Suzhou, China; May 20, 2019 -- CStone Pharmaceuticals (CStone; HKEX: 2616) today announced that the first patient has been dosed in China for the global Phase I clinical study of BLU-554 (CS3008), a highly potent and selective inhibitor of fibroblast growth factor receptor 4 (FGFR4) discovered by CStone's partner, Blueprint Medicines (Blueprint).  The study is part of Blueprint's ongoing global Phase I clinical trial in patients with advanced hepatocellular carcinoma (HCC), and is designed to evaluate the safety, tolerability, pharmacodynamics and preliminary antitumor activity of BLU-554 in advanced HCC patients.

Liver cancer is one of the most common malignant tumors in China, of which HCC accounts for approximately 85-90% of all cases[《原发性肝癌诊疗规范》2017年版 ]. China is burdened with 50% of worldwide liver cancer incidence and mortality[ Freddie Bray, Jacques Ferlay, Isabelle Soerjomataram, et al. Global Cancer Statistics 2018. ], with approximately 370,000 new diagnoses each year. In 2015, there were 326,000 deaths caused by liver cancer, making it China's second leading cause of cancer death[《2015年中国恶性肿瘤流行情况分析》]. The majority of liver cancer patients are not diagnosed until the advanced stages of the disease and are therefore not eligible for surgery[ Earl TM and Chapman WC. Hepatocellular carcinoma: Resection versus Transplantation. Semin Liv Dis 2013: 33(3):282-92 ]. Currently, treatment options for advanced HCC are extremely limited, presenting a significant unmet need for new therapies.

Based on preclinical studies, an estimated 30% of all HCC patients have aberrantly activated FGFR4 signaling, which is believed to be the driver for HCC in these patients. BLU-554 was specifically designed to inhibit FGFR4 with exquisite selectivity and treat patients with HCC caused by abnormal FGF19-FGFR4 signaling.

In June 2018, CStone entered into an exclusive collaboration and license agreement with Blueprint to develop and commercialize three therapeutic candidates, including BLU-554, in Mainland China, Hong Kong, Macau and Taiwan. Blueprint retains development and commercial rights to the licensed therapeutic candidates in the rest of the world.

Dr. Frank Jiang, Chairman and CEO of CStone, commented: “Precision therapy is an important part of CStone's pipeline strategy. Through global collaborations, CStone has brought to China a number of targeted-therapy candidates including BLU-554.  We will continue to deepen our exploration of BLU-554's potential as a mono therapy and in combination with PD-L1, with the aim of providing HCC patients with more effective treatments.”

“The U.S. FDA has already granted orphan drug designation to BLU-554 for the treatment of HCC. Previously presented data from the ongoing global Phase I trial showed that BLU-554 was generally well-tolerated and has the potential to provide clinical benefit to patients with advanced HCC caused by aberrant FGF19-FGFR4 pathway activation,” noted Dr. Jason Yang, Chief Medical Officer (CMO) of CStone.  “The study now underway in China is part of this ongoing global Phase I trial that we hope will produce positive results in the clinic.”

About BLU-554

BLU-554 is an orally available, potent, irreversible inhibitor of FGFR4. BLU-554 was specifically designed by Blueprint Medicines to inhibit FGFR4 with exquisite selectivity, thereby sparing the paralogs FGFR1, FGFR2 and FGFR3 and preventing potential adverse effects. Blueprint Medicines is developing BLU-554, an investigational medicine, for the treatment of patients with HCC caused by abnormal FGF19-FGFR4 signaling. Blueprint Medicines estimates that approximately 30% of patients with HCC have tumors with aberrantly activated FGFR4 signaling.

About CStone

CStone Pharmaceuticals (HKEX:2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, five late-stage candidates are at or near pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: www.cstonepharma.com.

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