As of May 31, 2016, Tocagen has treated 126 recurrent HGG patients with Toca 511 & Toca FC in three ongoing ascending dose Phase 1 clinical trials with three modes of delivery. In these trials, potential benefits were observed, including durable objective responses, extended overall survival and a favorable safety profile. To date, a dose-limiting toxicity has not been reached. Based on these Phase 1 clinical trial results, in November 2015 Tocagen initiated the Phase 2 portion of a Phase 2/3 clinical trial, which is designed to serve as a potential registrational trial in patients with first or second recurrent HGG undergoing resection.
The median overall survival of patients in the Phase 1 resection injection study, where Toca 511 was injected into the wall of the resection cavity after removal of the tumor, exceeds historical controls across a variety of previously reported clinical trials. As of the data cut-off of May 31, 2016, 43 patients in the resection trial had a median overall survival of 12.4 months that was approximately four months longer relative to results from clinical trials of drugs used as standard of care for recurrent HGG. Furthermore, as of the data cut-off of May 31, 2016, the subset of 24 patients in the Phase 1 resection injection trial that mirrors the entry criteria, clinical setting and dosing for patients in Toca 5, Tocagen’s Phase 2/3 clinical trial, had a median survival of 14.3 months, with an approximate six-month improvement in median overall survival relative to results from clinical trials of drugs used as standard of care for recurrent HGG.
The U.S. Food and Drug Administration (FDA) granted Toca 511 & Toca FC Breakthrough Therapy Designation for the treatment of recurrent HGG and the European Medicines Agency (EMA) has granted Toca 511 PRIME (PRIority MEdicines) designation for the treatment of HGG. These designations are granted to new products that treat serious diseases or conditions and demonstrate the potential to address an unmet medical need.
Tocagen has also obtained Orphan-Drug Designation for the treatment of malignant glioma. Orphan-Drug Designation is a designation for a product that treats a rare disease or condition and which, if the product receives the first FDA approval for that disease or condition, may result in a period of regulatory exclusivity, subject to some exceptions.