InnoCare Announces Orphan Drug Designation of Gunagrabtinib by US FDA for Treatment of Cholangiocarcinoma

InnoCare, a leading biopharmaceutical company focusing on cancer and autoimmune diseases, announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its pan-FGFR inhibitor gunagratinib (ICP-192) for the treatment of cholangiocarcinoma.

InnoCare's ICP-192 is a highly selective pan-FGFR inhibitor targeting multiple solid tumors with FGFR gene aberrations. Currently, several clinical studies are ongoing in China and the United States.

According to the latest clinical data presented at the 2021 American Society of Clinical Oncology (ASCO) annual meeting, as of February 2021, a total of 30 patients had received treatment of gunagratinib. Gunagratinib performed well in safety and tolerance, and the maximum tolerated dose (MTD) had not been reached. Among the 12 patients with FGF/FGFR gene aberrations who have completed at least one tumor assessment, the overall response rate (ORR) was 33.3%, including 1 patient (8.3%) of cholangiocarcinoma with complete response (CR), 3 patients (25%) with partial response (PR) and 7 patients having achieved stable disease (SD). The disease control rate (DCR) was 91.7%.

Dr. Jasmine Cui, the Co-founder, Chairwoman and CEO of InnoCare, said, "We are very proud that our solid tumor drug has been granted ODD by the FDA following that for our blood cancer drug. Gunagratinib demonstrated anti-tumor activity for multiple tumor types, including cholangiocarcinoma, in patients with FGF/FGFR gene aberrations. We will rapidly advance the multi-center, multi-indication clinical trials in both the US and China in order to benefit patients early."

The Orphan Drug Designation by the US FDA originates from the Orphan Drug Act, which was enacted in 1983 to encourage the development of innovative drugs to treat rare diseases with a target patient population of less than 200,000 in the US. Upon marketing approval, drugs with ODD qualify for seven-year market exclusivity. In addition, US FDA also rewards ODD drugs with comprehensive incentives including tax credit for clinical trial cost, waiver of marketing registration application fee, waiver or reduced annual product fee, and other benefits, such as clinical protocol assistance and qualification for expedited development programs.

Last December, FDA granted ODD to InnoCare's Bruton's tyrosine kinase (BTK) inhibitor orelabrutinib for the treatment of mantle cell lymphoma (MCL).

About InnoCare

InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancer and autoimmune diseases. We strategically focus on lymphomas, solid tumors, and autoimmune diseases with high unmet medical needs in China and worldwide. InnoCare has branches in China (Beijing, Nanjing, Shanghai, Guangzhou) , New Jersey and Boston.