BioLineRx Announces Oral Presentation on Data from Phase 1 Clinical Trial Evaluating Motixafortide for CD34+ Hematopoietic Stem Cell Mobilization for Gene Therapies in Sickle Cell Disease at ASH 2024
BioLineRx (NASDAQ: BLRX) announced positive initial results from a Phase 1 clinical trial evaluating motixafortide for stem cell mobilization in sickle cell disease (SCD) gene therapies. The study showed that motixafortide, both alone and combined with natalizumab, was safe and well-tolerated, potentially enabling collection of required stem cells in a single apheresis cycle. Motixafortide alone mobilized 198 CD34+ cells/μl median, while the combination achieved 231 CD34+ cells/μl. Notably, patients experienced 2.8-fold greater stem cell mobilization with motixafortide alone and 3.2-fold greater with the combination compared to current standard plerixafor.
BioLineRx (NASDAQ: BLRX) ha annunciato risultati iniziali positivi da uno studio clinico di Fase 1 che valuta motixafortide per la mobilitazione delle cellule staminali nelle terapie geniche per la malattia a cellule falciformi (SCD). Lo studio ha mostrato che il motixafortide, sia da solo che in combinazione con il natalizumab, era sicuro e ben tollerato, potenzialmente permettendo la raccolta delle cellule staminali necessarie in un singolo ciclo di aferesi. Il motixafortide da solo ha mobilizzato una media di 198 cellule CD34+/μl, mentre la combinazione ha raggiunto 231 cellule CD34+/μl. È interessante notare che i pazienti hanno sperimentato una mobilitazione di cellule staminali 2,8 volte maggiore con il motixafortide da solo e 3,2 volte maggiore con la combinazione rispetto allo standard attuale, il plerixafor.
BioLineRx (NASDAQ: BLRX) anunció resultados iniciales positivos de un ensayo clínico de Fase 1 que evalúa motixafortide para la movilización de células madre en terapias génicas para la enfermedad de células falciformes (SCD). El estudio mostró que el motixafortide, tanto solo como combinado con natalizumab, fue seguro y bien tolerado, lo que podría permitir la recolección de las células madre requeridas en un solo ciclo de aféresis. El motixafortide solo movilizó una mediana de 198 células CD34+/μl, mientras que la combinación alcanzó 231 células CD34+/μl. Notablemente, los pacientes experimentaron una movilización de células madre 2,8 veces mayor con el motixafortide solo y 3,2 veces mayor con la combinación en comparación con el estándar actual, plerixafor.
BioLineRx (NASDAQ: BLRX)는 motixafortide를 sickle cell disease (SCD) 유전자 치료를 위한 줄기 세포 동원 시험의 1단계 임상 시험에서 긍정적인 초기 결과를 발표했습니다. 이 연구는 motixafortide가 단독으로 또는 natalizumab과 결합하여 안전하고 잘 견딜 수 있으며, 단일 여과 사이클에서 필요한 줄기 세포를 수집할 수 있게 할 수 있음을 보여주었습니다. motixafortide 단독으로는 198 CD34+ 세포/μl의 중앙값을 동원했으며, 조합은 231 CD34+ 세포/μl에 도달했습니다. 특히, 환자들은 motixafortide 단독으로 2.8배 더 많은 줄기 세포 동원을 경험했으며, 조합으로는 현재의 표준인 plerixafor에 비해 3.2배 더 많았습니다.
BioLineRx (NASDAQ: BLRX) a annoncé des résultats initiaux positifs d'un essai clinique de Phase 1 évaluant motixafortide pour la mobilisation des cellules souches dans les thérapies géniques de la maladie à cellules falciformes (SCD). L'étude a montré que le motixafortide, seul ou combiné avec le natalizumab, était sûr et bien toléré, permettant potentiellement la collecte des cellules souches nécessaires en un seul cycle d'apheres. Le motixafortide seul a mobilisé une médiane de 198 cellules CD34+/μl, tandis que la combinaison a atteint 231 cellules CD34+/μl. Il est à noter que les patients ont connu une mobilisation des cellules souches 2,8 fois plus grande avec le motixafortide seul et 3,2 fois plus grande avec la combinaison par rapport à la norme actuelle, le plerixafor.
BioLineRx (NASDAQ: BLRX) gab positive erste Ergebnisse aus einer Phase-1-Studie bekannt, die motixafortide zur Mobilisierung von Stammzellen in Gen-Therapien bei Sichelzellenanämie (SCD) bewertet. Die Studie zeigte, dass motixafortide, sowohl allein als auch in Kombination mit Natalizumab, sicher und gut verträglich war, was möglicherweise die Sammlung der erforderlichen Stammzellen in einem einzigen Apherese-Zyklus ermöglicht. motixafortide mobilisierte allein median 198 CD34+ Zellen/μl, während die Kombination 231 CD34+ Zellen/μl erreichte. Bemerkenswerterweise erfuhren die Patienten mit motixafortide allein eine 2,8-fache höhere Mobilisierung von Stammzellen und mit der Kombination eine 3,2-fache höhere Mobilisierung im Vergleich zum aktuellen Standard plerixafor.
- Phase 1 trial demonstrated safety and tolerability of motixafortide
- Achieved 2.8-3.2x better stem cell mobilization compared to current standard treatment
- Could enable single-cycle collection vs. multiple attempts with current methods
- Shows potential to improve access to gene therapy for SCD patients
- None.
Insights
The Phase 1 trial results for motixafortide in sickle cell disease (SCD) stem cell mobilization are highly promising. The data shows 2.8-3.2x greater HSC mobilization compared to the current standard plerixafor. Most significantly, patients could potentially collect sufficient stem cells in a single apheresis cycle, versus multiple attempts with current methods.
The safety profile appears favorable with only Grade 1-2 adverse events and notably no vaso-occlusive events - a critical concern in SCD patients. The projected collection of 13.9-18.6x10⁶ HSCs in a single session meets the requirements for both FDA-approved gene therapies (16.5-22 million HSCs).
This could represent a significant advancement in making gene therapy more accessible to SCD patients by simplifying the stem cell collection process and potentially reducing associated costs and complications.
- 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
"Currently available gene therapies for sickle cell disease rely on the collection of significant quantities of CD34+ hematopoietic stem cells, posing challenges for many patients," said Zachary Crees, MD, principal investigator for the trial, Division of Oncology, Washington University School of Medicine. "The findings in this trial suggest that patients with sickle cell disease given motixafortide alone, or in combination with natalizumab, could mobilize and potentially collect the number of stem cells required for approved gene therapies in a single apheresis cycle. These are encouraging findings that we look forward to presenting in greater detail at ASH 2024."
"We are encouraged by the initial findings in this Phase 1 study showing that motixafortide is safe and well-tolerated and may hold potential to improve the overall treatment process and access to gene therapy for more people with SCD," said Philip Serlin, Chief Executive Officer of BioLineRx. "We look forward to continued collaboration with
The Phase 1 safety and feasibility study is evaluating 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. As reported in the abstract, five patients completed mobilization and apheresis with motixafortide alone, and four of five with motixafortide in combination with natalizumab.
Motixafortide alone, and in combination with natalizumab, were safe and well-tolerated in the trial. Common adverse events (AEs) were transient and included Grade 1-2 injection site (pruritis, tingling/pain) and systemic reactions (pruritis, hives). No Grade 4 AEs or vaso-occlusive events occurred.
Motixafortide alone, and in combination with natalizumab, resulted in robust CD34+ HSC mobilization to peripheral blood (PB). Motixafortide alone mobilized a median of 198 CD34+ cells/μl (range 77-690) to PB with median 3.49x10 CD34+ cells/kg as part of a single blood volume collection, projecting the collection of 13.9x106 HSCs in a normal, single-day four blood volume apheresis collection session. Motixafortide in combination with natalizumab mobilized a median of 231 CD34+ cells/μl (range 117-408), with median 4.64x10 CD34+ cells/kg collected as part of a single blood volume collection, projecting the collection of 18.6x106 CD34+ HSCs in a single day four blood volume apheresis collection session.
The two approved gene therapies for sickle cell disease in the
In the trial, patients who underwent prior mobilization with plerixafor, experienced 2.8- fold greater HSC mobilization with motixafortide alone, and 3.2-fold greater HSC mobilization with motixafortide in combination with natalizumab compared to plerixafor.
Oral Presentation at ASH 2024
Oral Presentation Details
Session Name: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Innovations in Mobilization, Collection, and Manufacturing for Cellular Therapies
Title: Motixafortide (CXCR4 Inhibition) Alone and in Combination with Natalizumab (VLA-4 Inhibition) As a Novel Regimen to Mobilize Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease: A First-in-Human, Proof-of-Principle Safety and Feasibility Study
Presenter: Zachary D. Crees, MD, Division of Oncology, Washington University School of Medicine,
Abstract ID#: 193210
Date: Saturday, December 7, 2024
Time: 12:00 PM
Location:
About the Clinical Trial of Motixafortide in Sickle Cell Disease (SCD)
The trial (ClinicalTrials.gov Identifier: NCT05618301) 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 enrolled 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 apheresis; and determining the kinetics of CD34+ HSPC 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 within 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.
About BioLineRx
BioLineRx Ltd. (NASDAQ: BLRX) (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, the potential benefits of APHEXDA, the execution of the launch of APHEXDA and the plans and objectives of management for future operations and expectations and commercial potential of motixafortide, as well as its potential investigational uses. 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
i LYFGENIA Prescribing Information; December 2023.
ii CASGEVY Prescribing Information; December 2023.
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