BREAKTHROUGH: New Daily Pill Delivers Stunning Results Against One of America’s Deadliest Cancers

Liberty Check

• New daily pill daraxonrasib shows unprecedented promise in early trial for pancreatic cancer, achieving disease control in approximately 90% of patients
• Drug targets RAS gene mutations found in over 90% of pancreatic cancers—something older treatments couldn’t effectively address
• Medical researchers call this one of the most important breakthroughs in solid tumor treatment, potentially doubling survival time in pretreated patients

A new drug for pancreatic cancer is showing promise in early testing. Daraxonrasib is a daily pill designed to block cancer signals linked to the RAS gene.

It has now finished an early-stage clinical trial—the first time it was tested in people—to evaluate both its safety and effectiveness. The clinical trial, led by the Dana-Farber Cancer Institute and published in The New England Journal of Medicine, tested the drug in 168 patients with advanced pancreatic cancer whose tumors had mutations in the RAS gene.

All study participants had previously received at least one chemotherapy treatment. The drug is designed to block multiple active cancer signals that help tumor cells grow.

This is especially important because more than 90% of pancreatic cancers carry these harmful mutations, researchers said. Existing and older drugs that target RAS mutations only work on certain types that are uncommon in pancreatic cancer, such as KRAS mutations.

At the 300-milligram dose—the amount that will be used in larger phase 3 trials—about 30% of patients saw a positive response, researchers noted. Overall, about 90% of patients had their cancer either shrink or stop getting worse.

There were some side effects reported—most commonly rash, mouth inflammation, nausea and diarrhea. Lead investigator Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber, commented in a press release statement that this development could change the future of cancer care.

“If supported by data from future clinical trials, daraxonrasib would be a targeted therapy relevant to nearly all patients with advanced pancreatic cancer,” he said.

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

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

In an interview, the researcher claimed that daraxonrasib represents “one of the most promising therapy advances we’ve seen in pancreatic cancer.” This is especially significant since pancreatic cancer has had “very few effective therapies” in the past, Wolpin noted.

“The study also showed disease control in approximately 90% of patients with metastatic pancreatic cancer, which is extremely exciting,” he added.

Wolpin noted that while side effects were common, most patients were able to tolerate treatment with “supportive care measures, and very few patients needed to stop therapy due to side effects.” As this was a phase 1/2 study, it does not “definitively prove” the superiority of daraxonrasib compared to chemotherapy, Wolpin added.

“The study did not include a randomized control arm that directly compared daraxonrasib with chemotherapy,” he said.

“That being said, the results for daraxonrasib looked substantially better than what we have seen in prior clinical trials of chemotherapy in patients with previously treated metastatic pancreatic cancer.”

It also remains unclear how the drug may perform earlier in the disease, as the trial included patients who had already received prior treatments. For patients and families affected by pancreatic cancer, Wolpin noted that daraxonrasib signals “real momentum” toward effective treatments, but it is still investigational and is not a cure.

“Pancreatic cancer remains a challenging disease, and additional research is needed to determine how best to sequence or combine therapies to provide the most durable responses and cures,” he said.

Brian Slomovitz, director of gynecologic oncology and co-chair of the Cancer Research Committee at Mount Sinai Medical Center in Miami Beach, applauded this development in a separate interview. “We are anxiously awaiting the upcoming plenary presentation of RASolute 302 at the ASCO meeting later this month,” said the expert, who was not involved in the study.

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

“If the full dataset results that will be reported later this month confirm what was earlier released, I believe this will be one of the most important breakthroughs in all solid tumors,” Slomovitz went on.

“Doubling the survival time in pretreated patients is unprecedented.”

The doctor added that the “magnitude of benefit” could “reshape the treatment landscape” and “establish a new standard of care.” “We will need to evaluate the full dataset for efficacy and safety,” Slomovitz added.

“I am more than cautiously optimistic, and I am truly excited for our patients and their families that suffer from this dreadful disease.”

Americans deserve better healthcare innovation—and research like this shows what’s possible when science focuses on results.