On March 6, 2023 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported a collaboration with Washington University School of Medicine in St. Louis to advance a Phase 1 clinical trial that will evaluate the safety and feasibility of the Company’s lead clinical candidate motixafortide to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease (SCD), one of the most common genetic diseases globally (Press release, BioLineRx, MAR 6, 2023, View Source [SID1234628180]). Currently available mobilization regimens can carry serious risks and side effects for patients with SCD or may not reliably yield optimal numbers of HSCs for gene therapy.

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The clinical trial is part of the Company’s long-term growth strategy for motixafortide across multiple therapeutic areas, including a potential future indication as a stem cell mobilization agent to support gene therapy development.

"Autologous hematopoietic stem cell-based gene therapies now offer curative potential for patients with SCD, but they are dependent upon the collection of significant quantities of stem cells, including early progenitor stem cells," said John DiPersio, MD, PhD, Professor of Medicine and Director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine, and principal investigator of the trial. "The common mobilization agent G-CSF is contraindicated in patients with sickle cell disease, significantly limiting their stem cell mobilization options. The development of novel mobilization regimens has the potential to overcome this unmet need for patients."

"We are excited to be advancing this important collaboration, which has the potential to ultimately support sickle cell disease gene therapy for patients," said Tami Rachmilewitz, MD, Chief Medical Officer at BioLineRx. "Motixafortide is being developed across multiple therapeutic areas, including stem cell mobilization for multiple myeloma, treatment of pancreatic cancer, and now in support of stem cell mobilization for gene therapy development for genetic disorders, which, we believe, demonstrates its broad potential."

The proof-of-concept trial, which will study motixafortide as a single agent and in combination with natalizumab, will assess the safety and tolerability of the two regimens as mobilization agents of CD34+ hematopoietic stem cells in patients with SCD. The study is anticipated to begin enrollment in the second half of 2023.

About the Clinical Trial of Motixafortide in Sickle Cell Disease (SCD)
The trial is a safety and feasibility study to evaluate motixafortide (CXCR4 inhibitor) as monotherapy and in combination with natalizumab (VLA-4 inhibitor) as novel regimens to mobilize CD34+ hematopoietic stem cells for gene therapies in SCD. The study will enroll five adults with a diagnosis of SCD who are receiving automated red blood cell exchanges via apheresis. The trial’s primary objective is to assess the safety and tolerability of motixafortide alone and the combination of motixafortide + natalizumab in SCD patients, defined by dose-limiting toxicities. Secondary objectives include determining the number of CD34+ hematopoietic stem and progenitor cells (HSPCs) mobilized via leukapheresis; and determining the kinetics of CD34+ HSPCs mobilization to peripheral blood in response to motixafortide alone and motixafortide + natalizumab in SCD patients.

About Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common genetic diseases globally, affecting millions of people throughout the world and disproportionately impacting persons of color. Sickle cell disease arises from mutations in the hemoglobin gene, ultimately leading to the production of abnormally shaped (sickle) red blood cells that tend to stick to blood vessels causing their occlusion. The clinical manifestations of SCD include anemia and blood vessel occlusion which can lead to both acute and chronic pain, as well as tissue ischemia across multiple organ systems (e.g., stroke, heart attack, respiratory failure), ultimately compromising end organ function. The cumulative impact of these complications significantly impacts morbidity and mortality for patients with SCD.