Ascentage Pharma, a global biopharmaceutical company, has received approval from the Center for Drug Evaluation (CDE) of China’s National Medical Product Administration (NMPA) for a global registrational Phase III study of their drug candidate olverembatinib (HQP1351). This study will focus on patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) who have previously failed systemic treatment. GIST is a common type of soft tissue sarcoma that affects the gastrointestinal tract, with KIT and PDGFRA mutations being key drivers in most cases.

Olverembatinib is a novel third-generation tyrosine kinase inhibitor (TKI) that has shown promising efficacy and safety in patients with SDH-deficient GIST. The drug has been granted Breakthrough Therapy Designation in China for the treatment of these patients. A global, multicenter Phase III study will evaluate the efficacy and safety of olverembatinib in this specific patient population, with the potential to support a future New Drug Application (NDA) for olverembatinib in SDH-GIST.

SDH-deficient GIST presents unique clinical and pathological characteristics, and patients with this subtype currently lack standard treatment options. Olverembatinib has demonstrated a clinical benefit rate of 92.3% in patients with SDH-deficient GIST, offering hope for improved outcomes in this challenging disease. The drug has already been approved for certain indications in China and has shown promising results in clinical studies presented at the American Society of Clinical Oncology (ASCO) Annual Meeting.

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, expressed excitement about the approval for the Phase III study, highlighting the potential for a clinical breakthrough in an area with unmet medical needs. The company remains dedicated to developing new treatment options for patients with solid tumors and addressing clinical gaps both in China and globally.

The Phase III study marks a significant milestone in Ascentage Pharma’s clinical development efforts, showcasing their commitment to advancing innovative therapies for cancer and other diseases. With a robust pipeline of clinical drug candidates and a focus on protein-protein interactions to promote cell death, Ascentage Pharma aims to address unmet clinical needs and improve patient outcomes worldwide.