The safety and efficacy study will enroll approximately 50 bone marrow or peripheral blood stem cell transplantation leukemia patients following chemotherapy.
The U.S.-based study will be conducted in five leading cancer centers including the Fred Hutchinson Cancer Research Center, Stanford University Medical Center, the Ohio State University Medical Center, University of California, San Diego and Massachusetts General Hospital/Harvard University.
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.
"The major problem with existing immunosuppressant therapy is the reduction of GvL," explained Haru Morita, President and Chief Executive Officer of REGiMMUNE. "We hope to demonstrate, in our Phase I/II study, that by inducing Treg-mediated transplantation tolerance, RGI-2001 can be developed as an effective treatment to prevent graft rejection, without the risks associated with current therapies."
$5 Million U.S. Financing Announced
REGiMMUNE also announced the closing of the first tranche of a Preferred A financing. The company raised $5 million U.S. in this tranche, which was led by Daiwa Enterprise Co. Ltd. and Japan Asia Investment Corporation (JAIC). Additionally, new investors Shinkin Capital Corporation and MediNet Co. Ltd. joined existing investor SMBC Venture Capital Co., Ltd., the VC arm of Sumitomo Mitsui Banking Corporation, in this round. Proceeds from this funding will be used primarily to support the company's first clinical stage product development. The company is continuing to seek investors for a second tranche later this year.