Tocagen's Promising Glioblastoma Therapy

John McCain died of glioblastoma last month. My father died of glioblastoma back in the 1970s when he was around 40 years old. Currently, for recurrent glioblastoma the standard of care (SOC) chemotherapies have a median survival of 8.4 months. There is a huge need for better treatment options.

Toca 511/Toca FC gene/immuno therapy is an extremely promising new option for treating glioblastoma and possibly more cancer types. Every time I look at the survival data above from Tocagen on its Toca 511/Toca FC's Phase 1 trial in glioblastoma, I am amazed. Six of 23 recurrent glioblastoma patients are alive for 3 years or more! And the side effects are much better than traditional chemotherapy because in the Toca therapy, an inactive precursor chemotherapy (Toca FC) is delivered systemically, that is enzymatically converted to an active form locally at the tumor site by cancer cells that have been genetically modified by Toca's special virus (present in Toca 511) to express a special enzyme. This Phase 1 trial was more robust than most, being conducted in many centers internationally with responses observed across 6 different clinical sites.

Toca 511/Toca FC's median survival of 14.4 months in the Phase 1 trial was a nice improvement on current SOC. However, as mentioned above and shown graphically above, what is most impressive are the long-term durable responders along with a really good safety profile. Six of 23 patients in the high dose cohort are alive for more than 3 yrs, one responder has remained cancer free for almost 5 years. This has earned Toca 511/Toca FC the coveted breakthrough therapy designation by the FDA and Prime designation by the European regulators, which should help this regimen get approved more quickly if the data continues to look significantly better than SOC.

As an investor in biopharma, it's good to see Tocagen's stock price start to run up as more analysts discover Tocagen and as they recently announced that their Phase 3 glioblastoma trial has reached full recruitment, over 3 months ahead of schedule. More importantly, this technology looks like a potential huge breakthrough for patients afflicted with glioblastoma. Plus, this technology may be more broadly applicable to other cancers. There is an exciting Phase 1 trial ongoing where Toca is attempting to deliver the Toca 511 virus intravenously in patients with a variety of cancers, especially colorectal cancer. We are excited to see this data continue to unfold through the end of 2019 and beyond.

#TOCA #AmpFree