A prospective, multicenter study of an etoposide plus G-CSF followed by peripheral blood CD34+ cell count-adapted plerixafor mobilization in transplant-eligible patients with multiple myeloma who have received daratumumab and/or lenalidomide containing-induction therapy: Catholic Research Network for Multiple Myeloma Study

Start Date01 Jan 2025

Sponsor / Collaborator-

NCT06506461 / Not yet recruitingPhase 1IIT

St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1

This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD.
Primary Objective
* To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
Secondary Objective
* To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.

Start Date01 Jan 2025

Sponsor / Collaborator

St. Jude Children's Research Hospital, Inc.

NCT06207799 / RecruitingPhase 2IIT

Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma

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 Date17 Jul 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

100 Clinical Results associated with Plerixafor

100 Translational Medicine associated with Plerixafor

100 Patents (Medical) associated with Plerixafor

2,167

Literatures (Medical) associated with Plerixafor

01 Jan 2025·PEDIATRIC BLOOD & CANCER

Use of plerixafor for short‐term management of leukopenia in a pediatric patient with WHIM syndrome

Letter

Author: Boucher, Maria ; Kasow, Kimberly A. ; Bies, Joshua J. ; Caceres‐Nazario, Beatriz ; Keene, Staci

31 Dec 2024·Annals of medicine

G-CSF + plerixafor versus G-CSF alone mobilized hematopoietic stem cells in patients with multiple myeloma and lymphoma: a systematic review and meta-analysis

Review

Author: Zhou, Ruixi ; Li, Yuyao ; Qiu, Xia ; Lei, Yupeng

AIM:

The combination of granulocyte-colony stimulating factor (G-CSF) and plerixafor is one of the approaches for hematopoietic stem cell mobilization in patients with multiple myeloma (MM), non-Hodgkin's lymphoma (NHL), and Hodgkin's lymphoma (HL). This systematic review and meta-analysis aimed to determine the ability of G-CSF + plerixafor to mobilize peripheral blood (PB) CD34+ cells and examine its safety profile.

METHODS:

We performed a database search using the terms 'granulocyte colony stimulating factor', 'G-CSF', 'AMD3100', and 'plerixafor', published up to May 1, 2023. The methodology is described in further detail in the PROSPERO database (CRD42023425760).

RESULTS:

Twenty-three studies were included in this systematic review and meta-analysis. G-CSF + plerixafor resulted in more patients achieving the predetermined apheresis yield of CD34+ cells than G-CSF alone (OR, 5.33; 95%, 4.34-6.55). It was further discovered that G-CSF + plerixafor could mobilize more CD34+ cells into PB, which was beneficial for the next transplantation in both randomized controlled (MD, 18.30; 95%, 8.74-27.85) and single-arm (MD, 20.67; 95%, 14.34-27.00) trials. Furthermore, G-CSF + plerixafor did not cause more treatment emergent adverse events than G-CSF alone (OR, 1.25; 95%, 0.87-1.80).

CONCLUSIONS:

This study suggests that the combination of G-CSF and plerixafor, resulted in more patients with MM, NHL, and HL, achieving the predetermined apheresis yield of CD34+ cells, which is related to the more effective mobilization of CD34+ cells into PB.

01 Dec 2024·TRANSFUSION

Comparison of the efficacy of a generic plerixafor versus Mozobil as adjunct peripheral blood stem cell mobilization agents in multiple myeloma patients

Article

Author: Yuan, Shan ; Kim, Hoim ; Wang, Shirong ; Chang, Shelley

Abstract:

Background:

Plerixafor is an adjunct peripheral blood stem cell (PBSC) mobilization agent with well‐demonstrated safety and efficacy. The routine use of the originator brand drug (Mozobil) has been limited by cost. This retrospective study was conducted to compare the mobilization efficacy of a lower‐cost generic plerixafor and Mozobil in multiple myeloma (MM) patients.

Study Design and Methods:

The study included two near‐concurrent cohorts of MM patients mobilized with brand (n = 64) or generic (n = 61) plerixafor in addition to filgrastim. Collection and early engraftment outcomes were compared.

Results:

The two cohorts had comparable distributions of sex, age, and weight. Previous treatment histories and proportions of upfront versus just‐in‐time plerixafor use were similar. There was no significant difference in their median overall cumulative total yield (106 CD34+ cells/kg) (brand, 5.91; generic, 5.80; p = .51). However, the generic cohort had a significantly higher median yield after the first dose (4.79 vs. 3.78, p = .03), and consequently lower median numbers of plerixafor doses (p = .001) and collection days (p = .002). Only 31.1% of patients in the generic arm required more than one dose versus 59.4% of patients in the brand arm (p = .006).All transplanted patients in the brand and generic arms (90.6% and 85.2% respectively, p = .42) achieved engraftment. There was no significant difference in their median times to platelet and neutrophil engraftment, nor their transfusion requirements during the first 30 days post‐transplant.

Conclusion:

The generic plerixafor produced comparable cumulative collection yields and early engraftment outcomes as Mozobil, but fewer doses and collection days were needed to reach collection goal.

News (Medical) associated with Plerixafor

25 Nov 2024

·www.prnewswire.com

BioLineRx Reports Third Quarter 2024 Financial Results and Provides Update on Transformation to Drive Shareholder Value

- Executed license agreement with Ayrmid Ltd. for APHEXDA® (motixafortide) for $10 million upfront, up to $87 million in commercial milestones, and 18-23% tiered royalties on sales - - Received $9 million equity investment from certain funds managed by Highbridge Capital Management, LLC - - Entered into agreement to reduce and restructure long-term debt by ~$16.5 million - - Annual operational expenses expected to decline by over 70% following out-license of APHEXDA® (motixafortide) commercial program to Ayrmid - - Company to continue to support motixafortide PDAC program while evaluating additional assets for development in rare diseases and oncology - - Management to host conference call today, November 25, at 8:30 am EDT - TEL AVIV, Israel, Nov. 25, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ/TASE: BLRX), a development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today reported its unaudited financial results for the third quarter ended September 30, 2024, and provided updates on strategic actions designed to drive shareholder value. "The license agreement for APHEXDA that we announced last week was made possible by the tremendous work of our commercial team, who through their hard work proved the significant value that APHEXDA can bring to transplant centers and patients," said Philip Serlin, Chief Executive Officer of BioLineRx. "Our launch progress attracted Ayrmid, who will now, through Gamida Cell, continue to build on the strong commercial foundation that has been laid. We would like to thank our employees for their outstanding contributions to APHEXDA growth and expect this innovative product to reach even more patients with the additional resources from Ayrmid. "Looking forward, our streamlined and nimble company has a new financial foundation supported by sales royalties and potential milestone payments, which will allow our experienced team to develop important new therapies in rare disease and oncology that address areas with high unmet need. We will also focus on advancing our motixafortide PDAC program through existing collaborations that require de-minimis investment. Through this strategy, we anticipate delivering near- and long-term value for our shareholders," Mr. Serlin concluded. Corporate Updates Executed license agreement with Ayrmid Ltd. to develop and commercialize APHEXDA® (motixafortide) in all indications except solid tumors, and across all territories except Asia License agreement included a $10 million upfront payment, up to $87 million in potential commercial milestones, and royalties on net sales ranging from 18% to 23% BioLineRx will supply motixafortide on a cost-plus basis, for both commercial and development supply Certain members of the BioLineRx U.S.-based commercial organization will be transitioned to Ayrmid Pharma Ltd. Received $9 million equity investment from certain funds managed by Highbridge Capital Management, LLC, to support BioLineRx's pipeline expansion Operating expense run-rate expected to decrease by more than 70% beginning January 1, 2025 through APHEXDA commercial program transfer and additional headcount reductions Company intends to evaluate additional asset opportunities in 2025, with a focus on early-stage clinical programs in oncology or rare diseases that address major areas of unmet need Financial Updates Executed repayment and restructuring agreement with BlackRock EMEA Venture and Growth Lending to repay $16.5 million of approximately $29 million in total debt due; remaining balance will be paid over the next three years at the existing fixed annual interest rate of 9.5 percent As of September 30, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $29.2 million Following the out-license to Ayrmid, the equity investment from Highbridge and the debt repayment to Blackrock, the Company's cash, cash equivalents and short-term bank deposits are expected to be approximately $20 million, which management believes will be sufficient to fund operations into 2026, as currently planned APHEXDA Launch Updates Aphexda achieved 10 percent market share milestone of total CXCR4 inhibitor usage in the U.S., which compares APHEXDA to branded MOZOBIL and generic plerixafor in all indications Institutions ordering APHEXDA increased by 40 percent in the third quarter Clinical Portfolio Updates Motixafortide Pancreatic Ductal Adenocarcinoma (mPDAC) Continued enrollment in the CheMo4METPANC Phase 2b clinical trial collaboration with Columbia University. In addition to Columbia, patient enrollment has begun at Brown University, and three additional sites are anticipated to begin enrollment over the next two quarters. Full enrollment in the randomized trial targeting 108 patients is anticipated in 2027, with a prespecified interim futility analysis planned when 40% of PFS events are observed Multiple Myeloma Collaboration partner Gloria Biosciences' stem cell mobilization bridging study IND was filed and approved by the Center for Drug Evaluation of the National Medical Products Administration in China. Anticipate initiation of pivotal clinical trial in 1H 2025 Gloria Biosciences has received regulatory approval to commercialize APHEXDA in the Boao Region of China and Macao, areas in Asia that do not require a bridging study Sickle Cell Disease (SCD) & Gene Therapy Announced oral presentation at ASH 2024 on initial results from a Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD). Sponsored by investigators at Washington University in St. Louis, the findings from this proof-of-concept study 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. The presentation will occur at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition taking place December 7-10, 2024, in San Diego, California Third Quarter 2024 Financial Results Total revenue for the three months ended September 30, 2024 was $4.9 million. The Company did not record any revenue during the third quarter of 2023. Revenue for the quarter reflects a portion of the upfront payment from the Gloria Biosciences license, which amounted to $3.2 million, as well as $1.7 million of net revenue from product sales of APHEXDA in the U.S. Cost of revenue for the three months ended September 30, 2024 was $0.8 million. The Company did not record any cost of revenue during the third quarter of 2023. Cost of revenue for the quarter primarily reflects the amortization of intangible assets, royalties on net product sales of APHEXDA in the U.S., and cost of goods sold on product sales Research and development expenses for the three months ended September 30, 2024 were $2.6 million, compared to $2.7 million for the same period in 2023. The decrease resulted primarily from lower expenses related to the termination of the development of AGI-134 and a decrease in payroll and share-based compensation Sales and marketing expenses for the three months ended September 30, 2024 were $5.5 million, compared to $8.1 million for the same period in 2023. The decrease resulted primarily from lower expenses of commercialization activities related to motixafortide. The higher expenses in the corresponding period of 2023 reflect the ramp-up of pre-commercialization activities related to motixafortide General and administrative expenses for the three months ended September 30, 2024 were $1.4 million, compared to $1.5 million for the same period in 2023. The decrease resulted primarily from small decreases in a number of G&A expenses Net loss for the three months ended September 30, 2024 was $5.8 million, compared to net loss of $16.0 million for the same period in 2023. The net loss for the 2024 period included $0.8 million in non-operating income, compared to non-operating expenses of $3.1 million for the same period in 2023, both primarily related to non-cash revaluation of warrants As of September 30, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $29.2 million. Third Quarter Results Conference Call and Webcast BioLineRx will report its third quarter 2024 results on November 25, 2024. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company's website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until November 27, 2024; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally. About BioLineRx BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a 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, which is being developed and commercialized by Ayrmid Ltd. (globally, excluding Asia) and Gloria Biosciences (in Asia). BioLineRx is utilizing its end-to-end expertise in development, regulatory affairs, manufacturing and commercialization to advance its innovative pipeline and ensure 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.  Cautionary Note Regarding Forward-Looking Statements (BioLineRx) 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 success of the license agreement with Ayrmid, expectations with regard to clinical trials of motixafortide, statements relating to the equity investment offering, including as to the consummation of the offering described above, the expected gross proceeds therefrom and the timing of the closings of the offering and the license agreement. 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] SOURCE BioLineRx Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1License out/inDrug ApprovalFinancial StatementPhase 2

05 Nov 2024

·www.businesswire.com

Ensoma Unveils Three Programs Leveraging In Vivo Hematopoietic Stem Cell Engineering Platform to Target Genetic, Immune and Oncological Diseases

BOSTON--( BUSINESS WIRE )--Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo treatments capable of precisely and durably engineering the hematopoietic system, today unveiled its three lead programs targeting genetic, immune and oncological diseases. These innovative programs leverage Ensoma’s in vivo hematopoietic stem cell (HSC) engineering platform and aim to address critical unmet needs in chronic granulomatous disease (CGD), sickle cell disease (SCD) and solid tumors. Ensoma’s in vivo HSC engineering approach has the potential to offer greatly improved patient access to therapy and a vastly reduced treatment burden. Ensoma’s in vivo HSC engineering platform combines an off-the-shelf delivery system with an advanced gene engineering toolkit that, together, offer durable and transformative therapies across a range of diseases. The delivery system is based on virus-like particles (VLPs) that preferentially bind to HSCs, efficiently deliver DNA to the nucleus and de-target the liver. With a 35-kilobase cargo capacity, these VLPs carry a diverse range of sophisticated genomic engineering tools capable of precise changes, from single base edits up to large multi-gene insertions. Importantly, the platform leverages HSCs as a self-renewing reservoir of engineered cells, providing durable therapies with improved patient access and outcomes across genetic and immune disorders, as well as oncology. Ensoma’s lead program is for individuals living with X-linked CGD (X-CGD), a condition that severely compromises immune function and leaves patients vulnerable to life-threatening infections. X-CGD is the most common form of the condition, affecting 60-70% of CGD patients. “Science and medicine have shown the curative impact of HSC-based therapies through bone marrow transplantation and ex vivo HSC-targeted gene therapy, but access to these treatments is limited and comes with significant burden for the patient and the healthcare system,” said Jim Burns, CEO of Ensoma. “At Ensoma, we believe the time for in vivo HSC engineering has come. Our proof-of-concept data in X-CGD demonstrate our in vivo approach for CGD and position us for an IND filing in the first half of 2025, which would make it the first in vivo HSC-directed therapy to be tested in humans. We are following CGD with preclinical programs in sickle cell disease and solid tumors, underscoring our dedication to advancing the future of medicine through precision in vivo treatment.” Ensoma will present key preclinical data at the Society for Immunotherapy of Cancer (SITC) 39th Annual Meeting and the 66 th American Society of Hematology (ASH) Annual Meeting, highlighting the potential of its in vivo HSC engineering platform in advancing treatments for CGD, SCD and solid tumors. Below is a summary of the findings and presentation details. Chronic Granulomatous Disease: Key Findings from ASH: Preclinical data to be presented at the ASH Annual Meeting will highlight proof-of-concept for Ensoma's innovative in vivo gene therapy, EN-374, designed to treat X-CGD. X-CGD is caused by a mutant CYBB gene that prevents white blood cells called neutrophils from producing functional NADPH oxidase, a protein important for fighting bacterial and fungal infections. Ensoma’s novel approach employs VLPs to efficiently engineer HSCs for sustained expression of a CYBB transgene in neutrophils, restoring function of the NADPH oxidase enzyme complex critical for immune defense. The study demonstrated therapeutic restoration of CYBB protein expression and NADPH oxidase activity in murine circulating neutrophils, achieving levels shown to confer meaningful clinical benefits for X-CGD patients. This research positions EN-374 as a pioneering therapy that addresses an unmet medical need and offers the potential to expand access to HSC-directed gene therapies for genetic, immune and oncological disorders. Presentation Details: Title : In Vivo Hematopoietic Stem Cell Engineering Restores the Function of NADPH Enzyme Complex in X-Linked Chronic Granulomatous Disease Model in Mice Abstract: 801 Poster Presentation Time/Date: 5:30-7:30 p.m. PT, Saturday, Dec. 7 Location: San Diego Convention Center, Halls G-H Presenter: Sravya Kattula, Ph.D., Ensoma Sickle Cell Disease: Key Findings from ASH: At the ASH Annual Meeting, Ensoma will also showcase compelling preclinical data demonstrating the safety and efficacy of its wholly owned single-dose truncated Gro-Beta (tGROβ), EN-145 (formerly MGTA-145), combined with plerixafor for mobilizing primitive HSCs in mouse models of SCD. This innovative approach addresses the limitations of current mobilization regimens, particularly the use of granulocyte colony-stimulating factor (G-CSF), which is contraindicated in SCD patients. In a SCD mouse model, the combination of EN-145 and plerixafor mobilized six-fold more long-term HSCs than plerixafor alone, significantly enhancing HSC yield for gene therapies. Studies in humanized NBSGW mice further demonstrated rapid and effective mobilization of human CD34+ cells, along with superior transduction efficiency—meaning that Ensoma’s VLPs were more effective at delivering the genetic material into the target cells, ensuring a higher percentage of modified cells with therapeutic potential. These findings highlight EN-145's potential use in a robust alternative HSC mobilization regimen in SCD, paving the way for improved outcomes in both ex vivo and in vivo gene therapies. Presentation Details: Title: Single Dose of tGROβ (EN-145)/Plerixafor Safely and Effectively Mobilizes Primitive HSCs in Mouse Models for In Vivo Gene Therapy of Sickle Cell Disease Abstract: 4770 Poster Presentation Time/Date: 6-8 p.m. PT, Monday, Dec. 9 Location: San Diego Convention Center, Halls G-H Presenter: Courtney Mercadante, Ph.D., Ensoma Solid Tumors: Key Findings from SITC: Preclinical data to be presented at SITC 2024 will highlight the transformative potential of Ensoma’s novel platform for in vivo engineering of multi-lineage CAR-immune cells, offering a new approach to overcoming the significant challenges in solid tumor therapies. Solid tumors, unlike hematological malignancies, present a complex microenvironment that limits access for current therapies and complicates immune cell infiltration and efficacy. Ensoma’s VLPs target CD46-positive cells, including both HSCs and mature immune cells, to enable efficient transduction and generate durable anti-tumor responses. In immune-competent mice, administration of VLPs encoding an anti-HER2 CAR generated CAR+ myeloid (M), NK and T cells within just a few days, followed by long-term HSC renewal of multi-lineage effectors. Upon challenge with HER2+ tumors, these engineered immune cells effectively controlled tumor growth, indicating robust and durable anti-tumor activity. Presentation Details: Title: In vivo VLP-based engineering generates multi-lineage CAR-immune cells that mediate potent and durable anti-tumor activity Abstract: 1098 Poster Presentation Time/Date: 3:30-5 p.m. CT, Thursday, Nov. 7 & 9 a.m. – 8:30 p.m. CT, Saturday, Nov. 9 Location: Grand Ballroom AB - George R. Brown Convention Center Presenter: Corinne Decker, Ph.D., Ensoma "At Ensoma, our commitment to addressing critical unmet needs in genetically driven diseases is reflected in the promising preclinical data from our lead programs, including for chronic granulomatous disease, where we are pioneering innovative solutions to restore immune function,” said Robert Peters, Ph.D., chief scientific officer of Ensoma. “Our VLPs enable the delivery of large, complex cargo, creating the unprecedented ability to engineer multi-lineage immune responses in vivo . Beyond initiating our first clinical program in X-CGD and demonstrating the safety and efficacy of our platform, our focus for 2025 is to progress two development candidates while continuing to innovate our technology. We are also actively exploring partnerships that can further expand the potential of our groundbreaking platform. Together, we aim to transform the landscape of genomic medicine and improve outcomes for patients suffering from these challenging conditions." About Ensoma Ensoma is developing curative medicines through precision in vivo cellular engineering. Our platform combines class-leading proprietary base editing and high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) to provide one-time and durable genetic medicines in a simple outpatient procedure. We are focused on in vivo engineering of hematopoietic stem cells (HSCs) to address genetic diseases, immune disorders and cancer. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genomic medicines. Ensoma is based in Boston. For more information, visit ensoma.com .

Gene TherapyASHImmunotherapy

05 Nov 2024

·www.prnewswire.com

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

- 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 San Diego, California - TEL AVIV, Israel and WALTHAM, Mass., Nov. 5, 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 an abstract including the initial results from a Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD) was accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition taking place December 7-10, 2024 in San Diego, California. The proof-of-concept study, conducted in collaboration with Washington University School of Medicine in St. Louis, is exploring alternative HSC mobilization strategies that could significantly improve the treatment journey of patients with sickle cell disease seeking gene therapy. "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 Washington University on this important research and our ongoing work to develop motixafortide for the potential benefit of patients with sickle cell disease." 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 U.S. require 16.5 million, and 22 million, total CD34+ HSCs, respectively.i,ii Unfortunately, granulocyte colony-stimulating factor (G-CSF), the most commonly used drug to support the collection of stem cells, is contraindicated in patients with SCD. The use of the mobilization agent plerixafor is the current standard of care for collecting HSCs for SCD gene therapies; however, plerixafor alone requires multiple mobilization attempts and often yields suboptimal HSC numbers. For some, gene therapy may be prohibitive due to the failure to obtain adequate numbers of HSCs. 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 San Diego Convention Center, San Diego, California 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, Saint Louis, MO Abstract ID#: 193210 Date: Saturday, December 7, 2024 Time: 12:00 PM Location: San Diego Convention Center, Room 25 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 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; 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] i LYFGENIA Prescribing Information; December 2023. ii CASGEVY Prescribing Information; December 2023. Logo - SOURCE BioLineRx Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 1Drug ApprovalASHCell Therapy

100 Deals associated with Plerixafor

External Link

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
Hematopoietic stem cell transplantation	JP	Sanofi KK	19 Dec 2016
Lymphoma	AU	Sanofi-Aventis Australia Pty Ltd.	18 May 2010
Multiple Myeloma	KR	Sanofi-Aventis Korea Co., Ltd.	26 Jan 2010
Stem cell mobilisation	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
Refractory Multiple Myeloma	Phase 3	FR	Genzyme Corp.	01 Sep 2008
Refractory Multiple Myeloma	Phase 3	DE	Genzyme Corp.	01 Sep 2008
Refractory Multiple Myeloma	Phase 3	IT	Genzyme Corp.	01 Sep 2008

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NA (EHA2024)	Not Applicable	acute leukemia CD34+	8	Plerixafor	jcfxhkorvs(bjnbbnmrrj) = xgyplrtalz tcqkqdvwgt (niyfxaatny ) View more	Positive	14 May 2024
EHA2024	Not Applicable	Hematologic Neoplasms	-	Intermediate Dose Cytarabine + G-CSF	hcsnafoyzt(ttunizmfcq) = 5% (2) of patients in the ID-AraC group tdgnjfobqi (apvgzwhtnh ) View more	Positive	14 May 2024
EHA2024	Not Applicable	Hematologic Neoplasms	-	Plerixafor + G-CSF		Positive	14 May 2024
NCT04177810 (CTgov)	Phase 2	Secondary malignant neoplasm of pancreas	25	Cemiplimab+Plerixafor	nfnzbhiunw(ndqcdwucig) = sjwbvzlvyl ygvkpjqcea (ufbycjlodw, hebxawqjdp - kpqxvxksug) View more	-	20 Feb 2024
NCT01696461 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Hodgkin's Lymphoma \| Acute Lymphoblastic Leukemia ... View more	127	Plerixafor	kalpuztttt(iujmlsgbxn) = guysnhximx zyayoehmjr (zqibkwphhh, loqqvxckjh - npkfugqpry) View more	-	14 Sep 2023
EHA2023	Not Applicable	Hematologic Neoplasms	85	Intermediate-dose AraC+G-CSF	ebtvodmfvc(mnyeurdozl) = brhplgcqwn tkdfwabmru (dzrittgges )	-	08 Jun 2023
EHA2023	Not Applicable	Hematologic Neoplasms	85	Cy+G-CSF	ebtvodmfvc(mnyeurdozl) = oiybgdjxez tkdfwabmru (dzrittgges )	-	08 Jun 2023
EBMT2023	Not Applicable	Bone Marrow Neoplasms \| Lymphoma	80	Plerixafor	ugftrbgvtn(dxmevppscn) = nczyjodhhd fztylxczfm (kowgzbdsru ) View more	-	23 Apr 2023
EBMT2023	Not Applicable	Bone Marrow Neoplasms \| Lymphoma	80	G-CSF	ugftrbgvtn(dxmevppscn) = arktiitosb fztylxczfm (kowgzbdsru ) View more	-	23 Apr 2023
P158 (EBMT2023)	Not Applicable	-	81	Plerixafor	ffajydpbod(ctmhiihxrj) = wmapjheeib xtzunpfcms (xjllqxvheb )	Positive	23 Apr 2023
P689 (EBMT2023)	Not Applicable	-	13	Plerixafor 0.24 mg/kg	cfolzprctv(hsnxchohfb) = Plerixafor was well tolerated by the donor in all cases and no side effects or adverse reactions occurred ddjsuhfvrt (suhjxznzeo )	-	23 Apr 2023
P689 (EBMT2023)	Not Applicable	-	13	G-CSF 10 mcg/kg		-	23 Apr 2023
P329 (EBMT2023)	Not Applicable	-	-	Plerixafor + G-CSF	kpenlkchzw(btvskdblnx) = dwxyqcnolu xympaexrru (cvstakxrip ) View more	-	23 Apr 2023
EHA2022	Not Applicable	-	84	Plerixafor	cykxjwquzk(ybfrpfekdt) = ejrsqnganu djrunxtlve (uqhqgeqpos )	-	12 May 2022

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