ToleroVax (alpha-galactosylceramide liposomal) / Regimmune, San Fu Chemical - LARVOL DELTA

Haplo Peripheral Blood Sct In GVHD Prevention (clinicaltrials.gov) - P1 | N=25 | Completed | Sponsor: Zachariah Michael DeFilipp | Active, not recruiting ➔ Completed

Trial completion • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • HLA-B • HLA-C

---RGI-2001 for the Prophylaxis of Acute Graft-Versus-Host Disease Following Allogeneic HCT. (PubMed, Blood) - P2 | "Forty-nine participants received RGI-2001 in combination with tacrolimus and methotrexate...These results support strategies that target NKT and Treg cell populations to augment immunological changes in allogeneic HCT recipients. This trial was registered at www.clinicaltrials.gov as NCT04014790."

Journal • Acute Graft versus Host Disease • Dental Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukopenia • Oncology • Stomatitis • Thrombocytopenia • Transplantation

RGI-2001 with CNI-Based Prophylaxis Demonstrates Better Acute Gvhd-Free Survival Following Myeloablative Allohct without Increased Relapse: Comparison of a Multi-Center Phase 2b Study with a Contemporaneous CIBMTR Cohort (TCT-ASTCT-CIBMTR 2024) - " RGI-2001-003, an open-label Phase 2b study, evaluated RGI-2001 added to a calcineurin inhibitor (CNI) with methotrexate (MTX) or mycophenolate mofetil for the prevention of aGVHD following myeloablative HCT (without T-cell depletion)... RGI-2001 added to tac/MTX resulted in less aGVHD without an increase in relapse compared to a CIBMTR cohort. RGI-2001 demonstrates a potential role in aGVHD prophylaxis in the myeloablative HCT setting. A Phase 3 study is planned."

Clinical • P2b data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

EFFICACY AND SAFETY OF THE ADDITION OF RGI-2001 IV INFUSION TO TACROLIMUS AND METHOTREXATE FOR ACUTE GVHD PREVENTION IN MYELOABLATIVE HSCT (EBMT 2023) - " RGI-2001-003 is an open-label, multi-center phase 2b study evaluating the efficacy and safety of RGI-2001 when added to a calcineurin inhibitor with methotrexate or mycophenolate mofetil (without T-cell depletion) for the prevention of aGvHD in subjects following myeloablative alloHCT... RGI-2001 IV infusion added to the standard-of-care tacrolimus / methotrexate GvHD prophylaxis shows promising efficacy in the prevention of aGvHD for HLA-matched donors (related or unrelated) with an acceptable safety profile. A phase III study is planned."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Dental Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Leukopenia • Pain • Stomatitis • Transplantation

Haplo Peripheral Blood Sct In GVHD Prevention (clinicaltrials.gov) - P1 | N=25 | Active, not recruiting | Sponsor: Zachariah Michael DeFilipp | Trial primary completion date: Dec 2022 ➔ Oct 2023 | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2023 ➔ Oct 2024

Enrollment closed • Post-transplantation • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • HLA-B • HLA-C

Advances in graft-versus-host disease prevention strategies: latest updates from the 2022 ASH annual meeting. (PubMed, Exp Hematol Oncol) - "The use of innovative agents and regimens, along with the conventional prophylactic approach of combining post-transplant cyclophosphamide and anti-thymocyte globulin, were discussed. The innovative agents and regimens highlighted in this review include abatacept, the first FDA-approved drug for acute GvHD prophylaxis; RGI-2001, which promotes the expansion of regulatory T cells; and cell therapies such as Orca-T and Orca-Q. These advancements provide promising strategies and options for GvHD prevention, offering hope for improved post-transplant patient outcomes in terms of survival rates."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplantation

RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=49 | Completed | Sponsor: Regimmune Corporation | Active, not recruiting ➔ Completed

Trial completion • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Myelodysplastic Syndrome • Transplantation

REGiMMUNE Limited Licenses the Rights to Develop and Commercialize RGI-2001 To San Fu Biotech in Major Asian Countries (Businesswire) - "REGiMMUNE...have entered a licensing agreement to develop and commercialize RGI-2001 for the prophylaxis of acute Graft-versus-host disease (aGvHD) in major Asian countries....Under the terms of agreement, REGiMMUNE and SFB will collaborate closely to accelerate the development of RGI-2001 in major Asian countries and will conduct clinical trials to study the efficacy of the drug in aGvHD. Together, both parties intend to submit an Investigational New Drug Application to the Taiwan Food and Drug Administration to initiate clinical trials in Taiwan for the prophylaxis of aGvHD before the end of 2023...planned to submit phase III IND to US FDA in Q4 of 2023."

IND • Licensing / partnership • New trial • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

RGI-2001 Infusion for Prevention of Acute Gvhd after Allogeneic Hematopoietic Cell Transplantation (ASH 2022) - P2 | " RGI-2001-003 (NCT04014790) is an open-label, multi-center phase 2b study to evaluate the potential efficacy and safety of RGI-2001 when added to calcineurin inhibitor with methotrexate or mycophenolate mofetil (without T-cell depletion) for the prevention of aGvHD in subjects following myeloablative alloHCT...Common myeloablative conditioning regimens included Fludarabine/ Busulfan (81.6%), Cyclophosphamide/TBI (12.2%), and Cytarabine/Cyclophosphamide/TBI (4%)... Intravenous administration of RGI-2001 added to standard-of-care tacrolimus / methotrexate showed promising efficacy in the prevention of clinically significant aGVHD with a favorable safety profile. A phase 3 study is planned."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Anemia • Chronic Graft versus Host Disease • Dental Disorders • Graft versus Host Disease • Gynecology • Hematological Disorders • Immunology • Infectious Disease • Musculoskeletal Pain • Pain • Respiratory Diseases • Septic Shock • Stomatitis • Transplantation

Efficacy of the Addition of RGI-2001 to Tacrolimus and Methotrexate for Acute Gvhd Prevention in Myeloablative HSCT Using HLA-Matched Donors (TCT-ASTCT-CIBMTR 2023) - "Methods : RGI-2001-003 is an open-label, multi-center phase 2b study evaluating the efficacy and safety of RGI-2001 when added to a calcineurin inhibitor with methotrexate or mycophenolate mofetil (without T-cell depletion) for the prevention of aGvHD in subjects following myeloablative alloHCT... RGI-2001 added to the standard-of-care tacrolimus / methotrexate GvHD prophylaxis shows promising efficacy in the prevention of aGvHD for HLA-matched donors (related or unrelated) with an acceptable safety profile. A phase III study is planned."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Dental Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Leukopenia • Pain • Stomatitis • Transplantation

REGiMMUNE Presented Positive Results of RGI-2001 in the Reduction of Acute GvHD from Phase 2b Study at the 2022 ASH Annual Meeting (Businesswire) - P2b | N=49 | NCT04014790 | Sponsor: Regimmune Corporation | REGiMMUNE Limited...announced the positive results of their Phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGvHD) after allogeneic hematopoietic cell transplantation (HCT) at the 2022 American Society of Hematology (ASH) Annual Meeting. RGI-2001 activates Regulatory T-cells (Tregs) through a novel mechanism that may have implications for the treatment of other autoimmune and chronic inflammatory diseases....REGiMMUNE concludes that intravenous administration of RGI-2001 added to standard-of-care tacrolimus/methotrexate showed promising efficacy in the prevention of clinically significant aGvHD with a favorable safety profile."

P2b data • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

REGiMMUNE to Present Results from the Phase 2b Study of RGI-2001 at the 2022 American Society of Hematology Annual Meeting (Businesswire) - "REGiMMUNE...today announced that the Phase 2b clinical data from 49 patients receiving RGI-2001 for the prevention of acute graft-versus-host disease (aGVHD) will be presented at the 2022 American Society of Hematology (ASH) Annual Meeting in New Orleans, Louisiana on December 12, 2022....Based on the analysis, RGI-2001 showed reduced incidence of aGVHD with an excellent safety profile. I am thrilled that the RGI-2001 study results have been selected for oral presentation at the ASH meeting."

P2b data • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=49 | Active, not recruiting | Sponsor: Regimmune Corporation | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2022 ➔ Apr 2023 | Trial primary completion date: Mar 2022 ➔ Aug 2022

Enrollment closed • Trial completion date • Trial primary completion date • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Myelodysplastic Syndrome • Transplantation

Chimerism through the activation of invariant natural killer T cells prolongs graft survival after transplantation of induced pluripotent stem cell-derived allogeneic cardiomyocytes. (PubMed, PLoS One) - "We conclude that mixed-chimera mice established using RGI-2001 showed prolonged graft survival after allogeneic iPSC-CM transplantation. This donor-specific immunotolerance might increase the efficacy of regenerative therapies for heart failure with allogeneic iPSC-CMs."

Journal • Bone Marrow Transplantation • Cardiovascular • Congestive Heart Failure • Heart Failure • Immunology • Transplantation • CD40LG

Haplo Peripheral Blood Sct In GVHD Prevention (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Zachariah Michael DeFilipp; Trial completion date: Feb 2023 ➔ Dec 2023; Trial primary completion date: Apr 2022 ➔ Dec 2022

Clinical • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2; N=50; Recruiting; Sponsor: Regimmune Corporation; Trial completion date: May 2022 ➔ Dec 2022; Trial primary completion date: Aug 2021 ➔ Mar 2022

Clinical • Trial completion date • Trial primary completion date • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Myelodysplastic Syndrome • Transplantation

[VIRTUAL] Pharmacokinetics and Safety of KRN7000 Following Intravenous Infusion of RGI-2001 in Allogeneic Transplant Patients (ASH 2020) - "Plasma levels of RGI-7000 were similar after the first dose and multiple doses. Its plasma half-life was approximately 36 hours."

Clinical • PK/PD data • Dermatology • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Mucositis • Oncology • Pruritus • Transplantation • IL4

Haplo Peripheral Blood Sct In GVHD Prevention (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Zachariah Michael DeFilipp; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

Haplo Peripheral Blood Sct In GVHD Prevention (clinicaltrials.gov) - P1; N=20; Not yet recruiting; Sponsor: Zachariah Michael DeFilipp

Clinical • New P1 trial • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

Regimmune phase 2a results of RGI-2001, an activator of invariant natural killer T cells, published in 'Biology of Blood and Marrow Transplantation' (MarketWired) - P2a, N=29; "REGiMMUNE Corporation...is pleased to announce the publication of its Phase 2a clinical trial results for RGI-2001...some patients treated with RGI-2001 had markedly increased the number of Tregs (CD4+CD25+CD127loFoxp3+) within 1 to 3 weeks after HCT. The patients who responded with an increased number of Tregs also had decreased GvHD onset compared with nonresponders."

P2a data • Graft versus Host Disease

Contrasting Effects of Conventional Immunosuppressants in Establishing Murine Transplantation Tolerance (KIDNEY WEEK 2019) - "Background Tacrolimus (TAC) is one of the most commonly used calcineurin inhibitors (CNIs) for immunosuppression maintenance after kidney transplantation...An inhibitor of the mammalian target of rapamycin (mTOR-I) such as everolimus (EVL) shows immunosuppressive activity by inhibiting other pathways...We previously reported an approach to induce mixed chimerism by stimulating invariant natural killer T cells with liposomal formation of alpha-galactosylceramide (RGI-2001) and CD40 ligand (CD40L) blockade...Conclusion TAC negatively impacted the regimen by interfering with Treg proliferation and activation. Funding Government Support - Non-U.S."

IO Biomarker • Preclinical

RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2; N=50; Recruiting; Sponsor: Regimmune Corporation; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Intravenous Administration of RGI-2001 in Patient Undergoing Allogenic Hematopoietic Stem Cell Transplantation (AHSCT) (clinicaltrials.gov) - P1/2; N=68; Completed; Sponsor: Regimmune Corporation; Active, not recruiting ➔ Completed

Trial completion

Japanese Biopharmaceutical firm relocating to Taiwan (Taipei Times) - "REGiMMUNE Ltd...yesterday inked an agreement with CTBC Securities Co...as part of its plans for an initial public offering (IPO) by 2021...It has decided to restructure itself and move its headquarters to Taiwan...company has gained approval from the US Food and Drug Administration (FDA) to conduct a phase-2b trial next month and plans to enroll 50 patients...The trial is expected to be completed by 2022..."

Clinical • New P2 trial • Trial completion date

RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2; N=50; Not yet recruiting; Sponsor: Regimmune Corporation

Clinical • New P2 trial