REGiMMUNE Begins Enrollment for Phase II Clinical Trial for Graft versus Host Disease

Tokyo, Japan – February 6, 2014 – REGiMMUNE Corporation announced today that it has begun a Phase II study of its proprietary compound RGI-2001 for GvHD associated with hematopoietic stem cell transplantation. The multicenter study is being conducted in six highly regarded US cancer centers including the Fred Hutchinson Cancer Research Center, Stanford University Medical Center, the Ohio State University Medical Center, University of California, San Diego, Massachusetts General Hospital/Harvard University and University of Miami Sylvester Comprehensive Cancer Center. 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 hemophilia and lysosomal diseases.”

RGI-2001 Clinical Development

The RGI-2001 study is a randomized, open-label, multicenter 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 1 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 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 hemophilia and lysosomal diseases.”

About REGiMMUNE

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. The company’s proprietary platform technology, reVax, induces immune tolerance in an antigen-specific manner through pharmacological induction of regulatory T (Treg) cells. Using its reVax technology, REGiMMUNE is developing RGI-2001, which may be the first drug in the class of Treg-inducing agents.

The company is also applying its reVax technology to develop a range of pipeline products, including its RGI-1000 series for allergy and its RGI-3100 series for type 1 diabetes. Additionally REGiMMUNE is developing products for preventing inhibitor formation in enzyme replacement therapies (ERT) and for celiac disease with undisclosed partners. The company is seeking pharmaceutical partnership opportunities for its products worldwide, exclusive of Japan. REGiMMUNE is headquartered in Tokyo, Japan and has a US operation in San Francisco, California.

Source: REGiMMUNE Co., Ltd.

Posted: February 2014

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