Amplia Therapeutics (ASX: ATX) has achieved a significant milestone in its ongoing ACCENT clinical trial, with 15 confirmed partial responses now recorded in patients with advanced pancreatic cancer. This level of response is sufficient to demonstrate that the combination of narmafotinib with standard chemotherapy is superior to chemotherapy alone, according to the company.
The ACCENT trial is evaluating narmafotinib, a highly potent and selective FAK (Focal Adhesion Kinase) inhibitor, in combination with the standard-of-care chemotherapies gemcitabine and Abraxane in patients with advanced pancreatic cancer. FAK is a protein that is overexpressed in pancreatic cancer and has gained increasing attention as a drug target for its role in solid tumors.
"We are extremely excited to have now recorded 15 confirmed partial responses in the ACCENT trial, demonstrating the benefit of adding narmafotinib to standard-of-care chemotherapy," said Amplia CEO and Managing Director Dr. Chris Burns. "With over 20 patients still on study, we are hopeful that further partial responses will be observed."
Trial Design and Response Criteria
The ACCENT trial is an open-label study in which all enrolled patients receive narmafotinib in combination with standard-of-care therapy. The data obtained is being compared to historical data for the combination of gemcitabine and Abraxane in pancreatic cancer, specifically from the MPACT study, which served as a model for the ACCENT trial design.
A confirmed partial response is defined as tumor shrinkage greater than 30% that is sustained for two or more months, with no new cancerous lesions detected. Given the highly aggressive nature of pancreatic cancer, complete responses are extremely rare, making these partial responses particularly meaningful.
At the outset of the study, statistical analysis determined that a cohort of 50 patients would be sufficient to evaluate the efficacy of the combination therapy with reasonable confidence if 15 or more responders were recorded. The trial has enrolled a total of 55 advanced pancreatic cancer patients since January 2024, with 21 patients still actively participating in the study.
Safety Profile
According to recent updates from Amplia, narmafotinib continues to be well tolerated by patients. The rate and type of adverse events observed with the narmafotinib combination are similar to those reported for chemotherapy alone, suggesting the addition of narmafotinib does not significantly increase toxicity.
Background on Narmafotinib
Narmafotinib was discovered at the Melbourne-based Cooperative Research Centre for Cancer Therapeutics. As a FAK inhibitor, it targets a mechanism believed to play a key role in cancer progression and treatment resistance. FAK is involved in cellular processes including adhesion, migration, and survival, making it a promising target for cancer therapy.
Trial Status and Next Steps
The Phase 2a portion of the ACCENT trial is now fully recruited, with top-line data expected in mid-Q3 2025. With 21 patients still on study, there is potential for additional responses to be observed before the final data readout.
Pancreatic cancer remains one of the most challenging malignancies to treat, with limited therapeutic options and poor survival rates. The positive signal from the ACCENT trial represents a potentially important advance in the treatment landscape for patients with this devastating disease.
If the final results confirm the current positive trend, narmafotinib could become an important addition to the treatment arsenal for pancreatic cancer, a disease where new therapeutic approaches are urgently needed.
