BioLineRx is seeking regulatory approval to advance into a Phase 3 clinical trial its treatment BL-8040, for blood stem cell mobilization to prepare for autologous transplants in patients with multiple myeloma.
The company hopes that the trial, called GENESIS, will allow BL-8040 to become an approved treatment for myeloma. The study is expected to launch before the end of the year.
“We are excited to move forward with BL-8040 into a Phase 3 registration study,” Philip Serlin, BioLineRx chief executive officer, said in a press release.
“We have previously reported positive results supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen for rapid mobilization of stem cells. This represents a significant improvement over the current treatment, which requires four-to-eight daily injections of G-CSF and one to-four apheresis sessions. We therefore hope that this Phase 3 trial will further support these results and help improve the standard of care for multiple myeloma patients,” he added.
Stem cell mobilization is a procedure used to gather stem cells from a donor or from affected patients. These cells, normally produced in the bone marrow, are forced to enter circulation with the help of a compound called G-CSF (granulocyte colony-stimulating factor). They are then gathered from the blood using a process called apheresis, allowing easier access compared to a spinal tap procedure.
But using G-CSF is a slow procedure linked to side effects such as bone pain.
The Phase 3 trial will explore if BL-8040 used with G-CSF can safely improve the treatment. The study will be conducted in two parts. First, researchers will determine the optimal dosing of BL-8040 in up to 30 patients.
Next, 180 patients will be randomized to receive BL-8040 or a placebo. Researchers will assess the ability of the treatment to mobilize enough stem cells after one administration of BL-8040 and G-CSF, with a maximum of two apheresis sessions.
Patients receiving the combination will be compared to those receiving a placebo and G-CSF.
A Phase 2 trial of the approach is currently ongoing, and interim results showed that a single injection of the combination mobilized stem cells to a similar extent as four to six injections of G-CSF alone, currently the standard approach. The company reported no safety issues at the time.
BL-8040 is a compound that has more use as a cancer treatment than as a stem cell mobilizing agent. The therapy acts to block a receptor called CXCR4, found in higher-than-normal levels in 70% of human cancers.
Studies show that by blocking CXCR4 with BL-8040, cancer cells also are forced to leave the bone marrow. In circulation, they are then sensitized to chemotherapy or biological cancer therapies. BL-8040 also directly kills cancer cells by turning on cellular self-destruction mechanisms.
“In parallel, we are continuing to expand the potential of our robust BL-8040 oncology platform, by advancing multiple clinical studies for additional indications that are ongoing or expected to commence during 2017,” Serlin said.
“These include a large, randomized, controlled Phase 2b study in AML, as well as several Phase 2 combination studies with immune checkpoint inhibitors in solid tumors and hematological malignancies,” he added.