Bellicum Announces Interim Results Showing Low Rates of Cancer Recurrence in Pediatric AML Patients Treated with BPX-501
- Category: DNA RNA and Cells
- Published on Tuesday, 13 March 2018 18:43
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Company also reports encouraging disease-free survival and overall survival rates in pediatric patients with primary immunodeficiencies
A pediatric registration trial is being planned
HOUSTON, TX, USA I March 13, 2018 I Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, today reported interim clinical data of BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies (PIDs). BPX-501 is an adjunct T cell therapy incorporating CaspaCIDe® administered after haploidentical hematopoietic stem cell transplant (haplo-HSCT) for the treatment of hematologic cancers and inherited blood diseases.
Data from the ongoing BP-004 trial suggest that BPX-501 T cells may contribute to a durable anti-leukemic effect in patients with AML. In the study, 38 pediatric AML patients in their first (n=13) or second (n=25) complete response underwent a haplo-HSCT followed by treatment with BPX-501. With a median follow-up of one year, rates of relapse-free survival and overall survival were 91.5% and 97.3%, respectively. This compares to one-year rates reported in the literature in pediatric AML patients undergoing alternate-donor HSCT of 60% to 80%.
“The recurrence of cancer is one of the most serious risks to AML patients receiving a stem cell transplant. These impressive results in children with AML suggest that BPX-501 may be effectively reducing or eradicating residual cancer cells following a haplo-transplant procedure,” commented Neena Kapoor, M.D., Director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and an investigator in the BP-004 trial.
Also from the BP-004 study, the Company reported high rates of disease-free survival and overall survival in pediatric patients with PIDs, including Severe Combined Immunodeficiency (“bubble boy disease”), Wiskott-Aldrich syndrome, Chronic Granulomatous Disease, and other rare immune deficiencies. Of 59 pediatric PID patients undergoing a haplo-HSCT and treatment with BPX-501, disease-free survival was reported at 88.1% and overall survival was reported at 88.6% with a median follow-up of one year.
Continued Dr. Kapoor: “Delayed immune reconstitution can lead to severe infectious complications, a major cause of morbidity and mortality in PID patients who undergo a T-depleted haplo-HSCT. BPX-501 donor T cells administered after a transplant support immune recovery in these patients, and the CaspaCIDe safety switch engineered into BPX-501 may provide a critical safety net to address the risk of uncontrolled GvHD from donor T cells.”
Based on these clinical data, Bellicum is working with the investigators and the U.S. Food and Drug Administration to develop a protocol for a potential U.S. registration study in pediatric patients. Pending regulatory clearances, the Company expects to initiate the study by the end of 2018. These clinical data have been submitted for presentation at an upcoming medical meeting.
About Bellicum Pharmaceuticals
SOURCE: Bellicum Pharmaceuticals