CStone Pharmaceuticals Receives NMPA Approval to Initiate Bridging Registrational Phase I Trial of Ivosidenib for IDH1-Mutant Relapsed or Refractory Acute Myeloid Leukemia in China

Acute myeloid leukemia (AML) is the most common type of acute leukemia in adults, characterized by rapid disease progression. There are approximately 20,000 new cases of AML annually in the United States, with a five-year survival rate of about 27%, whereas China sees 30,000 new cases each year, with a five-year survival rate below 20%. Most AML patients develop treatment resistance or experience relapse, progressing to relapsed/refractory AML (R/R AML). As life expectancy increases and the population ages in China, the incidence of AML is likely to rise significantly. Approximately 6%–10% of AML cases are associated with disrupted hematopoietic stem cell differentiation caused by IDH1 mutations. Currently approved AML therapies in China have shown limited efficacy in this patient population.

Ivosidenib was discovered and developed by CStone Pharmaceuticals’ partner, Agios Pharmaceuticals, Inc. (AGIO.US). It received FDA approval in July 2018 for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) harboring an IDH1 mutation, as detected by an FDA-approved test.

“Ivosidenib is the first and only approved precision therapy targeting IDH1 mutations in R/R AML in the United States, and it is the only product in CStone Pharmaceuticals’ portfolio to have received marketing authorization. The approval of our China registration study represents a significant milestone for us,” said Dr. Jiang Ningjun, Chairman and CEO of CStone Pharmaceuticals. He added that through precision medicine, patients can benefit from treatments targeting specific gene mutations that drive cancer.

CStone Pharmaceuticals’ Chief Medical Officer, Dr. Yang Jianxin, stated that ivosidenib was initially approved for the treatment of IDH1-mutant relapsed or refractory acute myeloid leukemia (R/R AML). Recently, the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application for ivosidenib to treat newly diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations who are aged 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy. In March 2019, the FDA granted Breakthrough Therapy Designation to the regimen of ivosidenib in combination with azacitidine for the same population of newly diagnosed AML patients. These milestones underscore the significant clinical potential of ivosidenib. CStone Pharmaceuticals will promptly initiate single-agent ivosidenib trials for R/R AML in China, aiming to generate robust clinical data to facilitate the drug’s timely entry into the Chinese market.

In the United States, TIBSOVO is indicated for the treatment of patients with acute myeloid leukemia (AML) who have a susceptible isocitrate dehydrogenase-1 (IDH1) mutation detected by an FDA-approved test, including:

Adult patients with newly diagnosed AML who are aged ≥75 years or unable to receive intensive induction chemotherapy due to comorbidities.

Adult patients with relapsed or refractory AML.

CStone Pharmaceuticals (HKEX: 2616) is a biopharmaceutical company focused on the development and commercialization of innovative immuno-oncology and precision medicine therapies to address the urgent medical needs of cancer patients in China and globally. Established in late 2015, CStone Pharmaceuticals has assembled a world-class management team with extensive experience in new drug research and development, clinical studies, and commercialization. Centered on combination therapies, the company has built a robust oncology pipeline comprising 15 candidate drugs. Currently, five late-stage candidates are in or approaching pivotal trials. Leveraging its experienced team, rich pipeline, strong clinical development-driven business model, and ample capital, CStone Pharmaceuticals aims to become a globally recognized leading Chinese biopharmaceutical company by delivering innovative oncology therapies to cancer patients worldwide.

AGIO is a U.S. biopharmaceutical company primarily developing therapeutic drugs in the field of cancer metabolism. It has also developed drugs focused on glycolysis, fatty acid metabolism, and autophagy. The company’s therapeutics are used in the areas of autoimmune, inflammatory, and neurological diseases.

                                                                                                       (Compiled by Tian Shuhang)