REGiMMUNE Limited, a clinical-stage biopharmaceutical company developing innovative immunotherapies for immune disorders and cancer, today 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.
Patients who undergo HCT have a high-risk of developing aGVHD which is associated with significant morbidity and mortality. Acute GVHD is characterized as a reaction of donor immune cells against host tissues. Despite the use of prophylactic immunosuppressive therapy, clinically significant aGvHD develops in 20%-40% of matched related and unrelated allogenic HCT and severe cases contribute to non-relapse mortality. RGI-2001, added to standard immunosuppression, is being evaluated for the potential to reduce or prevent aGvHD after HCT. Earlier studies had shown that a single dose of RGI-2001 given on the day of transplantation was safe and potentially contributed to aGvHD prevention.
“We are very excited about the RGI-2001 data and the potential to better prevent acute GVHD, which will benefit patients and reduce the risk of morbidity and mortality. RGI-2001 is an innovative therapy that may have a role in GVHD prevention.” said Dr. Yi-Bin Chen, Associate Professor of Medicine, Harvard Medical School and Director. Hematopoietic Cell Transplant and Cell Therapy Program, Massachusetts General Hospital.
“We are definitely encouraged by the positive results from this study; it also provides a strong foundation to confidently advance our plans for phase 3 study.” said Kenzo Kosuda, CEO of REGiMMUNE.
This phase 2b clinical trial was an open-label, multi-center, single-arm study to evaluate six weekly doses of RGI-2001 in combination with standard of care tacrolimus/methotrexate for the prevention of aGvHD in patients following allogenic HCT. RGI-2001 was administered at a dose of 100 ug/kg IV infusion weekly for 6 weeks, starting on the day of transplantation. The study included 49 patients and was conducted in the US at 7 centers.
Efficacy Results from the phase 2b trial in allogenic HSCT patients:
- All 49 patients had at least 100 days of follow-up after HCT, and 39 of 49 had complete follow-up to day 180. The median follow-up of survivors was 310 days (range 111-365 days)
- There were no cases of engraftment failure and no serious adverse events attributed to RGI-2001; and treatment emergent adverse events (>10%) include gastrointestinal AE such as diarrhea, stomatitis and nausea.
- Through day 100, the incidence of grades II-IV aGvHD was 20.4% (95% confidence interval 10.2-34.3%), only 2 cases of which were grades III-IV aGvHD [(4.1% (0.5-14.0%)).
- At day 180, estimates of overall survival, grades II-IV aGvHD-free survival, and disease relapse were 93.6% (81.6-97.9%), 75.2% (60.5-85.1%), and 4.1% (0.7-12.6%), respectively.
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. Based on these compelling phase 2b results, REGiMMUNE is strongly considering moving forward with a phase 3 study.
For more information, please find RGI-2001-003 ASH Oral Presentation.
RGI-2001 is a liposomal formulation of an alpha-galactosylceramide (alpha-GalCer) analog. Alpha-GalCer is a ligand for CD1d expressed on antigen presenting cells and invariant natural killer T cells (iNKT). Liposomal alpha-GalCer promotes tolerogenic immune cascades, resulting in the activation and expansion of regulatory T cells (Tregs).
RGI-2001 is REGiMMUNE’s lead drug candidate currently in Phase 3 planning for the prophylaxis of acute graft-versus-host disease (aGVHD).
About REGiMMUNE Limited
REGiMMUNE is a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies by harnessing the power of regulatory T cells (Tregs). REGiMMUNE is creating a pipeline of novel product candidates that either enhance Treg activities for immune diseases or suppress Treg activities for cancer.
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