dorocubicel (UM171 cell therapy) / ExCellThera - LARVOL DELTA

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

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 • Bone Marrow Transplantation • Cardiovascular • 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 • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Targeted Protein Degradation • Transplantation • MYC • TP53

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

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

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 | Recruiting ➔ Active, not recruiting | N=20 ➔ 30 | Trial completion date: Dec 2023 ➔ Oct 2027 | Trial primary completion date: Dec 2023 ➔ Oct 2024

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34 • FLT3 • HLA-DRB1 • JAK2 • TP53

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 • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • 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 • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34 • TP53

ExCellThera Announces Positive Phase 2 Study Results from UM171 Cell Therapy in Patients with High-risk Leukemias and Myelodysplasias at American Society of Hematology (ASH) 2023 Annual Meeting (GlobeNewswire) - P2 | N=52 | "The data were presented for the first time in an oral session at the 65th American Society of Hematology Annual Meeting & Exposition (ASH)...demonstrated excellent clinical outcomes at two years for this high and very high-risk cohort, including strong rates of overall survival (67%) and progression-free survival (63%), as well as low rates of non-relapse mortality (19%), relapse (18%) and moderate-to-severe chronic GVHD (7%)....Based on these highly compelling data, we are looking forward to advancing UM171 cell therapy forward to a registrational Phase 3 trial in high and very high-risk leukemias and myelodysplasias in 2024."

New P3 trial • P2 data • Hematological Malignancies • Leukemia • Oncology

ExCellThera announces new UM171 data to be presented at ASH 2023 (GlobeNewswire) - "ExCellThera Inc...announced today several UM171 presentations, including an oral session on new data from Phase 2 studies of investigational UM171 cell therapy in high-risk blood cancer patients, at the 65th American Society of Hematology Annual Meeting & Exposition (ASH), taking place December 9-12, 2023, in San Diego, California."

Clinical data • P2 data • Hematological Malignancies • Leukemia • Oncology

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: Jul 2025 ➔ Dec 2025 | Trial primary completion date: Jul 2023 ➔ Dec 2023

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

Improved Outcomes of UM171-Expanded Cord Blood Transplantation Compared with Other Graft Sources: Real-World Evidence. (PubMed, Blood Adv) - "Furthermore, UM171-expanded CB recipients experienced less grades III-IV acute GVHD and chronic GVHD compared to MUD graft recipients. Compared to real-world evidence with CB and MUD alloHCT, this study suggests that UM171-expanded CB recipients may benefit from lower NRM and higher GRFS."

HEOR • Journal • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: ExCellThera inc. | Trial completion date: Jun 2024 ➔ Sep 2025 | Trial primary completion date: Jun 2023 ➔ Sep 2023

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

A Pilot Study of UM171 Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Stem Cell Transplant in High and Very High-Risk Myeloma Patients. (TCT-ASTCT-CIBMTR 2023) - "GVHD prophylaxis consisted of tacrolimus and mycophenolate mofetil. In a high/very high-risk MM population, tandem auto/allo SCT using UM171 eCBs leads to prompt engraftment with a very low incidence of chronic GVHD and good CRFS. PFS will need to be confirmed in a Phase 2 study."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Oncology • Transplantation • CD34

Distinct Mechanisms Underlying the Ex Vivo Expansion of Human Cord Blood Stem Cells with Different Strategies Currently Used for Allogeneic Transplantation (ASH 2019) - P1; "Studies of the engraftment potential of SR1 expanded grafts are ongoing. In conclusion, our findings suggest that although VPA, UM171 and SR1 expand greater numbers of human HSCs that are useful for allogeneic stem cell transplantation, their underlying mechanisms of action and pathways involved are different."

Preclinical • AHR • CD133 • CD34

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: Apr 2024 ➔ Jul 2025 | Trial primary completion date: Apr 2023 ➔ Jul 2023

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 | Recruiting | Sponsor: ExCellThera inc. | N=20 ➔ 30 | Trial completion date: Dec 2022 ➔ Jun 2024 | Trial primary completion date: Jun 2022 ➔ Jun 2023

Enrollment change • 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

Study of expanded Cord Blood transplantation in patients with high-risk blood cancers (clinicaltrialsregister.eu) - P2 | N=30 | Ongoing | Sponsor: ExCellThera inc.

New P2 trial • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • HLA-DRB1

Improved Outcomes of UM171-Expanded Cord Blood Transplantation Compared with Other Graft Sources: Real World Evidence (ASH 2022) - "CB recipients who received anti-thymocyte globulin or alemtuzumab in the conditioning were excluded from the CIBMTR population. This real-word evidence suggests that UM171-expanded CB recipients have select improved outcomes compared to conventional CB and MUD PBSC recipients. While this study was limited by the small number of UM171 CB trial participants and inability to identify the planned number of controls for each case, UM171 expansion of CB appeared to achieve the desired outcome of maintaining CB's benefits while removing its disadvantages. Thus, prospective randomized studies of UM171-expanded CBT compared to MUD PBSC alloHCT are warranted to confirm these results."

Clinical • HEOR • Real-world evidence • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

ExCellThera announces completion of Phase 2 clinical trials for UM171-expanded cell therapy in high-risk blood cancers (GlobeNewswire) - "ExCellThera...announced today the completion of patient enrollment in its initial Phase 2 studies for high-risk leukemias and myelodysplastic syndromes (MDS)....The initial Phase 2 studies have enrolled 50 patients treated with ECT-001 Cell Therapy in the United States and Canada and are designed to assess its safety and efficacy in patients with high-risk leukemias and MDS. In light of the promising data from the ECT-001 Cell Therapy trials, extensions to these initial Phase 2 studies are being initiated in new clinical sites in the United States and Europe, thereby providing the opportunity for additional patients to receive treatment....Top-line data across all ECT-001 Cell Therapy Phase 1/2 trials, which will include over 100 patients as of the data lock date of November 30, 2022, are expected in the first half of 2023."

Clinical protocol • Enrollment closed • P1/2 data • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

ExCellThera announces 100th patient treated with UM171-expanded cell therapy (GlobeNewswire) - "ExCellThera Inc...announced today that its proprietary ECT-001 Cell Therapy has been administered to 100 patients. ECT-001 Cell Therapy is an investigational UM171-expanded cord blood cell therapy being evaluated in trials across multiple adult and pediatric hematological indications, including severe leukemias and myelodysplastic syndromes, in the United States and Canada. Additional clinical trials in Europe are expected soon...'Across all our clinical studies, we are seeing that ECT-001 Cell Therapy indicates promising results in delivering rapid engraftment, better HLA matching, low incidence of transplant-related mortality (TRM) and excellent graft-versus-host disease and relapse-free survival (GRFS)'..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology