Core Insights - Exicure, Inc. announced that results from its Phase 2 study of burixafor will be presented at the 67th ASH Annual Meeting in December 2025 [1][2] - Burixafor is a small molecule CXCR4 antagonist aimed at mobilizing hematopoietic progenitor cells for transplantation in multiple myeloma patients [2][5] Study Details - The Phase 2 trial (NCT05561751) is an open-label, multicenter study assessing burixafor in patients undergoing autologous hematopoietic cell transplantation [2][4] - The combination of burixafor, G-CSF, and propranolol showed an excellent safety profile, with 18 out of 19 patients proceeding to transplantation [3][4] Presentation Information - The oral presentation will take place on December 8, 2025, at 5:45-6:00pm EST, focusing on the safety and efficacy of burixafor in mobilizing hematopoietic progenitor cells [4] Product Overview - Burixafor (GPC-100) is designed to enhance the mobilization of hematopoietic stem cells from the bone marrow into peripheral blood for autologous stem cell transplant procedures [5] - The drug is also being explored for use in other conditions such as sickle cell disease and acute myeloid leukemia [5][6] Company Background - Exicure, Inc. is a clinical-stage biotechnology company focused on developing therapies for hematologic diseases, with burixafor as its lead program [6]

Core Insights - Exicure, Inc. announced that results from its Phase 2 study of burixafor will be presented at the 67th ASH Annual Meeting in December 2025 [1][2] - Burixafor is a small molecule CXCR4 antagonist aimed at mobilizing hematopoietic progenitor cells for transplantation in multiple myeloma patients [2][5] Study Details - The Phase 2 trial (NCT05561751) is an open-label, multicenter study assessing burixafor in patients undergoing autologous hematopoietic cell transplantation [2][4] - The combination of burixafor, G-CSF, and propranolol showed an excellent safety profile, with 18 out of 19 patients proceeding to transplantation [3][4] Presentation Information - The oral presentation will take place on December 8, 2025, at 5:45-6:00pm EST, featuring Dr. Jack Khouri as the presenter [4] Product Information - Burixafor (GPC-100) is designed to enhance the mobilization of hematopoietic stem cells from the bone marrow into peripheral blood for autologous stem cell transplant procedures [5] - The drug is also being explored for use in other diseases, including sickle cell disease and acute myeloid leukemia [5][6] Company Overview - Exicure, Inc. is a clinical-stage biotechnology company focused on developing therapies for hematologic diseases, with burixafor as its lead program [6]

Core Insights - Exicure, Inc. is advancing its lead program, burixafor (GPC-100), with a Phase 2 trial in multiple myeloma nearing key clinical readout and plans for expansion into sickle cell disease and acute myeloid leukemia [1][2] Phase 2 Trial in Multiple Myeloma - The ongoing Phase 2 study (NCT05561751) is a randomized, open-label, multicenter trial evaluating burixafor as a CXCR4 antagonist in autologous stem cell transplant for multiple myeloma [2][3] - Interim results show 100% of patients (10/10) achieved the primary endpoint of successful CD34+ stem cell mobilization, including those previously treated with daratumumab [3] - Burixafor demonstrates faster mobilization kinetics and a well-tolerated safety profile, allowing same-day administration of the mobilizing agent and leukapheresis, unlike FDA-approved agents requiring overnight pre-treatment [3] Upcoming Milestones - The final patient completed their last study visit in August, and the clinical database is locked, with topline data expected in Q4 2025 [4] - A full data publication from the ongoing Phase 2 trial is anticipated in 2026, alongside a publication from a previous Phase 2 study currently under peer review [5] Expansion into New Indications - Exicure is preparing to expand burixafor into sickle cell disease and acute myeloid leukemia (AML) [6][11] - Discussions are ongoing with key clinicians for an investigator-sponsored trial in sickle cell disease to improve stem cell mobilization during gene editing and autologous transplant [7] - A Phase 1 chemosensitization study in AML is planned, leveraging burixafor's ability to mobilize tumor cells from bone marrow niches to enhance chemotherapy efficacy [7] Leadership Appointments - New leadership appointments include Josephine (Pina) Cardarelli, Ph.D. as President and Chief Scientific Officer, bringing extensive drug development experience [7][8] - Niña Caculitan, Ph.D. has been appointed Head of Clinical, overseeing various clinical development activities [9] - Devki Sukhtankar, Ph.D. joins as Head of Preclinical Research and Translational Medicine, focusing on integrating clinical data and advancing the pipeline [10]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 were $300,000, a significant decrease from $6,900,000 in Q1 2024, primarily due to the transition of Effexxa's commercialization to AIRMET [18][19] - Net income for Q1 2025 was $5,100,000, compared to a net loss of $700,000 in Q1 2024, driven by non-operating income from the reevaluation of warrants [22][23] - Cash and cash equivalents as of March 31, 2025, were $26,400,000, providing a runway projected to fund operations through the second half of 2026 [10][23] Business Line Data and Key Metrics Changes - Effexxa generated sales of $1,400,000 in Q1 2025, resulting in $300,000 of royalty revenues to BioLineRx, indicating a return to growth after a transition period [17] - Research and development expenses decreased to $1,600,000 in Q1 2025 from $2,500,000 in Q1 2024, reflecting lower costs related to motixafortide due to the out-licensing agreement [20][21] Market Data and Key Metrics Changes - The overall market for Effexxa is estimated at approximately $300,000,000, with expectations that BioLineRx could capture a substantial portion of this market [43][44] - The company is focused on expanding its pipeline in oncology and rare diseases, leveraging expertise in drug development [7][8] Company Strategy and Development Direction - The company has shifted focus to evaluating early clinical stage and late preclinical stage therapeutic assets in oncology and rare diseases, aiming to leverage its drug development expertise [7][8] - The out-licensing agreement with AIRMET allows BioLineRx to return to its roots as an innovative company in complex drug development [6][10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing development of metixafortide in treating pancreatic cancer, highlighting strong scientific rationale for continued investment in this area [11][12] - The company is actively seeking new assets for in-licensing and development to expand its pipeline and create additional value for shareholders [68] Other Important Information - The company has reduced its operating cash burn by over 70%, from over $40,000,000 annually to less than $12,000,000 as of 2025 [10] - The ongoing development of metixafortide in sickle cell disease is supported by two phase one trials, with promising initial results [9][12] Q&A Session Summary Question: Does the cash runway include new asset development costs? - Yes, it does [27] Question: Have the costs from the November restructuring impacted the P&L? - Those costs were fully accrued by the end of 2024, with no further anticipated costs in 2025 [28] Question: What is the long-term opportunity for Effexxa? - The market is estimated at $300,000,000, with potential revenues in the $100 million range for BioLineRx [43][44] Question: What is the communication strategy for interim analysis results? - The company is limited by Columbia University's communication strategy and will release data once published by them [36] Question: What are the chances of regulatory approval based on the current study? - Regulatory approval is unlikely based on the current study, as it primarily measures progression-free survival [37] Question: What is the visibility on future milestone payments? - Milestones are primarily commercial-based, with some geographic and regulatory milestones included in the agreements [56][60] Question: What type of assets is the company looking for? - The company is looking for assets with a short clinical development pathway and lean programs to reach value-creating events [64][65]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 were $300,000, a significant decrease from $6,900,000 in Q1 2024, primarily due to the transition of Effexxa's commercialization to AIRMID [17][18] - Net income for Q1 2025 was $5,100,000, compared to a net loss of $700,000 in Q1 2024, driven by non-operating income related to the reevaluation of warrants [21] - Cash and cash equivalents as of March 31, 2025, stood at $26,400,000, providing a cash runway projected to fund operations through the second half of 2026 [21][9] Business Line Data and Key Metrics Changes - Effexxa generated sales of $1,400,000 in Q1 2025, resulting in $300,000 of royalty revenues to BioLineRx, with expectations of growth resuming after a transition period [15] - Research and development expenses decreased to $1,600,000 in Q1 2025 from $2,500,000 in Q1 2024, reflecting lower costs associated with motixafortide due to the out-licensing agreement [19] Market Data and Key Metrics Changes - The overall market for Effexxa is estimated at approximately $300,000,000, with expectations that BioLineRx could capture a substantial portion of this market, translating to potential revenues in the $100 million range [39][40] Company Strategy and Development Direction - The company is focused on evaluating early clinical stage and late preclinical stage therapeutic assets in oncology and rare diseases, leveraging expertise in drug development [6] - The strategic partnership with AIRMID is expected to provide significant commercial milestones and royalties, allowing the company to return to its roots in complex drug development [4][5] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing development of metixafortide in pancreatic cancer, highlighting strong scientific rationale for continued investment in this area [10][11] - The company aims to identify new assets for in-licensing and development to expand its pipeline and create additional value for shareholders [62] Other Important Information - The company has reduced its operating cash burn by over 70%, from over $40,000,000 annually to less than $12,000,000 as of 2025 [9] - The ongoing trials for sickle cell disease using metixafortide are expected to provide valuable data, with results from two phase one trials currently awaited [8] Q&A Session Summary Question: Does the cash runway include new asset development costs? - Yes, the cash runway includes potential costs associated with new assets, which may be offset by milestones and royalties [24] Question: Have the restructuring costs been fully accounted for in the P&L? - Yes, the restructuring costs were fully accrued by the end of 2024, with no further anticipated costs in 2025 [25] Question: What are the expectations for Effexxa's long-term opportunity? - The market for Effexxa is estimated at $300,000,000, with expectations of capturing a significant portion, leading to potential revenues exceeding $100 million [39][40] Question: What is the communication strategy for interim analysis results? - The communication strategy is aligned with Columbia University, which is leading the study, and data will be released following their publication [33] Question: What are the thoughts on the current trajectory of the cell and gene therapy fields? - There are headwinds in the gene therapy space, but the company views Effexxa as a long-term play with significant upside potential [42][45] Question: What is the visibility on future milestone payments? - Milestone payments are primarily commercial-based, with some geographic and regulatory milestones included in the agreements [51][55]

Financial Data and Key Metrics Changes - Total 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 [30] - Net loss for the year ended December 31, 2024, was $9.2 million compared to a net loss of $60.6 million for the year ended December 31, 2023 [34] - Cash, cash equivalents, and short-term bank deposits as of December 31, 2024, were $19.6 million, with approximately $29 million on a pro forma basis after financing completed in early January 2025 [34] Business Line Data and Key Metrics Changes - APHEXDA achieved a 10% market share of total CXCR4 inhibitor usage in the U.S. within less than 11 months of being on the market [26] - Generated more than $6 million of net APHEXDA product revenue in the U.S. through the closing of the Ayrmid transaction on November 21 [27] Company Strategy and Development Direction - The company has shifted its strategy to focus on evaluating early clinical stage and late pre-clinical stage therapeutic assets in oncology and rare diseases [6][7] - The licensing agreement with Ayrmid Pharma Ltd. allows the company to return to its roots in complex drug development while leveraging expertise in drug development [6][9] - The company aims to help as many patients as possible while creating enduring value for shareholders [20] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about evaluating several promising candidates and the potential for definitive announcements within the year [8] - The company has reduced its ongoing operating cash burn by approximately 70%, from over $40 million annually to approximately $12 million annually [18] - The company believes it is well-positioned to advance motixafortide in solid tumor indications while evaluating additional assets in oncology and rare diseases [19] Other Important Information - The Ayrmid agreement generated $10 million in upfront payments and potential milestones of $87 million, along with double-digit sales royalties ranging from 18% to 23% [16] - The company has shut down its U.S. operations and implemented a headcount reduction in Israel, transitioning a substantial portion of its commercial team to Ayrmid [17] Q&A Session Summary Question: Any more color on meaningful progress in evaluating assets? - Management indicated that they are having meaningful discussions regarding early-stage clinical assets in oncology and rare diseases but could not provide a timeline for announcements [38] Question: How are internal programs looking, especially with WashU and sickle cell? - Management noted that a significant win would be mobilizing enough cells in one cycle for gene therapy, reducing the number of apheresis sessions required [43][44] Question: Commentary on increased traction of APHEXDA for multiple myeloma? - Management expressed confidence in Ayrmid's team and their marketing efforts, stating that the transition has gone smoothly [49] Question: How many assets have been looked at in the acquisition process? - Management stated that they have looked at thousands of molecules over the company's history, focusing on early clinical stage assets in oncology and rare diseases [56][58] Question: Allocation of expenses during the acquisition process? - Management confirmed that expenses would initially be higher during the search process but would shift towards R&D once assets are acquired [59] Question: Internal versus external efforts in the acquisition process? - Management indicated that the majority of the work is done in-house, with external consultants used as needed for specific expertise [64]

Financial Data and Key Metrics Changes - Total 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 [30] - Net loss for the year ended December 31, 2024, was $9.2 million compared to a net loss of $60.6 million for the year ended December 31, 2023 [34] - Cash, cash equivalents, and short-term bank deposits as of December 31, 2024, were $19.6 million, with approximately $29 million on a pro forma basis after financing completed in early January 2025 [34] Business Line Data and Key Metrics Changes - Revenues in 2024 included $15 million from upfront and milestone payments under the Gloria license agreement and $6 million of net APHEXDA product revenues [31] - 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 [32] - 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 [33] Market Data and Key Metrics Changes - APHEXDA achieved a 10% market share of total CXCR4 inhibitor usage in the U.S. within less than 11 months of being on the market [26] - More than $6 million of net APHEXDA product revenue was generated in the U.S. through the closing of the Ayrmid transaction on November 21 [27] Company Strategy and Development Direction - The company has shifted its strategy to focus on evaluating early clinical stage and late pre-clinical stage therapeutic assets in oncology and rare diseases [6][7] - The exclusive license agreement with Ayrmid Pharma Ltd. for motixafortide allows the company to return to its roots in complex drug development while benefiting from Ayrmid's commercial potential [9][10] - The company aims to help as many patients as possible while creating enduring value for shareholders [20] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about evaluating promising candidates and making definitive announcements within the year [8] - The company has reduced its ongoing operating cash burn by approximately 70%, from over $40 million annually to approximately $12 million annually [18] - The company believes it is well-positioned to advance motixafortide in solid tumor indications while evaluating additional assets in oncology and rare diseases [19] Other Important Information - The Ayrmid agreement generated $10 million in upfront payments and potential milestones of $87 million, along with double-digit sales royalties ranging from 18% to 23% [16] - The company has shut down its U.S. operations and implemented a headcount reduction in Israel, allowing for a streamlined organization [17] Q&A Session Summary Question: Any more color on meaningful progress in evaluating assets? - Management indicated that while there are meaningful discussions ongoing, specific timelines for announcements cannot be provided [38] Question: How are internal programs, especially with WashU and sickle cell, looking? - Management noted that a significant win would be mobilizing enough cells in one cycle for gene therapy, reducing the number of apheresis sessions [43][44] Question: Commentary on increased traction of APHEXDA for multiple myeloma? - Management expressed confidence in Ayrmid's team and their marketing efforts, stating that the transition has gone smoothly [49] Question: How many assets have been looked at in the acquisition process? - Management stated that they have looked at thousands of molecules over the company's history, focusing on early clinical stage assets in oncology and rare diseases [56][58] Question: Allocation of expenses during the acquisition process? - Management confirmed that expenses would initially be higher during the search process, shifting towards R&D as acquisitions are made [59] Question: Internal versus external efforts in the acquisition process? - Management indicated that the majority of the work is done in-house, with external consultants used as needed [64]