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Bellicum Pharmaceuticals Announces BPX-501 Clinical Data Demonstrating Disease-Free Outcomes in Patients with Genetic Diseases

ASH poster presentations report on pediatric patients with beta thalassemia, SCID, Fanconi anemia, Wiskott-Aldrich Syndrome and other genetic diseases undergoing haploidentical stem cell transplant and genetically modified BPX-501 T-cell add-back

No transplant-related mortality

Significant reductions in time to hospital discharge and re-hospitalizations

Webcast of ASH investor/analyst event scheduled for December 7, 2015 at 12:15 p.m. ET

HOUSTON--(BUSINESS WIRE)--Dec. 5, 2015-- 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 the ongoing BP-004 Phase 1/2 clinical trial during the 57th Annual Meeting of the American Society of Hematology (ASH) in Orlando, Florida. Pediatric patients in the study with a variety of genetic diseases achieved disease-free outcomes following a haploidentical, T cell-depleted hematopoietic stem cell transplant (HSCT) followed by an add-back of BPX-501 donor T cells. The study is designed to evaluate whether this regimen is safe and improves immune reconstitution, infection control and overall outcomes.

Initial outcomes were reported from the 39 pediatric patients who have received the BPX-501 product (of a total of 49 enrolled) at the European trial site as of November 30. Twenty of these children had non-malignant genetic diseases including Fanconi anemia (5), beta thalassemia (4), severe combined immunodeficiency (SCID or “bubble boy” disease) (5), Wiskott-Aldrich Syndrome (3) and others. Nineteen additional patients had blood cancers, with acute lymphoblastic leukemia being the most common. (The cohort with blood cancers requires longer-term endpoints and will be reported on in more detail at a later date.)

“These interim results present strong evidence that the addition of BPX-501 modified donor T cells provides important immune support and improves outcomes in patients undergoing a T-depleted haploidentical stem cell transplant,” said lead investigator Dr. Franco Locatelli, Director, Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù. “Historically, haplo-transplants had to be given without T cells to avoid Graft versus Host Disease, increasing the risk of deadly infections and delayed immune recovery, and relapse in patients with malignant disease. An approach that addresses these risks without elevating the GvHD risk could shift the standard of care, making haplo-sourced transplants—almost always available from a family member—an attractive option for patients. I am very happy for the patients and the families who have benefited from participation in this BPX-501 clinical trial.”

The presented data show that treatment with BPX-501 is safe and well tolerated for patients with non-malignant and malignant diseases, and provides several important immune benefits compared to the clinical site’s historical controls. Highlights include:

  • Safety: No adverse events associated with infusion of BPX-501 were reported. The occurrence and severity of GvHD in study subjects was generally consistent with the historical control group. There were seven instances of Grade 1 or 2 GvHD which all resolved without requiring activation of the CaspaCIDe® safety switch with rimiducid.
  • Survival: There was no transplant-related mortality (TRM) in the 37 study patients with a minimum of 30 days follow-up. In particular, for non-malignant patients, this lack of TRM (0/18) compares favorably with 9% TRM in the historical non-malignant control patients (3/33). TRM, when it occurs, typically happens early in the post-transplant period in non-malignant transplant patients, primarily due to infection.
  • Immune Reconstitution: Non-malignant patients in the trial achieved a mean improvement of approximately 40 fewer days to reach a T-cell count of 500 cells/ul, showing immune recovery was significantly faster than historical controls.
  • Time in Hospital: Non-malignant patients in the trial were discharged significantly faster from the hospital, 21 days sooner on average following HSCT, compared to historical controls. The number of patients re-hospitalized was also substantially reduced.

“It’s exciting to see the progress and outcomes from this BPX-501 study that we initiated just a year ago,” said Annemarie Moseley, Ph.D., M.D. Chief Operating Officer and Executive Vice President of Clinical Development at Bellicum. “We are making preparations for dialogue with the regulators in Europe and the U.S. in the first half of 2016, with the goal of defining the path to regulatory approval initially for non-malignant pediatric diseases. We look forward to the further evaluation of BPX-501 in different transplant settings, and in accumulating longer-term data to assess relevant clinical outcomes in the malignant setting.”

Investor/Analyst Luncheon

Bellicum will also host an investor and analyst luncheon on Monday, December 7, 2015 from 12:15 – 1:15 p.m. EST at the Hyatt Regency Orlando. Management and select key opinion leaders, including lead Principal Investigator Professor Franco Locatelli, M.D., will review the BPX-501 Phase 1/2 clinical study data from the above-mentioned poster and additional data from the BP-004 study. 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 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 will 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. The Company is using its proprietary Chemical Induction of Dimerization, or CID, technology platform to engineer and control components of the immune system in real time. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or 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 such trials; the timing of an IND filing for BPX-501; our research and development activities and expenses relating to BPX-501, CaspaCIDe, CAR T-cell and TCR programs; 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, 2014 and our Report on Form 10-Q for the quarter ended June 30, 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.

Bellicum Pharmaceuticals
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