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Bellicum Pharmaceuticals Announces Successful Dosing of First Patient Cohort With BPX-501 T Cells Following Haplo-Identical Hematopoietic Stem Cell Transplant

BPX-501 is being evaluated as adjunct T cell therapy in patients with hematologic cancers and genetic blood diseases who lack a matched donor

HOUSTON--(BUSINESS WIRE)--Jan. 13, 2015-- Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies, today announced that the first cohort of patients in the BP-004 trial has completed dosing with the Company’s genetically engineered donor T cells (BPX-501), after receiving a partial T depleted haplo-identical allogeneic hematopoietic stem cell transplant (haplo-HSCT).

The trial in pediatric patients is evaluating whether BPX-501 T cells from partially mis-matched donors administered following HSCT are safe and can help speed immune reconstitution. BPX-501 contains the CaspaCIDe® safety switch, giving doctors the ability to eliminate the T cells should they cause Graft-versus-host disease, a more common occurrence with a haplo-HSCT than with a matched procedure. BPX-501 is designed to allow study doctors to restore patient immunity earlier with increasingly higher doses of haplo-identical T cells, in an effort to improve infection control and overall transplant outcomes.

“For hematological cancers and many orphan blood disorders such as severe primary immune deficiencies, inherited bone marrow failure syndromes, thalassemia and sickle cell disease, a hematopoietic stem cell transplant from a matched donor has resulted in a lifetime cure. But many patients lack a suitable, matched donor, eliminating this option,” said Prof. Francesco Locatelli, MD, lead study investigator and Full Professor of Pediatrics, University of Pavia, and Director, Department of Pediatric Hematology and Oncology, IRCCS Pediatrico Bambino Gesù Piazza, Rome, Italy. “Making haplo-identical transplants safer and more effective could make this curative therapy available to many times the number of patients eligible today.”

Commented Bellicum CEO Tom Farrell, “We’re pleased to have successfully launched a clinical program acceptable to U.S. and European regulatory agencies that allows us to include patients with blood cancers and up to 18 different non-malignant blood diseases under a single protocol. We believe BPX-501 may have the potential to make alternative donor haplo-identical stem cell transplants as routine as conventional transplants from matched donors, enabling a treatment known to be curative, and making it available for many more patients suffering from a wide range of deadly and life-long diseases.”

Study Details

The ongoing BP-004 trial is a Phase 1 / 2 multi-center, open label dose escalation study being held at leading transplant centers in Europe and in the U.S. The Phase 1 arm consists of three cohorts of three to six patients, each receiving escalating doses of BPX-501 T cells following haplo-HSCT. A Phase 2 extension arm will use the highest tolerated Phase 1 dose, with a maximum of 30 patients in both phases. The trial is enrolling pediatric patients, ages three months to 21 years, diagnosed with blood cancers(1), immune deficiencies(2) or inherited hematologic disorders(3), where hematopoietic stem cell transplants are historically curative.

The trial will also evaluate the treatment of Graft-versus-host disease (GvHD) in transplant patients who have received BPX-501 by infusion of the small molecule, dimerizer drug rimiducid (formerly AP1903). Rimiducid is designed to trigger rapid apoptosis and elimination of allo-reactive T cells. Patients who develop Grade III-IV acute GvHD, Grade II gut/liver GvHD, or Grade I-II acute GvHD (skin only) who progress or do not respond to standard treatment, will receive rimiducid.

Primary study endpoints include safety and optimal dosing of BPX-501 T cells, safety of rimiducid infusion in those patients who receive it to trigger elimination of BPX-501 T cells, and time to immune reconstitution. Primary endpoints for the Phase 2 extension also includes cumulative incidence of non-relapse mortality at 180 days and one year.

(1) Hematologic cancers include leukemias and lymphomas
(2) Immune deficiencies include severe combined immunodeficiency (SCID disorders), common variable immunodeficiency, alymphocytosis (“boy in a bubble” disease) and others
(3) Inherited hematological disorders include hemoglobinopathies including sickle cell, thalassemia, Fanconi’s anemia, and others.

About Bellicum Pharmaceuticals

Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for various forms of cancer, including hematological cancers and solid tumors, as well as 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. The Company is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or HSCT, CAR T cell therapy, and dendritic cell vaccines.

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. 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 our registration statement on Form S-1 and the related prospectus filed on December 17, 2014. 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.

Investors:
Bellicum Pharmaceuticals, Inc.
Alan Musso, 832-384-1116
Chief Financial Officer
amusso@bellicum.com
or
Media:
Brad Miles, 646-513-3125
bmiles@bmccommunications.com