Experimental Pancreatic Cancer Drug Doubles Survival Time

Pancreatic cancer remains one of the most challenging diseases to treat. Because the disease often produces no early symptoms, doctors diagnose roughly 80 percent of patients at an advanced or metastatic stage. For decades, treatment options have relied heavily on traditional chemotherapy.

Now, say experts with Baptist Health Miami Cancer Institute, an experimental drug called daraxonrasib is showing great promise in clinical trials, offering patients a chance at extended survival and an improved quality of life. The U.S. Food and Drug Administration (FDA) recently granted the drug an accelerated review, highlighting its potential to transform how we approach pancreatic cancer care.

How Daraxonrasib Targets Cancer Cells

Daraxonrasib is an oral targeted therapy that attacks a specific mutation found in many tumors. In about 90 percent of advanced pancreatic cancer cases, a “growth switch” called RAS becomes stuck in the “on” position. This constant signal tells cancer cells to grow and spread throughout the body.

Daraxonrasib works by binding to this active RAS protein and turning off the signal. This targeted approach slows or even shrinks the tumor.

“In simple terms, daraxonrasib is a targeted pill that goes after a key ‘engine’ inside most pancreatic cancers, rather than being another round of IV chemotherapy,” says Sarbajit Mukherjee, M.D., chief of Gastrointestinal Medical Oncology at Miami Cancer Institute.

Encouraging Clinical Trial Results

Recent clinical trials highlight the powerful impact of this new medication. In a large Phase 3 trial, researchers enrolled more than 500 patients with metastatic pancreatic ductal adenocarcinoma (PDAC) whose previous treatments had failed. Metastatic PDAC remains one of the leading causes of cancer-related deaths in the US, with a five-year survival rate of 3%.

Half of the participants took a daily 300 mg pill of daraxonrasib, while the other half received standard intravenous chemotherapy. The results demonstrated a significant improvement in patient outcomes.

Over more than 16 months of study in early trials, at least 29 percent of participants saw their tumors shrink. More than 90 percent experienced no new tumor growth. And many saw their survival time more than double, with median overall survival of 15.6 months—roughly twice as long as such patients typically fare with standard treatments.

“In the study, patients on daraxonrasib lived almost twice as long, which is something we have never seen before in this setting for pancreatic cancer,” Dr. Mukherjee notes.

Recently, Kyaw Aung, M.D., M.B.B.S., Ph.D., a medical oncologist at Miami Cancer Institute, was listed as a co-author on a landmark publication in the New England Journal of Medicine highlighting promising results from a major phase 1/2 clinical trial evaluating daraxonrasib for RAS-mutated cancers.

Dr. Aung’s involvement places Baptist Health Cancer Care alongside investigators from many of the nation’s leading cancer centers in advancing research for metastatic pancreatic cancer, one of the most difficult cancers to treat. The publication underscores both Dr. Aung’s contribution to this important national study and Baptist Health’s growing role in bringing innovative, precision-focused cancer research to patients facing complex and hard-to-treat disease. 

Daraxonrasib a "Miracle Drug"

The drug has already made a tangible difference for real people. Former U.S. Sen. Ben Sasse, who was diagnosed with stage 4 (terminal) pancreatic cancer last year, shared that he has been participating in a clinical trial for daraxonrasib. In a recent interview with 60 Minutes’ Scott Pelley, Mr. Sasse called it a “miracle drug.”

“I have much, much less pain than I had four months ago when I was diagnosed,” says Mr. Sasse. “And I have a massive 76 percent reduction in tumor volume over the last four months.”

Managing Treatment Side Effects

Like any potent cancer therapy, daraxonrasib comes with risks and limitations. Patients taking the drug may experience side effects such as rash, diarrhea, mouth sores and fatigue. Medical teams require patients to undergo regular blood tests and close monitoring throughout their treatment.

However, healthcare providers can typically manage these adverse effects effectively.

“The good news is that, in the trials so far, most of these problems have been manageable with dose adjustments and supportive medications,” Dr. Mukherjee explains.

The Future of Pancreatic Cancer Care

Researchers continue to explore how daraxonrasib might fit into earlier stages of treatment. Current trials are evaluating the drug as a first-line therapy, both on its own and combined with standard chemotherapy.

“Early results when it’s used as the first treatment, especially together with our usual chemotherapy, also look very encouraging, with more tumors shrinking and more patients doing well at six months than we would normally expect,” says Dr. Mukherjee.

If the FDA approves the medication, it will likely become a vital option for patients who stop responding to initial chemotherapy regimens. The drug represents a shift toward accessible and tailored healthcare solutions for individuals facing severe diagnoses.

Dr. Mukherjee emphasizes the broader significance of this medical advancement.

“It’s important to underscore that this is not a cure and not yet an approved option for everyone, but from my perspective, this is the first true ‘step-change’ drug we’ve seen for advanced pancreatic cancer in many years,” he says. “It gives patients more time, often with better quality of life. And it opens the door to much smarter, more personalized treatment in the future.”

Click here for more information about pancreatic cancer treatments available at Baptist Health Miami Cancer Institute.