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A drug recently approved for advanced pancreatic cancer, daraxonrasib, is showing encouraging results in early trials for patients with non-small cell lung cancer, according to new research published Wednesday (September 2) in the New England Journal of Medicine. The trial, led by Kathryn Arbour, MD, at Memorial Sloan Kettering Cancer Center, involved 136 patients whose tumors had RAS gene mutations and who had not responded to other therapies.
In the phase 1/2 clinical trial, more than 30% of participants experienced tumor shrinkage and improved health after taking daily doses of daraxonrasib for three weeks at a time. The drug’s effects lasted longer than standard chemotherapy, with a median progression-free survival of 8.3 months and a median overall survival of 16 months. The drug was generally well tolerated, and side effects were less severe than those from chemotherapy.
Daraxonrasib works by targeting RAS mutations, which are found in about 30% of lung cancer cases and are known to drive tumor growth. For decades, these mutations had been considered “undruggable,” but new approaches have allowed scientists to develop compounds that can disrupt their function. Daraxonrasib, developed by Revolution Medicines, acts as a “molecular glue” that binds to both normal and mutant RAS proteins, blocking their ability to signal uncontrolled cell growth, as explained in Nature.
The trial also established 200mg as the optimal daily dose for future studies, balancing effectiveness and long-term tolerance. The therapy offers patients the convenience of a once-daily oral pill, which allows for more flexibility and potentially a better quality of life compared to frequent chemotherapy infusions.
A larger phase 3 study is now underway, comparing daraxonrasib directly with standard chemotherapy to determine its effectiveness and safety over time. The U.S. Food and Drug Administration (FDA) is expected to review results from this ongoing trial before considering approval of daraxonrasib for lung cancer treatment.
Researchers hope that daraxonrasib’s ability to target a broad range of RAS mutations—unlike earlier targeted therapies that only worked on specific types—will help more patients, including those who develop resistance to other treatments. For pancreatic cancer, daraxonrasib nearly doubled average survival and was recently approved by the FDA under the brand name Rasonque.
“These are exciting times,” Dr. Arbour said, noting the potential for daraxonrasib to expand treatment options for people with difficult-to-treat cancers. More research will clarify how long the drug remains effective and whether it can benefit even more patients in the future, as outlined in the New England Journal of Medicine.