Exicure Announces Publication in Annals of Hematology Highlighting Rapid Stem Cell Mobilization with Burixafor in Patients with Multiple Myeloma and Lymphoma Undergoing Transplant

Exicure (Nasdaq: XCUR) announced publication in Annals of Hematology reporting Phase 2 data for burixafor (GPC-100/TG-0054), a selective CXCR4 inhibitor.

Rhea-AI Summary

Exicure (Nasdaq: XCUR) announced publication in Annals of Hematology reporting Phase 2 data for burixafor (GPC-100/TG-0054), a selective CXCR4 inhibitor. In a 12-participant study, 11 of 12 (92%) collected ≥5.0×10⁶ CD34+ cells/kg within two sessions; six achieved this in one session. Median neutrophil and platelet engraftment were 12 and 22 days. Peak CD34+ mobilization occurred within one hour, enabling same-day leukapheresis and marked lymphocyte increases (up to 11-fold in MM patients). Burixafor was generally well tolerated with two low-grade treatment-related adverse events. Exicure is advancing further Phase 2 studies including a propranolol combination.

Positive

• 92% of participants collected ≥5.0×10⁶ CD34+ cells/kg within two sessions
• Six participants achieved target collection in a single leukapheresis session
• Peak CD34+ mobilization occurred within 1 hour, enabling same-day leukapheresis
• Lymphocyte counts increased up to 11-fold in multiple myeloma patients

Negative

• Small sample size of 12 participants limits statistical robustness and generalizability
• Safety evidence is limited—only two low-grade treatment-related adverse events reported in a small cohort

Argus Feb 5 session

+4.41% close to close Open Argus

Details

News Market Reaction – XCUR

On Feb 5, the day this news came out, XCUR closed 4.41% above the previous close.

Data tracked by StockTitan Argus for the Feb 5 session.

Market Context

This announcement adds peer-reviewed validation of burixafor’s rapid mobilization profile, with 92% ...

Analysis

This announcement adds peer-reviewed validation of burixafor’s rapid mobilization profile, with 92% of patients meeting the cell collection endpoint and engraftment medians of 12 and 22 days. It complements recent Phase 2 topline results and a milestone-triggering clinical study report. Against a backdrop of past disclosures about limited cash and notable insider selling, investors may focus on how these data support future partnering or funding discussions.

Key Figures

Participants: 12 patients Primary endpoint success: 11 of 12 (92%) CD34+ collection target: ≥5.0 × 10^6 CD34+ cells/kg +5 more

Participants

12 patients

Phase 2 multi-center open-label study (NCT02104427)

Primary endpoint success

11 of 12 (92%)

Collected target CD34+ cells within two leukapheresis sessions

CD34+ collection target

≥5.0 × 10^6 CD34+ cells/kg

Primary endpoint threshold within two leukapheresis sessions

Single-session success

6 patients

Achieved CD34+ collection target in one leukapheresis session

Neutrophil engraftment

12 days (median)

Time to neutrophil engraftment after ASCT

Platelet engraftment

22 days (median)

Time to platelet engraftment after ASCT

Mobilization peak time

Within 1 hour

Peak CD34+ mobilization after burixafor dosing

Lymphocyte increase

Up to 11-fold

Increase in MM patient lymphocyte counts within hours of dosing

Historical Context

5 past events · Latest: Jan 21

5 events

1. Jan 21

   Phase 2 data poster

   24h Move

   +2.3%

   Positive Phase 2 burixafor data with high primary endpoint achievement and safety.

2. Dec 08

   Topline Phase 2 data

   24h Move

   +8.8%

   Positive topline Phase 2 burixafor results with rapid CD34+ mobilization and tolerability.

3. Nov 07

   Q3 2025 earnings

   24h Move

   -2.3%

   Low cash balance, ongoing losses, and statement that substantial additional financing is needed.

4. Nov 03

   ASH oral data notice

   24h Move

   -4.3%

   Announcement of completed Phase 2 burixafor data to be presented as ASH oral presentation.

5. Oct 06

   Program update

   24h Move

   +2.4%

   Update on burixafor progress, Phase 2 success, and plans for expansion into new indications.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

cxcr4, g-csf, autologous stem cell transplantation, leukapheresis, +4 more

8 terms

cxcr4 medical

"burixafor (GPC-100/TG-0054), a highly selective CXCR4 inhibitor, in the journal"

CXCR4 is a protein on the surface of many cells that acts like a lock for a specific chemical signal, guiding cells where to go and how to behave. It matters to investors because drugs that block or mimic this receptor can change immune responses, stop cancer cells from spreading, help mobilize stem cells for transplants, or affect viral entry; thus CXCR4-targeted therapies can drive clinical value, regulatory milestones, and licensing or partnership opportunities.

g-csf medical

"evaluating burixafor in combination with granulocyte colony-stimulating factor (G-CSF) in patients"

G‑CSF (granulocyte colony-stimulating factor) is a manufactured protein that prompts the bone marrow to produce neutrophils, a type of white blood cell that protects patients from infection after chemotherapy, bone marrow transplant, or severe illness. For investors, G‑CSF products matter because they fulfill a steady, clinical need—like safety gear hospitals must replenish—so changes in approvals, patents, manufacturing capacity, or competing biosimilars can meaningfully affect sales and company valuations.

autologous stem cell transplantation medical

"Hodgkin disease (HD) undergoing autologous stem cell transplantation (ASCT). In the study,"

A procedure that collects a patient’s own stem cells, stores them, and then returns them after intensive treatment that wipes out diseased or malfunctioning cells. Think of it like saving seeds before clearing a garden and replanting them afterward to restore growth. Investors watch this because demand, costs, and outcomes influence sales of related drugs, devices and services, reimbursement decisions, and the commercial potential of new therapies.

leukapheresis medical

"enables same-day leukapheresis, offering the potential to simplify treatment logistics,"

Leukapheresis is a medical procedure that separates and removes white blood cells from a person’s blood, like running blood through a specialized filter to pull out a specific ingredient. It matters to investors because those collected cells are often the raw material for advanced therapies and clinical trials, so the availability, cost, production speed, and regulation of leukapheresis can directly affect a biotech company’s ability to manufacture treatments and generate revenue.

car-t medical

"applicability in gene therapy, CAR-T, and other gene-editing workflows that require"

CAR-T is a type of cancer therapy that reprograms a patient’s own immune cells to seek and destroy specific cancer cells, like teaching guard dogs a new scent to track intruders. It matters to investors because CAR-T treatments can command high prices, drive strong revenue for successful developers, and carry regulatory and manufacturing risks that can sharply affect a company’s valuation and long-term growth prospects.

beta-adrenergic receptors medical

"crosstalk between CXCR4 and beta-adrenergic receptors, suggesting the potential for"

Proteins on the surface of many cells that act like locks while adrenaline and similar chemicals act as keys to change how the cell behaves — for example speeding or slowing the heartbeat, opening airways, or changing energy use. Investors care because these receptors are common drug targets; a medicine that safely blocks or activates them can become a large product, affect safety profiles, and drive regulatory approval, market value, or liability for healthcare companies.

hematopoietic cell transplantation medical

"multiple myeloma undergoing autologous hematopoietic cell transplantation (NCT05561751)."

A medical procedure that replaces a patient’s blood‑forming stem cells with healthy cells from the patient or a donor to rebuild the immune system and restore blood production. Think of it as replanting a damaged garden with new seeds so healthy plants can grow again; it matters to investors because the procedure’s demand, clinical success, regulatory approvals, supply chain for donor material or cell products, and treatment costs directly affect the revenues and risks of drug makers, medical device firms, hospitals and insurers.

gene-editing medical

"CAR-T, and other gene-editing workflows that require efficient peripheral blood cell"

Gene-editing is a set of laboratory techniques that change an organism’s DNA by adding, removing or altering specific genetic instructions—think of it as editing words in a document to fix a sentence. For investors, it matters because these tools can create new treatments, improve agricultural crops or reduce manufacturing costs, potentially driving product value, regulatory risk, research milestones and long-term revenue prospects for companies involved.

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Phase 2 data demonstrate differentiated CXCR4 inhibition kinetics enabling same-day cell collection, with rapid lymphocyte mobilization supporting potential therapeutic applications beyond transplant

REDWOOD CITY, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today 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.

The manuscript, titled “Burixafor, a CXCR4 Inhibitor with a Differentiated Kinetics Profile: Results of a Phase 2 Study for Rapid Cell Mobilization in Multiple Myeloma and Lymphoma Patients Undergoing Transplant,” reports results from a 12-participant, multi-center, open-label Phase 2 study (NCT02104427) evaluating burixafor in combination with granulocyte colony-stimulating factor (G-CSF) in patients with multiple myeloma (MM), non-Hodgkin’s lymphoma (NHL), and Hodgkin disease (HD) undergoing autologous stem cell transplantation (ASCT).

In the study, 11 of 12 participants (92%) met the primary endpoint of collecting ≥5.0 × 10⁶ CD34+ cells/kg within two leukapheresis sessions, with six participants achieving the target in a single session. Median time to neutrophil and platelet engraftment was 12 and 22 days, respectively. Burixafor was generally well tolerated, with only two treatment-related adverse events reported, both low grade.

Notably, peak mobilization of CD34+ cells occurred within one hour of burixafor administration, substantially faster than currently approved CXCR4 inhibitors, which typically peak 10-14 hours after dosing. This rapid mobilization profile enables same-day leukapheresis, offering the potential to simplify treatment logistics, reduce hospital resource utilization, and minimize patient burden.

In addition to robust stem cell mobilization, burixafor in combination with G-CSF led to marked increases in circulating white blood cell subsets, including lymphocytes. In participants with MM, lymphocyte counts increased by up to 11-fold within hours of burixafor administration, supporting potential applicability in gene therapy, CAR-T, and other gene-editing workflows that require efficient peripheral blood cell collection.

“ASCT remains a treatment cornerstone for multiple myeloma and certain lymphomas. Efficient mobilization of peripheral blood progenitor cells is critical to achieving reliable engraftment while minimizing patient burden and overall healthcare costs,” said Michael Schuster, MD, Clinical Professor of Medicine at Stony Brook University and a study author. “In this study, burixafor demonstrated enhanced mobilization kinetics that led to significantly faster attainment of stem cell collection goals, highlighting its differentiated profile and potential to meaningfully improve the efficiency of cell collection for ASCT. Beyond transplant, rapid and predictable mobilization of CD34-positive cells and lymphocytes may also be highly relevant for emerging gene-based and gene-editing approaches, including those being explored for conditions such as sickle cell disease.”

Exicure continues to advance burixafor’s clinical development and has recently completed an additional Phase 2 study evaluating burixafor in combination with G-CSF and propranolol in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (NCT05561751). The addition of propranolol was informed by previously published preclinical data demonstrating functional crosstalk between CXCR4 and beta-adrenergic receptors, suggesting the potential for enhanced mobilization through dual pathway blockade. Positive topline data from this recent study were presented at the American Society of Hematology (ASH) Annual Meeting in December 2025 and the Tandem Meetings in February 2026, further supporting burixafor’s differentiated and rapid mobilization profile in the transplant setting.

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 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 stem cell mobilization into the peripheral blood to support collection and use in autologous stem cell transplantation (ASCT).

Burixafor is being evaluated for its potential to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. In addition, Exicure is planning a chemosensitization trial in acute myeloid leukemia (AML), leveraging burixafor’s ability to mobilize malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy. Burixafor became part of Exicure’s pipeline following the company’s acquisition of GPCR Therapeutics, Inc. in January 2025. For more information, visit www.exicuretx.com.

Media Contact:
Sarah Ellinwood, PhD
Kendall Investor Relations
sellinwood@kendallir.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What were the key Phase 2 burixafor results reported by Exicure (XCUR) on February 5, 2026?

According to the company, the Phase 2 study (12 participants) showed 11 of 12 (92%) collected ≥5.0×10⁶ CD34+ cells/kg within two sessions, six in one session, with median neutrophil and platelet engraftment of 12 and 22 days, respectively.

How does burixafor’s mobilization speed compare to other CXCR4 inhibitors for XCUR investors?

According to the company, burixafor peaked in CD34+ mobilization within one hour versus typical 10–14 hour peaks for approved CXCR4 inhibitors, supporting same-day leukapheresis and potential reductions in hospital resource use and patient burden.

Does burixafor improve cell collection for gene therapies and CAR-T manufacturing (XCUR)?

According to the company, burixafor plus G-CSF produced marked lymphocyte increases—up to 11-fold in multiple myeloma patients—suggesting potential applicability for gene therapy, CAR-T, and gene-editing workflows requiring efficient peripheral blood cell collection.

What safety findings did Exicure report for burixafor in the Phase 2 study (XCUR)?

According to the company, burixafor was generally well tolerated in the 12-participant study, with only two treatment-related adverse events reported and both described as low grade; larger studies are needed to fully define safety.

What are Exicure’s next clinical steps for burixafor after the Annals of Hematology publication (XCUR)?

According to the company, Exicure has completed an additional Phase 2 study combining burixafor with G-CSF and propranolol (NCT05561751) and presented positive topline data at ASH December 2025 and the Tandem Meetings in February 2026 to support further development.