Bellicum Announces Initiation Of Patient Dosing With Controllable CAR T-Cell Product Candidate

- BPX-601 being studied in pancreatic cancers expressing PSCA

HOUSTON, Feb. 21, 2017 (GLOBE NEWSWIRE) -- Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, today announced it has dosed the first patient with BPX-601, the first CAR T-cell product candidate to enter clinical studies that is designed to enable control over the expansion and stimulation of the cells. BPX-601 targets solid tumors that express PSCA (prostate stem cell antigen), with an initial indication in non-resectable pancreatic cancer.

“We believe the initiation of the BPX-601 clinical study is an important milestone in the advancement of CAR T therapies,” said Rick Fair, Bellicum’s President and CEO. “The ability to control the intensity and duration of a cell-based treatment may help address the limitations that current CAR T therapies face, especially when targeting solid tumors. While our first study is in pancreatic cancer where there is great need for improved treatments, we intend to explore BPX-601 in other tumors known to express PSCA, including prostate, ovarian, bladder, esophageal and gastric cancers.”

BPX-601 incorporates GoCAR-TTM, a CAR T-cell modified to include Bellicum’s proprietary dual costimulatory domain MC (inducible MyD88/CD40) activation switch. With GoCAR-T, the level of stimulation and proliferation of BPX-601 cells can be refined by adjusting the administration schedule of rimiducid, a small molecule activator agent.

“This clinical trial is an important step in our ongoing research efforts to improve patient outcomes in one of the deadliest forms of cancer,” said Carlos Becerra, MD, Interim Chief of Oncology, Baylor University Medical Center at Dallas and principal investigator of the study. “The pancreatic cancer patients who will be eligible to participate in the study have progressed after standard treatments and have limited options. We hope that this trial will be an important step in developing safe and effective CAR T-cell therapy for solid tumors.”

About the BP-012 Study
The Phase 1 BP-012 study is an open-label, non-randomized, dose-finding trial designed to evaluate the safety and activity of BPX-601 and rimiducid in up to 30 patients with non-resectable pancreatic adenocarcinoma. Its purpose is to determine the safety of the administration of BPX-601, the safety of the rimiducid infusion and the persistence of the CAR T cells over time. The first of its kind trial for pancreatic cancer patients will take place at Baylor University Medical Center at Dallas, which is home to Baylor Scott & White Research Institute, one of the top translational research centers in the world. To learn more about the trial, visit

About GoCAR-T
Bellicum developed GoCAR-T to increase the efficacy and safety of CAR T-cell therapies in more challenging cancers, including solid tumors. Standard CAR T cells depend on the presence of cancer antigens for activation and proliferation. In GoCAR-T cells, a costimulatory signal is engineered into a rimiducid-controlled switch. This is designed to enable T-cell survival in the absence of antigen signaling, and full activation and proliferation in the presence of cancer antigens. In the event of side effects, the level of activation of GoCAR-T cells may be attenuated by reducing the rimiducid dosing schedule.

About Bellicum Pharmaceuticals
Bellicum is a leader in developing novel, controllable cellular immunotherapies for cancers and for orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), and CAR T and TCR cell therapies. More information can be found at

Forward-Looking Statement
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Bellicum may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," “designed,” "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research and development activities relating to rimiducid, inducible MyD88/CD40, MC, GoCAR-T, or its TCR programs; the effectiveness of rimiducid, inducible MyD88/CD40, MC, GoCAR-T, or its TCR programs, their possible range of application and potential curative effects and safety in the treatment of diseases; the timing and success of our clinical trials, including the rate and progress of enrollment in our BP-012 clinical trial or in any other clinical trials of BPX-601; the timing of regulatory filings for BPX-601 and for rimiducid; our research and development activities relating to BPX-601; and the potential applications and effectiveness of our product candidate BPX-601, including as compared to other treatment options and competitive therapies. Various factors may cause differences between Bellicum’s expectations and actual results as discussed in greater detail under the heading “Risk Factors” in Bellicum’s filings with the Securities and Exchange Commission, including without limitation our annual report on Form 10-K for the year ended December 31, 2015. Any forward-looking statements that Bellicum makes in this press release speak only as of the date of this press release. Bellicum assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Bellicum Pharmaceuticals
Alan Musso, CFO

BMC Communications
Brad Miles, 646-513-3125 

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