New pancreatic cancer drug almost doubles survival time

Historically, it has been difficult to develop drugs to treat pancreatic cancer, with genetically targeted treatments and immunotherapies having little impact on the disease. In addition, five-year survival rates are just 13% for all types of pancreatic cancer and only 8% for pancreatic adenocarcinoma, the most common form of the disease.

However, a new experimental drug from Revolution Medicines could represent a breakthrough in pancreatic cancer treatments.

The drug, which is called daraxonrasib, blocks a group of genes called RAS. Mutant forms of the protein, with the most well-known being KRAS, are found in roughly 30% of all cancers and over 90% of pancreatic cancers. Daraxonrasib works by binding to another protein in the cell to form a complex that can block RAS mutations, which are a key driver of tumor growth.

In a Phase 3 clinical trial, 501 patients with metastatic pancreatic ductal adenocarcinoma (PDAC) received either a daily oral dose of daraxonrasib or chemotherapy. These patients had previously received treatment, and most had a KRAS mutation.

Overall, patients who received daraxonrasib had a median overall survival of 13.2 months compared to 6.7 months for those who received chemotherapy. According to Revolution, daraxonrasib was "generally well tolerated, with a manageable safety profile in patients with PDAC and with no new safety signals."

Currently, Revolution is also running Phase 3 clinical trials to test daraxonrasib as a first-line therapy for pancreatic cancer, as well as an add-on therapy for patients whose tumors can be treated with surgery.

"[My CA 19-9 levels were] 8,100, north of 8,000, when I started on the trial," Sasse said. "This week [mid-March] I fell from 579 to 374. That's huge. Something like 60% tumor volume reduction. My pain is way, way down."

When Sasse was diagnosed, doctors said he had three to four months to live, but daraxonrasib may have already helped extend his life past that. "Given what the prognosis was, and given what the tables say about pancreatic cancer, this experience has seemingly extended both quantity and quality of life," he said.

Currently, Revolution plans to use data from the study to apply for FDA approval, though the company did not say when. Previously, the company received a Commissioner's National Priority Voucher (CNPV), which will allow daraxonrasib to be reviewed in just one or two months. The CNPV program, which launched last June, aims to expedite the review of treatments aligned with public health priorities, affordability, or supply chain resistance.

"This [drug] is incredibly important," said Leonid Timashev, an analyst at RBC Capital Markets. "We've had physicians describe this as potentially the biggest breakthrough in pancreatic cancer ever."

Aside from pancreatic cancer, Revolution is also testing daraxonrasib in lung cancer. The company also has three other similar drugs in development.