Wish I understood more about the science behind it all. What I do understand it will likely get to market as the results will be good.
Analyst Wangzhi highlights that TOCA is a Phase 3 clinical-stage biotech company developing novel viral gene therapy for cancer. It has developed a proprietary Retroviral Replicating Vector (RRV) platform to selectively deliver therapeutic genes in tumor cells. Lead program Toca 511 & Toca FC delivers cytosine deaminase to convert pro-drug 5-FC (5-fluorocytosine) to active 5-FU (5-Fluorouracil) selectively in tumor cells, which kills the tumor cells and also induces immune response.
Li views TOCA as an attractive investment opportunity with multiple upcoming catalysts and significant upside potential:
I. We see a good probability of success (POS) for Toca 511 & Toca FC pivotal trial with potential market launch in 2020 to address the high unmet need of rHGG ($1.2B+ market), given:
1. Toca 511 & Toca FC Phase 1 trials showed good safety and strong efficacy signal indicated by: 1) 5/23 (21.7%) pivotal trial eligible rHGG pts achieved rare durable CR (38.0+ to 57.5+ m) in rHGG with impressive mOS of 14.4m vs 6-10m of current TRx; 2) Late response onset and gradual improvement from PR to CR in 2 pts is consistent with the immune mechanism of action of Toca 511 & Toca FC; 3) Trend of dose response in OS; 4) Both FDA and EMA have granted Toca 511 & Toca FC BTD and PRIME designations after their diligent review of the Phase 1 results.
2. We see a good POS for the Toca5 pivotal trial to recapitulate the promising Phase 1 results in rHGG, based on: 1) Design of Toca5 pivotal trial recapitulates key Phase 1 signal with reasonable assumptions to provide decent buffer room for success; 2) Potential variables (injection procedures, resection extent and distribution of rGBM vs rAA pts, etc.) for Toca5 outcome appear to be in good control; 3) Strong regulatory support from FDA and EMA with BTD and PRIME designations; 4) Enrollment on track to complete by YE2018 with two interim analysis to provide earlier readouts in 2H18 and 1H19.
3. Toca 511 & Toca FC results appear highly competitive among products currently in active clinical development for rGBM/rHGG (page 15).
II. We see significant upside potential of expanding Toca 511 & Toca FC into other solid tumors.
III. We also see broad upside potential of expanding RRV platform to deliver a variety of anti-cancer viral gene therapy (e.g., local aPD-L1 by Toca 521).
