Core Insights - Ascentage Pharma announced promising results from a Phase Ib study of olverembatinib for treating SDH-deficient GIST, published in a high-impact journal [2][4][3] - The study is the largest prospective clinical trial to date for this rare tumor subtype, demonstrating significant clinical benefits and a novel mechanism of action [4][7][8] Company Overview - Ascentage Pharma is a global biopharmaceutical company focused on developing therapies for unmet medical needs in cancer [12] - The company has a diverse pipeline, including olverembatinib, which is a third-generation BCR::ABL1 inhibitor approved in China for specific indications [13][12] Clinical Study Details - The Phase Ib study involved 66 patients, including 26 with SDH-deficient GIST, showing an objective response rate (ORR) of 23.1% and a clinical benefit rate (CBR) of 84.6% [4][5] - The median progression-free survival (mPFS) for patients with SDH-deficient GIST was reported at 25.7 months [4][5] Mechanism of Action - Olverembatinib modulates lipid metabolism, inhibiting CD36 expression, which is linked to enhanced uptake of exogenous lipids in SDH-deficient tumors [6][7] - The study established a direct link between SDH deficiency and dysregulated lipid metabolism, providing a new therapeutic target for SDH-deficient GIST [7][6] Future Directions - Ascentage Pharma plans to continue clinical development of olverembatinib, with ongoing studies to validate its efficacy and safety in SDH-deficient GIST [9][10] - The drug has received Breakthrough Therapy Designation in China for this indication, indicating its potential as a new treatment option [10][11]
Ascentage Pharma Announces Publication of Olverembatinib Phase Ib Safety, Efficacy and Novel Mechanism Data in Gastrointestinal Stromal Tumors in Nature's Signal Transduction and Targeted Therapy