Aphexda (motixafortide) / BioLineRx, GenFleet Therap, Gloria Pharma, Ayrmid - LARVOL DELTA

Safety Outcomes of CXCR4 Antagonists for Stem Cell Mobilization in Patients with Multiple Myeloma (TCT-ASTCT-CIBMTR 2026) - "CXCR4 antagonists plerixafor and motixafortide, when combined with G-CSF, are widely used to mobilize hematopoietic stem and progenitor cells. Integrating structured nursing assessments into mobilization protocols enhances patient safety and the overall mobilization experience. Future nursing-led initiatives should aim to refine toxicity prophylaxis and management—particularly for motixafortide—to optimize safety and promote best practices in patient-centered mobilization care."

Clinical • Hematological Malignancies • Immunology • Multiple Myeloma

Ayrmid Reports Additional New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease and Beta-Thalassemia (ACCESS Newswire) - "Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies; 73% (11 of 15) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing with 5 patients receiving their gene therapy and appropriately engrafted; 73% (11 of 15) of patients had previously failed to collect sufficient cells with plerixafor; Results support the use of motixafortide as an effective single-agent mobilizer of HSC for people with sickle cell disease and in combination with G-CSF for beta-thalassemia."

Real-world • Beta-Thalassemia • Sickle Cell Disease

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

Adverse Drug Effects Following Motixafortide for Hematopoietic Progenitor Cell Mobilization in Patients with Multiple Myeloma (TCT-ASTCT-CIBMTR 2026) - "Introduction Despite the use of plerixafor, a low-affinity, short-acting CXCR4 inhibitor, over 25% of multiple myeloma (MM) patients are unsuccessful at mobilizing their target CD34+ hematopoietic progenitor cells (HPCs)...Standard premedications included acetaminophen, diphenhydramine, famotidine, and montelukast. Due to treatment emergent adverse events (TEAEs), intravenous dexamethasone 10 mg was added to the premedication regimen starting May 2025...Premedication, longer monitoring, and reaction treatment medication use with motixafortide impacts resource utilization for patients undergoing mobilization. (1) To describe the efficacy outcomes of using motixafortide for mobilization in a real world MM population (2) To describe the treatment emergent adverse effects of motixafortide when used in a real world MM population (3) To describe injection reaction rates with motixafortide following addition of dexamethasone as premedication"

Clinical • Dermatology • Hematological Malignancies • Immunology • Multiple Myeloma • Pruritus • Urticaria • CD34 • CXCR4

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

Motixafortide Is Effective in Hematopoietic Progenitor Cell Mobilization in Multiple Myeloma Patients Treated with Quadruplet Therapy (TCT-ASTCT-CIBMTR 2026) - "Majority of patients were lenalidomide-exposed (98.6%), anti- CD38 antibody-exposed (92.9%), and quadruplet therapy exposed (90%). In addition, this will increase apheresis space efficiency. (1) To describe efficacy outcomes in using motixafortide for hematopoietic progenitor cell mobilization in a real world patient population with multiple myeloma (2) To describe motixafortide efficacy for hematopoietic progenitor cell mobilization in patients receiving quadruplet induction therapy (3) To report engraftment outcomes in patients receiving transplant following mobilization with motixafortide"

Clinical • Hematological Malignancies • Multiple Myeloma • CXCR4

A Cohort Comparison of CXCR4 Antagonists for Multiple Myeloma Mobilization (TCT-ASTCT-CIBMTR 2026) - "Plerixafor and motixafortide are CXCR4 antagonists used with G-CSF to mobilize CD34⁺ stem cells by disrupting CXCL12–CXCR4. Further prospective and risk-balanced studies are needed to confirm these signals, identify patients most likely to benefit, and clarify operational and cost implications to guide agent selection. Compare Day 1 CD34 count in patients getting 2 different CXCR4 Antagonists"

Amyloidosis • Hematological Malignancies • Leukemia • Multiple Myeloma • Plasma Cell Leukemia • CD34 • CXCL12

Impact of Motixafortide on Apheresis Chair Utilization and Mobilization Efficiency Compared to Plerixafor in Autologous Stem Cell Transplant Candidates (TCT-ASTCT-CIBMTR 2026) - "Motixafortide plus G-CSF demonstrated comparable apheresis efficiency to plerixafor-based mobilization in the multiple myeloma patient population, with similar mean chair and procedure run times. These findings suggest both mobilization strategies provide equivalent operational efficiency in routine clinical practice."

Hematological Malignancies • Multiple Myeloma • Transplantation • CD34 • CXCR4

Collection and Manufacture of Autologous HSC Gene Therapy Cell Product for Patients with Sickle Cell Disease (SCD) and Transfusion Dependent Thalassemia (TDT): A Real World Gene Therapy (ReGenT) Consortium Report. (TCT-ASTCT-CIBMTR 2026) - "SCD received plerixafor mobilization while TDT received a combination of plerixafor and G-CSF. Two SCD patients received motixafortide... With ongoing data collection from the other centers, we demonstrate more significant challenges in mobilization, collection and manufacturing in the post-commercialization era as manufacturing scales nationally, compared to reports from the original clinical trial experience and the importance of need to improve manufacturing, increased apheresis days/cycle. Variability in CD34 + collection highlights the need for better predictive strategies to allow for optimal collection and allow for alternate mobilization and apheresis adjustments. 1."

Clinical • Gene therapy • Real-world • Real-world evidence • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34

Improved Hematopoietic Stem Cell Mobilization for Gene Therapy of Hemoglobinopathies Using Single Agent Motixafortide (TCT-ASTCT-CIBMTR 2026) - P1 | "HSC mobilization for SCD is limited to single agent plerixafor P , a CXCR4 antagonist, since G- CSF is associated with an increased risk of vaso-occlusive events VOEs and death...To mitigate known local injection site and potential systemic reactions to M, all patients received prophylactic H-1 and H-2 antagonists and acetaminophen, with 86% receiving additional montelukast...Additional studies are warranted to determine the ideal timing with apheresis and prophylactic medication regimen. 1- limitations of peripheral blood stem cell mobilization in sickle cell disease 2- Adverse effects of motixafortide especially in patients with SCD 3-Feasibility of using motixafortide for mobilization in beta thalassemia"

Gene therapy • Beta-Thalassemia • Dermatology • Gene Therapies • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Pruritus • Sickle Cell Disease • Urticaria

Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial. (PubMed, Nat Med) - P3 | "In conclusion, motixafortide + G-CSF mobilized significantly greater CD34 HSPC numbers within two apheresis procedures versus placebo + G-CSF while preferentially mobilizing increased numbers of immunophenotypically and transcriptionally primitive HSPCs. Trial Registration: ClinicalTrials.gov , NCT03246529."

Clinical • Journal • P3 data • Bone Marrow Transplantation • Dermatology • Hematological Malignancies • Multiple Myeloma • Oncology • Pain • Pruritus • Transplantation • CD34 • CXCR4

Mobilization of Stem Cells With Motixafortide (BL-8040) in Combination With G-CSF in Multiple Myeloma Patients (clinicaltrials.gov) - P3 | N=60 | Recruiting | Sponsor: Guangzhou Gloria Biosciences Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial completion date: Dec 2026 ➔ May 2027 | Trial primary completion date: Aug 2026 ➔ Dec 2026

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

MORPHEUS-EC: A Study of Multiple Immunotherapy-Based Treatment Combinations in Patients With Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction Cancer (G/GEJ) or Esophageal Cancer (Morpheus-Gastric and Esophageal Cancer) (clinicaltrials.gov) - P1/2 | N=214 | Completed | Sponsor: Hoffmann-La Roche | Active, not recruiting ➔ Completed | N=410 ➔ 214

Enrollment change • Trial completion • Esophageal Adenocarcinoma • Esophageal Cancer • Gastric Adenocarcinoma • Gastric Cancer • Gastroesophageal Junction Adenocarcinoma • Oncology • Solid Tumor • HER-2 • PD-L1 • TIGIT

A human iPSC-based neural spheroid platform for modelling glioblastoma infiltration using high-content imaging. (PubMed, Sci Rep) - "A proof-of-principle drug screen identified PF 573228 (FAK inhibitor) and motixafortide (CXCR4 inhibitor) as potent suppressors of GBM20 and GBM1 infiltration, respectively. Bulk RNA sequencing revealed gene expression profiles correlating with invasive behaviour and drug sensitivity. This platform offers a valuable model for studying glioblastoma infiltration along axons and provides proof-of-principle that migration can serve as a measurable and actionable phenotype to screen therapeutic vulnerabilities in glioblastoma."

Journal • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • CXCR4

Motixafortide + G-CSF hematopoietic stem cell mobilization in patients with multiple myeloma following quadruplet induction therapy (ASH 2025) - P1, P3 | "All patients received"enhanced" premeds with: loratadine (10 mg, PO), famotidine (20 mg, PO) and montelukast (10 mg, PO)daily on Day 1-5; plus acetaminophen (975mg, PO), hydrocortisone (200 mg, IV), diphenhydramine (25mg, IV) and famotidine (20 mg, IV) on Day 4 (30-45 mins pre-M). Meanwhile,enhanced premeds were associated with a low rate of AEs relative to standard premeds. Ongoingextended immunophenotyping may shed insight into the mechanism of impaired HSC mobilization post-quad induction, possibly via targeted depletion of a large population of CD38+ HSCs within the CD34+graft."

Clinical • Dermatology • Hematological Malignancies • Multiple Myeloma • Pruritus • CD34

Motixafortide (CXCR4 inhibition) alone and in combination with natalizumab (VLA-4 inhibition) to mobilize hematopoietic stem cells for gene therapy in sickle cell disease: A first-in-human, safety and feasibility study (ASH 2025) - P1 | "G-CSF is unsafe inSCD and the CXCR4 inhibitor (CXCR4i) plerixafor (P) does not reliably yield optimal HSC numbers. Remarkably, HSC transplant of non-SCD marrow into SCD mice (myeloablative cKit-ADCconditioning) reverted the enhanced mobilization phenotype in SCD mice to that of non-SCD mice,indicating enhanced CXCR4i-based mobilization in SCD may track with the hematopoietic system.In conclusion, our first-in-human trial demonstrates the potential of M and N+M as novel G-CSF-freeregimens to safely optimize HSC mobilization in SCD (median CD34+ cells/μl: P=73, M=189, N+M=312).Correlative FC and scRNA seq highlight regimen-specific mobilization of unique HSC subsets, includingincreased CLPs, ERPs and MEPs with N+M. Further mechanistic study of HSC mobilization biology maybuild upon our finding that Townes SCD mice and SCD humans share an enhanced mobilizationphenotype with CXCR4i +/- VLA4i."

Clinical • Combination therapy • First-in-human • Gene therapy • P1 data • Dermatology • Gene Therapies • Genetic Disorders • Hematological Disorders • Immunology • Pruritus • Sickle Cell Disease • Urticaria • CD34 • CXCR4

EMU-116: An oral CXCR4 antagonist as mobilizer of stem and immune cells in normal, neutropenic and sickle cell mice. (ASH 2025) - "The use of CXCR4 antagonists as cell mobilization agents has resulted in 3 FDA approved drugs for use inhematopoietic stem cell transplantation (Plerixafor, Motixafortide) and for treating neutropenia(Mavorixafor). In Swiss mice, EMU-116 dosed orally providesenhanced responses to cyclophosphamide indicating a potential use in chemotherapy-inducedneutropenia. In sickle cell mice, EMU-116 boosts LSKs when dosed orally and could possibly be utilizedfor a future in vivo gene therapy setting with superior effects to Plerixafor."

Immune cell • Preclinical • Bone Marrow Transplantation • Chemotherapy-Induced Neutropenia • Gene Therapies • Genetic Disorders • Hematological Disorders • Neutropenia • Sickle Cell Disease

Efficacy of motixafortide plus G-CSF versus plerixafor plus G-CSF for stem cell mobilization and collection in multiple myeloma: A single-center comparative analysis and interim Results from a prospective study in poor mobilizers (ASH 2025) - "At our institution, a real-world analysis (30MM patients per cohort) compared G-CSF plus motixafortide (prospectively) against G-CSF plusplerixafor (retrospectively, matched by age, gender, race, and IMiD/daratumumab exposure).There were no significant differences in overall mobilization, collection, or apheresis outcomes.Subgroup analysis suggested that patients with low peripheral blood CD34+ counts (pbCD34 ≤5/μL on Day 4 of G-CSF) may achieve a greater rise in CD34+ counts after motixafortide, raisingthe hypothesis that it may benefit "poor mobilizers."We initiated a prospective study to characterize motixafortide efficacy in poor mobilizers(defined as pbCD34+ ≤ 2/μL after 4 days of G-CSF). Full analysis including individual apheresis yields and total collection outcomesis in process. These data will inform future risk-benefit assessments of motixafortide use and helpoptimize treatment regimens in MM patients who mobilize poorly to initial G-CSF doses."

Clinical • Hematological Malignancies • Multiple Myeloma • CD34 • CXCR4

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease (ASH 2025) - P1 | "Currently, HSC mobilizationfor SCD is limited to single agent plerixafor (P), a CXCR4 antagonist, since G-CSF is associated with anincreased risk of vaso-occlusive events (VOEs) and death...The median total number of CD34+ cellscollected/day with P was 2.6 x 106/kg compared to 6.8 x 106/kg with M. M was used first line in 3 patientswith preexisting clinical concerns precluding multiple collection cycles...To mitigate known local injection site and potential systemicreactions to M, all patients received prophylactic H-1 and H-2 antagonists and acetaminophen, with 82%receiving additional montelukast...Ongoing clinical trials of M in SCD are examining safety, mobilization efficacy,optimal dose timing, and editing potential (NCT06442761; NCT05618301). Additional studies arewarranted to determine the ideal prophylactic medication regimen."

Gene therapy • Dermatology • Gene Therapies • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Pruritus • Sickle Cell Disease • Urticaria • CD34

Evaluating Premedication Regimens (Methylprednisolone vs Dexamethasone-based) for the Prevention of Systemic and Injection Site Reactions to Motixafortide in Patients With Multiple Myeloma Undergoing Stem Cell Mobilization, PARADE Trial (clinicaltrials.gov) - P4 | N=94 | Recruiting | Sponsor: Emory University | Not yet recruiting ➔ Recruiting

Enrollment open • Allergy • Hematological Malignancies • Multiple Myeloma • Oncology

Clinical Updates (PRNewswire) - "Continued to advance preparations for initiation of a Phase 1/2a clinical trial of GLIX1 in recurrent and newly diagnosed glioblastoma in the first quarter of 2026....Enrollment continues in the CheMo4METPANC Phase 2b clinical trial, which is being led by Columbia University, and supported by both Regeneron and BioLineRx. The CheMo4METPANC trial is evaluating motixafortide in combination with the PD-1 inhibitor cemiplimab and standard chemotherapy (gemcitabine and nab-paclitaxel)."

Enrollment status • New P1/2 trial • Glioblastoma • Pancreatic Ductal Adenocarcinoma

Cellular Therapy and HSCT Mobilizers – Motixafortide (ASH 2023) - No abstract available

Bone Marrow Transplantation

Novel Small Molecule VLA-4 Inhibitors Optimized for Extended Hematopoietic and Progenitor Cell Mobilization (ASH 2023) - "Importantly, a synergistic effect on HSPC mobilization was achieved when WU-106 was co-administered with CXCR4 inhibitors (Plerixafor or BL-8040). We have developed novel, potent PEGylated small molecule VLA-4 inhibitors that have excellent aqueous solubility and pharmacokinetics. The kinetics and magnitude of HSPC mobilization by our PEGylated inhibitors are superior to other previously described VLA-4 inhibitors and an antibody to alpha-4(anti-CD49d)."

Anemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CXCR4 • ITGA4 • VCAM1

Prolonged CXCR4 Receptor Occupancy By Motixafortide Following a Single Subcutaneous Injection Is Associated with Extended Mobilization of CD34+ Cells in Peripheral Blood for > 24 Hours (ASH 2023) - P1, P3 | "In vitro studies demonstrated complete receptor occupancy by motix starting at a concentration of 3nM. Increasing concentrations resulted in longer duration of receptor occupancy. Mobilization of CD34+ cells to PB was observed within 2 hours after motix injection (1."

Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CXCL12 • CXCR4

Use of Combination Premedication Prior to Motixafortide Administration Reduced Severity and Frequency of AEs in the Phase 3 Genesis Trial - a Single Center Analysis (ASH 2023) - "Motixafortide was safe and well tolerated with no episodes of anaphylaxis, no Grade 4 AEs and no deaths. Following implementation of a protocol amendment using combination pre-medication with an antihistamine H1 blocker, an H2 blocker, a leukotriene inhibitor and acetaminophen, the frequency and severity of hypersensitivity and injection site AEs associated with motixafortide were markedly improved."

Clinical • P3 data • Dermatology • Hematological Malignancies • Immunology • Multiple Myeloma • Oncology • Pruritus • Urticaria • CD34