On January 28, 2019 CStone Pharmaceuticals (CStone) reported that the National Medical Products Administration (NMPA) recently approved the clinical trial application (CTA) to start a Phase I clinical trial in China for BLU-554 (CS3008), an inhibitor of fibroblast growth factor receptor 4 (FGFR4) discovered by the company’s partner Blueprint Medicines (Press release, CStone Pharmaceauticals, JAN 28, 2019, View Source [SID1234532931]). CStone has exclusive rights to develop and commercialize BLU-554 in Mainland China, Hong Kong, Macau and Taiwan.
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The trial is part of a global Phase I trial for BLU-554 in patients with advanced hepatocellular carcinoma (HCC) not previously treated with a tyrosine kinase inhibitor (TKI). The aim is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of BLU-554 in this patient subset. CStone and Blueprint Medicines expect to enroll the first patient in the Phase I clinical trial in China soon.
HCC is the most common form of liver cancer. In China, HCC is the third most significant cause of cancer-related death, with approximately 466,000 new diagnoses and 422,000 deaths caused by the disease each year. It is estimated that approximately 30% of patients with HCC have tumors with aberrantly activated FGFR4 signaling. The majority of Chinese HCC patients (55%) are not diagnosed until the advanced stages of the disease. Currently, treatment options for advanced HCC patients remain limited.
BLU-554 is a potent and highly selective small-molecule inhibitor of FGFR4. Blueprint Medicines announced preliminary Phase I clinical data of BLU-554 at the European Society of Clinical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress, as of a data cutoff date of August 18, 2017. In heavily pre-treated patients with FGFR4-driven HCC (n=38), the data showed an objective response in 6 patients (16%; 1 complete response and 1 partial response pending confirmation), while disease control was achieved by 26 patients (68%), and 18 patients (49%) had a reduced tumor burden. In 5 patients who had not previously received TKI therapy, preliminary evidence of prolonged disease control was observed. BLU-554 was well-tolerated and most adverse events reported by investigators were Grade 1 or 2.
Dr. Frank Jiang, CEO and chairman of CStone, commented: "Compared with current treatments, BLU-554 has produced encouraging data in terms of tolerability and disease control rates. We also plan to conduct a Phase I trial of BLU-554 in combination with CS1001 for the treatment of advanced HCC patients in China in the second half of 2019. Our hope is to make more effective treatment options available to HCC sufferers."
About BLU-554
BLU-554 is an oral, highly selective and irreversible inhibitor of FGFR4 developed by Blueprint Medicines, with a precise selectivity for FGFR4 that spares the paralog kinases FGFR1, FGFR2 and FGFR3. Blueprint Medicines is developing BLU-554 for the treatment of HCC caused by FGFR4 activation, estimated to account for approximately 30% of patients with HCC tumors. BLU-554 has been granted orphan drug status by the U.S. Food and Drug Administration.
In June 2018, CStone and Blueprint Medicines entered into a license and collaboration agreement in which Blueprint Medicines granted CStone exclusive rights to develop and commercialize BLU-554 in Greater China. Blueprint Medicines retains development and commercial rights for BLU-554 in the rest of the world.