Bellicum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BPX-501, the Company’s lead product candidate.
The FDA designation is for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as “replacement T-cell therapy for the treatment of immunodeficiency and graft versus host disease (GvHD) after allogeneic hematopoietic stem cell transplant.” BPX-501 is an adjunct T-cell therapy incorporating the Company’s proprietary CaspaCIDe® safety switch.
Following an allogeneic transplant, a lack of sufficient mature T cells constitutes immune deficiency that can contribute to infections, viral reactivation, and relapse. The ability to correct this immune deficiency through the add-back of mature donor T cells, without raising the risk of uncontrollable GvHD, has the potential to fundamentally change the risk profile of allogeneic transplantation.
BPX-501 is currently being evaluated in multiple Phase 1/2 clinical trials in adults and pediatric patients with leukemias, lymphomas, and genetic blood diseases in the U.S. and Europe. Interim results presented at the 2015 ASHAnnual Meeting in pediatric patients with a variety of genetic diseases, including beta thalassemia, Wiskott-Aldrich and SCID, demonstrated disease-free outcomes following a haploidentical, T cell-depleted hematopoietic stem cell transplant, followed by an add-back of BPX-501 donor T cells. Results demonstrated reduced infection rates, faster immune reconstitution, and reductions in time to hospital discharge and in re-hospitalizations, compared to historical controls.
