Bellicum Pharmaceuticals Announces Successful Dosing of First Patient Cohort With BPX-501 T Cells Following Haplo-Identical Hematopoietic Stem Cell Transplant
BPX-501 is being evaluated as adjunct T cell therapy in patients with hematologic cancers and genetic blood diseases who lack a matched donor
The trial in pediatric patients is evaluating whether BPX-501 T cells from partially mis-matched donors administered following HSCT are safe and can help speed immune reconstitution. BPX-501 contains the CaspaCIDe® safety switch, giving doctors the ability to eliminate the T cells should they cause Graft-versus-host disease, a more common occurrence with a haplo-HSCT than with a matched procedure. BPX-501 is designed to allow study doctors to restore patient immunity earlier with increasingly higher doses of haplo-identical T cells, in an effort to improve infection control and overall transplant outcomes.
“For hematological cancers and many orphan blood disorders such as
severe primary immune deficiencies, inherited bone marrow failure
syndromes, thalassemia and sickle cell disease, a hematopoietic stem
cell transplant from a matched donor has resulted in a lifetime cure.
But many patients lack a suitable, matched donor, eliminating this
option,” said Prof.
Commented Bellicum CEO
The ongoing BP-004 trial is a Phase 1 / 2 multi-center, open label dose
escalation study being held at leading transplant centers in
The trial will also evaluate the treatment of Graft-versus-host disease (GvHD) in transplant patients who have received BPX-501 by infusion of the small molecule, dimerizer drug rimiducid (formerly AP1903). Rimiducid is designed to trigger rapid apoptosis and elimination of allo-reactive T cells. Patients who develop Grade III-IV acute GvHD, Grade II gut/liver GvHD, or Grade I-II acute GvHD (skin only) who progress or do not respond to standard treatment, will receive rimiducid.
Primary study endpoints include safety and optimal dosing of BPX-501 T cells, safety of rimiducid infusion in those patients who receive it to trigger elimination of BPX-501 T cells, and time to immune reconstitution. Primary endpoints for the Phase 2 extension also includes cumulative incidence of non-relapse mortality at 180 days and one year.
(1) Hematologic cancers include leukemias and lymphomas
(2) Immune deficiencies include severe combined immunodeficiency (SCID disorders), common variable immunodeficiency, alymphocytosis (“boy in a bubble” disease) and others
(3) Inherited hematological disorders include hemoglobinopathies including sickle cell, thalassemia, Fanconi’s anemia, and others.
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for various forms of cancer, including hematological cancers and solid tumors, as well as orphan inherited blood disorders. The Company is using its proprietary Chemical Induction of Dimerization, or CID, technology platform to engineer and control components of the immune system in real time. The Company is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or HSCT, CAR T cell therapy, and dendritic cell vaccines.
This press release contains forward-looking statements for purposes of
the safe harbor provisions of the Private Securities Litigation Reform
Act of 1995. We may, in some cases, use terms such as "predicts,"
"believes," "potential," "proposed," "continue," "estimates,"
"anticipates," "expects," "plans," "intends," "may," "could," "might,"
"will," "should" or other words that convey uncertainty of future events
or outcomes to identify these forward-looking statements.
Forward-looking statements include statements regarding our intentions,
beliefs, projections, outlook, analyses or current expectations
concerning, among other things: the timing of our clinical trials and of
our research and development activities relating to BPX-501. Various
factors may cause differences between Bellicum’s expectations and actual
results as discussed in greater detail in Bellicum’s filings with the