Biotechnology company REGiMMUNE Corporation has begun a phase II study of its proprietary compound RGI-2001 for graft versus host disease (GvHD) associated with hematopoietic stem cell transplantation. The multicentre study is being conducted in six highly regarded US cancer centres including the Fred Hutchinson Cancer Research Centre, Stanford University Medical Centre, the Ohio State University Medical Centre, University of California, San Diego, Massachusetts General Hospital/Harvard University and University of Miami Sylvester Comprehensive Cancer Centre.
The study was initiated following the successful completion of a phase I study in patients with bone marrow or peripheral blood stem cell transplantation leukemia patients following chemotherapy.
“Our proprietary immune-regulating technology is initially being tested in the clinic with RGI-2001 to demonstrate its potential as an effective treatment to prevent graft rejection, without the risks associated with current therapies,” explained Haru Morita, president and chief executive officer. “REGiMMUNE’s goal is to develop novel and proprietary products focused on therapeutic areas that can be addressed with immune-regulating technology including type 1 diabetes, celiac disease, and inhibitor formation in enzyme replacement therapies (ERT) such as haemophilia and lysosomal diseases.”
The RGI-2001 study is a randomized, open-label, multicentre phase II study being conducted in patients undergoing allogeneic hematopoietic stem cell transplantation (AHSCT). This clinical trial is the expansion of the company’s phase I dose-escalation study to evaluate the safety, tolerability and pharmacokinetic profile of RGI-2001 in patients undergoing AHSCT, with radiation or non-radiation myeloablative preparative treatment. The expansion phase will further evaluate the pharmacologic effects of either a maximum tolerated dose, maximum feasible dose or optimal pharmacologically active dose of RGI-2001. This phase II study will expand the enrollment criteria from phase I and allow transplantation by either related or unrelated donors. This study is designed to identify the dose range at which RGI-2001 has an acceptable safety profile, at which biologic activity is observed, and to guide possible dose levels to utilize in later phase studies based on biological activity. Results from the phase I study demonstrated very clean safety profile with no safety concerns observed up to the highest cohort.
RGI-2001 is a liposomal formulation of KRN7000, a synthetic derivative of alpha-galactosylceramide. The compound, which utilizes REGiMMUNE’s reVax technology, promotes transplantation tolerance by inducing regulatory T (Treg) cells. Treg has been shown to have significant potential for treating GvHD; in studies by independent researchers, Treg has proven to produce longer patient survival because it reduces rejection without reducing an anti-tumor graft versus leukemia (GvL) effect.
“Our proprietary immune-regulating technology is initially being tested in the clinic with RGI-2001 to demonstrate its potential as an effective treatment to prevent graft rejection, without the risks associated with current therapies,” explained Haru Morita, president and CEO. “REGiMMUNE’s goal is to develop novel and proprietary products focused on therapeutic areas that can be addressed with immune-regulating technology including type 1 diabetes, celiac disease, and inhibitor formation in enzyme replacement therapies (ERT) such as haemophilia and lysosomal diseases.”
REGiMMUNE is a biotechnology company focused on the discovery, development and commercialization of immune regulatory therapeutics to treat life-threatening and debilitating conditions, including allergies, autoimmune diseases and transplantation.