Plerixafor

WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients affected by this condition. ROME, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening immunodeficiency. WAS, seen almost exclusively in males, affects blood cells and cells of the immune system (the body's natural defenses). It is caused by abnormalities in the gene that produces the WAS protein found in blood cells and certain immune cells. Because people with the condition lack a functional WAS protein, their immune cells and blood cells do not develop and function normally. The FDA's approval of the BLA of Waskyra follows a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for the same product just a few weeks earlier. Developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Waskyra represents a major scientific and clinical achievement, offering new hope for patients affected by this condition. "The FDA's approval of Waskyra is an extraordinary achievement – not only for Italian research and for Fondazione Telethon, but for the global rare disease community," said Ilaria Villa, CEO of Fondazione Telethon. "It confirms the value of a patient-centered model that turns research into real treatments, especially where the market fails to act." "The approval of this gene therapy represents a decisive step forward and a tangible response to the needs of patients," commented Dr. Alessandro Aiuti, Deputy director clinical research at SR-Tiget, Chief of Pediatric Immunohematology at IRCCS Ospedale San Raffaele and Full Professor of Pediatrics at Università Vita-Salute San Raffaele. "Seeing years of scientific research and dedication translate into real therapeutic opportunities for people gives profound meaning to our work." The clinical trial phase was conducted at IRCCS Ospedale San Raffaele, a center of excellence in gene therapy for WAS and other diseases. Fondazione Telethon is the first non-profit organization to have successfully led the full pathway of an ex vivo gene therapy from laboratory research to regulatory approval, collaborating with industry partners to bring gene therapies from discovery to patients. FDA approval further recognizes Fondazione Telethon's excellence in research and strengthens its position as an international leader in rare genetic disease research and the development of advanced therapies. About Wiskott-Aldrich syndrome (WAS) Wiskott-Aldrich syndrome is a rare genetic blood disorder that causes immunodeficiency and low platelet count, resulting from mutations in the WAS gene. The disease manifests from early childhood with recurrent and persistent infections, bleeding episodes and eczema, and is associated with an increased risk of developing autoimmune diseases and lymphomas. It affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. Current treatment options include supportive therapies aimed at managing and preventing clinical manifestations. The only potentially curative option is hematopoietic stem cell transplantation, for which a compatible donor is not always available, and which is not without risks. About Waskyra™ (etuvetidigene autotemcel) gene therapy for Wiskott-Aldrich syndrome Waskyra consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene. Once corrected, the stem cells are reinfused into the patient, who undergoes chemotherapy beforehand to prepare the bone marrow to receive them. It has been shown that Waskyra reduces the frequency of severe and moderate bleeding events and serious infections in patients with WAS compared with the period prior to treatment. In cases where transplantation from a compatible family donor is not possible, a safe and efficacious gene therapy represents a potential therapeutic option for eligible patients. Indication and Important Safety Information What is Waskyra? Waskyra is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. What is the most important information I should know about Waskyra? During the clinical trials no adverse reactions attributable to Waskyra nor to conditioning regimen were reported. Some side effects are related to pre-treatment and other study procedures. Treatment with Waskyra is preceded by medical interventions, namely haematopoietic stem cell collection through peripheral blood mobilisation with G-CSF with or without plerixafor followed by apheresis, pre-treatment with rituximab (anti-CD20 monoclonal antibody) and reduced intensity conditioning, which carry their own risks. When assessing the safety of a treatment with Waskyra, the safety profile and product information of the medicinal products used for peripheral blood mobilisation, pre-treatment and reduced intensity conditioning should be considered, in addition to the risks linked to the gene therapy. Negative side effects of prescription drugs are encouraged to report to the FDA. Visit , or call 1–800-FDA-1088. For complete product information, please see the full Prescribing Information at Site: Package Insert: About Fondazione Telethon Fondazione Telethon is an Italian non-profit biomedical organization committed to advancing research on rare and complex genetic diseases. For over 35 years, it has supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions. 21% more press release views with Request a Demo

- Establishes joint venture with Hemispherian AS to advance GLIX1, a first-in-class, oral, small molecule targeting DNA damage response in glioblastoma and other cancers - - Phase 1/2a clinical trial of GLIX1 expected to commence in Q1 2026 - - Management to host conference call today, November 24th, at 8:30 am EST - TEL AVIV, Israel, Nov. 24, 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 unaudited financial results for the quarter ended September 30, 2025, and provided a corporate update. "The clear highlight of the third quarter was our announcement in September that we established a joint venture with Hemispherian, expanding our development pipeline into additional high-need cancer indications, leading with glioblastoma, in addition to our ongoing PDAC program," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Hemispherian's lead asset, GLIX1, is a versatile molecule with a novel mechanism of action that targets the DNA repair mechanism in cancer cells and has demonstrated compelling efficacy in numerous pre-clinical models. Importantly, the development path is straightforward and efficient, and we are eager to initiate a Phase 1/2a first-in-human study in the first quarter of next year while also advancing pre-clinical activities in support of future potential trials of GLIX1 in other cancers." "At the same time, the ongoing CheMo4METPANC Phase 2b clinical trial of motixafortide in metastatic pancreatic cancer, which is being led by Columbia University and supported by both Regeneron and BioLineRx, continues to progress, giving us a second opportunity to leverage our drug development expertise to bring true innovation to patients with difficult-to-treat cancers," Mr. Serlin concluded. Corporate Updates Announced formation of a joint venture to advance privately held Hemispherian's small molecule cancer therapeutic, GLIX1 GLIX1, a Phase 1-ready candidate that is being developed as a potential treatment for glioblastoma, estimated to be a greater than $3.7 billion global addressable market by 2030 that has seen little innovation since the current standard of care was developed in 2005. The compound is also expected to be evaluated in other cancers, with preclinical work beginning in 2026. Announced that it has received Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for a key patent covering GLIX1 for cancers in which cytidine deaminase (CDA) is not over-expressed beyond a specific threshold, estimated to be 90% of all cancers. Patent preserves BioLineRx's ability to evaluate GLIX1 in other cancers beyond glioblastoma, including both hematological and solid tumor cancer types. Patent further broadens and strengthens GLIX1's patent protection until 2040, with a possible patent-term extension of up to five years. Financial Updates With $25.2 million on its balance sheet as of September 30, 2025, BioLineRx is maintaining its cash runway guidance into the first half of 2027. Clinical Updates GLIX1 Continued to advance preparations for initiation of a Phase 1/2a clinical trial of GLIX1 in recurrent and newly diagnosed glioblastoma in the first quarter of 2026. World leading investigators in the field of glioblastoma, Dr. Roger Stupp and Dr. Ditte Primdahl of the Malnati Brain Tumor Institute of the Lurie Comprehensive Cancer Center at Northwestern University, will serve as principal investigators for the study. The Phase 1 part of the trial aims to establish a maximum tolerated dose (MTD) and/or a recommended dose based on safety, PK/PD and preliminary efficacy. The Phase 2a expansion part of the trial is planned to include three population cohorts: (1) GLIX1 as monotherapy in recurrent GBM, (2) GLIX1 on top of standard of care in newly diagnosed GBM patients (likely a "window of opportunity" study, with biopsies before and after treatment for PD assessment), and (3) GLIX1 in combination with PARP inhibitors in other solid tumors. Pre-clinical activities in support of potential clinical trials of GLIX1 in additional cancers are ongoing. Motixafortide Pancreatic Ductal Adenocarcinoma (mPDAC) Enrollment continues in the CheMo4METPANC Phase 2b clinical trial, which is being led by Columbia University, and supported by both Regeneron and BioLineRx. The CheMo4METPANC trial is evaluating motixafortide in combination with the PD-1 inhibitor cemiplimab and standard chemotherapy (gemcitabine and nab-paclitaxel). A prespecified interim analysis is planned when 40% of progression-free survival (PFS) events are observed. Sickle Cell Disease (SCD) & Gene Therapy Announced that a poster featuring final results from a Phase 1 clinical trial (NCT05618301) 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 presentation at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition taking place December 6-9, 2025, in Orlando, FL. The 10-subject proof-of-concept study, which was conducted in collaboration with Washington University School of Medicine, demonstrated that motixafortide alone, and in combination with natalizumab, were found to be safe and well- tolerated. Common adverse events were transient and included Grade 1-2 injection site and systemic reactions. No Grade 4 adverse events, dose limiting toxicities or complicated vaso-occlusive events occurred. Motixafortide alone, and in combination with natalizumab resulted in robust CD34+ HSC mobilization. Motixafortide alone mobilized a median of 189 CD34+ cells/μl (range 77-690) to the peripheral blood (PB), with a median yield of 4.22x106 CD34+ cells/kg following a single blood volume collection, projecting the collection of 16.9x106 cells/kg in a four-blood-volume apheresis collection session. Motixafortide in combination with natalizumab mobilized a median of 312 CD34+ cells/μl (range 117-447) to the PB, with a median yield of 4.89x106 CD34+ cells/kg following a single blood volume collection, projecting the collection of 19.6x106 CD34+ cells/kg in a four-blood-volume apheresis collection session. The collection yields of motixafortide alone and in combination with natalizumab are encouraging given that hematopoietic stem cell-based gene therapy for sickle cell disease requires sufficient HSCs (16.5-20x106 CD34+ cells/kg) to generate a product. In two subjects with prior plerixafor mobilization, motixafortide alone, and in combination with natalizumab, led to 2.7-2.8 fold higher CD34+ cells/μl mobilization to PB and 2.8-3.2 fold higher CD34+ cells/kg collection yield, respectively, than plerixafor. A second SCD study, sponsored by St. Jude Children's Research Hospital, continues to enroll patients. The study is a multi-center Phase 1 clinical trial evaluating motixafortide for the mobilization of CD34+ HSCs used in the development of gene therapies for patients with SCD. APHEXDA Performance Update APHEXDA generated sales of $2.4 million in the third quarter of 2025, providing royalty revenue to the Company of $0.4 million. Financial Results for the Quarter Ended September 30, 2025 Total revenues for the third quarter of 2025 were $0.4 million, reflecting the royalties paid by Ayrmid from the commercialization of APHEXDA in stem cell mobilization in the U.S. Total revenues in 2025 are not comparable to the same period in 2024, which included a portion of the upfront payment from Gloria Biosciences ($3.2 million) as well as direct commercial sales by BioLineRx ($1.7 million) prior to the Ayrmid transaction in November 2024. Cost of revenues for the third quarter of 2025 was immaterial, compared to cost of revenues of $0.8 million for the third quarter of 2024. The cost of revenues in 2025 reflects sub-license fees on royalties paid by Ayrmid from the commercialization of APHEXDA in stem cell mobilization in the U.S. The cost of revenues in 2024 primarily reflects 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 third quarter of 2025 were $1.7 million, a decrease of $0.8 million, or 33.0%, compared to $2.6 million for the third quarter of 2024. The decrease resulted primarily from lower expenses related to motixafortide due to the out-licensing of U.S. rights to Ayrmid, as well as a decrease in payroll and share-based compensation, primarily due to a decrease in headcount. There were no sales and marketing expenses for the third quarter of 2025, compared to $5.5 million for the third quarter of 2024. The decrease resulted primarily from the shutdown of U.S. commercial operations in the fourth quarter of 2024 following the Ayrmid out-licensing transaction. General and administrative expenses for the third quarter of 2025 were $0.8 million, a decrease of $0.6 million, or 40.2%, compared to $1.4 million for the third quarter of 2024. The decrease resulted primarily from lower payroll and share-based compensation, primarily due to a decrease in headcount, as well as small decreases in a number of general and administrative expenses. Non-operating income (expenses) for the third quarters of 2025 and 2024 primarily relate to fair-value adjustments of warrant liabilities on the Company's balance sheet, as a result of changes in its share price, offset by warrant offering expenses. Net financial income for the third quarter of 2025 was $0.1 million, compared to net financial expenses of $1.2 million for the third quarter of 2024. Net financial income (expenses) for both periods primarily relate to loan interest paid, partially offset by investment income earned on bank deposits and gains on foreign currency (primarily NIS) cash balances due to the strengthening of the NIS against the US dollar during the period. The significant decrease in financial expenses in the 2025 period results from a substantial paydown of the BlackRock loan balance in November 2024, following the transaction with Ayrmid. Net loss for the third quarter of 2025 was $1.0 million, compared to net loss of $5.8 million for the third quarter of 2024. As of September 30, 2025, the Company had cash, cash equivalents, and short-term bank deposits of $25.2 million, sufficient to fund operations, as currently planned, into the first half of 2027. 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 November 26, 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, APHEXDA® (motixafortide), is indicated in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma, and is being commercialized by Ayrmid Ltd. (globally, except Asia) and Gloria Biosciences (in Asia). BioLineRx has retained the rights to develop motixafortide in metastatic pancreatic cancer (PDAC) and has a Phase 2b PDAC trial currently ongoing under a collaboration with Columbia University. In addition, BioLineRx has established a joint venture with Hemispherian AS to develop GLIX1, a first-in-class, oral, small molecule targeting DNA damage response in glioblastoma and other solid tumors, for which a Phase 1/2a clinical trial is planned to be initiated in the first quarter of 2026. Learn more about who we are, what we do, and how we do it at , or on X 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 commercial potential of motixafortide, expectations with regard to clinical trials of motixafortide and GLIX1, 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. 21% more press release views with Request a Demo