BioLineRx Announces Clinical Trial Agreement with St. Jude Children's Research Hospital, Inc. to Evaluate Motixafortide for CD34+ Hematopoietic Stem Cell (HSC) Mobilization For Gene Therapy Applications in Sickle Cell Disease (SCD)
BioLineRx (NASDAQ: BLRX, TASE: BLRX) announced a Phase 1 clinical trial in collaboration with St. Jude Children's Research Hospital to evaluate motixafortide for mobilizing CD34+ hematopoietic stem cells (HSCs) in gene therapies for sickle cell disease (SCD). This study includes experts from St. Jude and two other clinical sites, focusing on new mobilization options due to the limitations of current strategies, like plerixafor, which require multiple cycles. Enrollment is set to start in a few months, and initial data from a related trial at Washington University is expected in late 2024. Motixafortide, BioLineRx's leading candidate, was FDA-approved in September 2023 for HSC mobilization in multiple myeloma patients.
- BioLineRx has initiated a new Phase 1 clinical trial in collaboration with a prestigious institution (St. Jude Children's Research Hospital), enhancing credibility.
- The study addresses a significant need in gene therapy for SCD, potentially increasing market demand for motixafortide.
- Motixafortide was FDA-approved in September 2023 for another indication, showcasing regulatory success.
- The trial includes experienced researchers, likely increasing the study's robustness.
- Enrollment starting soon suggests timely progress.
- There are existing limitations with current HSC mobilization strategies, indicating unknown challenges with motixafortide.
- Relies on future initial data from another trial, which introduces uncertainty.
- The trial is still in Phase 1, indicating a potentially long timeline before commercialization.
- Potential risks if motixafortide fails to show superior efficacy over current mobilization agents like plerixafor.
Insights
The announcement of a clinical trial agreement between BioLineRx and St. Jude Children's Research Hospital to evaluate motixafortide for CD34+ hematopoietic stem cell mobilization in gene therapy applications for sickle cell disease (SCD) holds substantial promise. This partnership leverages the expertise of leading researchers in SCD gene therapy clinical development, which could lead to significant advancements in treatment. Currently, the standard method of HSC mobilization using G-CSF is contraindicated in SCD patients due to potential complications and alternative strategies like plerixafor have limitations. This trial aims to address these gaps, potentially making gene therapy more accessible and effective for SCD patients.
In the short-term, the commencement of this trial could increase BioLineRx's visibility and credibility within the biopharmaceutical and rare disease community. For investors, the collaboration with a reputable institution like St. Jude Children's Research Hospital adds a layer of confidence in the scientific validity and potential clinical success of motixafortide.
In the long-term, successful trial results could lead to expanded indications for motixafortide, beyond its current approval for multiple myeloma. This could significantly enlarge the market size and revenue potential for BioLineRx. However, clinical trials are inherently risky and it is essential to monitor the progress and results carefully.
The initiation of this Phase 1 clinical trial is a strategic move for BioLineRx, particularly given the recent FDA approval of motixafortide for multiple myeloma. Expanding its application in gene therapy for SCD could provide a considerable boost to the company's pipeline and future revenue streams. The involvement of St. Jude Children's Research Hospital, a highly respected entity, further legitimizes the potential of this venture.
From a financial perspective, this trial could positively impact BioLineRx's stock performance. Clinical trials often lead to increased investor interest and potential stock price appreciation, particularly if interim data shows promise. However, investors should stay cautious as clinical trials are capital-intensive and the outcomes uncertain. The initial data from the ongoing trial at Washington University School of Medicine, expected in the second half of 2024, will be particularly important in evaluating the viability of this endeavor.
In summary, while the trial presents exciting opportunities, the financial risks associated with clinical developments should not be overlooked. Careful monitoring of the trial's progress and interim results will be important for making informed investment decisions.
As an oncologist, it is significant that BioLineRx's motixafortide, initially developed for multiple myeloma, is being tested for its efficacy in mobilizing hematopoietic stem cells (HSCs) for gene therapy in sickle cell disease (SCD). This is a critical area of need given the limitations of current HSC mobilization methods. G-CSF, the commonly used drug for HSC collection, is not suitable for SCD patients, making alternative strategies essential.
The potential for motixafortide to offer a reliable and efficient method of mobilizing sufficient quantities of HSCs could revolutionize gene therapy for SCD. This could lead to more successful genetic modifications and transplants, ultimately improving patient outcomes. The involvement of leading researchers and clinical sites further enhances the credibility and potential impact of this trial.
However, it is important to note that this is an early-phase trial. The path from Phase 1 to eventual clinical application is long and fraught with challenges. Therefore, while the scientific and clinical potential is exciting, it remains speculative until more concrete data is available.
- Investigator-initiated study includes leading researchers in SCD gene therapy clinical development from St. Jude Children's Research Hospital, Inc. and two other clinical sites -
- New trial to expand ongoing clinical research of motixafortide for mobilization of HSCs in patients with SCD -
Hematopoietic stem cell transplantation after genetic modification is potentially curative for patients with SCD. Significant quantities of HSCs (minimum 16.5-20 million cells/kg) are required for genetic manipulation and transplant success, however, the most commonly used drug for collection of stem cells, granulocyte colony-stimulating factor (G-CSF), is contraindicated in patients with SCD. Peripheral blood mobilization of stem cells using the mobilization agent plerixafor is the current strategy to collect HSCs for SCD gene therapies, however limitations exist including the need for multiple collection cycles to achieve the necessary HSC yields. For some, gene therapy may be prohibitive by the failure to obtain adequate numbers of HSCs.
"The recent FDA approvals of two gene therapies for sickle cell disease in the
Enrollment in the St. Jude Children's Research Hospital, Inc. study is expected to begin in the next few months. Initial data from the Washington University School of Medicine in
Motixafortide, BioLineRx's lead therapeutic candidate, was approved by the
About SCDSTEMM Clinical Trial with St. Jude Children's Research Hospital Inc.
The SCDSTEMM (Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide) Phase 1 clinical trial is an open-label, multi-center study evaluating the safety, tolerability, and feasibility of single-agent motixafortide (CXCR4 inhibitor) for the mobilization and collection of CD34+ HSCs in 12 patients (aged 18 and older) with SCD. The trial's primary objective is to assess the safety and tolerability of motixafortide in SCD patients, as determined by the incidence of adverse events. Secondary objectives include understanding CD34+ kinetics after motixafortide administration in patients with SCD and determining the number of CD34+ HSCs collected via leukapheresis. The study is designed in two parts: Part A (N=6) will evaluate single dose motixafortide mobilization followed by one apheresis session; Part B (N=6) will evaluate daily motixafortide administration over a two-day mobilization and apheresis regimen. Additional objectives include phenotype and cell function characterization, as well as assessment of the gene modifying potential and senescence of CD34+ cells.
About 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. SCD arises from mutations in the hemoglobin gene, ultimately leading to the production of abnormally shaped (sickle) red blood cells. 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., brain, lungs, heart, kidneys, spleen, liver, bones), ultimately compromising end organ function. The cumulative impact of these complications significantly impacts morbidity and mortality for patients with SCD.
About BioLineRx
BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases. The company's first approved product is APHEXDA® (motixafortide) with an indication in the
Learn more about who we are, what we do, and how we do it at www.biolinerx.com, or on Twitter and LinkedIn.
Forward Looking Statement
Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," and "would," and describe opinions about future events. These include statements regarding management's expectations, beliefs and intentions regarding, among other things, expectations with regard to clinical trials of motixafortide. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials, and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; whether BioLineRx's collaboration partners will be able to execute on collaboration goals in a timely manner; whether the clinical trial results for APHEXDA will be predictive of real-world results; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates, including the degree and pace of market uptake of APHEXDA for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients; whether access to APHEXDA is achieved in a commercially viable manner and whether APHEXDA receives adequate reimbursement from third-party payors; BioLineRx's ability to establish, operationalize and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for and ability to access sufficient additional financing, including any unexpected costs or delays in the commercial launch of APHEXDA; risks related to changes in healthcare laws, rules and regulations in
Contacts:
John Lacey
BioLineRx
IR@biolinerx.com
Moran Meir
LifeSci Advisors, LLC
moran@lifesciadvisors.com
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FAQ
What is the focus of BioLineRx's new clinical trial announced on May 30, 2024?
When is enrollment expected to begin for the new BioLineRx clinical trial?
What are the existing limitations in HSC mobilization for SCD that the new BioLineRx trial aims to address?
When is initial data from the related Washington University trial expected?
What was motixafortide approved for by the FDA in September 2023?
