Zemcelpro (dorocubicel) / ExCellThera - LARVOL DELTA

UM171-EXPANDED CORD BLOOD AS A SAFE AND POTENT GRAFT SOURCE FOR HIGH-RISK LEUKEMIAS AND MYELODYSPLASTIC SYNDROMES WITH DURABLE LONG-TERM SURVIVAL (EBMT 2026) - P2 | "UM171-expanded CBT provides rapid and durable engraftment and achieves promising long-term survival in adults with high and very high–risk AL/MDS, even when using intentionally selected 5/8 HLA-matched and low–cell-dose grafts. Particularly notable were the outcomes observed in patients with VH–risk features such as AL not in remission, TP53-mutated AML/MDS, or 2nd allo transplant. These VH-risk groups are historically associated with extremely poor post-transplant survival."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • TP53

US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov) - P1/2 | N=13 | Completed | Sponsor: ExCellThera inc. | Recruiting ➔ Completed | Trial completion date: Jun 2027 ➔ Dec 2025 | Trial primary completion date: Jun 2026 ➔ Dec 2025

Trial completion • Trial completion date • Trial primary completion date • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics • Transplantation • CD34 • HLA-DRB1

US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia (clinicaltrials.gov) - P2 | N=30 | Completed | Sponsor: ExCellThera inc. | Active, not recruiting ➔ Completed

Trial completion • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34

ECT-001 (UM171) Expanded Cord Blood Transplant to Treat High-risk Multiple Myeloma (clinicaltrials.gov) - P1/2 | N=19 | Completed | Sponsor: Ciusss de L'Est de l'Île de Montréal | Active, not recruiting ➔ Completed

First-in-human • Trial completion • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34

ExCellThera Inc…and its wholly owned subsidiary Cordex Biologics (Cordex), announce positive results from recently completed Phase 2 studies of Zemcelpro (dorocubicel), also known as UM171 Cell Therapy, in 60 adult patients with high- and very high-risk acute leukemias (AL) and myelodysplastic syndromes (PRNewswire.co.uk) - "Topline results will be presented on March 23, 2026 at the 52nd EBMT Annual Meeting in Madrid, Spain...At 24 months post-transplant, overall survival (OS), progression-free survival (PFS) and relapse-free survival (RFS) were 63.7%, 57.0% and 60%, respectively, among Zemcelpro (dorocubicel)-treated patients. The cumulative incidences of relapse and non-relapse mortality (NRM) were 22.5% and 20.5%, respectively."

P2 data • Leukemia • Myelodysplastic Syndrome

Zemcelpro (dorocubicel) receives NUB Status 1 in Germany (PRNewswire) - "The NUB designation for Zemcelpro (dorocubicel) enables hospitals to immediately apply for temporary, supplementary reimbursement for its use...Notably, 220 hospitals across Germany have expressed interest in accessing Zemcelpro (dorocubicel) through the NUB mechanism for the 2026 calendar year....In the interim, Cordex will engage with national health authorities to enable early access for eligible patients and is engaging with leading stem cell transplantation centres to establish a future network of treatment sites for Zemcelpro (dorocubicel) administration."

Reimbursement • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome

A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients. (PubMed, Transplant Cell Ther) - P1/2 | "UM171-expanded CB transplant in HR/ultra-HR myeloma patients is feasible and allows the use of single CB units with a low risk of cGVHD. Patients with negative pre-transplant MRD might benefit most from a UM171-expanded CB transplant."

Journal • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Multiple Myeloma • Oncology • Transplantation

ECT-001-CB-013: A Study Testing an Improved Dose of UM171 to Help Make Cord Blood Transplants More Effective and Safe. (clinicaltrials.gov) - P1/2 | N=7 | Recruiting | Sponsor: Ciusss de L'Est de l'Île de Montréal

New P1/2 trial • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

Dorocubicel: First Approval. (PubMed, Mol Diagn Ther) - "In patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation (HSCT), transplantation with dorocubicel has been shown to result in neutrophil and platelet engraftment, as well as rapid T cell reconstitution. This article summarizes the key milestones in the development of dorocubicel leading to this first approval for the treatment of adult patients with haematological malignancies who require an allogeneic HSCT following myeloablative conditioning, for whom no other type of suitable donor cells is available."

Journal • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Oncology • Transplantation • CD34

UM171 Expanded Cord Blood Transplantation Outcomes Compare Favorably to All Different Stem Cell Sources: An EBMT Registry Case-Control Analysis (ASH 2023) - "The different donor control cohorts were: 1) CB, single or double units, with a calcineurin inhibitor (CNI) and mycophenolate mofetil (MMF) for GVHD prophylaxis, 2) 10/10 HLA-matched unrelated donor (MUD) peripheral blood stem cells (PBSC) with a CNI and MMF or methotrexate (MTX), 3) 10/10 MUD bone marrow (BM) with a CNI and MMF or MTX, 4) 9/10 HLA-MUD BM or PBSC (9/10 UD), 5) T cell replete haploidentical donor (haplo) BM or PBSC, and 6) matched sibling donor (MSD) BM or PBSC. Exclusion criteria included in vitro manipulated grafts and CBT recipients who received anti-thymocyte globulin or alemtuzumab... This registry matched case-control analysis demonstrated real-word evidence suggesting UM171-expanded CBT recipients have improved outcomes compared to other donor types. More specifically and due to the absence of severe chronic GVHD with UM171 CB, GRFS was better with UM171 CB when compared to other graft sources except for CB and MUD BM. Also, OS and PFS were..."

Clinical • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

Infusion of UM171-Expanded Cord Blood Led to Excellent Survival in Patients with High-Risk Leukemias: Results from Two Independent Phase II Studies (ASH 2023) - "All patients received tacrolimus and mycophenolate mofetil for GVHD prophylaxis. The results of 2 independent Phase II clinical studies demonstrated that infusion of a UM171 single CBT led to excellent clinical outcomes in patients with high and very-high risk malignancies including patients with p53+ mutation. We also observed encouraging results for patients undergoing second/third transplants (33% of the entire cohort). Clinical outcomes were similar between the two Transplant Centers."

Clinical • P2 data • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • CD34 • TP53

CD33 Epitope Editing Unlocks UM171-Expanded Cord Blood Grafts for AML Immunotherapy. (PubMed, Mol Ther) - "Combining UM171-mediated expansion with adenine base editing (ABE), we precisely disrupted the P67 epitope of CD33, centered on phenylalanine 21, which is essential for gemtuzumab ozogamicin (GO) binding. Ultra-deep exome sequencing and GUIDE-seq revealed minimal off-target activity. Together, these findings establish CD33 P67 epitope-directed base editing as a precise, scalable strategy to generate immunotherapy-compatible, UM171-expanded CB grafts that safely enable GO integration into post-transplant care."

IO biomarker • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • CD33

Long Term Follow up of UM171 Expanded Cord Blood Transplantation Patients Does Not Reveal Any Late Toxicity (ASH 2024) - "Amongst these 31 patients, ≥ grade 3 AEs after 24 months included : portal hypertension (liver disease present prior to transplant), EBV viremia, anxiety, cataracts, left ventricular systolic dysfunction, non-severe visceral shingles, autoimmune hemolytic anemia (resolved after short course of prednisone), increase in ALT (resolved spontaneously), pneumothorax, pneumonia and sinusitis. There is no late cytopenia or graft failure and immune reconstitution is robust. In addition to the demonstrated efficacy of UM171 transplants in short term outcomes, these results confirm the long-term safety of UM171 expanded grafts, supporting its preferential effect on long-term HSCs."

Clinical • Anemia • Autoimmune Hemolytic Anemia • Cataract • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Herpes Zoster • Immunology • Infectious Disease • Leukemia • Mood Disorders • Myelodysplastic Syndrome • Oncology • Ophthalmology • Otorhinolaryngology • Pneumonia • Portal Hypertension • Psychiatry • Respiratory Diseases • Sinusitis • Transplantation • Varicella Zoster • CD4

UM171 Cord Blood Transplantation for TP53 Mutated and EVI1 Rearranged Myelodysplasia/Acute Myeloid Leukemia (ASH 2024) - "From 2016 to 2018, 22 patients with hematologic malignancies who lacked a donor were transplanted on a phase I-II clinical trial with a single UM171-expanded CB (Cohen S et al, Lancet Haematol 2019)...These results compare quite favorably to previous publications with OS usually being reported at ≈20% at 2 years. The main limitation of our study is the low number of patients, and thus more patients are needed to confirm these results."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Targeted Protein Degradation • Transplantation • MYC • TP53

UM171 Expanded Cord Blood In Patients With High-Risk Acute Leukemia/Myelodysplasia (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Ciusss de L'Est de l'Île de Montréal | Trial completion date: Oct 2027 ➔ Jun 2027 | Trial primary completion date: Oct 2024 ➔ Jun 2027

Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34 • FLT3 • HLA-DRB1 • JAK2 • TP53

Opening New Horizons: Advanced Hematopoietic Stem Cell Expansion Strategies Bridging Cord Blood Therapy from Bench To Bedside. (PubMed, Stem Cell Rev Rep) - "Novel expanded CB-derived hematopoietic stem and progenitor cells (HSPCs) therapies, including OMISIRGE (Omidubicel onlv.), Zemcelpro (Dorocubicel), and upcoming products with International Nonproprietary Name designations, introduce innovative concepts and comprehensive considerations for improving CB transplantation. This progress enables novel therapeutic options and represents a breakthrough in traditional CB transplants. In this context, we summarize and explore representative techniques and products to provide insights that inspire future developments in CB-derived HSPC therapies."

Journal • Review • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

ExCellThera Inc…announced today the conditional marketing authorization of Zemcelpro by the European Commission (EC) for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. (PRNewswire) - "This EC decision authorizes the marketing of Zemcelpro in all European Union member states, as well as Iceland, Norway and Liechtenstein."

EMA approval • Leukemia • Myelodysplastic Syndrome

Comparison of Outcomes after UM171-Expanded Cord Blood and Six Conventional Donor Source Transplants: A Matched Analysis from the EBMT Registry. (PubMed, Transplant Cell Ther) - "UM171-expanded CB recipients have equivalent or improved short- and long-term outcomes compared to patients transplanted from other graft sources."

Journal • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Oncology • Transplantation

Zemcelpro (UM171 Cell Therapy) receives positive CHMP opinion for treatment of blood cancer patients without access to suitable donor cells (PRNewswire.co.uk) - "ExCellThera Inc...announced today the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending granting conditional marketing authorization for Zemcelpro for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. The European Commission (EC) is expected to make a final decision within approximately two months following CHMP recommendation, and the decision will apply to all 27 European Union (EU) Member States, Iceland, Norway and Liechtenstein....The positive CHMP opinion was based on the conditional Marketing Authorization Application (MAA) for Zemcelpro. Additional filings are planned for Zemcelpro with other health authorities, including in the US, Canada, the UK, and Switzerland."

CHMP • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome

US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: ExCellThera inc. | Trial completion date: Jun 2026 ➔ Jun 2027

Trial completion date • Bone Marrow Transplantation • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics • Transplantation • CD34 • HLA-DRB1

ExCellThera Announces EMA’s Acceptance under Accelerated Assessment of Market Authorisation Application (MAA) for UM171 Cell Therapy for Patients with Hematological Malignancies who Lack a Readily Available Suitable Donor (GlobeNewswire) - "ExCellThera Inc...announced today that the Market Authorisation Application (MAA) for UM171 Cell Therapy (INN-dorocubicel) has been accepted under an accelerated assessment procedure by the European Medicines Agency (EMA), for adult patients with hematological malignancies requiring a stem cell transplant who lack a readily available suitable donor. The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted UM171 Cell Therapy accelerated assessment as it was deemed of major interest from a public health point of view....UM171 Cell Therapy has successfully completed Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias...The initiation of a Phase 3 trial in this patient population is planned for the second half of 2024."

EMA filing • New P3 trial • Hematological Malignancies • Oncology

A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in pediatric and young adult (<21year) Patients with High-Risk and Very High-Risk Myeloid Malignancies (ECT-001-CB.007) (clinicaltrialsregister.eu) - P2 | N=15 | Sponsor: Cordex Biolohics Inc

New P2 trial • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics • Transplantation • CD34 • HLA-DRB1 • TP53

US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: ExCellThera inc. | Trial completion date: Dec 2025 ➔ Jun 2026 | Trial primary completion date: Dec 2023 ➔ Jun 2026

Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics • Transplantation • CD34 • HLA-DRB1

US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: ExCellThera inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Nov 2025 ➔ Feb 2026 | Trial primary completion date: Nov 2023 ➔ Feb 2026

Enrollment closed • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34

ECT-001 (UM171) Expanded Cord Blood Transplant to Treat High-risk Multiple Myeloma (clinicaltrials.gov) - P1/2 | N=20 | Active, not recruiting | Sponsor: Ciusss de L'Est de l'Île de Montréal | Trial primary completion date: Sep 2021 ➔ Oct 2023

Trial primary completion date • Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34