On February 22, 2024 REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, reported the positive results of their phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation (alloHCT) at the 2024 Tandem Meeting of ASTCT and CIBMTR (Press release, REGimmune, FEB 22, 2024, View Source [SID1234642235]).
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aGVHD, primarily mediated by effector T-lymphocytes, is a major cause of morbidity and mortality after alloHCT. RG1-2001 contains the active moiety α-GalCer, which binds the CD1d receptor of antigen-presenting cells resulting in activation of iNK T cells, resulting in a cytokine-dependent Treg proliferation. This phase 2b trial is an open-label, multi-center, single-arm study. RGI-2001, added to standard immunosuppression, is being evaluated for the potential to reduce the incidence or severity of aGVHD in patients following alloHCT. The results are compared with a contemporaneous CIBMTR cohort (NCT04014790).
Through day 100, the incidence of grades II-IV aGVHD was 22.9% of subjects on RGI-2001 compared to 38.8% of those in the CIBMTR cohort. RGI-2001 also demonstrates higher rates of acute GVHD-free survival (72.9% in grade II-IV and 91.7% in grades III-IV compared with 50.7% and 77.3% in those on CIBMTR cohort, respectively) and overall survival (91.7% compared with 79.2% on CIBMTR cohort). Relapse rates between the 2 cohorts are similar, suggesting no compromise of the graft-versus-leukemia effect.
"We are thrilled to be selected for oral presentation at the Tandem Meeting. The results showed promising safety and efficacy outcomes using RGI-2001 in addition to the standard GVHD prevention medications, providing a strong foundation to advance our plans for phase 3 study." said Kenzo Kosuda, CEO of REGiMMUNE.
REGiMMUNE concludes that RGI-2001 added to standard-of-care shows positive results for acute GVHD prevention with survival benefit and no increase in relapse compared to CIBMTR control. Based on these results, a phase 3 randomized controlled study is being planned to confirm the efficacy and safety of RGI-2001 in alloHCT.
Details of presentation, please see RGI-2001 Tandem 2024 Oral Presentation