Exicure Highlights Recent Achievements and Near-term Strategic Priorities

Rhea-AI Summary

Exicure (Nasdaq: XCUR) reported progress on its lead program burixafor (GPC-100), highlighting that an ongoing Phase 2 multiple myeloma study achieved the primary endpoint in all evaluable patients (10/10) and that the clinical database is locked with topline data expected in Q4 2025. The company is preparing for potential Phase 3 planning and expanding development into sickle cell disease (investigator-sponsored trial discussions) and a planned Phase 1 AML chemosensitization study. Exicure also announced new senior drug-development hires to support clinical and translational execution.

Positive

• Primary endpoint met: 100% (10/10) achieved CD34+ mobilization
• Database locked and topline data due Q4 2025
• Same-day mobilization potential versus overnight competitors

Negative

• Small evaluable cohort: only 10 patients reported for primary endpoint
• Topline outcome pending: final readout could alter conclusions

Insights

Clinical Development Strategist

Burixafor Phase 2 shows promising interim mobilization results; topline readout due in Q4 2025.

Burixafor reported 100% (10/10) achievement of the trial’s primary endpoint for CD34+ stem cell mobilization, including patients with prior daratumumab exposure, and the database is locked ahead of a Q4 2025 topline announcement. The program’s same-day mobilization kinetics and tolerability are presented as differentiators versus agents that require overnight pre-treatment. Preparations for Phase 3 and planned publications in 2026 indicate organized development sequencing.

Key dependencies and risks include the final topline data integrity and the broader dataset beyond the reported interim cohort; the interim sample (10 patients) is small and limits certainty about consistency and safety across larger, more diverse populations. Expansion into Sickle Cell Disease and AML rests on investigator interest and preclinical signals, which must translate into reproducible clinical endpoints.

Watch the confirmed topline readout in Q4 2025, the planned Phase 3 start decision, and the full peer-reviewed publication expected in 2026. Leadership hires strengthen development execution capacity, but regulatory and larger-cohort efficacy/safety data will determine commercial and clinical impact over the next 12–24 months.

Burixafor (GPC-100) Phase 2 Trial in Multiple Myeloma Nears Key Readout

Preparing for Expansion into Sickle Cell Disease and Acute Myeloid Leukemia

New Leadership Appointments Bring Deep Drug Development Expertise

REDWOOD CITY, Calif., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today shared recent progress for its lead program, burixafor (GPC-100), outlined upcoming strategic priorities, and introduced new members of its leadership team who will help drive the company’s next phase of growth.

“The progress we’ve achieved since integrating GPCR Therapeutics earlier this year reflects both the strength and potential of the burixafor program and the dedication of our team,” said Andy Yoo, Chief Executive Officer of Exicure. “As we look ahead to our upcoming Phase 2 data readout and plan for expansion into new indications, I am energized by the opportunity to advance a program that could transform treatment approaches across multiple diseases.”

Burixafor Phase 2 Trial in Multiple Myeloma Nears Key Clinical Readout

Exicure’s ongoing Phase 2 study (NCT05561751) is a randomized, open-label, multicenter trial evaluating burixafor, a small molecule CXCR4 antagonist, in autologous stem cell transplant (ASCT) for multiple myeloma. By blocking CXCR4, burixafor is designed to mobilize hematopoietic stem cells out of the bone marrow and into the bloodstream, where they can be collected for use in transplant procedures.

Interim results to date have been highly encouraging, with 100% of patients (10/10) achieving the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. Burixafor has a faster kinetics of mobilization with a well-tolerated safety profile, enabling same-day administration of the mobilizing agent and leukapheresis. This differentiates burixafor from FDA-approved agents such as plerixafor and motixafortide, which require overnight pre-treatment.

In August, the company announced that the final patient had completed their last study visit. The clinical database has since been locked, and Exicure remains on track to report topline data in Q4 2025. Preparations are also underway for a potential Phase 3 trial.

A full data publication from the ongoing Phase 2 trial (NCT05561751) is anticipated in 2026. In addition, a publication from a previous Phase 2 study (NCT02104427) evaluating burixafor in combination with G-CSF in patients with multiple myeloma, non-Hodgkin lymphoma, and Hodgkin disease, is currently under peer review.

Expanding Development Opportunities

Building on progress in multiple myeloma, Exicure is preparing to expand burixafor into additional indications:

• Sickle Cell Disease: Exicure is in discussions with key clinicians at leading institutions to initiate an investigator-sponsored trial evaluating burixafor for improving stem cell mobilization in patients undergoing gene editing and autologous transplant.
• Acute Myeloid Leukemia (AML): The company is also planning a Phase 1 chemosensitization study in AML. Preclinical data suggest that burixafor may mobilize tumor cells from protective bone marrow niches, potentially enhancing the efficacy of chemotherapy.

New Leadership Strengthen Scientific and Clinical Capabilities

Following its acquisition of GPCR Therapeutics USA earlier this year, Exicure has been advancing the Phase 2 trial with dedicated internal drug development experts. These scientific leaders bring broad experience spanning from basic research to regulatory approval and have decades of proven track records in developing innovative medicines.

Josephine (Pina) Cardarelli, Ph.D. joined as President and Chief Scientific Officer. Dr. Cardarelli is a seasoned drug development executive whose career spans oncology, immunology, pharmacology, and translational medicine. She most recently served as Vice President of Cell Biology & Pharmacology at Bristol-Myers Squibb, where she played a key role in the approvals of Yervoy^® and Opdivo^® and Fucosyl GM-1 antibody program that is currently in Phase 3. Earlier in her career at Medarex, she advanced multiple programs into clinical development, including CXCR4 (Ulocuplumab), CXCL10, CD30, and CD19 antibodies and multiple ADCs. She also identified the lead antibody for the IFNα receptor program that ultimately became AstraZeneca’s FDA-approved Saphnelo™. With more than 100 global patents and over 50 peer-reviewed publications, Dr. Cardarelli brings extensive expertise in biologics, antibody-drug conjugates, IND strategy, and regulatory affairs. She received her Ph.D. in physiology from Albany Medical College.

Niña Caculitan, Ph.D. has been appointed Head of Clinical, overseeing activities spanning manufacturing, regulatory affairs, clinical development, clinical operations and data analysis. She brings over 15 years of experience from both academia and industry, with a strong background in antibody and small molecule chemistry for therapeutics in oncology and immune-mediated disorders. Previously, she was a key contributor to understanding cellular mechanisms of antibody drug conjugate processing at Genentech. Dr. Caculitan earned her Ph.D. in chemistry from the University of California, Berkeley.

Devki Sukhtankar, Ph.D. has joined as Head of Preclinical Research and Translational Medicine, guiding cross-functional translational research efforts with a focus on integrating clinical data and advancing the pipeline into new indications. She brings over 15 years of experience in oncology, neurology and inflammatory diseases, where she has led preclinical programs with a focus on pharmacology and contributed to 20 peer-reviewed publications. Dr. Sukhtankar earned her Ph.D. in cancer biology from the University of Arizona and has extensive experience collaborating with partner organizations to expand therapeutic opportunities.

About Burixafor (GPC-100)
Burixafor (GPC-100) is a highly selective small molecule antagonist of CXCR4, a chemokine receptor that plays a central role in retaining hematopoietic stem cells in the bone marrow niche. By blocking CXCR4, burixafor may enhance the mobilization of these cells into the peripheral blood for collection and use in autologous stem cell transplant (ASCT) procedures. Originally developed by GPCR Therapeutics, Inc., burixafor became part of Exicure’s pipeline following the company’s acquisition in January 2025. In addition to multiple myeloma, burixafor is also being considered in other diseases where improved stem cell mobilization could help enable more efficient and effective treatment approaches, such as sickle cell disease, rare diseases requiring autologous transplant, and cell and gene therapy settings. A chemosensitization trial in AML is also being planned, leveraging burixafor’s mechanism of mobilizing malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy.

About Exicure
Exicure, Inc. (Nasdaq: XCUR) is a clinical-stage biotechnology company developing therapies to address key challenges in hematologic diseases. The company’s lead program, burixafor (GPC-100), is being evaluated for its ability to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. It is also being studied as a potential chemosensitizing agent in acute myeloid leukemia (AML). For more information, visit www.exicuretx.com.

Contact:
Exicure, Inc.
847.673.1700 (Tel)
847.556.6411 (Fax)

FAQ

When will Exicure (XCUR) report topline Phase 2 burixafor data?

Topline Phase 2 burixafor data are expected in Q4 2025.

What were the Phase 2 CD34+ mobilization results for burixafor in multiple myeloma?

Interim Phase 2 results show 100% (10/10) of evaluable patients met the primary CD34+ mobilization endpoint.

Does burixafor enable same-day stem cell collection compared to plerixafor?

Exicure reports burixafor has faster mobilization kinetics enabling same-day administration and leukapheresis versus overnight pre-treatment for plerixafor.

Is Exicure planning additional trials of burixafor beyond multiple myeloma?

Yes. Exicure is preparing an investigator-sponsored sickle cell mobilization study and planning a Phase 1 AML chemosensitization trial.

Has Exicure completed enrollment in the Phase 2 burixafor study (NCT05561751)?

Yes. The company announced the final patient completed visits and the clinical database is locked.

How will recent leadership hires affect Exicure (XCUR) development plans?

New senior hires in scientific, clinical, and translational roles are intended to accelerate clinical execution and IND/Phase planning for burixafor.