A new pancreatic cancer drug, developed over more than 40 years, has demonstrated significant improvements in extending patient survival and controlling tumor growth compared with standard chemotherapy, according to results presented at the American Society of Clinical Oncology’s meeting in Chicago and published simultaneously in the New England Journal of Medicine.
The drug, known as daraxonrasib, targets the KRAS gene mutation, which is found in the majority of pancreatic cancer cases as well as some lung and colorectal cancers. KRAS has long been considered a difficult gene to target effectively in cancer therapy. In a clinical trial involving 500 patients whose pancreatic cancer had progressed after initial treatment, those receiving daraxonrasib lived for a median of 13.2 months, roughly twice as long as the 6.6 months observed in patients treated with traditional chemotherapy.
In addition to overall survival, the drug also doubled progression-free survival—the time before tumors began to grow or spread. Patients on daraxonrasib experienced 7.2 months of tumor control, compared to 3.6 months for those on chemotherapy.
Mark Goldsmith, chief executive of Revolution Medicines, the biotech company behind the pill, said the company is actively working to submit all required data to regulators through a rolling process and is prepared to launch the drug commercially pending approval. The U.S. Food and Drug Administration has already expanded access to daraxonrasib while the review continues.
Experts consider the results a landmark achievement in pancreatic cancer treatment, a disease that has historically offered few effective therapeutic options. Harsh Singh, program director at Mass General Brigham Cancer Institute, described the findings as possibly the greatest advance seen in pancreatic cancer to date. Researchers emphasize that while daraxonrasib is not a cure and resistance eventually develops, the drug establishes a foundation for developing more durable treatment combinations.
The progress is the culmination of decades of scientific research, much of it federally funded. Edward Scolnick, one of the scientists who first identified KRAS as a cancer-causing gene nearly 50 years ago, highlighted the importance of basic research alongside philanthropic and private sector support.
Patients participating in the trial reported notable side effects, such as rashes and gastrointestinal symptoms, but some also described improvements in their quality of life compared to chemotherapy. Helene Rubin, an 83-year-old patient from New Jersey who began the drug after lung nodules related to pancreatic cancer grew despite prior treatments, said that although she experienced mouth sores and cuts on her fingertips, daraxonrasib did not debilitate her as chemotherapy had, and her scans so far showed positive results.
Researchers are currently exploring the drug’s effectiveness as a first-line treatment and investigating combination therapies aimed at achieving longer-lasting responses. Anirban Maitra, director of the Perlmutter Cancer Center at NYU Langone Health, noted that the new drug offers a valuable platform from which science can develop more effective treatment strategies at an accelerating pace.