BPX-601: CAR T Phase 1 Trial Enrolling Patients with Pancreatic Cancer

BPX-601 is a GoCAR-Tproduct candidate containing Bellicum’s proprietary iMC, (inducible MyD88/CD40) activation switch. It is designed to treat solid tumors expressing prostate stem cell antigen, or PSCA—a cancer antigen expressed in many malignancies, including prostate, pancreatic, bladder, esophagus, and gastric cancers—for which there is a significant unmet need. The Company has launched BP-012, a Phase 1 BPX-601 GoCAR-T trial in the initial indication of non-resectable pancreatic cancer.

Our GoCAR-T technology consists of CAR T cells that are modified to include the proprietary dual co-stimulatory domain, MC. Unlike standard CARs, which depend on antigen for activation and proliferation, GoCAR-T places the costimulatory signal on a rimiducid-controlled switch, designed to allow control of T-cell survival in the absence of antigen signaling and full activation and proliferation in the presence of an antigen. In the event of side effects, the level of activation of the GoCAR-T cells can be refined by reducing the rimiducid administration schedule.

Preclinical data reported at ASH 2016 showed robust anti-tumor activity, and enhanced T-cell proliferation and persistence compared to traditional CAR T constructs. We believe this novel technology provides a powerful and unique solution for overcoming the efficacy and safety challenges of T-cell therapies, especially when targeting solid tumors.