A new pill developed to treat pancreatic cancer has stunned researchers with early trial results that show strong signs of effectiveness and tolerability.

The experimental drug, known as daraxonrasib, targets the RAS gene, which fuels tumor growth in most pancreatic cancers.

The early-stage clinical trial was the first time the treatment was tested in humans and was led by researchers at the Dana-Farber Cancer Institute.

Published in The New England Journal of Medicine, the trial evaluated the safety and efficacy of daraxonrasib in 168 patients who had advanced pancreatic cancer with RAS mutations.

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Each participant had previously undergone at least one round of chemotherapy before receiving the new drug.

In the study, patients received a daily pill meant to inhibit multiple cancer-promoting signals.

More than 90% of pancreatic cancers carry genetic mutations in the RAS pathway, making the target especially significant, according to the research team.

At the 300 milligram dose that will be used in upcoming phase 3 studies, about 30% of patients achieved measurable tumor shrinkage. Overall, nearly 90% of participants saw their disease stabilize or improve.

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The most commonly reported side effects were rash, mouth irritation, nausea, and diarrhea.

Most patients were able to continue treatment with supportive care, researchers noted.

Lead investigator Dr. Brian Wolpin, who directs the Hale Family Center for Pancreatic Cancer Research at Dana-Farber, said in a statement that the results could mark a turning point for cancer care if confirmed in later trials.

“This trial provides the first published data showing the safety and broad activity of a RAS(ON) multi-selective inhibitor in pancreatic cancer,” Wolpin said.

“If it proves effective in larger clinical trials, it would signify a substantial shift in how this disease is treated.”

He described the findings as “one of the most promising therapy advances we’ve seen in pancreatic cancer.”

Pancreatic cancer has historically had very few treatment options, making new developments particularly meaningful for patients and doctors.

However, Wolpin emphasized that the trial does not prove daraxonrasib is superior to chemotherapy.

The phase 1/2 study did not include a randomized control group, leaving further research necessary before any conclusions about comparative performance can be drawn.

“It remains unclear how the drug may perform earlier in the disease,” Wolpin explained, noting that the trial included patients who had already received prior therapies.

Additional trials will explore where the pill fits best in treatment sequencing or combination approaches.

Despite caution, the level of disease control achieved in the study has energized the research community.

“Approximately 90% of patients experienced disease control, which is extremely exciting,” Wolpin said, while also noting that more investigation is needed before it could be considered a cure.

Dr. Brian Slomovitz, director of gynecologic oncology and co-chair of the Cancer Research Committee at Mount Sinai Medical Center, said the findings could “reshape the treatment landscape” if future data confirm what researchers have seen so far.

“We are anxiously awaiting the upcoming plenary presentation of RASolute 302 at the ASCO meeting later this month,” Slomovitz said.

“Greater than 90% of pancreatic cancers have activation of kRAS, which is a major factor in the development and progression of these cancers.”

He called the early findings “unprecedented,” noting that doubling survival time in pretreated patients would mark a major step forward.

“If the full dataset results confirm what was earlier released, I believe this will be one of the most important breakthroughs in all solid tumors,” he said.

Wolpin added that with continued research, daraxonrasib might offer renewed hope for patients who face a disease known for its limited treatment options.

“Pancreatic cancer remains a challenging disease,” he said. “But this signals real momentum toward effective therapies.”