TOKYO, JAPAN--(Marketwired - Mar 9, 2017) - REGiMMUNE Corporation (RGI) is pleased to announce the publication of its Phase 2a clinical trial results for RGI-2001, a novel liposomal formulation of a synthetic derivative of alpha-galactosylceramide, a naturally occurring ligand that binds to CD1d and activates invariant natural killer T cells (iNKT). The company is developing RGI-2001 to ameliorate Graft versus Host Disease (GvHD) after allogeneic hematopoietic cell transplantation. The paper: "Increased Foxp3⁺Helios⁺ Regulatory T Cells and Decreased Acute Graft-versus-Host Disease after Allogeneic Bone Marrow Transplantation in Patients Receiving Sirolimus and RGI-2001, an Activator of Invariant Natural Killer T Cells" is now available from the online publication "Biology of Blood and Marrow Transplantation" http://www.bbmt.org/article/S1083-8791(17)30194-5/pdf.

This is the first clinical demonstration of a novel therapeutic that potentially expands Foxp3⁺regulatory T cells (Treg) mediated by the activation of iNKT cells in patients. RGI-2001, which utilizes REGiMMUNE's reVax technology, promotes transplantation tolerance by inducing Tregs. Tregs have been shown to have significant potential for treating GvHD; in studies by independent researchers, Tregs have proven to produce longer patient survival because they could suppress rejection without reducing an anti-tumor graft versus leukemia effect.

"The studies show that the administration of RGI-2001 was safe and resulted in increased numbers of Tregs vs. conventional CD4⁺ T cells after allogeneic transplantation in some patients, especially in those treated with the mammalian target of rapamycin inhibitor sirolimus as part of their GvHD prophylaxis regimen," commented Dr. Jack Bui, MD, PhD, Associate Professor, Director of Clinical Flow Cytometry and the Stem Cell Processing Laboratory, Department of Pathology, University of California San Diego.

The Phase 2a clinical trial of single-dose RGI-2001 on day 0 in the treatment of 29 patients undergoing allogeneic HCT demonstrated that some patients treated with RGI-2001 had markedly increased the number of Tregs (CD4⁺CD25⁺CD127^loFoxp3⁺) within 1 to 3 weeks after HCT. The patients who responded with an increased number of Tregs also had decreased GvHD onset compared with nonresponders. Some of the patients in this subset received sirolimus in addition to the standard of care using calcineurin inhibitors plus methotrexate for immunosuppression after HCT, suggesting potential synergy between sirolimus and RGI-2001 in Treg expansion in vivo.

A randomized study is being designed to further evaluate the impact of serial dosing of RGI-2001 on Treg biology and GvHD prevention.

About REGiMMUNE
REGiMMUNE Corporation 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 cells (Treg). 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; for inflammatory bowel disease; 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 Berkeley, California. For more information, visit www.regimmune.com.