REGiMMUNE Corp.

TAIPEI, Taiwan--( BUSINESS WIRE )--REGiMMUNE Limited (REGiMMUNE), a biotech company focused on creating innovative immunotherapies for immune disorders and cancer, and San Fu Biotech (SFB), a subsidiary of San Fu Chemical Co., Ltd. (4755.TW) have entered a licensing agreement to develop and commercialize RGI-2001 for the prophylaxis of acute Graft-versus-host disease (aGvHD) in major Asian countries. RGI-2001, with the novel mechanism to improve existing treatments, is a potentially first-in-class small molecule drug candidate targeted for preventing aGvHD, a life-threatening complication resulting from allogeneic hematopoietic stem cell transplantation (HSCT). GvHD is a systemic disorder that occurs when the graft's immune cells recognize the host as foreign and attack the recipient’s body cells or organs, and leads to skin rash, liver problems, abdominal pain or cramps, diarrhea, and increased risk for infections. Patients undergoing allogeneic HSCT have a high-risk of developing aGVHD associated with significant morbidity and mortality. Despite the use of prophylactic immunosuppressive therapy, clinically significant aGvHD develops in 20%-40% of HLA-matched related and unrelated allogeneic HSCT and severe cases contribute to non-relapse mortality. Under the terms of agreement, REGiMMUNE and SFB will collaborate closely to accelerate the development of RGI-2001 in major Asian countries and will conduct clinical trials to study the efficacy of the drug in aGvHD. Together, both parties intend to submit an Investigational New Drug Application to the Taiwan Food and Drug Administration to initiate clinical trials in Taiwan for the prophylaxis of aGvHD before the end of 2023. SFB will pay to REGiMMUNE development milestones and royalties on net profit upon successful commercialization of RGI-2001. SFB also retains the first rights of negotiation to develop and commercialize RGI-2001 for new indications in the authorized territories. RGI-2001 of REGiMMUNE, awarded Orphan Drug Designation by the US FDA in GvHD in 2012, has completed the phase II clinical trial targeting at the prophylaxis of aGvHD in the US. The efficacy and safety results were positive. Furthermore, RGI-2001 was selected for oral presentation at the American Society of Hematology (ASH) annual meeting in December 2022 and planned to submit phase III IND to US FDA in Q4 of 2023. Kenzo Kosuda , Chief Executive Officer and President, REGiMMUNE Limited, said : “ This agreement underscores our focus and commitment to improve the incumbent therapies of aGvHD prevention and treatment. The fast-growing patients with GvHD in Asia create a high unmet need for more effective treatments. ” “We are pleased to collaborate with SFB and San Fu Chemical Group which has a long history in Taiwan. The Group’s abundant biomedical resources, expertise and innovative strategy make San Fu a strong commercial partner for REGiMMUNE in Asia. Together, we look forward to expediting the development of RGI-2001 in this important market.” Simon H.H. Wu, SFB & San Fu Chemical Group Chairman, emphasized: “We are excited to add RGI-2001 to our growing portfolio of innovative therapeutic agents. This enhances our autoimmune pipeline and aligns with our long-term research and development strategy. RGI-2001 has demonstrated promising results in phase II studies in the US and we look forward to working together with REGiMMUNE to deliver treatments that will benefit the lives of patients.” About RGI-2001 RGI-2001 is a liposomal formulation of an alpha-galactosylceramide (alpha-GalCer) analog. Alpha-GalCer is a ligand for CD1d expressed on antigen presenting cells and invariant natural killer T cells (iNKT). Liposomal alpha-GalCer promotes tolerogenic immune cascades, resulting in the activation and expansion of regulatory T cells (Tregs). RGI-2001 is REGiMMUNE’s lead drug candidate currently in Phase 3 planning for the prophylaxis of acute graft-versus-host disease (aGVHD). About REGiMMUNE Limited REGiMMUNE is a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies by harnessing the power of regulatory T cells (Tregs). REGiMMUNE is creating a pipeline of novel product candidates that either enhance Treg activities for immune diseases or suppress Treg activities for cancer. About San Fu Biotech San Fu Biotech, a wholly-owned subsidiary of San Fu Chemical, develops first-in-class new drugs to address unmet medical needs, such as cancer tumors, autoimmune disease and ophthalmology, for benefiting patients around the world.

RGI-2001 was well tolerated and associated with a meaningful reduction in the incidence of severe acute GvHD TAIPEI, Taiwan--(BUSINESS WIRE)-- REGiMMUNE Limited, a clinical-stage biopharmaceutical company developing innovative immunotherapies for immune disorders and cancer, today announced the positive results of their Phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGvHD) after allogeneic hematopoietic cell transplantation (HCT) at the 2022 American Society of Hematology (ASH) Annual Meeting. RGI-2001 activates Regulatory T-cells (Tregs) through a novel mechanism that may have implications for the treatment of other autoimmune and chronic inflammatory diseases. Patients who undergo HCT have a high-risk of developing aGVHD which is associated with significant morbidity and mortality. Acute GVHD is characterized as a reaction of donor immune cells against host tissues. Despite the use of prophylactic immunosuppressive therapy, clinically significant aGvHD develops in 20%-40% of matched related and unrelated allogenic HCT and severe cases contribute to non-relapse mortality. RGI-2001, added to standard immunosuppression, is being evaluated for the potential to reduce or prevent aGvHD after HCT. Earlier studies had shown that a single dose of RGI-2001 given on the day of transplantation was safe and potentially contributed to aGvHD prevention. “We are very excited about the RGI-2001 data and the potential to better prevent acute GVHD, which will benefit patients and reduce the risk of morbidity and mortality. RGI-2001 is an innovative therapy that may have a role in GVHD prevention.” said Dr. Yi-Bin Chen, Associate Professor of Medicine, Harvard Medical School and Director. Hematopoietic Cell Transplant and Cell Therapy Program, Massachusetts General Hospital. “We are definitely encouraged by the positive results from this study; it also provides a strong foundation to confidently advance our plans for phase 3 study.” said Kenzo Kosuda, CEO of REGiMMUNE. This phase 2b clinical trial was an open-label, multi-center, single-arm study to evaluate six weekly doses of RGI-2001 in combination with standard of care tacrolimus/methotrexate for the prevention of aGvHD in patients following allogenic HCT. RGI-2001 was administered at a dose of 100 ug/kg IV infusion weekly for 6 weeks, starting on the day of transplantation. The study included 49 patients and was conducted in the US at 7 centers. Efficacy Results from the phase 2b trial in allogenic HSCT patients: All 49 patients had at least 100 days of follow-up after HCT, and 39 of 49 had complete follow-up to day 180. The median follow-up of survivors was 310 days (range 111-365 days) There were no cases of engraftment failure and no serious adverse events attributed to RGI-2001; and treatment emergent adverse events (>10%) include gastrointestinal AE such as diarrhea, stomatitis and nausea. Through day 100, the incidence of grades II-IV aGvHD was 20.4% (95% confidence interval 10.2-34.3%), only 2 cases of which were grades III-IV aGvHD [(4.1% (0.5-14.0%)). At day 180, estimates of overall survival, grades II-IV aGvHD-free survival, and disease relapse were 93.6% (81.6-97.9%), 75.2% (60.5-85.1%), and 4.1% (0.7-12.6%), respectively. REGiMMUNE concludes that intravenous administration of RGI-2001 added to standard-of-care tacrolimus/methotrexate showed promising efficacy in the prevention of clinically significant aGvHD with a favorable safety profile. Based on these compelling phase 2b results, REGiMMUNE is strongly considering moving forward with a phase 3 study. For more information, please find RGI-2001-003 ASH Oral Presentation. About RGI-2001 RGI-2001 is a liposomal formulation of an alpha-galactosylceramide (alpha-GalCer) analog. Alpha-GalCer is a ligand for CD1d expressed on antigen presenting cells and invariant natural killer T cells (iNKT). Liposomal alpha-GalCer promotes tolerogenic immune cascades, resulting in the activation and expansion of regulatory T cells (Tregs). RGI-2001 is REGiMMUNE’s lead drug candidate currently in Phase 3 planning for the prophylaxis of acute graft-versus-host disease (aGVHD). About REGiMMUNE Limited REGiMMUNE is a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies by harnessing the power of regulatory T cells (Tregs). REGiMMUNE is creating a pipeline of novel product candidates that either enhance Treg activities for immune diseases or suppress Treg activities for cancer. View source version on businesswire.com: Contacts Corporate: Charlie Hsu Chief Financial Officer REGiMMUNE Corporation Ltd. +886 2 2555 3377 #511 charlieh@regimmune.com Source: REGiMMUNE Limited View this news release online at:

DUBLIN, June 10, 2022 /PRNewswire/ -- The "Graft Versus Host Disease (GvHD) Treatment Market Forecast - Epidemiology & Pipeline Analysis 2022-2027" report has been added to ResearchAndMarkets.com's offering. The report considers the present scenario of the graft versus host disease market and its market dynamics for the period 2022-2027. It covers a detailed overview of several market growth enablers, restraints, and trends. The study covers both the demand and supply sides of the market. It also profiles and analyses leading companies and several other prominent companies operating in the market. The graft versus host disease market to grow at a CAGR of 7.44% during 2021-2027. SEGMENT ANALYSIS Corticosteroids accounted for the significant share of more than 28.97% in the global GvHD treatment market due to its wide recommendation in front line setting. The acute GVHD (aGVHD) accounted for a significant share of 34.73% in the global graft versus host disease (GVHD) treatment market due to 30% to 50% of hematopoietic stem cell transplant recipients develop acute GVHD. Males are accounted for the significant share of 59.64% in the global GvHD treatment market due to rise in number of hematopoietic stem cell transplantations. Key Report Features: Detailed overview of GVHD, including disease definition, classification, diagnosis, and treatment pattern Overview of the global graft versus host disease treatment market trends in the eight major markets (8MM) Historical, current, and projected patient pool of GVHD in the eight major markets (8MM) for the 2018 - 2027 period Disease, Gender, and Drug Class segmentations of the GVHD in the eight major markets (8MM) Historical, current, and projected market size & forecast of GVHD in eight major markets (8MM) for the 2018 - 2027 period In-depth analysis of the market segments, which includes products, treatment, and competitor analysis Market share of the market players, company profiles, product specifications, SWOT analysis, and competitive landscape Comprehensive data on emerging trends, market drivers, growth opportunities, and restraints Detailed overview of marketed drugs with key coverage of developmental activities, including sponsor name, approved indication, territory, collaborations, licensing, mergers and acquisitions, regulatory designations, and other product-related activities Detailed overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for GVHD across the complete product development cycle, including all clinical and non-clinical stages Detailed overview of clinical trial activities and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and Geography type for GVHD across all clinical stages Coverage of dormant and discontinued pipeline projects along with the reasons across GVHD Coverage of major milestones (product approvals/launches timelines, clinical trial result publications, regulatory designations, licensing & collaborations, research & development progress of pipeline assets) in the GVHD space Market Segmentation by Drug Class Corticosteroids Kinase Inhibitors Calcineurin Inhibitors TNF-Alpha Inhibitors Other Class of Drugs Market Segmentation by Treatment Type GVHD Prophylaxis Chronic GVHD Acute GVHD Market Segmentation by Gender Type Males Females GEOGRAPHIC ANALYSIS The United States dominated the graft versus host disease (GVHD) treatment market by geography. However, China is expected to grow faster with a high CAGR in the market. The rise in incidence and prevalence of GVHD patients, presence of key players, and rise in access to health care due to well-established healthcare infrastructure and extensive reach of novel therapeutics are largely driving the United States market. Market Segmentation by Geography North America US Europe France Germany Italy UK Spain APAC China Japan VENDOR ANALYSIS With more than 134+ molecules in various stages of development, it is expected that new vendors are likely to enter into the market with novel mechanism of action and better safety and efficacy profiles compared to the existing patented commercial drugs for treating GVHD. Key Players Incyte Pharmacyclics Bristol-Myers Squibb JCR Pharmaceuticals Co., Ltd. Takeda Pharmaceutical Co Ltd (Takeda) MaaT Pharma XeniKos Medac GmbH Mesoblast Kadmon Jazz Pharmaceuticals Gsk Novartis Syndax Pharmaceuticals Inc Other Prominent Vendors Neovii ElsaLys Biotech Mallinckrodt Regimmune Roche Altrubio CTTQ Equillium Janssen (johnson & johnson) ASC Therapeutics MSD Genentech Sun Pharma Mink Therapeutics Pluristem For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 SOURCE Research and Markets