BioLineRx to Present Phase 1 Trial Data on Motixafortide for Stem Cell Mobilization in Sickle Cell at ASH 2024

Findings suggest motixafortide alone, and in combination with natalizumab, could support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle. Data from proof-of-concept study shows that motixafortide was safe and well tolerated. Oral presentation at ASH 2024 on Saturday, December 7, 2024 in San Diego, California.

BioLineRx Ltd. announced that the initial results from a Phase 1 clinical trial evaluating motixafortide as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD) have been accepted for presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition. This event will take place from December 7-10, 2024, in San Diego, California. The study, conducted with Washington University School of Medicine in St. Louis, is investigating new strategies for HSC mobilization to improve the treatment process for SCD patients seeking gene therapy.

Dr. Zachary Crees, principal investigator for the trial from Washington University, highlighted that current gene therapies for SCD require the collection of substantial quantities of CD34+ hematopoietic stem cells, which is challenging for many patients. The trial's findings indicate that patients treated with motixafortide alone or combined with natalizumab could collect the necessary stem cells for gene therapies in one apheresis cycle. These promising results will be detailed at the upcoming ASH meeting.

Philip Serlin, CEO of BioLineRx, expressed optimism about the initial findings, noting that motixafortide has been shown to be safe and well-tolerated. The study suggests its potential to enhance the treatment process and improve access to gene therapy for more SCD patients. BioLineRx plans to continue collaborating with Washington University to advance this critical research.

The Phase 1 study aims to evaluate the safety and feasibility of motixafortide as a monotherapy and in combination with natalizumab for mobilizing CD34+ hematopoietic stem cells for gene therapies in SCD. According to the study’s abstract, five patients completed mobilization and apheresis with motixafortide alone, and four out of five patients did so with the combination treatment.

In terms of safety, motixafortide alone and in combination with natalizumab were well-tolerated. Common adverse events were mild and included transient reactions at the injection site and minor systemic reactions. No severe adverse events or vaso-occlusive episodes were reported.

The trial demonstrated that motixafortide, both alone and combined with natalizumab, effectively mobilized CD34+ HSCs into peripheral blood. Motixafortide alone mobilized a median of 198 CD34+ cells/μl with a median collection of 3.49x10 CD34+ cells/kg in a single blood volume collection. This suggests a projected collection of 13.9x10 CD34+ HSCs in a standard single-day apheresis session. When combined with natalizumab, a median of 231 CD34+ cells/μl was mobilized, with a median collection of 4.64x10 CD34+ cells/kg, projecting 18.6x10 CD34+ HSCs in a single-day session.

Currently, the two approved gene therapies for SCD in the U.S. require 16.5 million and 22 million total CD34+ HSCs, respectively. Granulocyte colony-stimulating factor (G-CSF) is contraindicated in SCD patients, making it necessary to find alternative methods for effective stem cell collection. Plerixafor is the current standard for collecting HSCs for SCD gene therapies but often requires multiple attempts and yields suboptimal results. The trial revealed that patients previously mobilized with plerixafor experienced significantly higher HSC mobilization with motixafortide alone and in combination with natalizumab.

The oral presentation of the trial’s findings will be part of the ASH 2024 meeting in San Diego, focusing on innovations in the mobilization, collection, and manufacturing of cellular therapies.

The clinical trial of motixafortide in Sickle Cell Disease (SCD) is a safety and feasibility study evaluating motixafortide alone and in combination with natalizumab as novel regimens to mobilize CD34+ hematopoietic stem cells for gene therapies in SCD. The study enrolled five adults with SCD, aiming to assess the safety and tolerability of these treatments and determine the number of mobilized CD34+ hematopoietic stem and progenitor cells.

Sickle cell disease (SCD) is one of the most common genetic disorders, disproportionately affecting people of color. It results from mutations in the hemoglobin gene, causing abnormally shaped red blood cells that can block blood vessels. SCD leads to anemia, pain, and compromised organ function, significantly impacting patient morbidity and mortality.

BioLineRx Ltd. is a biopharmaceutical company focused on developing therapies for oncology and rare diseases, including motixafortide for stem cell mobilization in multiple myeloma and other indications. The company is dedicated to advancing innovative treatments from development to commercialization.