On June 23, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that results will be presented today from its global BP-004 clinical trial of BPX-501 in the Presidential Symposium at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) being held in Madrid, Spain (Press release, Bellicum Pharmaceuticals, JUN 23, 2017, View Source [SID1234519659]). Clinical results reported in pediatric patients with a wide range of genetic blood diseases and hematologic cancers suggest that BPX-501 lowers the risks associated with haploidentical stem cell transplants, increasing their acceptability for more patients who could benefit but lack a matched donor. The EHA (Free EHA Whitepaper)’s Presidential Symposium showcases the top abstracts selected by the Scientific Program Committee each year.

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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. Franco Locatelli, MD, PhD, Director of the Department of Pediatric Hematology and Oncology, Ospedale Pediatrico Bambino Gesù (OPBG), Rome. "All cases of uncontrolled acute GvHD were resolved by administration of rimiducid to activate the CaspaCIDe safety switch incorporated in BPX-501. With low incidence of transplant-related mortality and manageable GvHD, these data suggest that the addition of BPX-501 T cells could represent a meaningful advance for patients in need of a transplant but who lack a matched related donor."

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 Europe, as well as on the subset of 61 patients at European sites.

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 Rome (OPBG) in patients undergoing an alpha/beta T-cell depleted haplo-HSCT without the addition of BPX-501 (n=107). Additionally, there were no reported adverse events associated with the use of BPX-501 or rimiducid. At six months of follow-up, the cumulative incidence of Grade 2-4 acute Graft versus Host Disease (GvHD) was 13 percent. The cumulative incidence of chronic GvHD was three percent at one year of follow-up.

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 Rick Fair, President and CEO of Bellicum Pharmaceuticals. "Data from this ongoing BP-004 study in Europe, along with comparative data from an observational trial of pediatric patients undergoing a stem cell transplant from matched unrelated donors, are expected to support the filing of a European Marketing Authorization Application in mid-2018."

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)
Other (n=14)

Hemoglobinopathies and Erythroid Disorders Poster Presentation
Also today at the 22nd Congress of EHA (Free EHA Whitepaper), updated results will be presented in patients with hemoglobinopathies and erythroid disorders, including five patients with sickle cell disease, in a poster presentation titled: "The Use of BPX-501 Donor T-Cell Infusion (with Inducible Caspase 9 Suicide Gene) Together with HLA-Haploidentical Stem Cell Transplant to Treat Children with Hemoglobinopathies and Erythroid Disorders."

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, June 23 at 3:45 – 4:00 PM CEST (9:45-10:00 AM EDT) and the poster (abstract P381) will be reviewed today at 5:15 – 6:45 PM CEST (11:15 AM – 12:45 PM EDT). Both presentations will be made available in the Events and Presentations section of the Bellicum website shortly after the time of the respective presentation.

About BPX-501
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.