BioLineRx Ltd. has announced the filing of regulatory submissions required to commence a Phase 2 trial for BL-8040 as a novel approach for mobilization and collection of bone marrow stem cells from the peripheral blood circulation. The trial is expected to commence shortly after receipt of regulatory approval, anticipated in the first quarter of 2016.
The Phase 2 open-label study will be conducted as an investigator-initiated study in collaboration with Washington University School of Medicine, Division of Oncology and Hematology, and will enroll up to 24 donor/recipient pairs. The study submission was made following a meeting with the FDA in October 2015 to discuss the BL-8040 stem cell mobilization development program.
The trial will evaluate the ability of BL-8040 to promote stem cell mobilization as a single agent in the allogeneic transplantation setting. Donors between 18 and 70 years of age and their HLA-matching recipients, diagnosed with advanced hematological malignancies that require stem cell transplantation, will be recruited to the study.
On the donor side, the primary endpoint of the study is the ability of a single injection of BL-8040 to mobilize sufficient amounts of cells for transplantation following up to two leukapheresis collections. On the recipient side the study aims to evaluate the functionality and engraftment following transplantation of the BL-8040 collected graft.
The safety and tolerability of BL-8040 in the healthy donors will be evaluated. In addition, graft durability, the incidence of grade 2-4 acute graft versus host disease (GvHD), and other recipient related parameters will be evaluated in the patients who have undergone transplantation of hematopoietic cells mobilized with BL-8040.
Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx, stated, “Stem cell mobilization is used increasingly as a method of collecting hematopoietic stem cells for transplantation, forming part of the treatment regimen for certain types of hematological cancers, as well as for severe anemia or immune deficiency disorders. We have already completed a successful Phase 1 safety and efficacy study in healthy volunteers, supporting BL-8040 as one-day, single-dose collection regimen, with the capacity to rapidly mobilize substantial amounts of stem cells, representing a significant improvement upon the current standard of care. Following a productive meeting with the FDA regarding the development program for BL-8040 as a stem cell mobilizer for allogeneic transplantation, and in light of the fact that there are no approved drugs for stem cell mobilization to support allogeneic transplant, we are looking forward to shortly commencing yet another Phase 2 trial for our lead oncology platform.”
“In parallel, BL-8040 is also undergoing a Phase 2 study for treating relapsed and refractory acute myeloid leukemia patients, and has recently initiated a Phase 2b study as an AML consolidation treatment and a Phase 1/2 study as a novel treatment for hMDS and AA, two bone marrow failure conditions. In addition, as we recently announced, we are also performing an extensive evaluation of BL-8040’s potential in the immuno-oncology space, as a combination treatment with immune checkpoint inhibitors,” added Dr. Savitsky.