BioLineRx and St. Jude to Test Motixafortide for Stem Cell Mobilization in SCD Gene Therapy

BioLineRx Ltd., a biopharmaceutical company specializing in oncology and rare diseases, has announced a promising new Phase 1 clinical trial to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) in patients with sickle cell disease (SCD). Sponsored by St. Jude Children's Research Hospital, Inc., the trial is set to expand ongoing research efforts in the field of SCD gene therapy.

SCD is a genetic disorder that leads to the formation of abnormally shaped red blood cells, causing significant health complications such as anemia, chronic pain, and organ damage. Hematopoietic stem cell transplantation, following genetic modification, offers a potential cure for SCD. However, the collection of a sufficient number of HSCs, crucial for successful transplantation, poses challenges. The commonly used drug for this purpose, granulocyte colony-stimulating factor (G-CSF), is unsuitable for SCD patients. Currently, the mobilization agent plerixafor is employed, though it often requires multiple collection cycles, which can be a limiting factor.

Motixafortide, BioLineRx’s lead therapeutic candidate, was previously approved by the U.S. Food and Drug Administration (FDA) in combination with G-CSF for mobilizing HSCs in multiple myeloma patients. This new trial aims to assess motixafortide’s safety and efficacy as a single agent in SCD patients. The study will involve 12 patients aged 18 and older and will be conducted across multiple centers.

The trial, known as SCDSTEMM (Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide), is structured into two parts. Part A will examine the effects of a single dose of motixafortide followed by one apheresis session. Part B will evaluate a two-day regimen of motixafortide administration and apheresis. Primary objectives include assessing the safety and tolerability of motixafortide based on the incidence of adverse events. Secondary goals involve understanding the kinetics of CD34+ cells post-administration and determining the number of HSCs collected.

Dr. Ella Sorani, Chief Development Officer at BioLineRx, emphasized the significance of recent FDA approvals of gene therapies for SCD in the U.S., highlighting the potential impact on the SCD community. She expressed enthusiasm for the collaboration with St. Jude and ongoing partnerships with other leading institutions, such as Washington University School of Medicine in St. Louis, to explore new mobilization options for SCD patients.

Enrollment for the St. Jude trial is anticipated to commence in the coming months, with initial data expected in the latter half of 2024. This trial, along with ongoing research, represents a concerted effort to overcome existing hurdles in SCD treatment and improve patient outcomes.

St. Jude Children's Research Hospital, Inc. and the other participating clinical sites bring substantial expertise in SCD gene therapy, positioning this study to potentially make significant strides in the field. By exploring motixafortide's capacity to mobilize HSCs efficiently, the trial aims to address a critical bottleneck in gene therapy for SCD.

This initiative underscores BioLineRx's commitment to advancing innovative treatments in rare diseases and oncology. With a strong pipeline of investigational medicines, the company's efforts in developing life-changing therapies continue to align with their mission of transforming patient care through scientific discovery and clinical excellence.