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35 children with genetic immune disorders and hemoglobinopathies are disease- and GvHD-free following haploidentical stem cell transplant with BPX-501
Webcast of ASH investor/analyst event scheduled for Monday, December
PID Highlights (Abstract #72)
“Outcome of Children with Primary Immune-Deficiencies (PIDs) Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501 Donor T Cells Genetically Modified with a Novel Suicide Gene (Inducible Caspase 9, iC9) After T-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (Haplo-HSCT)”
PIDs in the study include Severe Combined Immune Deficiency (“Bubble boy” disease) (n=11), Wiskott-Aldrich syndrome (n=6), Chronic Granulomatous Disease (n=2) and other rare Immune Deficiencies (n=4). Study outcomes for these patients included:
- All 23 PID patients engrafted with no secondary graft failure.
- All patients are alive and free of disease with a median follow-up of 404 days (range: 118-728 days).
- Median time to neutrophil and platelet recovery was 16 days and 10 days, respectively.
- Five children experienced Grade I or Grade II acute GvHD. Three of the cases resolved with either topical or systemic steroids. The other two GvHD cases resolved following infusion of rimiducid and activation of the CaspaCIDe® safety switch. There was one mild case of chronic GvHD, which also resolved. No one experienced severe Grade 3 or Grade 4 GvHD.
- There was a low rate of hospital re-admission to treat infections. No reported adverse events were associated with administration of BPX-501.
Hemoglobinopathy Highlights (Abstract #2286)
“Clinical Outcome and Immune Recovery after Adoptive Infusion of BPX-501 Cells (Donor T Cells Transduced with iC9 Suicide Gene) in Children with Hemoglobinopathies and Diamond-Blackfan Anemia Given a/b T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation (HSCT)”
Updated BP-004 study results were also reviewed in a poster session of 12 pediatric patients with Thalassemia Major β0/β0 (n=9), Sickle Cell Disease (n=1) and Diamond-Blackfan Anemia (n=2). HSCT is a potentially curative treatment for these patients, who otherwise typically require a lifetime of blood transfusions. Study results demonstrated that the BPX-501 cells expanded and persisted in the patients over time, contributing to overall immune reconstitution and successful transplant outcomes. Results reported in the poster included:
- All 12 patients attained full donor chimerism. One patient with secondary graft failure was re-transplanted from the same donor and then attained full donor chimerism.
- All patients are alive, GvHD-free and free of disease.
- The median initial hospital discharge occurred at 21 days (range: 14-55) and there were no re-hospitalizations.
- Grade I/II skin acute GvHD occurred in 2 patients, and was successfully treated with steroids. No chronic GvHD occurred.
- Median time to last red blood cell transfusion was 6.5 days (range: 4-33 days) post-transplant.
“Our BPX-501 program continues to gain momentum as additional centers
recognize its potential to improve haplo-HSCTs in both nonmalignant and
malignant diseases,” commented
Bellicum will host an investor and analyst luncheon on
Primary Immune Deficiencies (PIDs) are caused by genetic abnormalities that prevent the development of normal immune responses, which lead to an increased susceptibility to infections. A hematopoietic stem cell transplant (HSCT) is the current mainstay of treatment for severe forms of PIDs, including Severe Combined Immune Deficiency (SCID), Wiskott-Aldrich syndrome, Chronic Granulomatous Disease (CGD) and several others.
Hemoglobinopathies are a diverse group of genetic disorders caused by the abnormal structure or production of hemoglobin, a protein molecule in red blood cells that carries oxygen throughout the body. Depending on disease severity, standard treatments include blood transfusions and drugs to control symptoms, however a stem cell transplant is often preferred for severe cases.
BPX-501 is an adjunct T-cell therapy administered after allogeneic HSCT,
comprising genetically modified donor T cells incorporating Bellicum’s
CaspaCIDe® safety switch. It is designed to provide a safety
net to eliminate alloreactive BPX-501 T cells (via administration of
activator agent rimiducid) should uncontrollable GvHD occur. This
enables physicians to more safely perform stem cell transplants by
adding back BPX-501 engineered T cells to speed immune reconstitution
and provide control over viral infections, without unacceptable risk of
uncontrollable GvHD. The ongoing BP-004 Phase 1/2 clinical study of
BPX-501 is being conducted at transplant centers in the U.S. and
About Bellicum Pharmaceuticals
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), and CAR T and TCR cell therapies. More information can be found at www.bellicum.com.
This press release contains forward-looking statements for purposes
of the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. Bellicum may, in some cases, use terms such as
"predicts," "believes," "potential," "proposed," "continue," “designed,”
"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: our
research and development activities relating to BPX-501, rimiducid and
CaspaCIDe; the effectiveness of CaspaCIDe; the effectiveness of BPX-501,
its possible range of application and potential curative effects and
safety in the treatment of diseases including as compared to other
treatment options and competitive therapies; the timing and success of
our clinical trials, including the rate and progress of enrollment in
our clinical trials; and, the timing of regulatory filings for BPX-501
and for rimiducid. Various factors may cause differences between
Bellicum’s expectations and actual results as discussed in greater
detail under the heading “Risk Factors” in Bellicum’s filings with the
Alan Musso, CFO, 832-384-1116
Brad Miles, 917-570-7340
Amy Bonanno, 914-450-0349