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Bellicum Pharmaceuticals Announces BPX-501 Clinical Data Updates

EBMT oral presentations highlight encouraging preliminary outcomes from ongoing BP-004 clinical study in children with genetic diseases and cancers

HOUSTON--(BUSINESS WIRE)--Apr. 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 interim data from the lead site in Bellicum’s ongoing BP-004 Phase 1/2 clinical trial during the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in Valencia, Spain. Studies with BPX-501 were selected for two oral presentations, including one during the Presidential Symposium session where significant findings from the meeting are featured.

In the ongoing BP-004 clinical trial, malignant and nonmalignant pediatric patients receive a haploidentical, T cell-depleted hematopoietic stem cell transplant (HSCT) followed by an add-back of BPX-501 donor T cells. The trial is designed to evaluate whether this regimen is safe and improves immune reconstitution, infection control and overall outcomes.

In an oral presentation reviewing the preliminary outcomes from the BP-004 clinical trial in 17 high-risk pediatric patients with blood cancers, the data showed that BPX-501 cells expand in vivo and persist over time, contributing to adaptive immunity. There was no transplant-related mortality due to infections, GvHD or other transplant-related complications. Additionally, the relapse rate to date compares favorably with that of historical controls, with 16 of 17 patients in the trial with acute leukemias showing disease-free outcomes. The median follow-up period to date for these patients was approximately seven months.

In a feature presentation during the Presidential Symposium session, initial outcomes for nonmalignant patients were reviewed. The data included children with genetic diseases including Fanconi anemia (5), beta thalassemia major (5), severe combined immunodeficiency (SCID or “bubble boy” disease) (5), Wiskott-Aldrich Syndrome (4) and other orphan diseases (5). All 24 children treated remain disease-free with no treatment-related mortality (median follow-up period of approximately seven months), consistent with earlier results presented at ASH 2015.

“These interim results continue to be very encouraging and indicate that a haploidentical transplant, with the addition of BPX-501-modified donor T cells, can be an attractive option for children in need of a transplant,” said lead investigator Dr. Franco Locatelli, Director, Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome. “Future studies will address the role of repeated infusions or higher numbers of BPX-501 cells in malignant patients with resistant disease.”

“In both malignant and nonmalignant patients, the results show that treatment with BPX-501 appears safe, well-tolerated, and provides important immune benefits,” commented Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “These data also demonstrate high BPX-501 cell viability, expansion and persistence, and that the improvement of immune reconstitution is sustained. We look forward to sharing more results as these data mature, and providing updates following our plan to meet with the U.S. FDA and EMA during the second quarter of this year.”

A copy of the slides presented at the meeting is available in the Events & Presentations section of bellicum.com.

About BPX-501

BPX-501 is an adjunct T cell therapy of genetically modified donor T cells incorporating Bellicum’s proprietary CaspaCIDe® safety switch. The product candidate is designed to provide a safety net to eliminate the BPX-501 alloreactive T cells should severe GvHD occur, enabling physicians to more safely perform haploidentical stem cell transplants by adding back the BPX-501 genetically engineered T cells to speed immune reconstitution and provide control over viral infections.

BP-004 Clinical Trial Design

In December 2014, Bellicum initiated BP-004, a Phase 1/2 clinical trial in children with leukemias, lymphomas, or orphan inherited blood disorders, such as severe combined immunodeficiency (SCID), Wiskott-Aldrich Syndrome, beta thalassemia and sickle cell disease, all chronic life-long disorders for which HSCT is curative. The trial is being conducted in both European and U.S. pediatric transplant centers and plans to enroll up to 90 patients. The open label dose escalation trial is evaluating whether BPX-501 T cells from a haploidentical donor, typically the child’s mother or father, administered following a T-depleted HSCT, are safe and can enhance immune reconstitution.

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.

*CaspaCIDe® is a trademark registered with the U.S. Patent and Trademark Office.

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: the timing and success of our clinical trials, including the rate and progress of enrollment in our BP-004 clinical trial; the timing of regulatory filings for BPX-501; our research and development activities relating to BPX-501; the effectiveness of BPX-501, its possible range of application and its potential curative effects and safety in the treatment of blood cancers and inherited blood diseases; and the potential applications and effectiveness of our product candidates, 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.

Source: Bellicum Pharmaceuticals, Inc.

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