The highly anticipated results of a clinical trial for pancreatic cancer pill, daraxonrasib, have been released, giving doctors hope for treating the stubbornly lethal disease, reports The Washington Post.
The drug, which has been more than four decades in the making, has cracked one of the most stubbornly lethal cancers, extending people’s lives and keeping their tumours in check for twice as long as those on regular chemotherapy, say researchers.
The detailed results of the clinical trial, presented this past weekend at a plenary session of the American Society of Clinical Oncology’s meeting in Chicago and simultaneously published in The New England Journal of Medicine, are some of the most hotly anticipated medical results in cancer in years.
Oncologists who have traditionally had few options and little hope to offer patients are calling the results “unprecedented”, “compelling” and “spectacular”.
“It really blows it out of the water. It’s a remarkable and landmark study,” said Harsh Singh, Programme Director of Hepatobiliary and Pancreas Oncology at Mass General Brigham Cancer Institute. “This is possibly the biggest advance we have seen in pancreatic cancer, period.”
Scientists at Revolution Medicines, the biotech company that developed the pill, are now focused on finding ways to make those responses more durable, to give people more time. But the success shows the power of targeting a hard-to-hit gene called KRAS, which is the most common cancer-causing gene, driving most cases of pancreatic cancer and a fraction of lung and colorectal cancers.
In the trial, 500 people who had already been treated once for pancreatic cancer, only to see their disease progress, received either the experimental pill, called daraxonrasib, or traditional chemotherapy. Those taking the pill lived twice as long – for a median of 13.2 months, compared with 6.6 months for those on chemotherapy.
Another measure of cancer therapy’s effectiveness is “progression-free survival”, or the amount of time before tumours start to grow or spread. Daraxonrasib kept tumours in check for 7.2 months, compared with 3.6 months with chemotherapy.
Mark Goldsmith, chief executive of Revolution Medicines, said the company’s scientists have been working with urgency to provide all of the data to regulators through a rolling submission process, and are ready to launch the drug commercially if and when they get the approval.
“We’ve been preparing for this moment for a long time. We’ve been building out all of the various dimensions that are required to be successful in launching a product in the United States and globally,” Goldsmith said.
Last month, the Food and Drug Administration expanded access to the drug while review is ongoing.
The success is the product of decades of science, backed by federal funding, philanthropy and the private sector.
“The mutation in human pancreatic cancer is identical to the gene we discovered almost 50 years ago,” said Edward Scolnick, now 86, one of the scientists who revealed that KRAS was a cancer-causing gene while working at the National Cancer Institute. “This kind of basic science is being greatly threatened by current government policies.”
Helene Rubin (83) of New Jersey, is one of the patients taking the drug as part of a trial. She was diagnosed with pancreatic cancer in 2022 and was able to undergo surgery and chemotherapy to treat it.
But recently, regular medical scans showed that nodules in her lung had begun to grow. She started on daraxonrasib in February. The three-pill regimen initially caused her to vomit so much she ended up in the hospital, but her physician was able to reduce her dose to two pills per day.
She said that she has sores in her mouth and cuts on her fingertips, but much more energy than when she was on chemotherapy, and that her scans look good so far.
Doctors who treat patients with pancreatic cancer see this drug as the beginning of a revolution for the dire disease. It is still being tested as a first therapy for newly diagnosed patients, and there is hope that it will work even better when given up front. They are focused on ways to manage the side effects, which are common and can be significant, including a rash and gastrointestinal symptoms.
Physicians know that the treatment is not a cure and that the cancer will become resistant to it, but they see the opportunity to develop combinations that will provide longer-lasting responses.
“What we have is a wonderful foundation on which to now build more effective combinations for our patients,” said Anirban Maitra, a pancreatic cancer researcher and director of the Perlmutter Cancer Centre at NYU Langone Health.
“Science will drive these more effective combinations and that is already happening at breakneck speed.”
Study details
Daraxonrasib or Chemotherapy in Previously Treated Metastatic Pancreatic Cancer
Eileen O’Reilly, ZevWainberg, Andrew Hendifar et al.
Published in The New England Journal of Medicine on 31 May 2026
Abstract
Background
Current therapies offer limited benefit for patients with previously treated metastatic pancreatic ductal adenocarcinoma (mPDAC). Aberrant activation of the RAS pathway is the key driver of PDAC, with oncogenic RAS mutations present in more than 90% of cases. Daraxonrasib is an oral RAS(ON) multiselective, tri-complex inhibitor of the active guanosine triphosphate–bound state of mutant and wild-type RAS.
Method
In this phase 3, international, open-label, randomised trial, we randomly assigned patients with previously treated mPDAC to receive daraxonrasib or chemotherapy of the investigator’s choice. The dual primary end points were overall survival and progression-free survival in the subpopulation of patients with RAS G12 mutations (the RAS G12 population). Key secondary end points included overall survival and progression-free survival in the overall population (which included patients with RAS G12, G13, or Q61 mutations or with no RAS mutation identified) and objective response and patient-reported quality of life in the RAS G12 and overall populations. Safety was also assessed.
Results
A total of 500 patients, including 91.8% with RAS G12 mutations, were randomly assigned to receive daraxonrasib (248 patients) or chemotherapy (252 patients). The median overall survival in the RAS G12 population was 13.2 months with daraxonrasib and 6.6 months with chemotherapy, and the median overall survival in the overall population was 13.2 months and 6.7 months, respectively; the hazard ratio was 0.40 in both populations (P<0.001). The median progression-free survival in the RAS G12 population was 7.3 months with daraxonrasib and 3.5 months with chemotherapy, and that in the overall population was 7.2 months and 3.6 months, respectively; the hazard ratios were 0.45 and 0.49, respectively (P<0.001 for both comparisons). Adverse events that occurred after the start of treatment were reported in all the patients in the daraxonrasib group and in 97.7% of those in the chemotherapy group; the incidence of adverse events of grade 3 or higher was 61.8% and 69.6%, respectively. Treatment-related adverse events that led to treatment discontinuation occurred in 1.2% of the patients in the daraxonrasib group and in 11.2% of those in the chemotherapy group.
Conclusions
Among patients with previously treated mPDAC, treatment with daraxonrasib led to significantly longer overall survival and progression-free survival than chemotherapy.
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