Clinical Trial Shows 100% Success Rate for Exicure's Multiple Myeloma Drug

Exicure, Inc. (Nasdaq: XCUR) Nears Completion of Phase 2 Study of GPC-100 in Multiple Myeloma

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04/14/2025 - 06:11 PM

REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Exicure, Inc. (Nasdaq: XCUR) today announced that GPCR Therapeutics USA, a subsidiary of Exicure Inc., has dosed the 19th patient in its ongoing Phase 2 clinical trial evaluating GPC-100 (burixafor), a small molecule CXCR4 inhibitor, for the mobilization of stem cells in multiple myeloma (MM) patients undergoing autologous stem cell transplant (ASCT) (NCT05561751).

The study is an open-label, multi-center trial evaluating the safety and efficacy of GPC-100 and propranolol in combination with G-CSF. Data from an interim analysis of 10 patients were previously presented at the 2024 American Society of Hematology (ASH) Annual Meeting.

Preliminary results are encouraging, with 100% of patients achieving the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. Notably, GPC-100 enabled same-day administration of the mobilizing agent and leukapheresis, offering faster kinetics compared to FDA-approved agents such as plerixafor and motixafortide, which require overnight pre-treatment. Median times to neutrophil and platelet engraftment were 11 and 14 days, respectively, which is consistent with standard of care.

This trial is expected to finish patient recruitment at the end of April.

About Exicure, Inc.

Exicure, Inc. has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its restructuring and suspension of clinical and development activities, the Company is exploring strategic alternatives to maximize stockholder value. For further information, see .