The study results are set to be presented orally at the American Society of Hematology (ASH) Annual Meeting on December 7, 2024, in San Diego, California.

Motixafortide alone achieved a median mobilization of 198 CD34+ cells/µl, projecting around 13.9 million stem cells in a single session, while the combination with natalizumab resulted in a median of 231 CD34+ cells/µl, projecting approximately 18.6 million stem cells.

The need for effective mobilization is critical, as current FDA-approved gene therapies for SCD require between 16.5 million and 22 million total CD34+ stem cells, which can be challenging to collect.

This trial was conducted in collaboration with Washington University School of Medicine in St. Louis, aiming to enhance treatment options for SCD patients.

The trial also found that patients previously mobilized with plerixafor had significantly greater mobilization with motixafortide, achieving a 2.8-fold to 3.2-fold increase in stem cells collected.

The trial indicates that motixafortide, whether used alone or in combination with natalizumab, can facilitate the collection of sufficient stem cells in a single apheresis cycle, a significant advancement for gene therapy accessibility.

Results showed that motixafortide significantly improved hematopoietic stem cell (HSC) mobilization compared to plerixafor, the current standard treatment, which often requires multiple attempts and yields suboptimal results.

In the trial, five patients successfully mobilized stem cells with motixafortide alone, while four out of five achieved mobilization with the combination treatment, underscoring motixafortide's robust mobilization capabilities.

BioLineRx's CEO, Philip Serlin, expressed optimism about these findings, highlighting their potential to improve access to gene therapy for SCD patients.

BioLineRx Ltd. has announced promising findings from a Phase 1 clinical trial of motixafortide, a treatment aimed at mobilizing stem cells for patients with sickle cell disease (SCD).

Initial data from the trial indicates that motixafortide is safe and well tolerated among patients, with common adverse events being mild and transient.

Zachary Crees, MD, the principal investigator, emphasized the ongoing challenge of collecting adequate quantities of CD34+ stem cells necessary for existing gene therapies.