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Bellicum Pharmaceuticals Announces GoCAR-T and GoTCR Preclinical Presentations at the American Society of Hematology 2016 Annual Meeting

Results demonstrate unique control of cell proliferation and persistence may improve outcomes in solid tumors

SAN DIEGO--(BUSINESS WIRE)--Dec. 5, 2016-- Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, today announced the presentation of preclinical results on the Company’s GoCAR-T and GoTCR technologies at the 58th American Society of Hematology Annual Meeting in San Diego, California.

“We are pleased to report further supportive preclinical data on the utility of our proprietary iMC activation switch, which is incorporated into our BPX-601 GoCAR-T product candidate now entering a Phase 1 clinical study,” commented Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “We believe our novel technology provides a powerful and unique solution for overcoming the efficacy and safety challenges of T-cell therapies, especially when targeting solid tumors.”

The Company’s GoCAR-T platform incorporates an inducible MyD88/CD40 (iMC) costimulatory switch, which requires presence of both a target antigen and rimiducid to trigger the full effect of CAR T cell activity. The presence of rimiducid and antigen results in upregulation of cytokines such as IL2, leading to T-cell proliferation, persistence and improved anti-tumor efficacy. Unlike traditional CAR T constructs, GoCAR-T is designed to support persistence of CAR T cells in the body in the absence of cancer antigen to provide continued anti-tumor surveillance.

In a poster presentation titled, “Inducible MyD88/CD40 (iMC) Costimulation Provides Ligand-Dependent Tumor Eradication By CD123-Specific Chimeric Antigen Receptor T Cells,” Bellicum scientists targeted CD123, which is highly expressed in acute myeloid leukemia (AML) and leukemic stem cells. Results demonstrated that GoCAR-T removed CD123-positive leukemic cells in animal models through rimiducid-activated costimulation. Conversely, infrequent costimulation with rimiducid led to reduced activity of CAR T cells, supporting the technology’s potential to provide control over the activation, expansion and persistence of cells to achieve a desired level of safety and anti-tumor potency.

Additional data were presented on the Company’s GoTCR technology, which also uses an iMC costimulatory switch. The presence of GoTCR and rimiducid triggers the release of cytokines that upregulate MHC (major histocompatibility complex) on tumor cells, exposing them to potent immune response by both engineered and endogenous T cells. In the study outlined in a poster presentation titled, “Inducible MyD88/CD40 (iMC) Enhances Proliferation and Survival of Tumor-Specific TCR-Modified T Cells and Improves Anti-Tumor Efficacy in Myeloma,” T cells were engineered to express tumor antigen-specific T-cell receptors (TCRs) targeting preferentially-expressed antigen in melanoma (PRAME) or Bob1. PRAME is overexpressed in a wide variety of cancers including melanoma, sarcoma and several types of leukemias. Bob1 is also found to be highly expressed in certain leukemias, along with lymphomas and myelomas. Results demonstrated that the rimiducid-driven iMC costimulatory switch provided potent T-cell activation, proliferation and persistence, synergizing with signals from PRAME- or Bob1-targeted TCRs for improved anti-tumor efficacy in vitro and in vivo.

About Bellicum Pharmaceuticals

Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and 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 www.bellicum.com.

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, CAR-T, GoCAR-T, TCR and GoTCR programs; the effectiveness of BPX-601, its possible range of application and potential curative effects and safety in the treatment of diseases including as compared to other treatment options and competitive therapies; the timing and success of our clinical trials, including the rate and progress of enrollment in our clinical trials; and, the timing of regulatory filings for BPX-601 and for rimiducid. 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.

Source: Bellicum Pharmaceuticals, Inc.

Investors:
Bellicum Pharmaceuticals
Alan Musso, CFO
832-384-1116
amusso@bellicum.com
or
Media:
BMC Communications
Brad Miles, 917-570-7340
bmiles@bmccommunications.com
or
BMC Communications
Amy Bonanno, 914-450-0349
abonanno@bmccommunications.com