An experimental pill, daraxonrasib, nearly doubled survival time for advanced pancreatic cancer patients to 13.2 months versus 6.7 for chemotherapy in a major clinical trial. The drug targets the KRAS gene mutation, present in over 90% of cases and long considered "undruggable." Hailed as a "game changer" by oncologists, the drug is now available to some patients via an FDA expanded access program ahead of an expected expedited approval.
Daraxonrasib Phase 3 trial results
- ▪The clinical trial involved 500 patients with metastatic pancreatic cancer whose disease had stopped responding to prior treatment
- ▪In a Phase 3 trial, the drug daraxonrasib extended median survival for advanced pancreatic cancer patients to 13.2 months, compared to 6.7 months for chemotherapy
- ▪Oncologists hailed the results as a "game changer" and "unprecedented," with one expert reporting she cried with joy upon seeing the data
- ▪The most common severe side effects for daraxonrasib were a severe rash and mouth sores
- ▪Patients taking daraxonrasib experienced fewer severe side effects (43.6%) than those on chemotherapy (57.5%)
KRAS mutation targeting mechanism
- ▪The KRAS protein was long considered "undruggable" because its smooth structure made it difficult for drugs to attach
- ▪Daraxonrasib targets a mutated KRAS protein that fuels tumor growth in over 90% of pancreatic cancer cases
- ▪Daraxonrasib acts as a "molecular glue," binding with a protein called cyclophilin A to then attach to and shut down the KRAS protein
Pancreatic cancer survival rates
- ▪The American Cancer Society estimates 67,000 new U.S. cases and over 52,000 deaths from pancreatic cancer will occur this year
- ▪For patients with metastatic pancreatic cancer, where the disease has spread, the five-year survival rate is 3%
- ▪Pancreatic cancer has one of the highest mortality rates of all major cancers, with a 13% five-year overall survival rate
Patient experiences with daraxonrasib
- ▪Former U.S. Senator Ben Sasse reported a 76% reduction in tumor volume and less pain after four months on daraxonrasib
- ▪Patients taking daraxonrasib reported less pain and a better quality of life as their tumors shrank
- ▪Debby Orcutt, a clinical trial participant, experienced an 80% reduction in her pancreatic tumor and the disappearance of a cancerous spot on her liver
FDA expanded access program
- ▪The FDA has granted an "expanded access" program, allowing some patients to receive daraxonrasib outside of clinical trials before full approval
- ▪The expanded access program has triggered a "deluge" of patient requests for the drug at cancer centers
- ▪The FDA plans to expedite its review of daraxonrasib, which could lead to full approval within a month or two after the manufacturer files its application
Future applications across cancers
- ▪The KRAS gene mutation is also a driver in other cancers, including lung and colorectal cancer, suggesting potential for daraxonrasib's broader use
- ▪Researchers plan to study daraxonrasib's use earlier in the disease to potentially shrink tumors enough for surgery
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