spanlecortemlocel (MGTA-456) / Dianthus Therap - LARVOL DELTA

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Masonic Cancer Center, University of Minnesota | Completed ➔ Active, not recruiting | Trial completion date: Dec 2023 ➔ Dec 2026

Enrollment closed • Trial completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom Macroglobulinemia • CEBPA • FLT3 • HLA-DRB1 • KMT2A • NPM1

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2 | N=22 | Completed | Sponsor: Masonic Cancer Center, University of Minnesota | Active, not recruiting ➔ Completed

Trial completion • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom Macroglobulinemia • CEBPA • FLT3 • HLA-DRB1 • KMT2A • NPM1

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2024 ➔ Jun 2025

Trial completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom Macroglobulinemia • CEBPA • FLT3 • HLA-DRB1 • KMT2A • NPM1

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Masonic Cancer Center, University of Minnesota | Trial completion date: Apr 2023 ➔ Jun 2024

Trial completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom Macroglobulinemia • CEBPA • FLT3 • HLA-DRB1 • KMT2A • NPM1

Mgta-456, a Cell Therapy Utilizing Expansion of CD34+ Hematopoietic Stem Cells (HSC), Improves HLA Matching for Adult Recipients, Promotes Faster Hematopoietic Recovery and Enables Uniform Engraftment with Less Acute Graft-Vs-Host Disease (GVHD) (TCT-ASTCT-CIBMTR 2020) - P2; "Patients received cyclophosphamide 120 mg/kg, fludarabine 75 mg/m2 and total body irradiation 1320 cGy or a busulfan-based regimen for children <3 years of age prior to MGTA-456 with cyclosporine/mycophenolate mofetil prophylaxis. Cryopreserved MGTA-456 cell therapy resulted in rapid and 100% engraftment with speed of neutrophil recovery correlating with CD34+CD90+ cell dose in patients with high risk hematologic malignancy. Expansion of smaller CBUs increases the chance of finding a better HLA match, particularly for adults, thus reducing the barriers associated with low cell dose and poor HLA match in CBU transplantation."

Clinical • AHR • CD34

Mgta-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-Vs-Host Disease (GVHD) (ASH 2019) - P2; "Conditioning consisted of cyclophosphamide 120 mg/kg, fludarabine 75 mg/m2 and total body irradiation 1320 cGy (total doses) with cyclosporine and mycophenolate mofetil as immunoprophylaxis. Transplantation of cryopreserved MGTA-456 resulted in complete engraftment and rapid recovery with speed of neutrophil recovery correlating with the CD34+CD90+ cell dose. Based on the marked expansion that is now possible, units with fewer cells can now be considered, increasing the probability of finding a better HLA matched unit, particularly for adults. Availability of MGTA-456 could reduce the barriers associated with cell dose and poor HLA match previously limiting the successful use of UCB in transplantation."

AHR • CD34 • IL6

Mgta-456 Cell Therapy in Inherited Metabolic Disease (IMD) Yields Rapid and Durable Long-Term Improvement of Disease-Specific Outcomes in a Phase 2 Trial (TCT-ASTCT-CIBMTR 2020) - P=N/A, P2; "Treatment with MGTA-456 in patients with IMDs shows early, robust engraftment and immune reconstitution, features that are highly associated with improved clinical outcomes. Disease-specific outcomes up to one-year post-transplant in MPS I and cALD patients show crucial, durable stabilization in biochemical, MRI and clinical/neurodevelopmental assessments. In summary, the high CD34+ cell doses delivered by MGTA-456 shows compelling potential to rapidly and durably improve outcomes in IMD patients."

P2 data • CD34 • CD8 • MRI

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Masonic Cancer Center, University of Minnesota | Trial completion date: Aug 2023 ➔ Apr 2023 | Trial primary completion date: Jul 2023 ➔ Mar 2023

Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom Macroglobulinemia • CEBPA • FLT3 • HLA-DRB1 • KMT2A • NPM1

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) (clinicaltrials.gov) - P2; N=8; Completed; Sponsor: Magenta Therapeutics, Inc.; Active, not recruiting ➔ Completed

Clinical • Trial completion • Bone Marrow Transplantation • Metabolic Disorders • Transplantation

[VIRTUAL] MGTA-456, A CD34 Expanded Cord Blood Product, Permits Selection of Better HLA Matched Units and Results in Rapid Hematopoietic Recovery, Uniform Engraftment and Reduced Graft-Versus-Host Disease in Adults with High-Risk Hematologic Malignancies (TCT-ASTCT-CIBMTR 2021) - P2 | "All patients >3 were conditioned with cyclophosphamide 120 mg/kg, fludarabine 75 mg/m2 and total body irradiation 1320 cGy. GVHD prophylaxis was tacrolimus and mycophenolate mofetil... Marked expansion of CD34+CD90+ cells in MGTA-456 results in rapid and sustained hematopoietic recovery, complete engraftment in all subjects, and improved HLA match in adults weighing >60 kg. The availability of MGTA-456 reduces the barriers of low cell dose and poor HLA match previously limiting the successful use of CB as a graft source."

Clinical • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation • CD34 • IL6

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 (clinicaltrials.gov) - P=N/A; N=3; Terminated; Sponsor: Magenta Therapeutics, Inc.; Active, not recruiting ➔ Terminated; Due to low enrollment

Clinical • Clinical data • Trial termination • Metabolic Disorders • Transplantation • MRI

Magenta Therapeutics to Present Additional Data from Phase 1 MGTA-145 Stem Cell Mobilization Program and Preclinical Updates on Targeting Conditioning Program at Transplantation and Cellular Therapy (TCT) 2021 Annual Meeting (Businesswire) - "Magenta Therapeutic...announced data presentations across its stem cell mobilization and targeted conditioning programs at the Transplantation and Cellular Therapy (TCT) Annual Meeting...Oral Presentation Showcasing Preclinical Study of MGTA-117 Targeted ADC Conditioning Program; Poster Presentation Highlighting Preclinical Data of CD45-ADC Targeted Conditioning Program; Oral Presentation of MGTA-456 Stem Cell Therapy Expansion Program in Patients with Blood Cancer..."

Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov) - P2; N=22; Active, not recruiting; Sponsor: Masonic Cancer Center, University of Minnesota; Recruiting ➔ Active, not recruiting; N=40 ➔ 22

Clinical • Enrollment change • Enrollment closed • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Burkitt Lymphoma • Chronic Myeloid Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Waldenstrom’s Macroglobulinaemia

[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (EBMT 2020) - " For mouse transplant studies, bone marrow cells were transplanted into wild-type or Idua-/- mice following myeloablative busulfan or treosulfan conditioning and analyzed weekly from 1-16 weeks post-HSCT. We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia."

CNS Disorders • Gene Therapies • Hurler Syndrome • Metabolic Disorders • Transplantation • CD34

[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (EBMT 2020) - " For mouse transplant studies, bone marrow cells were transplanted into wild-type or Idua-/- mice following myeloablative busulfan or treosulfan conditioning and analyzed weekly from 1-16 weeks post-HSCT. We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia."

CNS Disorders • Gene Therapies • Hurler Syndrome • Metabolic Disorders • Transplantation • CD34

[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (EBMT 2020) - " For mouse transplant studies, bone marrow cells were transplanted into wild-type or Idua-/- mice following myeloablative busulfan or treosulfan conditioning and analyzed weekly from 1-16 weeks post-HSCT. We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia."

CNS Disorders • Gene Therapies • Hurler Syndrome • Metabolic Disorders • Transplantation • CD34

[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (EBMT 2020) - " For mouse transplant studies, bone marrow cells were transplanted into wild-type or Idua-/- mice following myeloablative busulfan or treosulfan conditioning and analyzed weekly from 1-16 weeks post-HSCT. We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia."

CNS Disorders • Gene Therapies • Hurler Syndrome • Metabolic Disorders • Transplantation • CD34

MGTA-456, A Cell Therapy Utilizing an Aryl Hydrocarbon Receptor Antagonist (AHRa) Culture, Promotes Expansion of CD34+CD90+Cord Blood (CB) Hematopoietic Stem Cells (HSC), Resulting in Rapid Hematopoietic Recovery, Uniform Engraftment and Better HLA Matched Grafts for Larger Recipients (ASGCT 2020) - P2 | "Conditioning consisted of cyclophosphamide 120 mg/kg, fludarabine 75 mg/m2 and total body irradiation 1320 cGy in 10 patients with a busulfan-based regimen in two children aged 2 yrs. GVHD prophylaxis was cyclosporine and mycophenolate mofetil, and G-CSF was started on d+1 after MGTA-456 infusion until neutrophils exceeded 2500/uL for 3d... Per protocol, all patients received >10 x106 nucleated cells/kg in the expanded product. Median CD34+ cell fold-expansion was 421-fold (r, 219-1476). Patients received a median CD34+ and CD34+CD90+ cell dose of 2.6 x 107 /kg (r, 0.9-13.5 x 107 /kg) and 1.3 x 106 /kg (r, 0.5-7.0 x 106 /kg), respectively."

Graft versus Host Disease • Hematological Malignancies • Oncology • AHR • CD34 • CSF3 • IL6

[VIRTUAL] A PHASE 2 TRIAL OF MGTA-456 CELL THERAPY DEMONSTRATES RAPID AND DURABLE LONG-TERM IMPROVEMENT IN DISEASE OUTCOMES IN INHERITED METABOLIC DISEASE (IMD) PATIENTS (EBMT 2020) - P=N/A, P2 | "Treatment with MGTA-456 in patients with IMDs shows early, robust engraftment and immune reconstitution, features that are highly associated with improved clinical outcomes. Disease-specific outcomes up to one-year post-transplant in MPS I and cALD patients show crucial, early and durable stabilization in biochemical, MRI and clinical/neurodevelopmental assessments. In summary, MGTA-456 shows compelling potential to rapidly and durably improve outcomes in IMD patients through high CD34+ cell doses."

Clinical • P2 data • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hurler Syndrome • Metabolic Disorders • Neutropenia • CD34 • CD8 • MPS1 • MRI

[VIRTUAL] A PHASE 2 TRIAL OF MGTA-456 CELL THERAPY DEMONSTRATES RAPID AND DURABLE LONG-TERM IMPROVEMENT IN DISEASE OUTCOMES IN INHERITED METABOLIC DISEASE (IMD) PATIENTS (EBMT 2020) - P=N/A, P2 | "Treatment with MGTA-456 in patients with IMDs shows early, robust engraftment and immune reconstitution, features that are highly associated with improved clinical outcomes. Disease-specific outcomes up to one-year post-transplant in MPS I and cALD patients show crucial, early and durable stabilization in biochemical, MRI and clinical/neurodevelopmental assessments. In summary, MGTA-456 shows compelling potential to rapidly and durably improve outcomes in IMD patients through high CD34+ cell doses."

Clinical • P2 data • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hurler Syndrome • Metabolic Disorders • Neutropenia • CD34 • CD8 • MPS1 • MRI

[VIRTUAL] A PHASE 2 TRIAL OF MGTA-456 CELL THERAPY DEMONSTRATES RAPID AND DURABLE LONG-TERM IMPROVEMENT IN DISEASE OUTCOMES IN INHERITED METABOLIC DISEASE (IMD) PATIENTS (EBMT 2020) - P=N/A, P2 | "Treatment with MGTA-456 in patients with IMDs shows early, robust engraftment and immune reconstitution, features that are highly associated with improved clinical outcomes. Disease-specific outcomes up to one-year post-transplant in MPS I and cALD patients show crucial, early and durable stabilization in biochemical, MRI and clinical/neurodevelopmental assessments. In summary, MGTA-456 shows compelling potential to rapidly and durably improve outcomes in IMD patients through high CD34+ cell doses."

Clinical • P2 data • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hurler Syndrome • Metabolic Disorders • Neutropenia • CD34 • CD8 • MPS1 • MRI

[VIRTUAL] A PHASE 2 TRIAL OF MGTA-456 CELL THERAPY DEMONSTRATES RAPID AND DURABLE LONG-TERM IMPROVEMENT IN DISEASE OUTCOMES IN INHERITED METABOLIC DISEASE (IMD) PATIENTS (EBMT 2020) - P=N/A, P2 | "Treatment with MGTA-456 in patients with IMDs shows early, robust engraftment and immune reconstitution, features that are highly associated with improved clinical outcomes. Disease-specific outcomes up to one-year post-transplant in MPS I and cALD patients show crucial, early and durable stabilization in biochemical, MRI and clinical/neurodevelopmental assessments. In summary, MGTA-456 shows compelling potential to rapidly and durably improve outcomes in IMD patients through high CD34+ cell doses."

Clinical • P2 data • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hurler Syndrome • Metabolic Disorders • Neutropenia • CD34 • CD8 • MPS1 • MRI

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 (clinicaltrials.gov) - P=N/A; N=3; Active, not recruiting; Sponsor: Magenta Therapeutics, Inc.; Enrolling by invitation ➔ Active, not recruiting; N=12 ➔ 3; Trial completion date: May 2025 ➔ Dec 2020; Trial primary completion date: Dec 2024 ➔ Dec 2020

Clinical • Clinical data • Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Metabolic Disorders • Transplantation • MRI

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) (clinicaltrials.gov) - P2; N=8; Active, not recruiting; Sponsor: Magenta Therapeutics, Inc.; Recruiting ➔ Active, not recruiting; N=12 ➔ 8; Trial completion date: Apr 2022 ➔ Jan 2021; Trial primary completion date: Jun 2021 ➔ Feb 2020

Clinical • Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Metabolic Disorders • Transplantation

Safety and Tolerability of HSC835 in Patients With Hematological Malignancies (clinicaltrials.gov) - P1/2; N=35; Recruiting; Sponsor: Novartis Pharmaceuticals; Trial primary completion date: Aug 2017 ➔ Dec 2017

Trial primary completion date • Acute Lymphocytic Leukemia • Biosimilar • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology