tocagen has received grant support from leading brain cancer foundations, including Accelerate Brain Cancer Cure (ABC2), National Brain Tumor Society (NBTS), American Brain Tumor Association (ABTA), Musella Foundation and Voices Against Brain Cancer (VABC). For more information, visit www.tocagen.com or follow @Tocagen.