Ascentage Pharma Group International released the results of a Phase Ib study (NCT03594422) evaluating olverembatinib (HQP1351) in patients with succinate dehydrogenase‑deficient gastrointestinal stromal tumors (SDH‑deficient GIST). The study enrolled 66 patients, including 26 who had progressed on prior tyrosine‑kinase inhibitors, making it the largest prospective trial to date for this rare subtype.
The data show an objective response rate of 23.1 % and a clinical benefit rate of 84.6 % in the SDH‑deficient cohort, with a median progression‑free survival of 25.7 months and a median follow‑up of 14.5 months. These figures represent a substantial benefit in a population that currently has no standard therapy, underscoring the clinical relevance of the findings.
Safety outcomes were favorable, with only manageable adverse events reported over the 14.5‑month follow‑up. A robust safety profile is critical for regulatory approval and for positioning olverembatinib as a first‑in‑class therapy in this indication.
Translational analyses revealed that olverembatinib suppresses CD36‑mediated lipid uptake, establishing a novel mechanism of action distinct from other tyrosine‑kinase inhibitors. This mechanistic insight could serve as a biomarker for patient selection and may differentiate the drug in a crowded oncology market.
The publication supports ongoing regulatory submissions and could open a new revenue stream for Ascentage Pharma. The data reinforce the company’s strategy to expand olverembatinib beyond chronic myeloid leukemia and Ph+ ALL, strengthening its oncology pipeline and positioning the drug for Breakthrough Therapy Designation in China for SDH‑deficient GIST.
Dr. Yifan Zhai, Chief Medical Officer, said the results “signal a potential breakthrough in addressing an indication with urgent unmet clinical need.” Prof. Ruihua Xu added that the study “provides encouraging efficacy and safety results, suggesting that olverembatinib may offer a new treatment option for this rare tumor type.”
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