The highly anticipated results of a clinical trial for the pill, daraxonrasib, have been released, giving doctors hope for treating the stubbornly lethal disease.

A pancreatic cancer drug more than four decades in the making has cracked one of the most stubbornly lethal cancers, extending people’s lives and keeping their tumors in check for twice as long as those on regular chemotherapy.

The detailed results of the clinical trial, presented Sunday 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 really remarkable and landmark study,” said Harsh Singh, the program 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” — the amount of time before tumors start to grow or spread. Daraxonrasib kept tumors 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 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. Earlier this 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 Randolph, 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.

“It’s not that I don’t have a physical response to these pills, but what I’m getting is something that doesn’t debilitate me really much,” Rubin said.

Doctors who treat patients with pancreatic cancer see this drug as the beginning of a revolution for the dire disease. The drug 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,” Anirban Maitra, a pancreatic cancer researcher and director of the Perlmutter Cancer Center at NYU Langone Health, wrote in an email. “Science will drive these more effective combinations and that is already happening at breakneck speed.”