“We are now back in the mode of rescheduling trips,” says Jim, a patient in the daraxonrasib trial. A tumor in his liver, which had spread from his pancreas, shrank by 50% after he started the drug. “Which was incredible news,” he told ABC’s “Good Morning America.”
Another trial patient, Carla Walker, said the medication cut her tumors by 40% and kept the mass in her pancreas stable. She now spends her time with her grandsons. “I would love, love to make it to their graduation from high school,” Walker said. “I’m going to be there, no matter what.”
Their stories sit on top of trial numbers that drew attention at the American Society of Clinical Oncology annual meeting in Chicago. The findings were presented there and published at the same time in the New England Journal of Medicine.
The trial results
The global Phase 3 study enrolled 500 patients with metastatic pancreatic cancer who had already received one line of chemotherapy for metastatic disease. Those given daraxonrasib had a median overall survival of 13.2 months. Those given chemotherapy had 6.7 months. The drug reduced the risk of death by 60%.
Tumors responded too. About 31% of all trial participants saw their tumors shrink or disappear, against 11% on chemotherapy. Among people with a RAS mutation called G12, the figure was about 33% against about 12%, and tumors stayed under control for a median of about 7 months on the drug versus about 3 months on chemotherapy.
Daraxonrasib is an oral drug taken once a day, and 300 mg was selected as the Phase 3 dose. It is the first in a new class called RAS(ON) inhibitors, which target variants of the RAS gene that drive cancer growth. That gene mutation turns up in 92% of pancreatic cancer cases.
Side effects and what specialists say
Side effects were reported in 96% of participants who received 300 mg or less, and about a third were grade 3 or higher. Christina Annunziata, senior vice president of Extramural Discovery Science at the American Cancer Society, noted that they “were lower compared to standard chemotherapy typically used in this second-line treatment setting.”
Her one worry is access. “The only concern will be the availability of this drug to people who need it,” she said. “This will depend on the cost and level of insurance coverage.”
Dr. Brian Wolpin of the Dana-Farber Cancer Institute said: “I think many of us would consider this a big win.” He said the aim of the trial was to help people “live as long as they can and actually live better with less side effects, less symptoms from the cancer.”
Diane Simeone, director of the UC San Diego Moores Cancer Center, called it “an extraordinarily hopeful moment for the pancreatic cancer field.” Mark Goldsmith, chief executive of manufacturer Revolution Medicines, said the results “represent a potentially transformative advance for patients.”
Where approval stands
The U.S. Food and Drug Administration has not yet approved the drug. It did grant permission for an expanded access program, which lets some patients with previously treated metastatic pancreatic cancer receive daraxonrasib while the regulatory review goes on.
The disease is among the deadliest cancers. The American Cancer Society reports that 68,000 Americans will be diagnosed this year and roughly 53,000 will die. Annunziata said there is currently no screening method for the general population. People with a strong family history, or with hereditary syndromes such as BRCA or Lynch, should talk with their doctor about regular screening with endoscopic ultrasound or MRI.
Sources
- Pancreatic Cancer: Experimental Pill Nearly Doubles Survival Rates
- Experimental pancreatic cancer drug offers new hope in major trial
