Exicure's Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Exicure (XCUR).
Over the past two years, Exicure has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
burixafor. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Burixafor FDA Regulatory Timeline and Events
Burixafor is a drug developed by Exicure for the following indication: In Multiple Myeloma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- burixafor
- Announced Date:
- February 5, 2026
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc. announced the publication of results from a prior Phase 2 clinical study evaluating burixafor (GPC-100/TG-0054), a highly selective CXCR4 inhibitor, in the journal Annals of Hematology.
AI Summary
Exicure, Inc. announced publication in the Annals of Hematology of Phase 2 results for burixafor (GPC-100/TG-0054), a selective CXCR4 inhibitor. In 12 participants, 11 (92%) collected ≥5.0 × 10^6 CD34+ cells/kg within two leukapheresis sessions; six did so in one session. Median neutrophil and platelet engraftment were 12 and 22 days. Burixafor was generally well tolerated, with only two treatment‑related adverse events reported, both low grade.
Peak CD34+ mobilization occurred within one hour, much faster than CXCR4 inhibitors that typically peak at 10–14 hours. This could enable same‑day leukapheresis, simplify logistics, and reduce hospital resource use and patient burden. Combined with G‑CSF, burixafor caused marked rises in white blood cells; lymphocytes increased up to 11‑fold in multiple myeloma participants. These findings support potential use in gene therapy, CAR‑T, and gene‑editing workflows that need efficient peripheral blood cell collection.
Read Announcement- Drug:
- burixafor
- Announced Date:
- January 21, 2026
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc. announced that it will present a poster at the upcoming 2026 Tandem Meetings | Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood and Marrow Transplant Research (CIBMTR), taking place February 4-7, 2026, in Salt Lake City, UT.
Read Announcement- Drug:
- burixafor
- Announced Date:
- December 8, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc. announced positive results from its completed Phase 2 trial evaluating burixafor (GPC-100) in combination with propranolol and granulocyte colony-stimulating factor (G-CSF) for the mobilization of hematopoietic progenitor cells (HPCs) in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT).
AI Summary
Exicure reported positive Phase 2 results of burixafor (GPC-100) given with propranolol and G-CSF to mobilize hematopoietic progenitor cells (HPCs) in multiple myeloma patients preparing for autologous transplant. In the open-label, multicenter study (NCT05561751) presented at ASH, 17 of 19 participants (89.5%) collected ≥2 × 10^6 CD34+ cells/kg within two leukapheresis sessions; two needed a third session. Among those who proceeded to transplant, median time to neutrophil engraftment was 13 days and to platelet engraftment 17.5 days.
Burixafor blocks CXCL12 binding to CXCR4, rapidly moving stem cells into the blood, with peak CD34+ levels seen within one hour—enabling same-day dosing and apheresis. Propranolol appeared to boost mobilization via β2-adrenergic receptor inhibition. Sixteen participants had prior daratumumab; 14 of those (87.5%) met the primary endpoint, including 12 of 14 (85.7%) who had also received lenalidomide. The regimen was well tolerated, with no burixafor-related adverse events above Grade 2.
Read Announcement- Drug:
- burixafor
- Announced Date:
- October 6, 2025
- Indication:
- In Multiple Myeloma
Announcement
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.
AI Summary
Exicure’s Phase 2 trial (NCT05561751) is testing burixafor, a small molecule CXCR4 antagonist, in autologous stem cell transplant for multiple myeloma. This randomized, open-label, multicenter study aims to boost stem cell collection by moving cells from bone marrow into the blood.
So far, all 10 patients have met the main goal of collecting enough CD34+ stem cells, including those previously treated with daratumumab. Burixafor works faster than other agents and allows same-day administration of the mobilizing drug and leukapheresis.
The drug has a well-tolerated safety profile with no serious side effects reported to date. This differs from FDA-approved options like plerixafor and motixafortide, which require overnight pre-treatment.
Enrollment is complete, and the study database is locked. Exicure expects topline results in Q4 2025 and is preparing for a Phase 3 trial if the data support further testing. A full data report is planned.
Read Announcement- Drug:
- burixafor
- Announced Date:
- August 1, 2025
- Target Action Date:
- Q4 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc. announced that the Topline results from the study are expected in the fourth quarter of 2025.
AI Summary
Exicure, Inc. announced that it has completed the last patient’s final visit in its Phase 2 clinical trial of GPC-100 (burixafor) combined with propranolol and G-CSF for multiple myeloma patients undergoing autologous stem cell transplant (ASCT). Topline results from this study are expected in the fourth quarter of 2025.
The randomized, open-label, multicenter trial is designed to see if GPC-100 can boost CD34+ hematopoietic stem cell mobilization from the bone marrow into the blood, making collection by leukapheresis more efficient. Early data presented at ASH 2024 showed that GPC-100 mobilizes stem cells faster than current treatments.
Investigators noted that, unlike traditional methods requiring an injection the night before collection, GPC-100 is given only 45 minutes ahead of the procedure. This shorter timeline may reduce logistical challenges and improve the experience for frail patients.
Exicure is also planning a Phase 1 study in acute myeloid leukemia and exploring collaborations to use GPC-100’s rapid mobilization in cell and gene therapy settings.
Read Announcement- Drug:
- burixafor
- Announced Date:
- August 1, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc announced it has completed the last patient, last visit in its ongoing Phase 2 clinical trial (NCT05561751) evaluating the safety and efficacy of GPC-100 (burixafor) in combination with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT).
AI Summary
Exicure, Inc. announced it has completed the last patient, last visit in its ongoing Phase 2 clinical trial (NCT05561751) of GPC-100 (burixafor) combined with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT). Topline results are expected in the fourth quarter of 2025.
The randomized, open-label, multicenter study is testing whether GPC-100, a small molecule CXCR4 antagonist, can boost CD34+ hematopoietic stem cell mobilization from bone marrow into the blood for collection by leukapheresis. Early data presented at ASH 2024 showed GPC-100 mobilizes stem cells faster than current approved treatments.
Dr. Muthalagu Ramanathan of UMass Memorial Health noted that GPC-100 only needs to be given 45 minutes before stem cell collection, simplifying scheduling and easing the experience for frail myeloma patients. Exicure is also exploring GPC-100 in acute myeloid leukemia and cell and gene therapy settings.
Read Announcement- Drug:
- burixafor
- Announced Date:
- May 5, 2025
- Target Action Date:
- H2 2025
- Estimated Target Date Range:
- July 1, 2025 - December 31, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc announced that Topline results expected H2 2025
AI Summary
Exicure, Inc. announced that topline results for its Phase 2 clinical trial are expected in the second half of 2025. The study is evaluating the safety and efficacy of GPC-100 (burixafor) in combination with propranolol and G-CSF for patients with multiple myeloma who are undergoing autologous stem cell transplant. GPC-100 is designed to improve the mobilization of hematopoietic stem cells, which is key for a successful transplant procedure.
The efficient collection of stem cells can help reduce delays and additional procedures, benefiting both patients and healthcare providers. Exicure’s trial focuses on improving stem cell mobilization, and the upcoming results could lead to new treatment approaches for various indications. The company is also exploring additional studies and potential applications of GPC-100 in other blood disorders, further highlighting its commitment to advancing innovative therapies in hematologic diseases.
Read Announcement- Drug:
- burixafor
- Announced Date:
- May 5, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc. announced it has completed patient enrollment in its ongoing Phase 2 clinical trial (NCT05561751) evaluating the safety and efficacy of GPC-100 (burixafor) in combination with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT).
AI Summary
Exicure, Inc. announced it has finished enrolling patients for its ongoing Phase 2 clinical trial (NCT05561751), which is testing the safety and effectiveness of GPC-100 (burixafor) combined with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT). This randomized, open-label, multicenter study focuses on improving the collection of CD34+ hematopoietic stem cells by mobilizing them from the bone marrow to the peripheral blood. The goal is to enhance the efficiency of stem cell collection during treatment, which can reduce delays, decrease the number of apheresis sessions needed, and lower overall treatment costs. Topline results from the trial are expected in Fall 2025, and the study highlights the potential for GPC-100 to provide a safer and more effective stem cell mobilization option for patients undergoing ASCT.
Read Announcement- Drug:
- burixafor
- Announced Date:
- April 14, 2025
- Indication:
- In Multiple Myeloma
Announcement
Exicure, Inc 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).
AI Summary
Exicure, Inc. announced that GPCR Therapeutics USA, a subsidiary of Exicure, has dosed the 19th patient in its ongoing Phase 2 clinical trial. The study is evaluating GPC-100 (burixafor), a small molecule CXCR4 inhibitor, for mobilizing stem cells in multiple myeloma patients undergoing autologous stem cell transplant. The trial, identified as NCT05561751, is open-label and multi-center, and it tests the safety and effectiveness of GPC-100 combined with propranolol and G-CSF.
Preliminary results are promising, with all patients achieving successful CD34+ stem cell mobilization. Notably, GPC-100 allows for same-day administration of the treatment and leukapheresis, offering faster mobilization compared to other approved agents. The trial is on track to complete patient recruitment at the end of April.
Read Announcement
Exicure FDA Events - Frequently Asked Questions
As of now, Exicure (XCUR) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Exicure (XCUR) has reported FDA regulatory activity for burixafor.
The most recent FDA-related event for Exicure occurred on February 5, 2026, involving burixafor. The update was categorized as "Publication," with the company reporting: "Exicure, Inc. announced the publication of results from a prior Phase 2 clinical study evaluating burixafor (GPC-100/TG-0054), a highly selective CXCR4 inhibitor, in the journal Annals of Hematology."
Currently, Exicure has one therapy (burixafor) targeting the following condition: In Multiple Myeloma.
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FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:XCUR last updated on 2/5/2026 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
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