BPX-501 in Blood Cancers &
Inherited Blood Disorders


BPX-501 Phase 1/2 Clinical Trials

We are currently conducting four Phase 1/2 clinical trials of BPX-501 at leading transplant centers in the United States and Europe:

  • BP-001 and BP-005, in adults with hematological cancers in which BPX-501 is administered after initial allogeneic HSCT;
  • BP-003, a single site study in children with orphan inherited blood disorders in which BPX-501 is administered after initial allogeneic HSCT;
  • BP-004, in children with hematological cancers or orphan inherited blood disorders in which BPX-501 is administered after initial allogeneic HSCT;
  • BP-008, to treat post-transplant relapse in adults and children with blood cancers and evaluate the potential for a titrated dose of rimiducid to resolve uncontrolled GvHD while preserving BPX-501 cells

We are planning to initiate additional Phase 1/2 clinical trials in the U.S. and Europe, as part of our strategy to pursue global regulatory approvals and expand the potential addressable patient population for BPX-501. More information about our BPX-501 trials can be found at clinicaltrials.gov.

Clinical studies published in the medical journal Blood in 2015 (DOTTI trial) and in The New England Journal of Medicine in 2011 and Blood in 2014 (CASPALLO trial), showed that CaspaCIDe-modified donor T cells administered 30+ days after T cell depleted haplo-HSCT, improved immune reconstitution and reduced viral infections. A single dose of rimiducid was administered to the 8 of 22 patients who developed acute GvHD, which was resolved within 24-48 hours. Anti-viral immunity was preserved after rimiducid administration and the patients required no further treatment.

The Texas Children’s Hospital, in collaboration with Baylor College of Medicine, under agreements with us, conducted the two Phase 1 DOTTI and CASPALLO clinical trials. The studies had similar parameters, except that in the CASPALLO trial the CaspaCIDe T cells were depleted of alloreactive cells prior to transplant, and in the DOTTI trial the donor T cells were non-allodepleted.