Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BL-8040 · 4 trials · 15 indications
Response is determined by assessment of target lesions identified in CT or MRI imaging. The ORR is assessed according to RECIST 1.1, defined as the sum of PRs (Partial Responses) and CRs (Complete Responses) determined according to best response RECIST 1.1 criteria. PR is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter. CR is defined as disappearance of all target lesions.
95% confidence interval (CI) will be calculated for subjects who achieve Complete response (CR), Partial response (PR), for all AA and MDS subjects, and hematological improvement (HI) for MDS subjects only. Measurement in percentages (%)
| Arm | Type | Description |
|---|---|---|
| Arm 1: Donors | EXPERIMENTAL | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections. |
| Arm 2: Recipients | EXPERIMENTAL | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Cohort 1: BL-8040 + Pembrolizumab (Keytruda®) | EXPERIMENTAL | BL-8040 monotherapy 1.25 mg/kg subcutaneous (SC) injections daily on Days 1-5 of Week 1 of treatment. Combination therapy period begins following monotherapy treatment and consists of: * Pembrolizumab 200 mg once every three weeks. * Beginning on Day 10, BL-8040 three times a week |
| BL-8040 + Pembrolizumab + Chemotherapy | EXPERIMENTAL | BL-8040 monotherapy 1.25 mg/kg subcutaneous (SC) injections daily on days 1-5 of week 1 of treatment. Combination therapy period begins following monotherapy treatment and consists of: * IV Onivyde® 70 mg/m2 over 90 minutes followed by IV leucovorin (LV) 400 mg/m2 over 30 minutes or according to local standard, followed by IV fluorouracil (5-FU) 2400 mg/m2 over 46 hours, every 2 weeks. * Pembrolizumab 200mg once every three weeks. * Beginning on Day 10, BL-8040 twice a week and following the chemotherapy dosing. |
| BL-8040 plus hATG, methylprednisolone, cyclosporine | EXPERIMENTAL | BL-8040 0.75mg/kg will be administered Subcutaneously (SC ) on Days 1-10, then on first 5 days of months 2-6. Standard therapy: hATG: Days 11-14: 40mg/kg/day IV over 6-8 hours, in all AA subjects, or in MDS subjects less than 55 years old. 35mg/kg/day IV over 6-8 hours in MDS subjects 55 years and older. Standard therapy: methylprednisolone: Days 11-14: 40mg/kg/day IV over 6-8 hours, in all AA subjects, or in MDS subjects less than 55 years old. 35mg/kg/day IV over 6-8 hours in MDS subjects 55 years and older. Standard therapy: cyclosporine: 5mg/kg/day orally, given in 2 divided doses, starting on day 11 and continuing through Month 6 Day 30 (end of treatment) |
| Cohort A | EXPERIMENTAL | Each subject will receive a BL-8040 or Placebo on in a randomized double-blind fashion |
| Cohort B | EXPERIMENTAL | Each subject will receive a BL-8040 or Placebo on in a randomized double-blind fashion |
| Cohort C | EXPERIMENTAL | Each subject will receive a BL-8040 or Placebo on in a randomized double-blind fashion |
| Name | Type | Description |
|---|---|---|
| BL-8040 | DRUG | - |
| Leukapheresis | PROCEDURE | - |
| Hematopoietic cell transplant | PROCEDURE | - |
| Pembrolizumab | DRUG | Pembrolizumab will be given as a 30-minute IV infusion |
| Chemotherapy | DRUG | • IV Onivyde® followed by IV leucovorin (LV), followed by IV fluorouracil (5-FU), every 2 weeks. |
| horse anti-thymocyte globulin (hATG) | DRUG | From Day 11 through Day 14 of first month, subjects will receive hATG infusion over 6-8 hours each day. |
| Methylprednisolone | DRUG | From Day 11-14 of first month, subjects receive infusion of methylprednisolone 30 minutes prior to hATG infusion. Treatment with methylprednisolone will continue for 30 days. (After day 14, subjects may receive oral prednisone dose equivalent to IV methylprednisolone dose. The oral dose will be tapered off over 30 days. |
| Cyclosporine | DRUG | From Day 11 through end of treatment (Month 6 Day 30), subjects will receive oral dose of cyclosporine. |
| Placebo | DRUG | - |
Inclusion Criteria (DONOR): * Age 18 to 70 years of age. * ECOG performance status of 0 or 1. * PART 1: Donor must be a 5/6 or 6/6 HLA-matched sibling willing to donate PBSC for transplant. * PART 2: Donor must be a 5/6 or 6/6 HLA-matched sibling or 3/6 or 4/6 HLA haploidentical donor willing to do...
BL-8040 is an investigational small molecule being developed for multiple oncology and hematology indications, including metastatic pancreatic adenocarcinoma, acute myelogenous leukemia, aplastic anemia, multiple myeloma, and mobilization of hematopoietic stem cells to peripheral blood. It is currently in clinical development and is not approved by the FDA.
BL-8040 is being developed by BioLineRx Ltd., a biopharmaceutical company traded on the NASDAQ under the ticker BLRX. The company is conducting clinical trials to evaluate the drug's safety and efficacy across several indications.
BL-8040 is in Phase 2 clinical development for most indications, with one Phase 3 trial active for stem cell mobilization in multiple myeloma. It remains investigational and has not received FDA approval. The Phase 3 trial is active but not recruiting participants.
BL-8040 has been studied in several trials, including NCT02073019 (Phase 1, healthy volunteers), NCT02462252 (Phase 2, aplastic anemia), NCT02639559 (Phase 2, hematological malignancies), and NCT03246529 (Phase 3, multiple myeloma). These trials are completed or active, with a total enrollment of 180 in the Phase 3 study.
BL-8040 is also known as BL-8040 1.25 mg/kg + G-CSF, which refers to the combination of BL-8040 with granulocyte colony-stimulating factor (G-CSF) at a specific dose. This combination is being studied for stem cell mobilization in multiple myeloma.