On June 23, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported data from its ongoing multicenter BP-004 trial of BPX-501 and rimiducid in a cohort of pediatric patients with acute leukemias who lack a matched donor (Press release, Bellicum Pharmaceuticals, JUN 23, 2017, View Source [SID1234519662]). Results from 47 patients demonstrated that administering BPX-501 following an alpha/beta T-cell depleted haploidentical hematopoietic stem cell transplant (alpha/beta T-cell depleted haplo-HSCT) produced rapid immune reconstitution, low incidence of acute and chronic Graft versus Host Disease (GvHD), and a low rate of disease relapse. The data will be reviewed in an oral presentation at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) at 5 p.m. CEST on June 24 in Madrid, Spain.

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"These data demonstrate that BPX-501-modified T cells can provide rapid hematological reconstitution and a potentially stronger anti-leukemic effect in children with high-risk disease than traditional T-cell depleted transplants," said Franco Locatelli, MD, PhD, Director of the Department of Hematology and Oncology at Ospedale Pediatrico Bambino Gesù in Rome, Italy. "The incidence of GvHD was low, and managed by standard treatments or the administration of rimiducid. Patients who received rimiducid experienced a recovery of the beneficial, non-reactive BPX-501 T cells. These are very encouraging early outcome results for children who need a stem cell transplant and lack a matched donor."

Summary of Results (Abstract S495)

Investigators evaluated the safety and efficacy of an infusion of BPX-501 in children with high-risk acute lymphoblastic leukemia (ALL) (n=28) and acute myeloid leukemia (AML) (n=19) following an alpha/beta T-cell depleted haplo-HSCT. Patients had a median follow-up of approximately eight months (11 months in surviving patients). Results from the three European trial sites showed:

Disease relapse rate of 14.72% in BPX-501 treated patients
Median neutrophil recovery = 16 days (9-24); median platelet engraftment = 11 days (8-19); median time to hospital discharge = 21.5 days (14-103)
Acute GvHD Grade 2-4 was 11.83%; acute GvHD Grade 3-4 was 4.70%
Chronic GvHD Grade Moderate-Severe was 3.23%
Non-relapse mortality (NRM) of 3.23% in BPX-501 patients
Re-expansion of infection-fighting BPX-501 cells after a sharp decrease following infusion of rimiducid, with no reoccurrence of GvHD
Results compare favorably to historic controls1
Commented Rick Fair, President and CEO of Bellicum Pharmaceuticals, "We are pleased by the profile observed with BPX-501 in pediatric patients receiving a haploidentical transplant, and by its potential to reduce disease relapse. These results provide important insights into the potential long-term benefits of BPX-501, and support our plans to begin a mid-stage study this year in adults with hematologic cancers, for whom lack of a matched donor and disease relapse are significant treatment challenges."

The presentation slides (abstract S495) will be made available in the Events and Presentations section of the Bellicum website shortly after the time of the presentation.

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 administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect, without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.