Bellicum Pharmaceuticals Announces BPX-501 Clinical Data Demonstrating Disease-Free Outcomes in Patients with Genetic Diseases
ASH poster presentations report on pediatric patients with beta thalassemia, SCID, Fanconi anemia, Wiskott-Aldrich Syndrome and other genetic diseases undergoing haploidentical stem cell transplant and genetically modified BPX-501 T-cell add-back
No transplant-related mortality
Significant reductions in time to hospital discharge and re-hospitalizations
Webcast of ASH investor/analyst event scheduled for December 7, 2015
Initial outcomes were reported from the 39 pediatric patients who have
received the BPX-501 product (of a total of 49 enrolled) at the European
trial site as of
“These interim results present strong evidence that the addition of
BPX-501 modified donor T cells provides important immune support and
improves outcomes in patients undergoing a T-depleted haploidentical
stem cell transplant,” said lead investigator Dr.
The presented data show that treatment with BPX-501 is safe and well tolerated for patients with non-malignant and malignant diseases, and provides several important immune benefits compared to the clinical site’s historical controls. Highlights include:
- Safety: No adverse events associated with infusion of BPX-501 were reported. The occurrence and severity of GvHD in study subjects was generally consistent with the historical control group. There were seven instances of Grade 1 or 2 GvHD which all resolved without requiring activation of the CaspaCIDe® safety switch with rimiducid.
- Survival: There was no transplant-related mortality (TRM) in the 37 study patients with a minimum of 30 days follow-up. In particular, for non-malignant patients, this lack of TRM (0/18) compares favorably with 9% TRM in the historical non-malignant control patients (3/33). TRM, when it occurs, typically happens early in the post-transplant period in non-malignant transplant patients, primarily due to infection.
- Immune Reconstitution: Non-malignant patients in the trial achieved a mean improvement of approximately 40 fewer days to reach a T-cell count of 500 cells/ul, showing immune recovery was significantly faster than historical controls.
- Time in Hospital: Non-malignant patients in the trial were discharged significantly faster from the hospital, 21 days sooner on average following HSCT, compared to historical controls. The number of patients re-hospitalized was also substantially reduced.
“It’s exciting to see the progress and outcomes from this BPX-501 study
that we initiated just a year ago,” said
Bellicum will also host an investor and analyst luncheon on
BPX-501 is an adjunct T cell therapy of genetically modified donor T cells incorporating Bellicum’s proprietary CaspaCIDe safety switch. The product candidate is designed to provide a safety net to eliminate the BPX-501 alloreactive T cells should severe GvHD occur, enabling physicians to more safely perform haploidentical stem cell transplants by adding back the BPX-501 genetically engineered T cells to speed immune reconstitution and provide control over viral infections.
BP-004 Clinical Trial Design
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and 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. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or HSCT, and CAR T and TCR cell therapies. More information can be found at www.bellicum.com.
*CaspaCIDe® is a trademark registered with the U.S. Patent and Trademark Office.
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: the
timing and success of our clinical trials, including the rate and
progress of enrollment in such trials; the timing of an IND filing for
BPX-501; our research and development activities and expenses relating
to BPX-501, CaspaCIDe, CAR T-cell and TCR programs; the effectiveness of
BPX-501, its possible range of application and its potential curative
effects and safety in the treatment of blood cancers and inherited blood
diseases; and the potential applications and effectiveness of our
product candidates, including as compared to other treatment options and
competitive therapies. 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, 832-384-1116
Brad Miles, 917-570-7340
Amy Bonanno, 914-450-0349