Amplia’s ACCENT on Success: Pancreatic Trial Hits Key Efficacy Milestone

By all accounts, Amplia Therapeutics (ASX: ATX) has reason to be chipper this week, as its flagship ACCENT trial in advanced pancreatic cancer has delivered a clinically significant result—fifteen confirmed partial responses (PRs), enough to claim superiority over chemotherapy alone.

In the biotech game, particularly in a field as daunting as pancreatic cancer, that’s no small beer.

Amplia’s ACCENT trial is assessing the company’s focal adhesion kinase (FAK) inhibitor narmafotinib (AMP945) in combination with the well-known chemotherapy duo gemcitabine and Abraxane. The trial, which commenced in January 2024, is now fully enrolled with 55 patients, 21 of whom remain on treatment. It is modelled after the MPACT study that originally validated the gemcitabine-Abraxane pairing.

According to the company, the fifteen confirmed partial responses—defined as tumour shrinkage exceeding 30% sustained over at least two months without new metastases—were enough to pass the pre-set efficacy threshold. That statistical line in the sand had been drawn at 15 PRs or complete responses (CRs) in the 50-patient cohort.

Notably, pancreatic cancer is infamous for its dismal prognosis and resistance to treatment, so even a partial response holds considerable weight. A complete response remains vanishingly rare.

CEO and MD Dr Chris Burns put it plainly: “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. With over 20 patients still on study we are hopeful that further PRs will be observed.”

That optimism may not be misplaced. If additional responses are clocked among the remaining patients, it would strengthen the already encouraging signal and bolster the drug’s case as a potential frontline enhancer.

The current ACCENT trial is open-label and single-arm, meaning there’s no randomised control group receiving chemotherapy alone. Instead, the company is benchmarking results against historical data, particularly from the MPACT trial published in the New England Journal of Medicine in 2013. While purists may grumble about the lack of a placebo arm, this approach is not uncommon in oncology—especially in indications where ethics would frown upon withholding treatment.

So far, the safety profile remains favourable. As noted in a prior April update, narmafotinib continues to be well tolerated, with adverse events in line with those expected from chemo alone. For a drug targeting a new biological pathway—FAK, which is overexpressed in pancreatic and other fibrotic cancers—that’s an encouraging sign.

For investors, the timing is propitious. Top-line data from the fully enrolled Phase 2a portion of the ACCENT trial is expected by mid-Q3 2025, making Amplia a stock to watch as the biotech reporting season heats up. It’s worth noting that the FAK space is garnering increasing attention globally, particularly in relation to solid tumours and fibrotic diseases.

Amplia’s approach targets the tumour microenvironment—essentially making the cancer’s ‘home turf’ less hospitable for growth and metastasis. It’s a promising frontier, and narmafotinib’s specificity and potency may give it a leg-up over first-generation FAK inhibitors.

That said, Amplia remains a development-stage biotech. Revenue is nil, and commercial success will hinge on successfully navigating further clinical trials, securing partnerships or licensing deals, and eventually wading through the regulatory minefield. The company will need to keep its powder dry—or top up the coffers—between now and a potential Phase 3.

Still, for a company that listed back in 2019 and has largely flown under the radar, Amplia is starting to make some serious clinical noise. If the forthcoming Q3 data echoes the current results, Amplia could soon find itself with more than just an encouraging acronym on its hands—it may have a pipeline-driving asset worth betting the house on. Or at least, worth watching closely.

No investment advice here—but keep your eyes on the ACCENT.