Press Release Details

FDA Grants Orphan Drug Designation to Bellicum’s BPX-501 for the Treatment of Immunodeficiency and GvHD Following Stem Cell Transplant

02/22/16 at 7:00 AM EST

HOUSTON--(BUSINESS WIRE)--Feb. 22, 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 that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BPX-501, the Company’s lead product candidate.

The FDA designation is for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as “replacement T-cell therapy for the treatment of immunodeficiency and graft versus host disease (GvHD) after allogeneic hematopoietic stem cell transplant.” BPX-501 is an adjunct T-cell therapy incorporating the Company’s proprietary CaspaCIDe® safety switch.

Following an allogeneic transplant, a lack of sufficient mature T cells constitutes immune deficiency that can contribute to infections, viral reactivation, and relapse. The ability to correct this immune deficiency through the add-back of mature donor T cells, without raising the risk of uncontrollable GvHD, has the potential to fundamentally change the risk profile of allogeneic transplantation.

“Expanding the availability of hematopoietic stem cell transplants to more patients with rare genetic diseases and blood cancers is a primary goal of our BPX-501 program,” commented Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “The FDA’s approval of our request for orphan drug designation is an important milestone that supports our efforts to address key risks and improve outcomes in allogeneic transplant which could make the procedure more viable for patients who lack a perfect match donor.”

BPX-501 is currently being evaluated in multiple Phase 1/2 clinical trials in adults and pediatric patients with leukemias, lymphomas, and genetic blood diseases in the U.S. and Europe. Interim results presented at the 2015 ASH Annual Meeting in pediatric patients with a variety of genetic diseases, including beta thalassemia, Wiskott-Aldrich and SCID, demonstrated disease-free outcomes following a haploidentical, T cell-depleted hematopoietic stem cell transplant, followed by an add-back of BPX-501 donor T cells. Results demonstrated reduced infection rates, faster immune reconstitution, and reductions in time to hospital discharge and in re-hospitalizations, compared to historical controls.

About Orphan Drug Designation

The FDA’s Office of Orphan Products Development (OOPD) provides orphan designation to drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S. Orphan designation also qualifies a company for various development incentives, including tax credits for qualified clinical testing and marketing exclusivity for a period of seven years.

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.

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

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. We may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "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 of our clinical trials and of our research and development activities relating to BPX-501; the effectiveness and success of our research and development activities relating to our CaspaCIDe platform; and the effectiveness of BPX-501 and its possible range of applications and potential curative effects and safety profile. Various factors may cause differences between Bellicum’s expectations and actual results as discussed in greater detail in Bellicum’s filings with the Securities and Exchange Commission, including without limitation, under the heading “Risk Factors” in our annual report on Form 10-K for the year ended December 31, 2014 and our Report on Form 10-Q for the quarter ended June 30, 2015. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume 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.

Bellicum Pharmaceutical
Alan Musso, CFO, 832-384-1116
BMC Communications
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