UPDATE: Bellicum Pharma Announces Successful First Dosing for Patient Cohort with BPX-501

Bellicum Pharmaceuticals, Inc.

, 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.