Bellicum Pharmaceuticals reported safety and activity of BPX-601 in part 1 of a Phase 1/2 dose-escalation study in patients with advanced, metastatic pancreatic cancer expressing prostate stem cell antigen, or PSCA. BPX-601 is a novel GoCAR-T cell candidate incorporating Bellicum’s co-activation domain, iMC, designed to boost T cell proliferation and persistence via administration of rimiducid. Data were reviewed during an oral presentation at the European Society for Medical Oncology Immuno-Oncology Congress, or ESMO-IO. A total of 12 patients with advanced metastatic pancreatic cancer expressing PSCA were treated with escalating doses of BPX-601 cells in a 3+3 design. Nine of 12 patients received a single dose of rimiducid following BPX-601 treatment to evaluate its effect on cell expansion and persistence. Patients in the study received a reduced conditioning regimen consisting of cyclophosphamide only. The administration of BPX-601 without rimiducid resulted in limited expansion of cells, but the cells did not persist. A single dose of rimiducid seven days following BPX-601 administration resulted in significant expansion of cells in four patients in spite of a reduced conditioning regimen. BPX-601 cells persisted longer than three weeks in three patients after a single dose of rimiducid. Increases in key cytokine levels were observed in patients receiving higher doses of BPX-601 cells and rimiducid. Four of six efficacy-evaluable patients treated with BPX-601 and a single dose of rimiducid had stable disease, with two patients demonstrating tumor shrinkage greater than 20%. No cytokine release syndrome or neurotoxicity of any grade was reported. The most frequently observed AEs were consistent with those experienced by advanced cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies. Patients continue to be evaluated in the study.
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