35 children with genetic immune disorders and hemoglobinopathies are disease- and GvHD-free following haploidentical stem cell transplant with BPX-501

Webcast of ASH investor/analyst event scheduled for Monday, December 5 at 12:15 p.m. PST

SAN DIEGO--(BUSINESS WIRE)--Dec. 3, 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 results from the BP-004 multicenter clinical trial which includes children with Primary Immune Deficiencies (PIDs) and hemoglobinopathies who received an add-back of BPX-501 modified T cells following a haploidentical, T cell-depleted hematopoietic stem cell transplant (haplo-HSCT). The data were presented in oral and poster sessions today during the 58^th Annual Meeting of the American Society of Hematology (ASH). Results show that all 35 children treated are alive, free from GvHD and cured of their underlying disease.

According to Neena Kapoor, MD, Director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and who reviewed study results of patients with PIDs in an oral presentation, “These results show that the inclusion of BPX-501 T cells may make haplo-HSCT a first-line option for children with PIDs who lack a suitable, HLA-matched donor.” Dr. Kapoor added, “Delayed immune reconstitution leading to severe infectious complications is the primary cause of morbidity and mortality in PID patients who undergo a T-depleted haplo-transplant. The data reflect that the add-back of BPX-501 donor T cells provides faster immune recovery and lower rates of re-hospitalizations due to infection. The CaspaCIDe safety switch, engineered into BPX-501 T cells, provides a critical safety net to address the risk of uncontrolled acute GvHD.”

PID Highlights (Abstract #72)

“Outcome of Children with Primary Immune-Deficiencies (PIDs) Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501 Donor T Cells Genetically Modified with a Novel Suicide Gene (Inducible Caspase 9, iC9) After T-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (Haplo-HSCT)”

PIDs in the study include Severe Combined Immune Deficiency (“Bubble boy” disease) (n=11), Wiskott-Aldrich syndrome (n=6), Chronic Granulomatous Disease (n=2) and other rare Immune Deficiencies (n=4). Study outcomes for these patients included:

• All 23 PID patients engrafted with no secondary graft failure.
• All patients are alive and free of disease with a median follow-up of 404 days (range: 118-728 days).
• Median time to neutrophil and platelet recovery was 16 days and 10 days, respectively.
• Five children experienced Grade I or Grade II acute GvHD. Three of the cases resolved with either topical or systemic steroids. The other two GvHD cases resolved following infusion of rimiducid and activation of the CaspaCIDe^® safety switch. There was one mild case of chronic GvHD, which also resolved. No one experienced severe Grade 3 or Grade 4 GvHD.
• There was a low rate of hospital re-admission to treat infections. No reported adverse events were associated with administration of BPX-501.

Hemoglobinopathy Highlights (Abstract #2286)

“Clinical Outcome and Immune Recovery after Adoptive Infusion of BPX-501 Cells (Donor T Cells Transduced with iC9 Suicide Gene) in Children with Hemoglobinopathies and Diamond-Blackfan Anemia Given a/b T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation (HSCT)”

Updated BP-004 study results were also reviewed in a poster session of 12 pediatric patients with Thalassemia Major β0/β0 (n=9), Sickle Cell Disease (n=1) and Diamond-Blackfan Anemia (n=2). HSCT is a potentially curative treatment for these patients, who otherwise typically require a lifetime of blood transfusions. Study results demonstrated that the BPX-501 cells expanded and persisted in the patients over time, contributing to overall immune reconstitution and successful transplant outcomes. Results reported in the poster included:

• All 12 patients attained full donor chimerism. One patient with secondary graft failure was re-transplanted from the same donor and then attained full donor chimerism.
• All patients are alive, GvHD-free and free of disease.
• The median initial hospital discharge occurred at 21 days (range: 14-55) and there were no re-hospitalizations.
• Grade I/II skin acute GvHD occurred in 2 patients, and was successfully treated with steroids. No chronic GvHD occurred.
• Median time to last red blood cell transfusion was 6.5 days (range: 4-33 days) post-transplant.

“Our BPX-501 program continues to gain momentum as additional centers recognize its potential to improve haplo-HSCTs in both nonmalignant and malignant diseases,” commented Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “The reliable and expedient performance of our molecular switch technology, combined with an advanced method of T-depleted stem cell transplantation, have the potential to make curative haplo-transplants available to many more patients suffering from genetic diseases and blood cancers.”

Investor/Analyst Luncheon

Bellicum will host an investor and analyst luncheon on Monday, December 5, 2016 from 12:15 – 1:15 p.m. PST at the San Diego Marriott Gaslamp Quarter Hotel. Management and investigators Dr. Franco Locatelli (Ospedale Pediatrico Bambino Gesù), Dr. Neena Kapoor (Children’s Hospital of Los Angeles), and Dr. Kris Mahadeo (Montefiore Medical Center) will discuss BPX-501 Phase 2 clinical data in the nonmalignant and malignant settings. The luncheon will be webcast live and may be accessed from the News & Events section of the Bellicum website. An archived version of the webcast will be available for replay for at least two weeks following the event.

About PIDs

Primary Immune Deficiencies (PIDs) are caused by genetic abnormalities that prevent the development of normal immune responses, which lead to an increased susceptibility to infections. A hematopoietic stem cell transplant (HSCT) is the current mainstay of treatment for severe forms of PIDs, including Severe Combined Immune Deficiency (SCID), Wiskott-Aldrich syndrome, Chronic Granulomatous Disease (CGD) and several others.

About Hemoglobinopathies

Hemoglobinopathies are a diverse group of genetic disorders caused by the abnormal structure or production of hemoglobin, a protein molecule in red blood cells that carries oxygen throughout the body. Depending on disease severity, standard treatments include blood transfusions and drugs to control symptoms, however a stem cell transplant is often preferred for severe cases.

About BPX-501

BPX-501 is an adjunct T-cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe^® safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. This enables physicians to more safely perform stem cell transplants by adding back BPX-501 engineered T cells to speed immune reconstitution and provide control over viral infections, without unacceptable risk of uncontrollable GvHD. The ongoing BP-004 Phase 1/2 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

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 BPX-501, rimiducid and CaspaCIDe; the effectiveness of CaspaCIDe; the effectiveness of BPX-501, 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-501 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.

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Source: Bellicum Pharmaceuticals, Inc.

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