On December 14, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported safety and promising activity of BPX-601 in Part 1 of a Phase 1/2 dose-escalation study in patients with advanced, metastatic pancreatic cancer expressing PSCA (prostate stem cell antigen) (Press release, Bellicum Pharmaceuticals, DEC 14, 2018, View Source [SID1234532061]). 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 (ESMO-IO) held in Geneva, Switzerland from December 13-16.

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"These initial results in advanced pancreatic cancer suggest that having greater control over the expansion and persistence of therapeutic cells may provide unique treatment benefits. We have observed promising initial clinical activity with BPX-601 in patients with one of the most challenging solid tumors, where there is a critical need for better treatments," commented Carlos R. Becerra, M.D., lead study investigator and oncologist and medical director of the Innovative Clinical Trials Center at Baylor University Medical Center at Dallas. "I am looking forward to the next phase of our study, which allows for a standard lymphodepletion regimen and administration of multiple doses of rimiducid to further increase the expansion and persistence of BPX-601 cells and the potential for greater impact against pancreatic and other tumor types."

Study Overview
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.

Interim Results:

The administration of BPX-601 without rimiducid resulted in limited expansion of cells, but the cells did not persist; A single dose of rimiducid 7 days following BPX-601 administration resulted in significant expansion of cells (three-fold to 20-fold) 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; 4 of 6 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 percent; 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.
"These results provide the first clinical evidence suggesting that our GoCAR-T technology drives expansion and persistence of therapeutic T cells in patients," said Rick Fair, President & CEO of Bellicum Pharmaceuticals. "We are excited about BPX-601 and the broader application of our technology intended to improve the safety and efficacy of cell therapies. With these promising results, we’re preparing to begin Part 2 of the BPX-601 study, adding patients with prostate and gastric cancers, allowing multiple doses of rimiducid and a more complete conditioning regimen. We’re looking forward to reporting updated results in 2019."

About BPX-601
BPX-601, the Company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence, and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, prostate and gastric cancers.