To learn if giving elranatamab before and after an autologous stem cell transplant (ASTC) can help to control newly diagnosed, high-risk MM. An ASTC is a type of transplant in which a person's own stem cells are collected, preserved, and returned to them.

Start Date30 Jul 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

NCT06414889 / RecruitingEarly Phase 1IIT

Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder

To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.

Start Date20 May 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NCT05343572 / RecruitingEarly Phase 1IIT

Bone Marrow Derived Stem Cells Mobilization for Treatment of Asherman's Syndrome, Atrophic Endometrium, and Recurrent Implantation Failure

This study will assess the use of autologous bone marrow stem cells mobilization using 1,1'-[1,4-phenylenebis-(methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (PLERIXAFOR) as an effective medical therapy for the treatment of Asherman's Syndrome (AS), Atrophic Endometrium (AE) and Recurrent Implantation Failure (RIF).

Start Date01 Nov 2023

Sponsor / Collaborator-

100 Clinical Results associated with Plerixafor

100 Translational Medicine associated with Plerixafor

100 Patents (Medical) associated with Plerixafor

2,185

Literatures (Medical) associated with Plerixafor

01 Mar 2024·Blood cells, molecules & diseases

Effect of voxelotor on murine bone marrow and peripheral blood with hematopoietic progenitor cell mobilization for gene therapy of sickle cell disease

Article

Author: Bao, Weili ; Mendelson, Avital ; Liu, Yunfeng ; Shi, Patricia A

In preparation for hematopoietic stem cell mobilization and collection, current ex vivo gene therapy protocols for sickle cell disease require patients to undergo several months of chronic red cell transfusion. For health care equity, alternatives to red cell transfusion should be available. We examined whether treatment with GBT1118, the murine analog of voxelotor, could be a safe and feasible alternative to red cell transfusion. We found that 3 weeks of treatment with GBT1118 increased the percentage of bone marrow hematopoietic stem cells and upon plerixafor mobilization, the percentage of peripheral blood hematopoietic stem cells. Our data suggest that voxelotor should be further explored for its potential safety and utility as preparation for hematopoietic stem cell mobilization and collection.

01 Feb 2024·Cytotechnology

Study on the mechanism of CXCL12/CXCR4-axis-mediated upregulation of IL-8 and IL-6 on the biological function of acute T lymphocyte leukaemia cells

Article

Author: Chen, Zhe ; Qian, Guanglei ; Zhao, Hui ; Zhou, Liping ; Li, Dong ; Zhang, Chao

Blocking the CXCL12/CXCR4 axis can alter the biological functions of leukaemia cells. We hypothesise that interleukin (IL)-8 and IL-6 play an important role in this process. To test this hypothesis, we established a co-culture model of leukaemia cells and bone marrow stromal cells. Treatment of cells with AMD3100, a CXCR4 antagonist, and G-CSF blocked the CXCL12/CXCR4 axis, inducing biological changes in the leukaemia cells and altering IL-8 and IL-6 levels. Subsequently, after stimulating the CXCL12/CXCR4 axis, specific pathway blockers were employed to assess the role of four candidate signalling pathways in this process. ELISA results confirmed that MG-132 (10 μm) inhibits IL-8 expression and that the NF-κB signalling pathway contributes to this effect. Moreover, treatment with Perifosine, an AKT inhibitor, inhibited IL-6 expression. In addition, changes in the NF-κB signalling pathway inhibited IL-8 expression. Treatment with SP600125, a Jun N-terminal kinase inhibitor, and Perifosine also inhibited IL-8 expression; however, this effect occurred later. IL-6 expression was also lower in the Perifosine group; hence, inhibiting the PI3K/AKT signalling pathway can reduce IL-6 expression. This process requires the participation of multiple signalling pathways to regulate IL-8 and IL-6 expression. Therefore, the associated mechanism is likely to be highly intricate, with potential cross-effects that may impact leukaemia pathogenesis. IL-6 and IL-8 are physiologically regulated by the CXCL12/CXCR4 axis, while the NF-κB and JNK/AP-1 pathways are required for IL-8 expression in T-cell acute lymphoblastic leukaemia. Accordingly, by upregulating IL-8, the bone marrow microenvironment and CXCL12/CXCR4 axis may contribute to T-cell acute lymphoblastic leukaemia pathogenesis.

15 Jan 2024·Internal medicine (Tokyo, Japan)

Hematopoietic Progenitor Cell Counts of the Leukapheresis Product Determined Using Sysmex XN Analyzers Predict a Sufficient Number of CD34<sup>+</sup> Stem Cells in a Peripheral Blood Stem Cell Harvest for Autologous Transplantation

Article

Author: Kurahashi, Shingo ; Imoto, Naoto ; Uchida, Kazuho ; Kondo, Yuka

Objective Several institutions outsource CD34⁺ cell counting of leukapheresis products, limiting rapid measurements, as results are obtained the next day. This problem is compounded with plerixafor use, a stem cell-mobilizing drug that increases leukapheresis efficiency but requires administration the day before leukapheresis. Use of this drug for a second leukapheresis procedure before the first-day leukapheresis CD34⁺ count results are confirmed causes unnecessary leukapheresis and expensive plerixafor administration. We investigated whether or not measuring hematopoietic progenitor cells in leukapheresis products (AP-HPCs) using a Sysmex XN-series analyzer could resolve this problem. Methods We retrospectively compared the absolute AP-HPC value per body weight with the CD34⁺ (AP-CD34⁺) count in 96 first-day leukapheresis product samples obtained between September 2013 and January 2021. Comparisons were also conducted according to regimen: granulocyte colony-stimulating factor (G-CSF) monotherapy, chemotherapy plus G-CSF, or plerixafor mobilization. Results AP-CD34⁺ and AP-HPC counts correlated strongly (r_s=0.846) overall and, in particular, under chemotherapy plus G-CSF (r_s=0.92) but correlated mildly under G-CSF monotherapy (r_s=0.655). AP-HPCs could not completely be dichotomized based on an AP-CD34⁺ threshold of 2×10⁶/kg for any stimulation procedure. In most cases with AP-HPCs >6×10⁶/kg, the AP-CD34⁺ count exceeded 2.0×10⁶/kg, but in 5.7% of these cases, the AP-CD34⁺ count was <2.0×10⁶/kg. A cut-off of AP-HPCs >4.843×10⁶/kg yielded a sensitivity of 71% and specificity of 96% for predicting AP-CD34⁺≥2×10⁶/kg. Conclusion AP-HPCs can identify cases in which sufficient stem cells have been collected.

News (Medical) associated with Plerixafor

30 May 2024

·www.prnewswire.com

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)

- 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 - TEL AVIV, Israel, May 30, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced a multi-center Phase 1 clinical trial sponsored by St. Jude Children's Research Hospital, Inc. to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with sickle cell disease (SCD). Investigators in the trial from St. Jude Children's Research Hospital, Inc. and two other clinical sites have extensive SCD gene therapy clinical development experience and are recognized leaders in the field. 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 U.S. is an exciting development for the sickle cell community," said Ella Sorani, PhD, Chief Development Officer at BioLineRx. "Through this new trial with St. Jude, and our ongoing collaboration with investigators at Washington University School of Medicine in St. Louis, we are excited to be working with leaders in gene therapy and stem cell mobilization to evaluate potential new mobilization options for patients with SCD." 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 St. Louis sponsored clinical trial (ClinicalTrials.gov Identifier: NCT05618301) is anticipated in the second half of 2024. Motixafortide, BioLineRx's lead therapeutic candidate, was approved by the U.S. Food & Drug Administration (FDA) in September 2023, in combination with filgrastim (G-CSF), to mobilize HSCs for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA®. 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 U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside. Learn more about who we are, what we do, and how we do it at , 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 the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; and the impact of the COVID-19 pandemic, the Russian invasion of Ukraine, the declared war by Israel against Hamas and the military campaigns against Hamas and other terrorist organizations, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 26, 2024. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law. Contacts: United States John Lacey BioLineRx [email protected] Israel Moran Meir LifeSci Advisors, LLC [email protected] Logo - SOURCE BioLineRx Ltd.

Phase 1Gene TherapyDrug Approval

06 May 2024

·www.prnewswire.com

BioLineRx Announces Poster Presentation on Economic Model Data for APHEXDA® (motixafortide) as part of CD34+ Hematopoietic Stem Cell Mobilization in Patients with Multiple Myeloma at ISPOR 2024

- Presentation Today, Monday, May 6, 2024 in Atlanta, Georgia - TEL AVIV, Israel, May 6, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that new economic model data will be presented on APHEXDA® (motixafortide) for CD34+ hematopoietic stem cell (HSC) mobilization in patients with multiple myeloma at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) – The Professional Society for Health Economics and Outcomes Research – 2024 conference, taking place May 5-8, 2024, in Atlanta, Georgia. Autologous stem cell transplantation (ASCT) is part of the standard of care treatment paradigm for multiple myeloma and prolongs survival for patients with this cancer type.1 Historically, depending on induction regimens and mobilization strategies, approximately 50% to 75% of patients required more than one apheresis session to collect a target number of cells.2,3 Uncertainty in stem cell mobilization and the possible need for multiple apheresis sessions may result in psychological and logistical burden on patients, in addition to financial and operational inefficiencies for apheresis centers. The new economic model assessed the cost and healthcare resource utilization impacts of multiple apheresis attempts via a comparison between daily filgrastim (G-CSF) alone and in combination with APHEXDA, and an indirect comparison between daily filgrastim used with either plerixafor or APHEXDA, using drug costs from Micromedex, procedure costs from CMS.gov and data for apheresis days obtained from clinical trials and product labels. The data are expected to be published in Value in Health, Volume 27, Issue 6, S1 (June 2024.) Poster Presentation at ISPOR 2024 Georgia World Congress Center, Atlanta, Georgia Poster Session Details Poster: Number EE148 Title: The Institutional Level Impact of Additional Apheresis Days for Multiple Myeloma Patients Undergoing Autologous Stem Cell Transplantation on Costs and Healthcare Resource Utilization Presenter: Jeffrey R Skaar, PhD, Trinity Life Sciences, New York, NY and Jennifer L Lessor, MPH, BioLineRx USA, Inc., Waltham, MA Poster Session: Economic Evaluation Date: Monday, May 6, 2024 Time: 3:30 PM - 6:30 PM The new economic model data to be presented at ISPOR 2024 builds on the evaluation of APHEXDA for CD34+ HSC mobilization in patients with multiple myeloma in apheresis center operations. About the GENESIS Trial GENESIS (NCT 03246529) is a 2-part, Phase-3, randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of APHEXDA (motixafortide) plus filgrastim (G-CSF), compared to placebo plus filgrastim, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. Part 1 was a single center, lead-in, open-label study involving 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. Part 2 involved 122 patients who were randomized 2:1 in a double-blind, placebo-controlled, multicenter study. The primary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in one apheresis session. About Multiple Myeloma Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow. According to the American Cancer Society, in 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and nearly 13,000 people will die from the disease in the U.S.4 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections. About APHEXDA® APHEXDA (motixafortide) is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours) that, in combination with filgrastim (G-CSF), enables mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation in patients with multiple myeloma.5 INDICATION AND IMPORTANT SAFETY INFORMATION INDICATION APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS APHEXDA is contraindicated in patients with a history of serious hypersensitivity reactions to motixafortide. WARNINGS AND PRECAUTIONS Anaphylactic Shock and Hypersensitivity Reactions: Anaphylactic shock and hypersensitivity reactions have occurred. Premedicate all patients with a triple drug premedication regimen that includes an H1-antihistamine, an H2 blocker, and a leukotriene inhibitor approximately 30-60 minutes prior to each dose of APHEXDA. Administer APHEXDA in a setting where personnel and therapies are immediately available for treatment of anaphylaxis and other systemic reactions. Monitor patients for 1 hour following APHEXDA administration and manage reactions promptly. Patients receiving negative chronotropic drugs (e.g., beta-blockers) may be more at risk for hypotension in the event of a hypersensitivity reaction and these drugs, when appropriate, should be replaced with non-chronotropic drugs. Injection Site Reactions: Injection site reactions (73%) including pain (53%), erythema (27%), and pruritus (24%) have occurred. Severe reactions occurred in 9% of patients. Premedicate with an analgesic premedication (e.g., acetaminophen) prior to each APHEXDA dose. Use analgesic medication and local treatments post-dose, as needed. Tumor Cell Mobilization in Patients with Leukemia: For the purpose of hematopoietic stem cell (HSC) mobilization, APHEXDA may cause mobilization of leukemic cells and subsequent contamination of the apheresis product. Therefore, APHEXDA is not intended for HSC mobilization and harvest in patients with leukemia. Leukocytosis: Administering APHEXDA in conjunction with filgrastim increases circulating leukocytes as well as HSC populations. Monitor white blood cell counts during APHEXDA use. Potential for Tumor Cell Mobilization: When APHEXDA is used in combination with filgrastim for HSC mobilization, tumor cells may be released from the marrow and subsequently collected in the leukapheresis product. The effect of potential reinfusion of tumor cells has not been well-studied. Embryo-fetal Toxicity: Based on its mechanism of action, APHEXDA can cause fetal harm. Advise pregnant women of the potential risk to the fetus. Verify pregnancy status in females of reproductive potential prior to initiating treatment with APHEXDA and advise use of effective contraception during treatment and for 8 days after the final dose. ADVERSE REACTIONS The most common adverse reactions (incidence >20%) in patients treated with APHEXDA were injection site reactions [73%, including pain (53%), erythema (27%), pruritus (24%)]; pruritus (38%); flushing (33%); back pain (21%). USE IN SPECIFIC POPULATIONS Pregnancy: Please see the important information in Warnings and Precautions under Embryo-fetal Toxicity. Lactation: There are no data on the presence of motixafortide in human milk, the effects on the breastfed child, or the effects on milk production. Advise females that breastfeeding is not recommended during treatment with APHEXDA and for 8 days after the final dose. Pediatric Use: The safety and effectiveness of APHEXDA have not been established in pediatric patients. Please see the accompanying full Prescribing Information. 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 U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside. Learn more about who we are, what we do, and how we do it at , 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 the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; the impact of any resurgence of the COVID-19 pandemic, the Russian invasion of Ukraine, the declared war by Israel against Hamas and the military campaigns against Hamas and other terrorist organizations, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 26, 2024. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law. Kumar SK, et al. Blood. 2008;111(5):2516-2520. Dhakal B, Zhang M, Burns L, et al. Haematologica. 2023;106(8):2257-2260. Ahmed N, Li L, Rojas P, et al. Bone Marrow Transplant. 2021;56(6):1458-1461. American Cancer Society. Key Statistics About Multiple Myeloma. Atlanta, Ga: American Cancer Society; 2024. APHEXDA. Prescribing Information. BioLineRx Ltd; 2023. Contacts: United States John Lacey BioLineRx [email protected] Israel Moran Meir LifeSci Advisors, LLC [email protected] SOURCE BioLineRx

Phase 3Drug ApprovalClinical ResultNDA

17 Apr 2024

·www.prnewswire.com

BioLineRx Announces Poster Presentation on Apheresis Center Efficiency and CXCR4 Antagonists including APHEXDA® (motixafortide) in Patients with Multiple Myeloma at the ASFA 2024 Annual Meeting

TEL AVIV, Israel, April 17, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced a poster presentation on apheresis center efficiency and CXCR4 antagonists including APHEXDA (motixafortide) in patients with multiple myeloma. The poster will be presented at the American Society for Apheresis (ASFA) 2024 Annual Meeting, taking place April 17-19, 2024, in Las Vegas, Nevada. Autologous stem cell transplantation (ASCT) is part of the standard of care treatment paradigm for multiple myeloma and prolongs survival for patients with this cancer type.1 Historically, depending on induction regimens and mobilization strategies, approximately 50% to 75% of patients required more than one apheresis session to collect a target number of cells.2,3 The model in the poster at ASFA analyzed the number of apheresis days needed to collect ≥6 million CD34+ cells/kg using different mobilization regimens based on product-specific Phase 3 studies. A direct comparison was used between daily filgrastim alone and in combination with APHEXDA based on the Phase 3 GENESIS trial that supported the U.S. Food and Drug Administration (FDA) approval of APHEXDA. In the absence of head-to-head Phase 3 studies, an indirect comparison was made between daily filgrastim, plerixafor in combination with filgrastim, and APHEXDA in combination with filgrastim. The calculations were based on data from the MOZOBIL® (plerixafor) US Prescribing Information and local laboratory assessments in the GENESIS trial.4 "Variability in the time to mobilize sufficient stem cells for ASCT is a significant operational challenge for apheresis centers that can cause suboptimal experiences for patients, as well as delays in care and cost impact," said Edmund K. Waller, MD, PhD, FACP, Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University. "Research of this type supports clinical and institutional decision making and we look forward to presenting the model at the poster session at ASFA." Poster Presentation at the ASFA 2024 Annual Meeting The Resorts World, Las Vegas, NV Poster Session Details Poster: Number P-28. See abstract in Journal of Clinical Apheresis. Title: Enhancing Apheresis Center Efficiency with CXCR4 Antagonists: Evidence from the Phase 3 Trials Authors: Edmund K. Waller, MD, PhD, FACP, Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, GA Date: April 17-19, 2024 About Multiple Myeloma Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow. According to the American Cancer Society, in 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and nearly 13,000 people will die from the disease in the U.S.5 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections. About the GENESIS Trial GENESIS (NCT 03246529) is a 2-part, Phase-3, randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of APHEXDA (motixafortide) plus filgrastim (G-CSF), compared to placebo plus filgrastim, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. Part 1 was a single center, lead-in, open-label study involving 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. Part 2 involved 122 patients who were randomized 2:1 in a double-blind, placebo-controlled, multicenter study. The primary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in one apheresis session. The study showed that APHEXDA combined with filgrastim (G-CSF) significantly enhanced the rate of mobilizing ≥6 × 106 CD34+ cells/kg in up to 2 apheresis days compared to placebo + filgrastim. Central laboratory assessments were used for the efficacy results. Local laboratory results were used for clinical treatment decisions. About APHEXDA® APHEXDA (motixafortide) is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours) that, in combination with filgrastim (G-CSF), enables mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation in patients with multiple myeloma.6 INDICATION AND IMPORTANT SAFETY INFORMATION INDICATION APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS APHEXDA is contraindicated in patients with a history of serious hypersensitivity reactions to motixafortide. WARNINGS AND PRECAUTIONS Anaphylactic Shock and Hypersensitivity Reactions: Anaphylactic shock and hypersensitivity reactions have occurred. Premedicate all patients with a triple drug premedication regimen that includes an H1-antihistamine, an H2 blocker, and a leukotriene inhibitor approximately 30-60 minutes prior to each dose of APHEXDA. Administer APHEXDA in a setting where personnel and therapies are immediately available for treatment of anaphylaxis and other systemic reactions. Monitor patients for 1 hour following APHEXDA administration and manage reactions promptly. Patients receiving negative chronotropic drugs (e.g., beta-blockers) may be more at risk for hypotension in the event of a hypersensitivity reaction and these drugs, when appropriate, should be replaced with non-chronotropic drugs. Injection Site Reactions: Injection site reactions (73%) including pain (53%), erythema (27%), and pruritus (24%) have occurred. Severe reactions occurred in 9% of patients. Premedicate with an analgesic premedication (e.g., acetaminophen) prior to each APHEXDA dose. Use analgesic medication and local treatments post-dose, as needed. Tumor Cell Mobilization in Patients with Leukemia: For the purpose of hematopoietic stem cell (HSC) mobilization, APHEXDA may cause mobilization of leukemic cells and subsequent contamination of the apheresis product. Therefore, APHEXDA is not intended for HSC mobilization and harvest in patients with leukemia. Leukocytosis: Administering APHEXDA in conjunction with filgrastim increases circulating leukocytes as well as HSC populations. Monitor white blood cell counts during APHEXDA use. Potential for Tumor Cell Mobilization: When APHEXDA is used in combination with filgrastim for HSC mobilization, tumor cells may be released from the marrow and subsequently collected in the leukapheresis product. The effect of potential reinfusion of tumor cells has not been well-studied. Embryo-fetal Toxicity: Based on its mechanism of action, APHEXDA can cause fetal harm. Advise pregnant women of the potential risk to the fetus. Verify pregnancy status in females of reproductive potential prior to initiating treatment with APHEXDA and advise use of effective contraception during treatment and for 8 days after the final dose. ADVERSE REACTIONS The most common adverse reactions (incidence >20%) in patients treated with APHEXDA were injection site reactions [73%, including pain (53%), erythema (27%), pruritus (24%)]; pruritus (38%); flushing (33%); back pain (21%). USE IN SPECIFIC POPULATIONS Pregnancy: Please see the important information in Warnings and Precautions under Embryo-fetal Toxicity. Lactation: There are no data on the presence of motixafortide in human milk, the effects on the breastfed child, or the effects on milk production. Advise females that breastfeeding is not recommended during treatment with APHEXDA and for 8 days after the final dose. Pediatric Use: The safety and effectiveness of APHEXDA have not been established in pediatric patients. Please see the accompanying full Prescribing Information. 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 U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside. Learn more about who we are, what we do, and how we do it at , 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 the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; the impact of any resurgence of the COVID-19 pandemic, the Russian invasion of Ukraine, the declared war by Israel against Hamas and the military campaigns against Hamas and other terrorist organizations, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 26, 2024. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law. Kumar SK, et al. Blood. 2008;111(5):2516-2520. Dhakal B, Zhang M, Burns L, et al. Haematologica. 2023;106(8):2257-2260. Ahmed N, Li L, Rojas P, et al. Bone Marrow Transplant. 2021;56(6):1458-1461. Data on file. American Cancer Society. Key Statistics About Multiple Myeloma. Atlanta, Ga: American Cancer Society; 2024. APHEXDA. Prescribing Information. BioLineRx Ltd; 2023. ©BioLineRx USA, Inc. 2024. All Rights Reserved. APHEXDA® is a registered trademark of BioLineRx Ltd. All names and trademarks are property of their respective owners. SOURCE BioLineRx

Phase 3Clinical ResultDrug ApprovalASH

100 Deals associated with Plerixafor

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KEGG	Wiki	ATC	Drug Bank
D08971	Plerixafor	L03AX16	DB06809

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Peripheral Stem Cell Transplantation	FR	Seacross Pharmaceuticals Ltd	28 Feb 2023
Malignant Solid Neoplasm	EU	Accord Healthcare SLU	16 Dec 2022
Malignant Solid Neoplasm	IS	Accord Healthcare SLU	16 Dec 2022
Malignant Solid Neoplasm	LI	Accord Healthcare SLU	16 Dec 2022
Malignant Solid Neoplasm	NO	Accord Healthcare SLU	16 Dec 2022
Hematopoietic stem cell transplantation	JP	Sanofi KK	19 Dec 2016
Stem cell mobilisation	CA	Sanofi	26 Mar 2012
Lymphoma	EU	Sanofi	30 Jul 2009
Lymphoma	IS	Sanofi	30 Jul 2009
Lymphoma	LI	Sanofi	30 Jul 2009
Lymphoma	NO	Sanofi	30 Jul 2009
Multiple Myeloma	US	Genzyme Corp.	15 Dec 2008
Non-Hodgkin Lymphoma	US	Genzyme Corp.	15 Dec 2008

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hodgkin's Lymphoma	Phase 3	FR	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	DE	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	IT	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	NL	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	ES	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	SE	Genzyme Corp.	01 Sep 2008
Hodgkin's Lymphoma	Phase 3	GB	Genzyme Corp.	01 Sep 2008
COVID-19	Phase 2	BG	4Living Biotech	19 Jul 2022
COVID-19	Phase 2	FR	4Living Biotech	19 Jul 2022
Respiratory Distress Syndrome, Acute	Phase 2	BG	4Living Biotech	19 Jul 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03746080 (Phase II study of plerixafor combined with whole brain radiation therapy (WBRT) for patients with newly diagnosed glioblastoma, ASCO2024)	Phase 2	Glioblastoma IDH wildtype \| pMGMT unmethylated	17	Plerixafor + WBRT	roherqmsqx(jnvjmludhz) = lnqmpmvjjh ifkgoerqpw (idbiphmdfj ) View more	Negative	24 May 2024
EHA2024	Not Applicable	Hematologic Neoplasms	-	Intermediate Dose Cytarabine + G-CSF	hvrivfptrz(eqxjotztka) = 5% (2) of patients in the ID-AraC group vbmgfrwcuz (khbziuefvo ) View more	Positive	14 May 2024
EHA2024	Not Applicable	Hematologic Neoplasms	-	Plerixafor + G-CSF		Positive	14 May 2024
EHA2024	Not Applicable	Multiple Myeloma \| Lymphoma CD34-positive	33	plerixafor	ihzakbfkkt(odikigloxn) = grade I-II diarrhea and myalgias (15%) olsgwkrcev (wubnentkuy )	Positive	14 May 2024
NA (EHA2024)	Not Applicable	acute leukemia CD34+	8	Plerixafor	woobtkjoyu(ceqckbebgv) = sktpuzjrbt icyorxsuaj (kwkvpwesbz ) View more	Positive	14 May 2024
NCT04177810 (CTgov)	Phase 2	Secondary malignant neoplasm of pancreas	25	Cemiplimab+Plerixafor	iujflbamdm(eivendjssi) = itvgiydkdp ajodbgvlrn (ebasunjkaj, zqzepfutzl - mtbhmequje) View more	-	20 Feb 2024
NCT01696461 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Hodgkin's Lymphoma \| Acute Lymphoblastic Leukemia ... View more	127	Plerixafor	sfybijxizh(gslvlzowoz) = qjfamduzfd crofcrthuq (dzuqiigklz, dcgfaqayvo - jrbocvvlch) View more	-	14 Sep 2023
EHA2023	Not Applicable	Hematologic Neoplasms	85	Intermediate-dose AraC+G-CSF	coodmmygmm(mkqltidfqv) = ocvymbbnjn kzifhudgbq (ttpctfulpl )	-	08 Jun 2023
EHA2023	Not Applicable	Hematologic Neoplasms	85	Cy+G-CSF	coodmmygmm(mkqltidfqv) = dahdhcxikz kzifhudgbq (ttpctfulpl )	-	08 Jun 2023
P329 (EBMT2023)	Not Applicable	-	-	Plerixafor + G-CSF	nwwuvxzpyc(jmcdiovdqi) = bbldiolyzu tquyrxnhcm (lpqnjdptqc ) View more	-	23 Apr 2023
EBMT2023	Not Applicable	Bone Marrow Neoplasms \| Lymphoma	80	Plerixafor	tujkfqpean(bkpxxdauiy) = exyuacvtai cozqcpydwp (pzqfrgctpo ) View more	-	23 Apr 2023
EBMT2023	Not Applicable	Bone Marrow Neoplasms \| Lymphoma	80	G-CSF	tujkfqpean(bkpxxdauiy) = aqlzqpopzo cozqcpydwp (pzqfrgctpo ) View more	-	23 Apr 2023
P689 (EBMT2023)	Not Applicable	-	13	Plerixafor 0.24 mg/kg	tmzhfxbajv(ldruzduokn) = Plerixafor was well tolerated by the donor in all cases and no side effects or adverse reactions occurred akqkgzncfk (hydcvfyrub )	-	23 Apr 2023
P689 (EBMT2023)	Not Applicable	-	13	G-CSF 10 mcg/kg		-	23 Apr 2023

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