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“The vast majority of patients in this study experienced successful engraftment, no or low grade GvHD, rapid immune recovery and early hospital discharge,” said Dr.
Updated Results of BP-004 Study in Global and EU Populations
Investigators reported on pediatric patients with hematologic cancers and genetic blood diseases who underwent treatment with BPX-501 after an alpha/beta T-cell depleted haploidentical hematopoietic stem cell transplant (alpha/beta T-cell depleted haplo-HSCT) and had at least six months of follow-up. Investigators reviewed overall data on 98 patients from 12 centers in the U.S. and
At the 180-day measurement time point, cumulative incidence of transplant-related mortality (TRM) was 5% in the overall patient population. A significant reduction in viral infections and reinfections was observed with BPX-501 compared to results from a study in
The administration of rimiducid was required in 11 patients with acute GvHD who did not respond to standard treatments. In all 11 cases, rimiducid rapidly resolved the GvHD. In addition, the non-reactive T cells recovered, with no recurrence of GvHD.
Data from the subset of 61 patients from European sites were also reviewed in the presentation. At six months of follow-up there was no transplant-related mortality. Cumulative GvHD incidence remains low, with incidence rates of 10 percent for Grade 2-4 acute GvHD, and three percent for Grade 3-4 acute GvHD. There was no persistent chronic GvHD.
“Data from our global BP-004 study continue to show consistent and strong overall outcomes across a wide range of diseases, suggesting the potential to enable curative stem cell transplants from partially matched donors for more children,” said
Patient Populations Included in Study, By Disease Type
|Genetic Blood Diseases (n=59)||Hematologic Cancers (n=39)|
|Primary immune deficiencies (n=26)||Acute lymphoblastic leukemia (ALL) (n=21)|
|Thalassemia major (n=8)||Acute myeloid leukemia (AML) (n=14)|
|Hemophagocytic lymphohistiocytosis (HLH) (n=6)||Other (n=4)|
|Sickle cell disease (n=5)|
Hemoglobinopathies and Erythroid Disorders Poster Presentation
Also today at the 22nd
All 15 patients in the cohort showed normal hemoglobin levels and remained transfusion-free at 180 days, with no transplant-related mortality.
The Presidential Symposium oral presentation (abstract S146) will take place today,
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 administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemia effect, without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.
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 our BPX-501, CaspaCIDe, CID, CAR T and TCR programs; the effectiveness of BPX-501 and rimiducid; their possible range of application and potential curative effects and safety in the treatment of diseases; and, the timing and success of our clinical trials. 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
Bellicum Pharmaceuticals, Inc. Alan Musso, CFO 832-384-1116 firstname.lastname@example.org Media: BMC Communications Brad Miles646-513-3125 email@example.com