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June 9, 2017

Exclusive License Agreement for Avacopan, a selective inhibitor of the Complement C5a Receptor, for Rare Kidney Diseases, in Japan

Kissei Pharmaceutical Co., Ltd. (Head office: Matsumoto-city, Nagano, Chairman and CEO: Mutsuo Kanzawa; "Kissei") and Vifor Fresenius Medical Care Renal Pharma (Headquarters: St. Gallen, Switzerland, CEO: Stefan Schulze; "VFMCRP") have signed an agreement for exclusive license to develop and market the complement C5a receptor*1 (C5aR) inhibitor "Avacopan" (international nonproprietary name) in Japan.

Under the terms of this agreement, Kissei obtains exclusive development and marketing rights of Avacopan in Japan from VFMCRP, who holds the global commercial rights for the drug (excluding the US and China). Kissei will conduct development in Japan and exclusively market Avacopan upon acquisition of marketing approval.

Avacopan, an orally-administered small-molecule agent for the treatment of rare kidney diseases, has been developed by ChemoCentryx, Inc., in the US. It inhibits C5a receptors in leukocytes, including neutrophils, and exhibits anti-inflammatory properties by preventing the migration of leukocytes as well as the expression and induction of adhesion molecules. ANCA (anti-neutrophil cytoplasmic auto-antibody)-associated vasculitis*2, which is one of the targets of Avacopan, is a rare and severe autoimmune disease that is designated as an intractable disease by the Ministry of Health, Labour, and Welfare in Japan. The number of patients with ANCA-associated vasculitis has increased rapidly in recent years, and in many cases, they present necrotizing glomerulonephritis. Currently, Phase III clinical trials are being conducted by ChemoCentryx in Europe, the United States and other countries, for the indication of ANCA-associated vasculitis. Phase II clinical trials around C3 glomerulopathy*3 and atypical hemolytic uremic syndrome*4 are also planned.

Kissei is working to expand its portfolio of products in the urology and renal/dialysis areas, as well as in other areas with high unmet medical needs. With the signing of this agreement, Kissei will further enhance its efforts in those areas and strive to deliver Avacopan for the treatment of rare diseases to patients at the earliest.


《Reference》

*1: The complement C5a receptor:

Complement is a system of proteins found in blood and is involved in various immune responses and preventing infection. There are many kinds of complements, generally expressed as C by taking the initial letter of complement. Of these, C5a acts as a chemokine (chemotactic factor), attracting neutrophils to the inflamed area. Avacopan is thought to exhibit anti-inflammatory properties by inhibiting the C5a receptor, thereby suppressing the activity of neutrophils that damage blood vessels.

*2: ANCA (anti-neutrophil cytoplasmic auto-antibody)-associated vasculitis (AAV):

AAV is a rare and severe autoimmune disease characterized by no or very little immune complex deposition, necrotizing inflammation of the small blood vessels, and high ANCA-positive rates. It damages various organs, including the kidneys, lungs, and nervous system. The number of patients with AAV is estimated to be over 10,000 in Japan (based on the number of recipients of the certificates for special disease treatment in 2015). The current standard treatment for AAV is the concurrent use of adrenocorticosteroids and immunosuppressants. Steroid replacement with Avacopan is expected to avoid the adverse events associated with steroid use.

*3: C3 glomerulopathy (C3G):

C3G, a designated intractable disease, is a type of primary membranoproliferative glomerulonephritis. It is caused by renal tissue disorders induced by abnormalities in the complement pathway. In Japan, the number of patients with primary membranoproliferative glomerulonephritis is estimated to be over 40 (based on the number of recipients of the certificates for special disease treatment in 2015). Currently, there are no drugs approved for the indication of C3 glomerulopathy.

*4: Atypical hemolytic uremic syndrome (aHUS):

aHUS is a designated intractable disease caused by abnormalities in complement factors. It is characterized mainly by 1) hemolytic anemia, 2) decrease in platelets, and 3)acute nephropathy. In Japan, the number of patients with aHUS is estimated to be over 40 (based on the number of recipients of the certificates for special disease treatment in 2015). The biopharmaceutical eculizumab is currently used to treat aHUS.

Vifor Fresenius Medical Care Renal Pharma Ltd. (VFMCRP):

Vifor Pharma Group, formerly Galenica Group, is a global specialty pharmaceuticals company. It aims to become the global leader in iron deficiency, nephrology and cardio-renal therapies. Headquartered in Switzerland, Vifor Pharma Group is listed on the Swiss Stock Exchange. VFMCRP belongs to the Vifor Pharma Group and is a common company of Vifor Pharma Group and Fresenius Medical Care, develops and commercializes innovative and high quality therapies to improve the life of patients suffering from chronic kidney disease (CKD) worldwide. VFMCRP has also given Kissei the license for sucroferric oxyhydroxide (sold as Velphoro® in Europe) for hyperphosphatemia.

ChemoCentryx, Inc.:

ChemoCentryx, Inc. is a biopharmaceutical company headquartered in California, US, and it is listed on NASDAQ. The company is focused on discovering, developing, and commercializing orally-administered therapeutics in autoimmune diseases, inflammatory disorders, and cancer.