Shares of Biolinerx Ltd. (NASDAQ:BLRX) shot up 53% to $4.88 May 4 on news that adding its lead candidate, motixafortide, to standard of care G-CSF for hematopoietic stem cell mobilization helped achieve significantly better mobilization than G-CSF alone in a phase III trial. The company said it's working "aggressively" to gain regulatory approval to market the drug for use in autologous bone marrow transplants for multiple myeloma (MM) patients, with plans to make an NDA submission in the first half of 2022.

High-dose chemotherapy followed by autologous stem cell transplant has become an established treatment for a variety of blood cancers, including MM, with about 45,000 autologous transplants conducted in the EU and U.S. in 2018, according to Biolinerx. However, while stem cells can often be mobilized from a patient's bone marrow with G-CSF alone, where mobilization fails – an estimated 5% to 30% of the time in MM – Mozobil (plerixafor, Sanofi SA) is generally used to improve it for easier collection of the cells with apheresis.

Motixafortide’s potential was already illuminated in large part in October 2020, when the Tel Aviv, Israel-based company stopped enrollment in the trial early due to evidence favoring the candidate. Now, an analysis of data on all 122 patients enrolled in Biolinerx's phase III Genesis study suggest motixafortide may be able to serve the same function, and possibly with improved convenience. If approved, it would be the company's first commercial product.

Motixafortide, also called BL-8040, is a short synthetic peptide that targets CXCR4, a chemokine receptor that is overexpressed in many human cancers. CXCR4 plays a role in tumor growth and its overexpression is often correlated with a poor prognosis.

A combination of motixafortide and G-CSF demonstrated a 4.9-fold increase vs. G-CSF alone in achieving the trial's primary endpoint of target mobilization in up to two apheresis sessions, the company reported Tuesday, meeting all primary and secondary endpoints (p<0.0001).

According to top-line data reported by the company, 70% of trial participants who received just one administration of motixafortide after G-CSF mobilized at least 6 million CD34+ cells/kg in up to two apheresis sessions vs. just 14.3% of participants who received a placebo after G-CSF.

On a key secondary endpoint of the study, the addition of motixafortide to G-CSF allowed 88.3% of patients to undergo transplantation after just one apheresis session vs. 10.8% in the G-CSF arm, the company said.

The results are "a real game-changer" that will provide a basis for the candidate to become the new standard of care on top of G-CSF, Biolinerx CEO Philip Serlin said in a conference call held to present the results. To prove its case, the company is already pursuing a pharmacoeconomic analysis to support the contention. "Eliminating the need for additional apheresis sessions may improve patients' quality of life, reduce costs and allow for more efficient use of resources," he said.

Even as it charges ahead with the stem cell mobilization program, Biolinerx is also working to move ahead with other programs, including an evaluation of motixafortide for the treatment of pancreatic cancer and testing of AGI-134, an immunotherapy candidate for multiple solid tumors now in phase I/IIa testing.