An experimental daily pill has demonstrated the ability to control disease progression in 90% of patients with advanced pancreatic cancer, according to early-stage clinical trial results published in The New England Journal of Medicine.
The drug, known as daraxonrasib, targets RAS mutations present in more than 90% of pancreatic cancers—a genetic driver that has historically had few effective therapies. While older medications typically target only rare mutations, daraxonrasib is designed to block multiple active cancer signals that allow tumor cells to grow.
In a study of 168 patients led by the Dana-Farber Cancer Institute, researchers found that at a 300-milligram dose—the level set for upcoming Phase 3 trials—30% of patients showed a positive response. Overall, 90% of participants experienced “disease control,” meaning their tumors either shrank or stabilized.
“If supported by data from future clinical trials, daraxonrasib would be a targeted therapy relevant to nearly all patients with advanced pancreatic cancer,” said lead investigator Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber.
Outside experts have noted the potential for the drug to reshape the treatment landscape. Dr. Brian Slomovitz, director of gynecologic oncology at Mount Sinai Medical Center, who was not involved in the study, called the possibility of doubling survival time in pretreated patients “unprecedented.”
The U.S. Food and Drug Administration (FDA) has already fast-tracked daraxonrasib as it prepares for larger-scale testing. However, researchers cautioned that as a phase 1/2 study, the trial did not include a randomized control arm to definitively prove superiority over standard chemotherapy.
While the drug represents significant momentum, it is not considered a cure. Common side effects reported during the trial included rash, mouth inflammation, nausea, and diarrhea, though researchers noted that very few patients were forced to discontinue treatment due to these issues.
Further data is expected to be presented at the American Society of Clinical Oncology (ASCO) meeting later this month.