Exicure Highlights Recent Achievements and Near-term Strategic Priorities

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]