Bellicum Announces First Reported Use of CaspaCIDe® Safety Switch to Mitigate CAR-T Cell Toxicity

HOUSTON, March 04, 2021 (GLOBE NEWSWIRE) -- Bellicum Pharmaceuticals, Inc. BLCM, a leader in developing novel, controllable cellular immunotherapies for cancers, today announced the publication of a case report using the company's CaspaCIDe (inducible caspase-9, or iC9) safety switch. The letter to the editor titled "Utility of Safety Switch to Abrogate CD 19 CAR T Cell-Associated Neurotoxicity" appeared as an ahead-of-print publication in the digital edition of Blood, a journal published by The American Society of Hematology. Link to article.

The publication detailed a case report from an investigator-sponsored trial (IST) at the University of North Carolina Lineberger Comprehensive Cancer Center, where a Phase 1/2 trial is ongoing to test the safety and efficacy of autologous T lymphocytes genetically modified to express a CD19 chimeric antigen receptor (CAR) and iC9. A patient in the dose expansion cohort of this trial experienced a high grade neurologic adverse event with persistence of grade 3-4 immune effector cell-associated neurotoxicity syndrome (ICANS) for 72 hours despite standard care, and rimiducid was subsequently given per protocol to activate iC9.

Within twelve hours of rimiducid administration, ICANS grade improved from 3 to 1 and was fully resolved after four days. This publication marked the first reported clinical use of the iC9 safety switch to mitigate a severe CAR-T-mediated adverse event refractory to standard of care treatment.

"Given the risks of ICANS and other CAR-T cell-mediated adverse events, the inclusion of the iC9 switch has the potential to dramatically improve the safety of cellular immunotherapies," said Matthew Foster, M.D., University of North Carolina Lineberger Comprehensive Cancer Center. "We are also encouraged by the anti-leukemic response we observed in this patient even with the use of the iC9 switch."

"We remain enthusiastic about our switch technology, which as shown in this case report and in the extensive CaspaCIDe experience from the rivo-cel program, can improve the overall benefit/risk profile of cell therapy," said Rick Fair, President and CEO of Bellicum Pharmaceuticals. "We believe this clinical experience validates our continued efforts to incorporate this technology into a growing number of internal and collaborator programs."

About CaspaCIDe^®

CaspaCIDe (inducible caspase-9, or iC9) is Bellicum's chemical induction of dimerization (CID) safety switch technology designated to eliminate cells in the event of toxicity. The CaspaCIDe switch consists of the CID-binding domain coupled to the signaling domain of caspase-9, an enzyme that is part of the apoptotic pathway. Infusion of rimiducid is designed to trigger activation of this domain of caspase-9, which in turn leads to selective apoptosis of the CaspaCIDe-containing cells. Because CaspaCIDe is designed to be permanently incorporated into Bellicum's cellular therapies, the safety switch has the potential to be available for use long after the initial therapy is delivered.

About Bellicum Pharmaceuticals

Bellicum is a clinical stage biopharmaceutical company striving to deliver cures through controllable cell therapies. The company's next-generation product candidates are differentiated by powerful cell signaling technologies designed to produce more effective CAR-T cell therapies. Bellicum's GoCAR-T^® product candidates, BPX-601 and BPX-603, are designed to be more efficacious CAR-T cell products capable of overriding key immune inhibitory mechanisms. More information about Bellicum can be found at www.bellicum.com.

Forward-Looking Statements

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: the potential for the CaspaCIDe safety switch and rimiducid to improve safety of cell therapies; and the potential useful life of the CaspaCIDe safety switch. 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 Bellicum's quarterly report on Form 10-Q for the three months ended September 30, 2020 and Bellicum's annual report on Form 10-K the year ended December 31, 2019. 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

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