GFH375, an experimental oral drug, is showing promising results for patients with advanced KRAS G12D–mutant pancreatic cancer, a mutation present in roughly 40% of pancreatic ductal adenocarcinomas. The early-phase trial involved daily 600 mg doses in patients who had already received multiple treatments.
The therapy produced tumor shrinkage in 40.7% of patients, all partial responses, and nearly 96.7% achieved disease control. Most patients (91.5%) experienced some tumor reduction, with median progression-free survival of 5.5 months. Four-month survival was 92.2%, and median overall survival has not yet been reached. GFH375 selectively targets KRAS G12D, a key driver of tumor growth.
Side effects were generally manageable, including diarrhea, nausea, low neutrophil counts, vomiting, and anemia, with 13.6% experiencing serious events and 31.8% having Grade 3 or higher adverse effects. These results support further development of GFH375 as a potential targeted therapy for this high-risk patient group.