Tokyo, Japan-(OCT. 28, 2012) – REGiMMUNE Corporation announced today that RGI-2001, the company’s liposomal formulation of alpha-GalCer, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for the treatment of Graft-versus-Host Disease (GvHD) associated with hematopoietic stem cell transplantation.
Orphan Drug Designation is a special status granted by FDA to a novel product to treat a rare disease or condition on the basis of Orphan Drug Act (ODA) In order to give incentive to biotech companies to tackle treatments for race diseases, the company of the product is qualified for some benefits such as tax credit or waiver of a prescription drug user fee.

About RGI-2001

RGI-2001 is a liposomal formulation of alpha-GalCer (a CD1d ligand), which induces regulatory T cells (Treg), a key player in immune tolerance. Because the compound promotes transplantation tolerance, Treg has been shown to have significant potential for treating Graft-versus-Host Disease (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. Unlike immunosuppressents that destroy entire T cell subsets, RGI-2001 induces Treg while maintaining normal immune cell functions and has the potential to reduce GvHD and improve survival for patients.

About Graft-versus-Host Disease (GvHD)

GvHD is a serious complication that results from rejection of the donor Human Stem Cell Transplantation (HSCT). GvHD is caused by the donor’s immune system recognizing the cells/organs derived from the patient (recipient) as pathogens (foreign body). A low grade of GvHD is regarded as positive, because it reflects a normal immune reaction while trying to eliminate residual cancer cells. However, a more severe grade of GvHD can be life threatening as it may significantly damage skin cells, fast growing cells in the gastrointestinal (GI) tract and liver cells.


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 its RGI-4000 series for vaccine adjuvants. The company is seeking pharmaceutical partnership opportunities for its products worldwide, exclusive of Japan. REGiMMUNE is headquartered in Tokyo, Japan and has a U.S. operation in Santa Clara, California

For more information about RGI-2001, please contact us.