CK0801 / Cellenkos - LARVOL DELTA

A Phase Ib, Open-Label Study of Add on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib (ASH 2023) - P1 | "Adoptive therapy with allogeneic, cord blood-derived regulatory T cells (Tregs) (CK0801- HLA 3/ 6 match, fresh infusion) showed safety and early clinical signal in MF (Kadia et al., Blood. The preliminary analysis of this study evaluating CK0804 (CXCR4 enriched T regs cell therapy) as addition to ruxolitinib shows initial safety with no myelosuppressive adverse events and promising clinical activity. The study is actively enrolling participants (NCT05423691). MD Anderson Cancer Center has an institutional conflict of interest with Cellenkos related to the research presented herein."

Clinical • Fibrosis • Immunology • Myelofibrosis • Oncology • CXCR2 • IL13 • TGFA • TGFB1

Cellenkos Announces US FDA Orphan Drug Designation Granted to CK0801 (Allogeneic Cord Blood derived T regulatory Cell Product) for Treatment of Aplastic Anemia (PRNewswire) - "Cellenkos Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its novel cell therapy product, CK0801....CK0801 is planned for Registration trial in H2 2025."

New trial • Orphan drug • Aplastic Anemia

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial completion date: May 2024 ➔ May 2027 | Trial primary completion date: May 2024 ➔ May 2027

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes. (PubMed, NEJM Evid) - P1 | "In previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure. (Funded by Cellenkos Inc.; Clinicaltrials.gov number, NCT03773393.)."

Journal • P1 data • P1 data • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Oncology

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial completion date: Jan 2024 ➔ May 2024 | Trial primary completion date: Jan 2024 ➔ May 2024

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial completion date: Jan 2023 ➔ Jan 2024 | Trial primary completion date: Jan 2023 ➔ Jan 2024

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

Adoptive Therapy with Allogeneic Cord Blood T Regulatory Cells Improves Transfusion Requirement in Bone Marrow Failure Syndromes (ASH 2021) - P1 | "All AA pts continued their immunosuppressive therapies and the 2 MF patients continued their Ruxolitinib. A single infusion of allogeneic CB derived Treg cells (CK0801) is well tolerated, feasible, and may be associated with clinical improvement in patients with immune related bone marrow disorders. Clinical response appears to correlate with a decrease in inflammatory biomarkers. Expansion of the study, including multiple repeated Treg dosing in BMF is planned."

Anemia • Aplastic Anemia • Graft versus Host Disease • Hematological Disorders • Immunology • Inflammation • Myelodysplastic Syndrome • Myelofibrosis • CD4 • CD8

Phase I Clinical Trial of CK0801 (cord blood regulatory T cells) in Patients with Bone Marrow Failure Syndrome (BMF) Including Aplastic Anemia, Myelodysplasia and Myelofibrosis (ASH 2019) - P1; "Serum will be analyzed for inflammatory cytokines: IL1, IL2, IL4, IL6, IL7, IL8, IL10, IL17, IFN-gamma, ST2, REG3a, OPN, Follistatin, Elafin, TGF-beta. Optional exploratory cytokines: SCF, G-CSF, GM-CSF, HGF, VEGF, SDF1a, MCP1, MCP2, TARC, MIP3a, TECK, CTACK, CCL28, FGF, PDGF, EGF, TGF-α, TLR. The first patient is already consented with a planned total of 9 patients."

Clinical • P1 data • CSF2 • CSF3 • CXCL8 • HGF • IFNG • IL10 • IL15 • IL17A • IL2 • IL4 • IL6 • IL7 • REG3A • VEGFA

A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS) (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Cellenkos, Inc. | N=18 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Complement-mediated Rare Disorders • HLA-B • HLA-DRB1

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial completion date: Aug 2022 ➔ Jan 2023 | Trial primary completion date: Aug 2022 ➔ Jan 2023

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial primary completion date: Mar 2022 ➔ Aug 2022

Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Cellenkos, Inc. | Trial completion date: Sep 2021 ➔ Aug 2022 | Trial primary completion date: Sep 2021 ➔ Mar 2022

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

[VIRTUAL] Adoptive Therapy with Allogeneic Cord Blood T Regulatory Cells Show Safety and Early Clinical Signal in Primary Myelofibrosis (ASH 2020) - P1 | "This is the first study to show that a single infusion of allogeneic CB derived Treg cells (CK0801) is well tolerated, feasible, and may be associated with clinical improvement in patients with immune related bone marrow disorders. Determining the optimal dose and schedule of the Treg cell infusion is ongoing."

Clinical • Aplastic Anemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Inflammation • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Pain • JAK2

A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS) (clinicaltrials.gov) - P1; N=18; Not yet recruiting; Sponsor: Cellenkos, Inc.; Trial completion date: Feb 2022 ➔ Feb 2027; Trial primary completion date: Feb 2021 ➔ Feb 2025

Clinical • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • HLA-DRB1

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1; N=18; Active, not recruiting; Sponsor: Cellenkos, Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Aplastic Anemia • Hematological Disorders • Myelodysplastic Syndrome

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1; N=18; Not yet recruiting; Sponsor: Cellenkos, Inc.

New P1 trial • Biosimilar • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1; N=18; Recruiting; Sponsor: Cellenkos, Inc.; Trial completion date: Jun 2021 ➔ Sep 2021; Trial primary completion date: Jun 2020 ➔ Sep 2021

Clinical • Trial completion date • Trial primary completion date

A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS) (clinicaltrials.gov) - P1; N=18; Not yet recruiting; Sponsor: Cellenkos, Inc.; Trial completion date: Dec 2020 ➔ Feb 2022; Initiation date: Mar 2019 ➔ Feb 2020; Trial primary completion date: Dec 2019 ➔ Feb 2021

Clinical • Trial completion date • Trial initiation date • Trial primary completion date

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) (clinicaltrials.gov) - P1; N=18; Recruiting; Sponsor: Cellenkos, Inc.; Not yet recruiting ➔ Recruiting; Trial completion date: Dec 2020 ➔ Jun 2021; Initiation date: Jan 2019 ➔ May 2019; Trial primary completion date: Dec 2019 ➔ Jun 2020

Clinical • Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date

A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS) (clinicaltrials.gov) - P1; N=18; Suspended; Sponsor: Cellenkos, Inc.; Not yet recruiting ➔ Suspended

Clinical • Trial suspension