BPX-501: Encouraging Results to Date in Haploidentical Hematopoietic Stem Cell Transplantation

Lead product candidate designed to improve outcomes for patients undergoing stem cell transplant who lack a matched donor  

BPX-501 is being studied to improve the outcomes of patients undergoing haploidentical (partial match) hematopoietic stem cell transplants (HSCT). Our lead product candidate was designed to address the controllability issues associated with haplo-HSCT, and has demonstrated impressive clinical results to date. While stem cell transplants can be life-saving for patients with orphan inherited blood disorders and hematologic malignancies who lack a matched donor, they also carry the risk of life-threatening infections and uncontrolled graft versus host disease (GvHD). The potential benefit of BPX-501 is multi-fold: the patient gets the benefit of having T cells to fight infection, support engraftment, and prevent disease relapse and, should GvHD occur, the CaspaCIDe® safety switch can be activated to kill the toxic T cells. We believe BPX-501 can improve outcomes and broaden patient eligibility for a haplo transplant.

The European Commission (EC) granted orphan drug designations for BPX-501 for treatment in HSCT, and for activator agent rimiducid for the treatment of GvHD. BPX-501 and rimiducid also have orphan drug status from the U.S. Food and Drug Administration (FDA) as a combination replacement T-cell therapy for the treatment of immunodeficiency and graft versus host disease (GvHD) after allogeneic hematopoietic stem cell transplant.

European BP-004 Pivotal Clinical Trial Progressing

During the 59th Annual Meeting of the American Society of Hematology (ASH), data from a study of 112 pediatric patients within the ongoing Phase 1/2 BP-004 trial were reported in an oral presentation. Patients in the study were treated with BPX-501 following an alpha/beta T cell and CD19+ B cell depleted haploidentical hematopoietic stem cell transplant (haplo-HSCT). Results demonstrated that donor BPX-501 cells infused after transplant expanded in vivo and persisted over time, contributing to improved immune recovery for these patients as compared to historical controls from the same transplant center.

Outcomes from the BP-004 trial, along with those from a comparator observation study to be conducted in the MUD (matched unrelated donor) transplant setting, are expected to be the basis for filings of Marketing Authorization Applications (MAAs) in Europe for BPX-501 and rimiducid. The MAAs will be for treatment of pediatric patients with orphan inherited blood disorders or treatment-refractory hematological cancers.

Registrational Trials of BPX-501 in the U.S.
We are finalizing plans for the design of our registrational trials of BPX-501 in the U.S. Our current plans include conducting a controlled clinical trial in adult patients with acute myeloid leukemia (AML). In the pediatric non-malignant setting, we are designing a registrational trial to evaluate BPX-501 in a distinct subset of orphan inherited blood disorders.