This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.
PRIMARY OBJECTIVE
* To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs).
SECONDARY OBJECTIVES
* To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg).
* To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts
* To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects

Start Date14 Nov 2024

Sponsor / Collaborator

St. Jude Children's Research Hospital, Inc.

[+1]

NCT06547112

/ RecruitingPhase 1IIT

A Pharmacodynamic Study of the Apheresis Product of Multiple Myeloma Patients Undergoing Quad-induction Followed by Motixafortide + G-CSF Mobilization

This study includes extended CD34+ profiling on the apheresis product of multiple myeloma patients undergoing standard-of-care quad-induction followed by motixafortide + G-CSF mobilization, and in addition, assesses the pharmacodynamics (PD) of motixafortide following "standard" (
12 hours) vs "early" (
16 hours) dosing. The investigators hypothesize that quad-induction may alter the stem cell subsets within the mobilized graft. The investigators further hypothesize that standard and early dosing strategies will result in comparable mobilization and stem cell collection rates.

Start Date31 Oct 2024

Sponsor / Collaborator

Washington University School of Medicine

[+1]

NCT06514508

/ Not yet recruitingPhase 3

A Phase Ⅲ, Randomized, Double-Blinded Study Evaluating the Safety and Efficacy of Combination Treatment of Motixafortide and G-CSF as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects With Multiple Myeloma

This is a randomized, double-blinded, placebo-controlled, multi-center phase Ⅲ bridging clinical study designed to evaluate the efficacy, safety, and pharmacokinetic and pharmacodynamic profiles of Motixafortide (BL-8040) + G-CSF vs placebo + G-CSF mobilized hematopoietic stem cells for autologous transplantation in Chinese patients with multiple myeloma.

Start Date30 Sep 2024

Sponsor / Collaborator

Guangzhou Gloria Biosciences Co., Ltd.

100 Clinical Results associated with Motixafortide

100 Translational Medicine associated with Motixafortide

100 Patents (Medical) associated with Motixafortide

100

Literatures (Medical) associated with Motixafortide

03 Apr 2025·EXPERT OPINION ON THERAPEUTIC PATENTS

Small molecule and peptide CXCR4 antagonists. A patent review from 2019 to 2024

Review

Author: Wilson, Lawrence J. ; Liotta, Dennis C. ; Tahirovic, Yesim A. ; Geng, Jiafeng ; Sahin, Zafer

INTRODUCTION:

The chemokine receptor CXCR4 has been under intense study due to the central role it plays in immune system regulation and the pathology of human disease. Although the first CXCR4 drug plerixafor emerged over a decade ago (2007), recently the first peptide (motixafortide, 2023) and the first oral small molecule (mavorixafor, 2024) CXCR4 antagonists became FDA approved.

AREAS COVERED:

This article describes patent documents published during the period of 2019 through 2024 for both small molecule and peptides. This IP includes few new chemotypes, with most being extensions of existing structural classes. There is also less significant IP covering peptide-based therapeutics than those covering small molecules. Notably, multiple therapeutic uses have also emerged. Patents were searched from SciFinder (CAS) and Google Patents with the term CXCR4 antagonists. Patents were selected according to whether they fit into the classification of small molecules or peptides.

EXPERT OPINION:

In the last 5 years there has been significant advancement in CXCR4 antagonists as gauged by the FDA approval of two drugs. The search for second and third generation compounds will be the focus of future efforts with new uses and better properties which likely could come from some of the IP described herein.

01 Nov 2024·LEUKEMIA

CXCR4 as a therapeutic target in acute myeloid leukemia

Review

Author: Chlubek, Dariusz ; Bosiacki, Mateusz ; Barczak, Katarzyna ; Kupnicka, Patrycja ; Korbecki, Jan ; Baranowska-Bosiacka, Irena

Extensive research on the CXCL12-CXCR4 axis in acute myeloid leukemia (AML) has resulted in the incorporation of novel anti-leukemia drugs targeting this axis into therapeutic strategies. However, despite this progress, a comprehensive and up-to-date review addressing the role of the CXCL12-CXCR4 axis in AML's oncogenic processes is lacking. In this review, we examine its molecular aspects influencing cancer progression, such as its impact on autonomous proliferation, apoptotic regulation, chemoresistance mechanisms, and interactions with non-leukemic cells such as MSCs and T_reg cells. Additionally, we explore clinical implications, including prognosis, correlation with WBC count, blast count in the bone marrow and peripheral blood, as well as its association with FLT3-ITD, NPM1 mutations, and FAB classification. Finally, this paper extensively discusses drugs that specifically target the CXCL12-CXCR4 axis, including plerixafor/AMD3100, ulocuplumab, peptide E5, and motixafortide, shedding light on their potential therapeutic value in the treatment of AML.

01 Oct 2024·Journal of oncology pharmacy practice : official publication of the International Society of Oncology Pharmacy Practitioners

A comparative review of Aphexda and Mozobil for mobilization prior to stem cell collection

Review

Author: Dietz, Lauren ; Hashmi, Hamza ; Marini, Jessica ; McGann, Mary

High-dose chemotherapy followed by autologous hematopoietic stem cell transplantation is the standard of care for eligible patients with newly diagnosed multiple myeloma leading to prolonged progression free and overall survival. Successful engraftment following hematopoietic stem cell infusion requires adequate stem cell doses. Current mobilization regimens include granulocyte colony-stimulating factor (G-CSF) with or without plerixafor. Motixafortide is a recently approved agent that can be used in combination with G-CSF for mobilization. In the absence of any head-to-head trials comparing the two products, this article aims to outline the similarities and differences of these two agents. Though moxitafortide has a more favorable pharmacokinetic profile in comparison to plerixafor, in clinical trials, the agents demonstrated similar efficacy. In addition, the use of motixafortide in clinical practice may be limited by product cost as well as administration and monitoring requirements.

News (Medical) associated with Motixafortide

31 Mar 2025

·www.prnewswire.com

BioLineRx Reports 2024 Financial Results and Provides Corporate Update

- Reports meaningful progress in the evaluation of assets for potential in-licensing and development in the areas of oncology and rare diseases - - Executed license agreement with Ayrmid Pharma Ltd. for APHEXDA® (motixafortide) with $10 million upfront payment, up to $87 million in commercial milestones, and high double-digit royalties on net sales - - Completed financings raising combined gross proceeds of $19 million and reduced operating expense run rate by 70%, extending the Company's cash runway through H2 2026 - - Management to host conference call today, March 31st, at 8:30 am EDT - TEL AVIV, Israel, March 31, 2025 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today reported its audited financial results for the year ended December 31, 2024, and provided a corporate update. "It has been just over four months since we implemented a major strategy shift, highlighted by the transformational exclusive licensing agreement that we entered into with Ayrmid Ltd., granting it the rights to commercialize APHEXDA® (motixafortide) in all non-solid-tumor indications and all territories other than Asia," said Philip Serlin, Chief Executive Officer of BioLineRx. "Since then, we implemented cost efficiencies across the Company, including the shutdown of our U.S. commercial operations, that have resulted in an approximate 70% reduction in our operating expense base, which, together with recent financings, have put us on a firm footing with a cash runway through the second half of 2026." "As we return to our roots as a lean drug development company, with a highly validated development platform focused on oncology and rare diseases, we believe these actions help ensure that we remain nimble and capable of seizing the opportunities in front of us. Our strategy moving forward is to in-license additional assets over the next year that we can advance through clinical proof-of-concept, funded in part by milestones and royalties from our out-licensing transactions. To that end, I am pleased to report that we are evaluating numerous promising candidates. This process is methodical and steady to ensure that our due diligence is thorough as we look for new chemical entities. Based on our deep and validated experience in drug development, I believe we are well positioned to create sustained value for our shareholders. I am excited about what the future holds for our Company this year and beyond," Mr. Serlin concluded. Corporate Updates Executed license agreement with Ayrmid Pharma 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% Announced receipt of a Notice of Allowance from the U.S. Patent & Trademark Office (USPTO) for a patent, titled "Composition of BL-8040," which strengthens BioLineRx's robust intellectual property (IP) estate and extends its patent protection on motixafortide (BL-8040) in the U.S. through December 2041 Financial Updates Completed two financings in past few months which raised combined gross proceeds of $19 million Reduced operating expense run rate by approximately 70% beginning January 1, 2025 through the APHEXDA program transfer to Ayrmid and the resulting shutdown of the Company's U.S. commercial operations in Q424, as well as additional headcount and other operating expense reductions Significantly reduced outstanding debt and restructured the remainder on favorable terms to the Company APHEXDA 2024 Performance Update Aphexda achieved 10 percent market share of total CXCR4 inhibitor usage in the U.S., which compares APHEXDA to branded MOZOBIL and generic plerixafor in all indications BioLineRx generated more than $6 million in net product sales year-to-date through the November 2024 completion of the Ayrmid out-licensing transaction Clinical Updates Motixafortide Pancreatic Ductal Adenocarcinoma (mPDAC) Additional trial sites activated for the CheMo4METPANC Phase 2b clinical trial being led by Columbia University. 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 Sickle Cell Disease (SCD) & Gene Therapy First patient dosed in the multi-center Phase 1 clinical trial evaluating motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with Sickle Cell Disease (SCD). The trial is sponsored by St. Jude Children's Research Hospital. Oral presentation delivered at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition detailing 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 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. Financial Results for the Year Ended December 31, 2024 Revenues for the year ended December 31, 2024 were $28.9 million, an increase of $24.1 million, or 502.1%, compared to $4.8 million for the year ended December 31, 2023. The revenues in 2024 primarily reflect a portion of the up-front payment received, and a milestone payment achieved, under the Gloria license, which collectively amounted to $15.0 million, as well as the up-front payment received under the Ayrmid license and $6.0 million of net revenues from product sales of APHEXDA in the U.S. The revenues in 2023 (all of which were recorded in the fourth quarter of 2023) primarily reflect a portion of the up-front payment received under the Gloria license of $4.6 million, as well as $0.2 million of revenues from product sales of APHEXDA in the U.S. Cost of revenues for the year ended December 31, 2024 were $9.3 million, an increase of $5.6, or 151.4%, compared to $3.7 million for the year ended December 31, 2023. The cost of revenues in 2024 primarily reflects amortization of intangible assets, Biokine's share of the up-front payment received under the Ayrmid license, sub-license fees accrued on a milestone payment recorded under the Gloria license, as well as royalties on net product sales of APHEXDA in the U.S. and cost of goods sold on product sales. The cost of revenues in 2023 primarily reflects Biokine's share of the up-front payment received under the Gloria license and of the net sales. Research and development expenses for the year ended December 31, 2024 were $9.2 million, a decrease of $3.3 million, or 26.4%, compared to $12.5 million for the year ended December 31, 2023. The decrease resulted primarily from lower expenses related to motixafortide NDA supporting activities, termination of the development of AGI-134 and a decrease in payroll and share-based compensation. Sales and marketing expenses for the year ended December 31, 2024 were $23.6 million, a decrease of $1.7 million, or 6.7%, compared to $25.3 million for the year ended December 31, 2023. The decrease resulted primarily from the shutdown of U.S. commercial operations in the fourth quarter of 2024 following the Ayrmid license. General and administrative expenses for the year ended December 31, 2024 were $6.3 million, similar to the year ended December 31, 2023. Net non-operating income amounted to $18.4 million for the year ended December 31, 2024, compared to net non-operating expenses of $10.8 million for the year ended December 31, 2023. Non-operating income for the year ended December 31, 2024 primarily relates to non-cash, fair-value adjustments of warrant liabilities on the Company's balance sheet, as a result of changes in the Company's share price, offset by warrant offering expenses. Non-operating expenses for the year ended December 31, 2023 primarily relate to non-cash, fair-value adjustments of warrant liabilities on the Company's balance sheet. Net financial expenses amounted to $7.3 million for the year ended December 31, 2024, compared to net financial expenses of $0.1 million for the year ended December 31, 2023. Net financial expenses for both periods primarily relate to interest paid on loans, which increased in 2024 due to a one-time $4.0 million charge to interest expense in connection with the November 2024 amendment to the loan agreement with BlackRock, partially offset by investment income earned on bank deposits. Net loss for the year ended December 31, 2024 was $9.2 million, compared to $60.6 million for the year ended December 31, 2023. As of December 31, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $19.6 million (approximately $29.0 million on a pro-forma basis, following the financing completed at the beginning of January 2025). A copy of the Company's annual report on Form 20-F for the year ended December 31, 2024 has been filed with the U.S. Securities and Exchange Commission at and posted on the Company's investor relations website at Company will deliver a hard copy of its annual report, including its complete audited consolidated financial statements, free of charge, to its shareholders upon request at [email protected]. Conference Call and Webcast Information 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 April 2, 2025; 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.  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 success of the license agreement with Ayrmid and the commercial potential of motixafortide, expectations with regard to clinical trials of motixafortide, the expected cash runway, and BioLineRx's business strategy. 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 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; 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 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; 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, manage, and maintain corporate collaborations, as well as the ability of BioLineRx's collaborators to execute on their development and commercialization plans; BioLineRx's ability to integrate new therapeutic candidates and new personnel as well as new collaborations; 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; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; BioLineRx's ability to maintain the listing of its ADSs on Nasdaq; and statements as to the impact of the political and security situation in Israel on BioLineRx's business, 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 31, 2025. 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 Irina Koffler LifeSci Advisors, LLC [email protected] Israel Moran Meir LifeSci Advisors, LLC [email protected] 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

Phase 1License out/inASHDrug ApprovalFinancial Statement

23 Jan 2025

·www.businesswire.com

CORRECTING and REPLACING Exicure, Inc. Announces Purchase Agreement with GPCR Therapeutics Inc.

CHICAGO--( BUSINESS WIRE )--Please replace the release dated January 22, 2025 with the following corrected version due to multiple revisions. The updated release reads: EXICURE, INC. ANNOUNCES PURCHASE AGREEMENT WITH GPCR THERAPEUTICS INC. Exicure, Inc. (Nasdaq: XCUR, “the Company”, “Exicure”), today announced that on January 19, 2025, Exicure Inc. (“the Company”) entered into a Share Purchase Agreement with GPCR Therapeutics Inc., a Korean corporation (“GPCR”), pursuant to which the Company acquired from GPCR all of the issued and outstanding equity securities of GPCR Therapeutics USA Inc., a California corporation (“GPCR USA”). The transactions completed under the Share Purchase Agreement closed concurrently with execution. GPCR USA was, until immediately prior to closing under the Share Purchase Agreement, a wholly owned subsidiary of GPCR. In connection with the closing of the Share Purchase Agreement, the Company and GPCR entered into a License and Collaboration Agreement (“L&C Agreement”) to further develop and commercialize GPCR’s technologies related to certain intellectual properties and patents. The L&C Agreement requires the Company to make milestone payments to GPCR upon the achievement of specific milestone events relating to clinical trials, marketing authorizations, and net sales, as well as for the Company to pay a recurring royalty payment based on at least 10% of net sales, as set forth in the L&C Agreement. About GPCR USA, Inc. GPCR USA is a biotech company that is currently led by Dr. Pina Cardarelli, the CSO and president of GPCR USA. The company has an ongoing Phase 2 clinical trial focused on blood cancer patients, particularly those eligible for hematopoietic stem cell transplantation (HSCT). The trial involves the combined administration of G-CSF, GPC-100 (Burixafor) and propranolol. Per Dr. Cardarelli, "Two additional patients will receive treatment in January 2025. GPCR USA plans to complete the administration of GPC-100 to 20 patients by the end of April and aim to announce the clinical trial results by September." In December of last year, GPCR USA presented results at the American Society of Hematology conference evaluating the impact of inhibiting CXCR4 and ADRB2 (beta-2 adrenergic receptor) on stem cell mobilization. Interim data from 10 patients for burixafor in combination with propranolol and G-CSF is promising. Results suggest that the treatment regimen can mobilize CD34 + stem cells sufficiently for autologous stem cell transplant. Notably, burixafor allowed for same day administration of both mobilizing agent and leukapheresis. This fast kinetics of mobilization is differentiated from the FDA approved plerixafor or motixafortide, which require overnight pre-treatment prior to leukapheresis. The median times to neutrophil and platelet engraftment, 11 and 14 days respectively, are slightly less than what has been observed for standard of care. Importantly, 100% of the patients met the primary objective and the combination regimen exhibited excellent safety profiles in patients receiving the new front line blood cancer drug, Daratumumab. Dr. Cardarelli stated, "After completing patient treatment, we will conduct follow-up observations until July and then start data analysis," adding that positive results from the GPC-100 trial could have a beneficial impact on attracting additional investment and enhancing corporate value. Currently, the ongoing clinical trials have met all primary end points and are at the stage of deciding whether to proceed to Phase 3. A proposed double-blind clinical trial comparing the mobilization agent Plerixafor with GPC-100 is under discussion. Additionally, research has shown that the combination of GPC-100 and propranolol increases the number of specific T cells, which are favorable for enhancing CAR-T (Chimeric Antigen Receptor T-cell) response rates, and relevant papers are being published. Thus, this could assist Exicure in expanding its additional pipeline for cell and gene therapies. Dr. Cardarelli suggested that in the future, it will be essential to prioritize discussions with various partners based on the likelihood of success and high return on investment. She concluded, "If the hypotheses presented to GPCR USA are validated, there may be potential G-Protein Coupled Receptor targets that could be applicable to various diseases. We are considering steps such as establishing collaborations with companies possessing antibody manufacturing technologies, testing those technologies, and conducting joint research on multiple G-Protein Coupled Receptors.” Dr. Cardarelli was an inventor of the CXCR4 antibody, Ulocuplumab developed by BMS, and in 2019 she joined GPCR Therapeutics evaluating the combination of CXCR4 and ADRB2. She later served as the Chief Scientific Officer (CSO) of GPCR USA, contributing to the establishment of GPCR USA, talent acquisition, formation of a scientific advisory board, and the introduction of cell analysis and high-throughput screening technologies. She also successfully designed clinical trials for GPC-100 and secured FDA orphan drug designation. About Exicure, Inc. Exicure, Inc. is an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its recent restructuring and suspension of clinical and development activities, the Company is exploring strategic alternatives to maximize stockholder value, both with respect to its historical biotechnology assets and more broadly. For further information, see www.exicuretx.com . Forward-Looking Statements This press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. Forward looking statements are those which are not historical in nature. They are often identified by their inclusion of words such as “will,” “anticipate,” “estimate,” “plan,” “should,” “expect,” “believe,” “intend” and similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual outcomes to differ materially from the outcomes expressed or implied by this report. Such risks include, among others, the accuracy of projections, targets, or plans, the possibility of listing deficiencies, and the risk that Nasdaq will ultimately delist the Company’s common stock. All such factors are difficult to predict and may be beyond the Company’s control. The Company undertakes no obligation and does not intend to update or revise any forward-looking statements contained herein, except as required by law or regulation. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report.

Clinical ResultLicense out/inASHPhase 2Orphan Drug

21 Jan 2025

·www.prnewswire.com

BioLineRx Issues Letter to Shareholders

- Company outlines strategic long-term vision and financial outlook - TEL AVIV, Israel, Jan. 21, 2025 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today issued the following letter to shareholders. To my fellow shareholders, It has been approximately two months since we announced a major strategy shift with the signing of an exclusive license agreement with Ayrmid Ltd. to commercialize APHEXDA® (motixafortide), the next-generation stem cell mobilization agent that we successfully shepherded from early clinical development through FDA approval and commercial launch in September 2023. Following such a transformational transaction, I wanted to provide more granularity regarding our vision for our company moving forward, as we remain as committed as ever to maximizing value for our shareholders. To fully understand where we plan to go, it is important to recap the salient terms of the license agreement with Ayrmid Ltd. (Ayrmid), as well as the previously announced license agreement that we entered into with Guangzhou Gloria Biosciences Co., Ltd. (GloriaBio). Both agreements provided significant non-dilutive upfront capital, while allowing us to retain upside potential from royalties on future sales of APHEXDA, as well as from potential future commercial and development milestones in the respective territories. We expect these collaborations to provide a growing stream of cash flows into our company over time while we work to rebuild our pipeline with new assets. Ayrmid The license agreement with Ayrmid, which we announced in November 2024, gives Ayrmid the right to develop and commercialize APHEXDA (motixafortide) across all indications, excluding solid tumor indications, and in all territories other than Asia. In exchange for the license, we received a $10 million upfront payment, and are eligible to receive up to an additional $87 million in potential commercial milestones, plus royalties ranging from 18% to 23% on net sales of APHEXDA. In addition, certain funds managed by Highbridge Capital Management LLC, Ayrmid's principal shareholder, executed a $9 million equity investment in the Company. GloriaBio The license agreement with GloriaBio, which we first announced in August 2023, gives GloriaBio the right to develop and commercialize motixafortide across all indications in the Asia region, beginning with stem cell mobilization (SCM), followed by pancreatic ductal adenocarcinoma (PDAC). In exchange, we received a $15 million upfront payment, and are eligible to receive up to ~$50 million in potential development and regulatory milestones in China and Japan, as well as up to ~$200 million in potential commercial milestones based on defined sales targets. We are also eligible to receive tiered double-digit royalties on net sales. In addition, a $14.6 million equity investment in the Company was executed here as well. BioLineRx 2.0 As it pertains to BioLineRx, or what I now like to call 'BioLineRx 2.0', we have returned to our roots as a lean drug development company, leveraging the many years of experience and the track record of success that our team brings, as evidenced by our successful development of motixafortide, which we advanced from early-stage clinical development through a successful Phase 3 trial, and culminating with FDA approval in September 2023. We have retained the rights to develop motixafortide across all solid tumor indications, in all territories other than Asia, including in pancreatic ductal adenocarcinoma (PDAC), for which an investigator-initiated Phase 2b trial, sponsored by Columbia University, and supported equally by BioLineRx and Regeneron, is ongoing at a relatively minimal cost to BioLineRx. A second Phase 2b trial in PDAC is being planned by GloriaBio, at no cost to us. We expect these pipeline programs to continue to advance without any significant expense to our Company, while providing the potential for a meaningful future upside. At the same time, a key component of our strategy moving forward is to in-license additional assets in oncology and/or rare diseases over the next two years that we can advance through clinical development. We have substantial experience in scouting and assessing assets in transactions with back-ended, success-based consideration, which can be acquired or licensed for a modest upfront payment, and with relatively modest and affordable clinical development programs. We are actively working on this initiative and are being presented with many promising opportunities that meet these criteria. Visibility on our progress with this initiative, like most companies, will be minimal until we have a tangible agreement to announce. Our longer-term vision is to develop innovative assets with significant potential value whose development costs have been offset by the royalties and milestones from our existing motixafortide partnerships. We aim to continue pursuing new partnerships on these programs to create additional value for our shareholders. Strengthened Balance Sheet and Streamlined Cost Structure Provide Runway through H2 2026 Proceeds from the Ayrmid and GloriaBio transactions, together with the opportunistic $10 million equity financing that we completed in early January and our significantly reduced cost structure (our currently planned operating burn rate going forward is ~$12 million per year) are expected to provide us with a current cash runway to execute upon our goals through the second half of 2026. This includes the relatively minor costs associated with continuing to support the PDAC trials, while also engaging in our planned pipeline expansion activities. Furthermore, it does not take into account any potential revenue upside generated from sales royalties or commercial milestones under our out-licensing agreements that I just described. ADS Ratio Change Maintains Nasdaq Listing Compliance Finally, to regain compliance with Nasdaq's minimum bid price requirement, we announced just a few days ago our intent to change the ratio of our American Depositary Shares to ordinary shares, from 15 ordinary shares per ADS to 600 ordinary shares per ADS. This is equivalent to a 1-for-40 reverse stock split, and will go into effect on January 30, 2025. As we continue to execute on our long-term strategy, including the potential in-licensing of new assets, maintaining our Nasdaq exchange listing is a critical objective. By implementing this change, we hope to regain compliance with all of Nasdaq's applicable requirements, allowing us to continue to enjoy all the benefits that such a listing confers. In closing, we are entering 2025 with a renewed focus on innovative drug development within our core competency areas, a strengthened balance sheet with $29.5 million of cash, a very lean cost structure, and a cash runway based on our current operating plan through the second half of 2026. With these strategic actions completed, I believe we are well positioned to create enduring value for our shareholders while introducing novel therapeutics for patients suffering from cancer or rare diseases. I am excited about what the future holds for BioLineRx, both for this year and beyond, and I look forward to keeping you apprised of our continued progress, beginning with our fourth quarter 2024 results release in March. Thank you for your continued support. Sincerely, Phil Serlin Philip Serlin Chief Executive Officer 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 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 its future prospects, including BioLineRx's cash runway. 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, 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 Irina Koffler LifeSci Advisors, LLC [email protected] Israel Moran Meir LifeSci Advisors, LLC [email protected] 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

License out/inPhase 2Phase 3Drug Approval

100 Deals associated with Motixafortide

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB14939

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Myeloma	United States	Ayrmid Pharma Limited	08 Sep 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Pancreatic Ductal Carcinoma	Phase 2	China	BioLineRx Ltd.	28 Jun 2022
Pancreatic Ductal Carcinoma	Phase 2	China	Genfleet Therapeutics (Shanghai), Inc.Startup	28 Jun 2022
Adult Lymphoblastic Lymphoma	Phase 2	United States	National Institutes of Health	02 Dec 2016
Refractory T Acute Lymphoblastic Leukemia	Phase 2	United States	National Institutes of Health	02 Dec 2016
Metastatic Pancreatic Cancer	Phase 2	United States	BioLineRx Ltd.	01 Sep 2016
Metastatic Pancreatic Cancer	Phase 2	Israel	BioLineRx Ltd.	01 Sep 2016
Metastatic Pancreatic Cancer	Phase 2	South Korea	BioLineRx Ltd.	01 Sep 2016
Metastatic Pancreatic Cancer	Phase 2	Spain	BioLineRx Ltd.	01 Sep 2016
Pancreatic adenocarcinoma metastatic	Phase 2	United States	BioLineRx Ltd.	01 Sep 2016
Pancreatic adenocarcinoma metastatic	Phase 2	Israel	BioLineRx Ltd.	01 Sep 2016

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02907099 (P328, CTgov)	Phase 2	Pancreatic adenocarcinoma metastatic \| Metastatic Pancreatic Cancer	20	BL-8040+Pembrolizumab	blllycsvtz = vikvzsilxd wilupisqtk (khdyrmhtjo, kriurqrrsr - xdcuvwjwmo) View more	-	19 Feb 2025
NCT05293171 (Tandem Meetings ASTCT and CIBMTR2024)	Phase 1	CD34+	-	Motixafortide 1.25mg/kg	vowzxtvkks(hgmbyqrsuh) = krmkmzkvpc jyaemdnrga (bukrmsulbx ) View more	-	01 Feb 2024
				G-CSF + Motixafortide 1.25mg/kg	vowzxtvkks(hgmbyqrsuh) = zthahdvsxk jyaemdnrga (bukrmsulbx ) View more
NCT03246529 (GENESIS, CTgov)	Phase 3	Multiple Myeloma	180	G-CSF+BL-8040 (BL-8040 + G-CSF Part 1)	ktccuplput = mtorrewuvp rrxfmzwnzz (twsjwkjbmk, xyztglpceq - csqzxhznlu) View more	-	07 Nov 2023
				G-CSF+BL-8040 (BL-8040 + G-CSF Part 2)	yanihcngqy = yexjcastjl twtapovdlm (ehfncdefnp, yldxnqeoat - wasuuteclt) View more
NCT02763384 (CTgov)	Phase 2	Refractory T Acute Lymphoblastic Leukemia \| Adult Lymphoblastic Lymphoma	12	BL-8040+Nelarabine	ggtqonhehx = tyeufvlhaz mvblyxximr (srirrdvfpf, azmhqopsof - iyrqbfgcst) View more	-	31 Oct 2023
NCT03246529 (GENESIS, FDA) Manual	Phase 3	Multiple Myeloma	122	APHEXDA+Filgrastim	wzziyejrvz(degdhxmwrz) = ygrgxaredc ottpubmjbn (fqrnvioeuq ) View more	Positive	08 Sep 2023
				Placebo+Filgrastim	wzziyejrvz(degdhxmwrz) = zbjbtifzjr ottpubmjbn (fqrnvioeuq ) View more
CC2 (AACR2021)	Phase 2	Pancreatic adenocarcinoma metastatic Second line	43	Motixafortide (BL-8040)	pqnrymtbtd(mibdhdyhtr) = oaddmstmup whkpqzafit (sppopbfqnd ) View more	-	01 Jul 2021
NCT03246529 (GENESIS, PRNewswire) Manual	Phase 3	Multiple Myeloma	122	G-CSF+Motixafortide	bloakkyxoo(chpebybzww) = upfqcpqugx cyeomhbpot (sokjufdirz ) View more	Positive	04 May 2021
				G-CSF+Placebo	bloakkyxoo(chpebybzww) = flebjzdudm cyeomhbpot (sokjufdirz ) View more
NCT01838395 (Pubmed \| Cancer) Manual	Phase 2	Acute Myeloid Leukemia	42	HiDAC+Motixafortide	krwyrnyhzh(umujrniboo) = bveomdysyq xlxzhhkizf (hzraacjfsz ) View more	Positive	15 Apr 2021

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis