burixafor (GPC-100) / TaiGen, GPCR Therap, Exicure, Adbiotech - LARVOL DELTA

An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma (ASH 2025) - P2 | "Mediantimes to neutrophil and platelet engraftment were 11 days (range 11-17 days) and 15 days (range 11-27days), respectively.ConclusionsBurixafor, in combination with propranolol and G-CSF, demonstrated an excellent safety profile andeffectively mobilized sufficient HPCs for AHCT, including patients previously treated with lenalidomideand daratumumab. Notably, burixafor enabled same-day administration with leukapheresis, offering akey advantage over other CXCR4 inhibitors, such as plerixafor and motixafortide through its rapidmobilization kinetics. Its favorable safety profile, characterized by minimal adverse events, supporting itspotential clinical utility."

Clinical • IO biomarker • P2 data • Cardiovascular • Constipation • Diabetes • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hypotension • Multiple Myeloma • Musculoskeletal Diseases • Musculoskeletal Pain • CD34 • CXCR4

Exicure…announced that it has entered into a co-development agreement with Adbiotech…, a Korea-based biotechnology company, to explore combination therapies based on Burixafor (GPC-100) across multiple therapeutic areas (GlobeNewswire) - "The collaboration will focus on evaluating combination strategies involving Burixafor (GPC-100) in indications including sickle cell disease (SCD), acute myeloid leukemia (AML), and solid tumors. Under the agreement, Adbiotech will conduct in vivo studies and support preclinical validation and translational research, while Exicure will provide Burixafor and lead clinical and regulatory strategy....The parties intend to conduct in vivo validation studies and, subject to further agreement, may advance selected programs into IND-enabling studies and clinical trials. The parties also intend to secure funding to support the advancement of future clinical development."

Licensing / partnership • Acute Myelogenous Leukemia • Sickle Cell Disease • Solid Tumor

Toward optimizing CXCR4 inhibition with beta adrenergic blockade to enhance chemotherapy response in acute myeloid leukemia (AACR 2024) - "The CXCR4 inhibitor plerixafor is used to increase mobilization of peripheral blood stem cells in combination with filgrastim...These have included use of plerixafor with 7+3 or MEC, BL-8040 with cytarabine, and ulocuplumab with MEC...The objective of this preclinical study is to identify the AML patient population most likely to respond to CXCR4 inhibition, and the role of combined beta-adrenergic blockade, as the latter has been shown pre-clinically to augment mobilization of HSCs when combined with a CXCR4 inhibitor, GPC-100 (Sukhtankar et al... These studies support further investigation of whether simultaneous blockade of CXCR4 and ADBR2 may potentiate chemotherapy response in AML by limiting microenvironment mediated chemotherapy protection and reveal that patients with new diagnosis may be more susceptible to this approach."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ADRB2 • CD123 • CD34 • CXCL12 • CXCR4 • FLT3 • IL3RA • NPM1 • PTPRC

Ph2, Study to Assess the Safety and Efficacy of GPC 100 and Propranolol With and Without G-CSF for the Mobilization of Stem Cells in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplant (clinicaltrials.gov) - P2 | N=30 | Completed | Sponsor: GPCR Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

An Open-Label, Multi-Center Phase 2 study to Assess the Safety and Efficacy of Burixafor (GPC-100) and Propranolol with G-CSF for the Mobilization of Hematopoietic Progenitor Cells in Patients with Multiple Myeloma (TCT-ASTCT-CIBMTR 2026) - P2 | "All received lenalidomide-based induction therapy, with 70% also receiving daratumumab. Assess the efficacy of the above treatment combination. Demonstrate the fast kinetics of mobilization of burixafor, allowing for leukapheresis within an hour after burixafor administration, as opposed to 10-12 hours with other CXCR4 inhibitors, plerixafor and motixafortide."

Clinical • IO biomarker • P2 data • Cardiovascular • Constipation • Diabetes • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hypotension • Multiple Myeloma • Musculoskeletal Diseases • Musculoskeletal Pain • CD34 • CXCR4

Exicure Announces Publication in Annals of Hematology Highlighting Rapid Stem Cell Mobilization with Burixafor in Patients with Multiple Myeloma and Lymphoma Undergoing Transplant (The Manila Times) - "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...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."

P2 data • Hodgkin Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma

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 Manila Times)

Trial completion • Multiple Myeloma

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. (PubMed, Ann Hematol) - No abstract available

Journal • P2 data • Hematological Malignancies • Lymphoma • Multiple Myeloma • Oncology • Transplantation • CXCR4

Exicure to Present Data from Burixafor Phase 2 Trial at the 2026 Tandem Meetings (GlobeNewswire) - "The presentation will serve as an encore of data reported previously, highlighting results from Exicure’s open-label, multicenter Phase 2 trial (NCT05561751) evaluating burixafor in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT)."

P2 data • Bone Marrow Transplantation • Multiple Myeloma

Exicure Presents Positive Topline Phase 2 Data for Burixafor in Multiple Myeloma at 2025 ASH Annual Meeting (GlobeNewswire) - "In the open-label, multicenter Phase 2 trial (NCT05561751), 17 of 19 participants (89.5%) achieved the primary endpoint of collecting ≥2 × 10⁶ CD34+ cells/kg within two leukapheresis sessions....Notably, 16 of 19 participants (84.2%) had prior exposure to daratumumab, a therapy associated with reduced mobilization, yet 14 of those 16 participants (87.5%) achieved the primary endpoint, including 12 of 14 participants (85.7%) who had received both daratumumab and lenalidomide....Burixafor administered in combination with propranolol and G-CSF was well tolerated and demonstrated an excellent safety profile."

P2 data • Multiple Myeloma

Combination of Propranolol and a Novel CXCR4 Antagonist Burixafor (GPC-100) for Enhanced Hematopoietic Cell Mobilization (ASH 2023) - P2 | "In cell-based assays, co-activation of both receptors with their respective ligands, CXCL12 and epinephrine, leads to a synergistic increase in calcium flux (Figure 1) as well as ß-arrestin recruitment, indicating functional consequences of the heteromerization...For comparison, plerixafor increased CD8+ T cell mobilization by only 2-fold...We anticipate that our clinical studies can fill the gap in the current standard of care without adding a significant financial burden or co-morbidities. Furthermore, enhanced mobilization of CD8+ T cells can provide extended opportunities for improved adoptive cell therapies."

Genetic Disorders • Hematological Disorders • Hematological Malignancies • Immunology • Lymphoma • Multiple Myeloma • Oncology • Sickle Cell Disease • CD8 • CXCL12

Trial in Progress: An Open-Label, Multi-Center Phase 2 Study to Assess the Safety and Efficacy of Burixafor (GPC-100) and Propranolol with and without G-CSF for the Mobilization of Stem Cells in Patients with Multiple Myeloma Undergoing Autologous Stem Cell Transplant (ASH 2023) - P2 | "Key secondary objectives include: assessment of safety and tolerability of GPC-100 and propranolol with and without G-CSF, evaluation of the pharmacodynamics by determining levels of circulating CD34 + cell counts and levels of the chemokine CXCL12 in peripheral blood, and assessment of mean time to ANC and platelet count recovery after ASCT. Exploratory objectives are also included to discover key biomarkers for patient selection, to evaluate cancer/stem/immune cell profiles, and to evaluate treatment impact on patient quality of life."

Clinical • P2 data • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Immunology • Lymphoma • Multiple Myeloma • Oncology • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease • Transplantation • CD34 • CXCL12 • CXCR4

Combined CXCR-4 Inhibition with Novel Agent GPC-100 (burixafor) and Beta 2 Adrenergic Receptor Blockade Enhances Cytarabine Response for Acute Myeloid Leukemia Blasts on Stroma (ASH 2024) - P1 | "The CXCR4 inhibitor plerixafor improves yields of mobilized normal hematopoietic stem cells (HSCs) in combination with filgrastim (G-CSF). Clinical trials of CXCR4 inhibitors have been conducted to mobilize AML out of the protected BM niche, including plerixafor with 7+3 or MEC, BL-8040 with cytarabine (araC), LY2510924 with idarubicin/araC, and ulocuplumab (human IgG4 antibody) with MEC...In addition, high throughput drug screening of AML on stroma, but not in suspension or on CXCL12 coated plates, revealed that combination of the CXCR4 inhibitor GPC-100 and beta blocker propranolol, with araC, increased drug sensitivity (reduced IC50 by ≥4 to >10 fold) as compared to araC alone for AML cells on HS-5 human stromal cell line or autologous patient mesenchymal stromal cells...Conclusions : These studies support further investigation of whether simultaneous blockade of CXCR4 and ADRB2 may potentiate chemotherapy response in AML, perhaps by disrupting..."

Stroma • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ADRB2 • CD58 • CXCL12 • CXCR4 • FLT3 • NPM1 • TP53

An Open-Label, Multi-Center Phase 2 Study to Assess the Safety and Efficacy of Burixafor (GPC-100) and Propranolol with G-CSF for the Mobilization of Stem Cells in Patients with Multiple Myeloma Undergoing Autologous Stem Cell Transplant (ASH 2024) - P2 | "Newer therapies, such as daratumumab, may also have a negative impact on mobilization, supporting a need for an alternative mobilization regimen. Notably, burixafor allowed for same day administration of both mobilizing agent and leukapheresis. This quick kinetics of mobilization is differentiated from the FDA approved plerixafor or motixafortide, which require overnight pre-treatment prior to leukapheresis."

Clinical • P2 data • Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34 • CXCR4

Strategy to Improve Functional T-Cell Yield in Peripheral Blood By Co-Inhibition of Beta-2 Adrenergic and CXCR4 Signaling Pathways (ASH 2024) - P2 | "A significant increase in PB lymphocyte count was only observed when propranolol was combined with GPC-100, but not with AMD3100 or BL8040...Treatment of stimulated PBMC with epinephrine inhibited IFNy release, which was fully reversed when propranolol and GPC-100 were added...Therefore, we propose that propranolol can be safely combined with GPC-100 for improving CAR-T efficiency. Comprehensive analysis of immune cell subsets mobilized by propranolol and GPC-100 in our clinical study (NCT05561751) is also ongoing to evaluate this treatment for improving current approaches to cell and gene therapy."

Gene Therapies • Hematological Malignancies • Multiple Myeloma • Oncology • CD4 • CD8 • CXCR4 • GZMB • IFNG

New Burixafor Clinical Data to be Presented as an Oral Presentation at the 2025 ASH Annual Meeting (GlobeNewswire) - "The presentation will highlight additional patient data from the open-label, multicenter Phase 2 trial (NCT05561751) assessing burixafor in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT)....The combination of burixafor, G-CSF and propranolol demonstrated an excellent safety profile and was able to ensure adequate and efficient mobilization of hematopoietic progenitor cells, with all patients who chose to undergo transplant (18/19) proceeding to AHCT."

P2 data • Multiple Myeloma

Burixafor Phase 2 Trial in Multiple Myeloma Nears Key Clinical Readout (The Manila Times) - "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...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."

P2 data • Hodgkin Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma

Burixafor has a faster kinetics of mobilization with a well-tolerated safety profile, enabling same-day administration of the mobilizing agent and leukapheresis. (The Manila Times) - "Preparations are also underway for a potential Phase 3 trial."

New P3 trial • Multiple Myeloma

Building on progress in multiple myeloma, Exicure is preparing to expand burixafor into additional indications: (The Manila Times) - "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 trial • Acute Myelogenous Leukemia • Sickle Cell Disease

Molecular targets of neuroplasticity in ischemic stroke: insights from GEO database, single-cell analysis and immune infiltration analysis. (PubMed, Front Aging Neurosci) - "Finally, the Leronlimab, Ulocuplumab, Burixafor, and MSX-122 are promising drugs to treat IS via targeting on CCR5 and CXCR4. In conclusion, our findings suggest that CCR5 and CXCR4 are promising targets for enhancing neuroplasticity post-ischemic stroke, thus providing potentially effective and reliable therapeutic targets for future interventional strategy."

Journal • Cardiovascular • Ischemic stroke • CD8 • CXCR4 • GRM1 • TIMP1

Exicure Completes the Last Patient, Last Visit in Phase 2 Study of GPC-100 for Stem Cell Mobilization in Multiple Myeloma Patients Undergoing Autologous Transplant (Businesswire) - "Exicure...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). Topline results from the study are expected in the fourth quarter of 2025."

P2 data • Trial status • Multiple Myeloma

Ph2, Study to Assess the Safety and Efficacy of GPC 100 and Propranolol With and Without G-CSF for the Mobilization of Stem Cells in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplant (clinicaltrials.gov) - P2 | N=40 | Active, not recruiting | Sponsor: GPCR Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025

Enrollment closed • Trial completion date • Trial primary completion date • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Exicure Completes Patient Enrollment in Phase 2 Study of GPC-100 for Stem Cell Mobilization in Multiple Myeloma Patients Undergoing Autologous Transplant (Businesswire) - "Exicure...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). The randomized, open-label, multicenter study is designed to assess whether GPC-100, a small molecule CXCR4 antagonist, can improve CD34+ hematopoietic stem cell mobilization from the bone marrow into the peripheral blood, where they can be collected via leukapheresis for use in ASCT. Topline results from the study are expected in Fall 2025."

Enrollment closed • P2 data • Multiple Myeloma

Exicure, Inc. (Nasdaq: XCUR) Nears Completion of Phase 2 Study of GPC-100 in Multiple Myeloma (Businesswire) - "Exicure...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....This trial is expected to finish patient recruitment at the end of April."

Trial status • Multiple Myeloma

Exicure, Inc. (Nasdaq: XCUR) Announces Their Next Step in Planning for a New Clinical Trial in Acute Myeloid Leukemia (AML) (Businesswire) - "Exicure...shared updates on its lead asset, GPC-100 (burixafor), a small molecule CXCR4 inhibitor. Exicure...is planning for a clinical trial in Acute Myeloid Leukemia (AML) with GPC-100. The company believes that GPC-100, when paired with modern AML treatment regimens, can further improve outcomes in this high unmet need clinical indication....In addition to AML, GPC-100 is currently being evaluated in an ongoing Phase 2 trial in patients with multiple myeloma (MM) undergoing autologous stem cell transplant (ASCT; NCT05561751). Clinical trial results for this study are expected in Q4 2025."

New trial • P2 data • Acute Myelogenous Leukemia • Multiple Myeloma