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:
- 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:
- August 1, 2025
- Estimated Event Date Range:
- October 1, 2025 - December 31, 2025
- Target Action Date:
- Q4 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:
- May 5, 2025
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- H2 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 August 1, 2025, involving burixafor. The update was categorized as "Provided Update," with the company reporting: "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)."
Currently, Exicure has one therapy (burixafor) targeting the following condition: In Multiple Myeloma.
More FDA Event Resources from MarketBeat
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
This page (NASDAQ:XCUR) was last updated on 8/2/2025 by MarketBeat.com Staff
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