Positive Topline Data from Pivotal Phase-III ADVOCATE Trial Demonstrating Avacopan's Superiority over Standard of Care in ANCA Vasculitis

Kissei Pharmaceutical Co., Ltd. (Chairman and CEO: Mutsuo Kanzawa; "Kissei") announced today that Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and ChemoCentryx, Inc., (CCXI), have obtained positive topline data from the pivotal phase-III ADVOCATE trial of avacopan for the treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis).

The ADVOCATE trial of avacopan was a global double-blind phase-III trial of 331 patients with ANCA-associated vasculitis in 20 countries. Its primary endpoints were remission at 26 weeks and sustained remission at 52 weeks, assessed by the Birmingham Vasculitis Activity Score, or BVAS. The study met both of its primary endpoints, the avacopan treatment group has demonstrated statistically significant non-inferiority to the current standard of care (glucocorticoids) remission at 26 weeks. Furthermore, the avacopan treatment arm achieved statistically significant superiority to standard of care at week 52 and exhibited the additional benefit of avoidance of harm compared to glucocorticoids. Avacopan demonstrated a safe and well-tolerated profile. A full analysis of the data is under way.

For details on the announcements of VFMCRP and CCXI, please refer to the press release on the website of both companies.

VFMCRP： http://www.viforpharma.com/en/investors
CCXI： https://ir.chemocentryx.com/investor-relations

In June 2017, Kissei entered into the agreement with VFMCRP for an exclusive sub-license to develop and commercialize Aavacopan in Japan and Kissei has participated in the Japanese part of the ADVOCATE trial suponsored by CCXI. After further analysis of these clinical results, we will proceed with discussions with the Pharmaceuticals and Medical Devices Agency for NDA in Japan.

Kissei is working to expand its portfolio of products in the urology and renal disease areas, as well as rare diseases area. Cooperating with the overseas partners, we will strive to deliver avacopan for the treatment of rare diseases to patients at the earliest.

These clinical results have negligible effects on Kissei's consolidated business forecasts for the fiscal year ended in March 2020.

《Reference》

About Avacopan:

Avacopan, an orally-administered small-molecule agent for the treatment of rare kidney diseases, has been developed by CemoCentryx. 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-associated vasculitis, 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.

ChemoCentryx is responsible for the discovery and development of avacopan and owns and retains the commercial rights to the drug in the United States. VFMCRP has an exclusive license to commercialize the drug in all countries outside the United States. VFMCRP has granted KISSEI PHARMACEUTICAL CO LTD., an exclusive sub-license to develop and commercialize the drug in Japan.

About 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.

About 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 2017). The current standard of care is the concomitant use of glucocorticoids and immunosuppressants. Avacopan is expected to avoid the adverse events associated with glucocorticoids.

About 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 (SIX Swiss Exchange, VIFN, ISIN: CH0364749348). 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 granted Kissei the license for sucroferric oxyhydroxide (sold as Velphoro® in Europe) for the treatment of hyperphosphatemia.

About ChemoCentryx, Inc.:

ChemoCentryx is a biopharmaceutical company developing new medications targeted at inflammatory and autoimmune diseases and cancer, which is located in California, US, and is listed on NASDAQ (NASDAQ: CCXI). ChemoCentryx targets the chemokine and chemoattractant systems to discover, develop and commercialize orally-administered therapies.