Bellicum Pharmaceuticals Announces Presentations of its Controllable Switch Technology Platform at the American Society of Hematology 2017 Annual Meeting
“These data continue to support our excitement over the technology’s potential to make cell therapies safer and more effective in more tumor types,” said Rick Fair, Bellicum’s President & Chief Executive Officer. “We are currently validating our platform in the clinic in three different product candidates, and look forward to reporting results on these programs in 2018. With the most advanced controllable cell technologies in our industry, we believe we are well positioned to move additional preclinical CAR-T projects into clinical trials that have the potential to be best-in-class.”
The Company’s novel technology platform is designed to enable full control over the activation, persistence, and elimination of cell therapies to safely elicit the full effect of CAR-T and TCR activity in the body. Unlike traditional approaches, Bellicum’s controllable CAR-T and TCR constructs are designed to provide anti-tumor surveillance, even in the absence of cancer antigen. The switch technologies covered in the posters include:
| Technology | Description |
| GoCAR-T | CAR-T cells incorporated with the inducible MyD88/CD40 (iMC) costimulatory switch to provide ligand-regulated control over the activation and persistence of cells |
| CIDeCAR | CAR-T construct that includes the MC costimulatory domain with the CaspaCIDe® safety switch |
| Dual-Switch CAR-T | CAR-T cells with both the iMC costimulatory switch and CaspaCIDe safety switch to provide greater control over the activation and persistence of therapeutic cells, as well as the ability to rapidly eliminate them by activating the safety switch |
Summary of Study Results
“Dual-Switch CAR-T cells: Orthogonal Molecular Switches to Control Activation and Elimination of CAR-T Cells to Target CD123+ Cancer” (Abstract 3184)
Researchers targeted CD123—which is highly expressed in acute myeloid leukemia (AML) and leukemic stem cells—with a novel construct consisting of a first-generation CAR combined with regulated activation and apoptotic signaling elements. T cell costimulation was controlled by rimiducid, and a rapamycin-controlled pro-apoptotic safety switch was designed to induce rapid dimerization of caspase-9 to mitigate possible CAR-T cell toxicity. Results demonstrate that when combined with a first-generation CD123-specific CAR, these molecular switches enable controlled, robust expansion of engineered T cells to control tumor growth in vitro and in vivo, and provide a rapid and efficient safety mechanism to block excessive cytokine release.
“Inducible MyD88/CD40 (iMC) Costimulation Enhances Polyclonal Epstein-Barr Virus-Specific Cytotoxic T Lymphocyte (EBV-CTL) Proliferation and Anti-Tumor Activity” (Abstract 3337)
Using peripheral blood mononuclear cells from healthy donors, researchers generated EBV-specific T cells, which were genetically modified with iMC. They concluded that modifying EBV-CTL with iMC resulted in increased T cell proliferation and persistence and improved anti-tumor efficacy, suggesting that iMC may have broad applications, such as modifying tumor-infiltrating lymphocytes, virus-specific T cells and other polyclonal T cell products to increase their potency.
“MyD88/CD40 enhanced CD19-specific CAR-T cells maintain therapeutic efficacy following resolution of cytokine-related toxicity using inducible caspase-9” (Abstract 4615)
Scientists demonstrated that CD19-specific CAR-T cells modified with a constitutively active form of the potent fusion protein MC were effective at eliminating aggressive tumors, with efficacy associated with cytotoxic cytokine release. However, this toxicity was effectively resolved with rimiducid-mediated activation of co-expressed iC9 or by selecting distinct T cell populations without affecting long-term efficacy of the CAR-T treatment.
The presentations can be found in the Investors & Media section of the Company’s website.
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, CaspaCIDe, the CID platform, iMC, dual switch, HSCT, CAR-T and TCR programs; the possible range of application of the CID platform and its 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; and, our research and development activities relating to our GoCAR-T and TCR technologies. 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
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Media:
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Source:
Source: Bellicum Pharmaceuticals, Inc.
