An experimental medication for treating pancreatic cancer has achieved unprecedented results in clinical trials, offering new hope for patients battling one of the most lethal forms of cancer, according to research findings released Sunday.
The once-daily pill, called daraxonrasib and developed by Revolution Medicine, demonstrated remarkable effectiveness in a study involving 500 participants. Patients taking the medication lived significantly longer than those receiving conventional chemotherapy treatments, with researchers finding the drug cut the overall death risk by 60% for those with advanced pancreatic cancer.
The clinical trial focused on patients whose cancer had not responded to an initial round of chemotherapy. Results showed the experimental treatment stopped or reversed cancer progression in nearly one-third of patients, compared to just 10% of those receiving standard chemotherapy. These findings were unveiled Sunday at the American Society of Clinical Oncology conference.
“It ticks all of the boxes,” stated Dr. Rachna Shroff from the University of Arizona Cancer Center, who serves as an expert in pancreatic cancer for the medical organization. She emphasized that such dramatic improvements in survival rates and death risk reduction have never before been observed in patients whose cancer continued advancing after chemotherapy.
Earlier data released on April 13 revealed the medication extended patient survival from diagnosis to 13.2 months, compared to 6.7 months for those receiving standard chemotherapy. This announcement caused the company’s stock value to surge by 40%.
Dr. Brian Wolpin from Harvard’s Dana-Farber Cancer Institute, who led the clinical trial, predicted significant impact from these findings. “These results will change how scientists, clinicians, and patients think about treatment for pancreatic cancer,” he said.
While the medication showed promise, researchers noted that 86.3% of patients developed skin rashes after beginning treatment. However, Dr. Wolpin indicated this side effect can typically be managed effectively using antibiotics and topical steroids.
Pancreatic cancer ranks among the deadliest of all major cancer types. The American Cancer Society projects approximately 68,000 Americans will receive pancreatic cancer diagnoses this year, with roughly 53,000 expected to die from the disease.
Statistics show only 3% of patients whose cancer has metastasized beyond the pancreas survive five years. About 80% of cases are identified when the disease has already reached advanced or metastatic stages.
Revolution Medicines’ chief executive Mark Goldsmith revealed the company is currently conducting additional trials testing daraxonrasib in earlier disease stages and in combination therapies, aiming to further “significantly elevate” patient survival rates.
Among those participating in expanded trials is Menta “Steve” Wallace, a 74-year-old Houston, Texas resident. After experiencing abdominal pain, he received his pancreatic cancer diagnosis in January and began the experimental treatment on February 12 through the University of Texas MD Anderson Cancer Center.
Following initial symptoms including nausea, diarrhea, and what he described as a rash that was “not bad at all,” Wallace reports feeling well and being “very pleased” with his progress. His most recent medical scan revealed his tumor had decreased in size by 46%.
The improvement has allowed Wallace, who enjoys traveling, to resume retirement plans with his wife that had been postponed. After receiving medical clearance last week, he intends to bring his medication on ice during a Caribbean cruise planned for late June.
Daraxonrasib represents the first medication in a new category called RAS(ON) inhibitors, which target specific variants of the RAS gene responsible for driving cancer growth.
For patients carrying a particular RAS mutation known as G12, tumors remained controlled for an average of 7.3 months among those taking daraxonrasib, versus 3.5 months with chemotherapy. Similar results were observed across the entire study population.
Among patients with the G12 mutation, approximately 33.2% experienced tumor shrinkage or disappearance, compared to about 11.8% in the chemotherapy group. In the broader study population, 31.6% of patients saw their tumors shrink or disappear, versus 11.2% who received chemotherapy.
The most frequently reported side effects included mouth inflammation, nausea, diarrhea, and skin rash, with 86.3% of patients experiencing rashes. Serious or life-threatening side effects occurred in 43.6% of patients taking daraxonrasib, compared to 57.5% in the chemotherapy group, representing higher rates than observed in earlier, smaller studies.
Skin rash was the most common serious side effect, affecting 14% of patients, followed by mouth sores and inflammation in 12% of cases. A Jefferies analyst had hoped to see severe rash rates below 10% before the results were announced.
Only 1.2% of patients taking daraxonrasib discontinued the trial due to side effects, compared to 11.2% of those receiving chemotherapy.
On May 1, the Food and Drug Administration approved expanded access to the medication and has committed to conducting an expedited review process.
Dr. Shubham Pant from MD Anderson, who served as a co-principal investigator, shared an example of treatment benefits. He described one patient, an enthusiastic golfer who had been forced to abandon the sport, who was able to decrease his narcotic pain medication dependence and return to playing golf after one month of treatment.
“I have multiple patients like that,” he noted.
