Amplia Therapeutics (ASX: ATX) shares gain after 31% response rate in pancreatic cancer trial; U.S. Phase 2 greenlit

How is Amplia Therapeutics progressing in its pancreatic cancer trials and expanding to the U.S.?

Amplia Therapeutics Limited (ASX: ATX) saw a sharp uptick in investor interest after confirming a 31% objective response rate in its flagship ACCENT clinical trial for advanced pancreatic cancer. The Australian biotech company also secured U.S. ethics clearance for a new Phase 2 trial combining its lead FAK inhibitor narmafotinib with FOLFIRINOX, a widely used chemotherapy regimen in North America. These back-to-back milestones, disclosed across three ASX releases between June 24 and July 11, 2025, have reshaped market sentiment, reinforcing Amplia Therapeutics’ strategy to position narmafotinib as a potential first-line adjunct therapy in one of oncology’s most challenging indications.

Narmafotinib (AMP945) is a highly selective focal adhesion kinase (FAK) inhibitor that has shown promising results when paired with chemotherapy in both preclinical models and clinical settings. The molecule targets the FAK signaling pathway, which is implicated in tumor progression, immune suppression, and fibrosis—particularly in hard-to-treat cancers like pancreatic and ovarian malignancies.

Institutional investors are now viewing Amplia Therapeutics as a biotech with a differentiated oncology pipeline and credible global trial strategy. With 17 confirmed partial responses out of 55 patients in its ongoing ACCENT trial and U.S. FDA-cleared IND status for the upcoming FOLFIRINOX trial, Amplia Therapeutics is gaining traction as a contender in the next generation of combination therapies for pancreatic cancer.

What are the latest findings from Amplia Therapeutics’ ACCENT trial in pancreatic cancer patients?

The July 11 update from Amplia Therapeutics confirmed the 17th partial response in its Phase 1b/2a ACCENT trial, bringing the overall objective response rate to 31%—a meaningful clinical signal for an indication with historically low treatment efficacy. The ACCENT trial pairs narmafotinib with gemcitabine and Abraxane®, a standard chemotherapy combination, and is being conducted at seven trial sites across Australia and five in South Korea.

The newly recorded partial response followed a similar announcement on July 2, when Amplia Therapeutics reported its 16th PR and a 29% response rate. According to the trial design, a “partial response” requires tumor shrinkage greater than 30%, sustained for at least two months, with no emergence of new lesions.

This puts the Australian biotech ahead of historical benchmarks. The MPACT study, a global standard for chemotherapy-alone performance in pancreatic cancer, showed a 23% response rate. In contrast, Amplia Therapeutics’ ACCENT data already surpasses this figure even with 20 patients still on study.

The ACCENT trial’s Phase 1b segment concluded in November 2023, determining the optimal oral dose of narmafotinib in combination with the chemotherapeutic backbone. The current Phase 2a segment aims to assess broader efficacy outcomes including progression-free survival (PFS) and overall survival (OS), while continuing to monitor safety and tolerability.

Why is Amplia Therapeutics now pursuing a separate U.S.–Australia trial with FOLFIRINOX?

On June 24, Amplia Therapeutics received ethics clearance from a central U.S. Institutional Review Board (IRB) to initiate a new Phase 2 clinical trial of narmafotinib in combination with the chemotherapy regimen FOLFIRINOX. This trial differs materially from the ACCENT trial not only in treatment backbone but also in geographic reach and regulatory framework.

The new study, titled “A Phase 1b/2a, Multicenter, Open Label Study of the Safety, Efficacy and Pharmacokinetics of narmafotinib in Combination with modified FOLFIRINOX in Pancreatic Cancer Patients,” will be conducted under an active Investigational New Drug (IND) application from the U.S. Food and Drug Administration. The protocol adheres to the U.S. FDA’s Project Optimus guidance, which mandates more detailed dose-finding assessments in early oncology trials.

While gemcitabine–Abraxane remains a standard of care in many geographies, FOLFIRINOX has become the preferred front-line therapy for newly diagnosed metastatic pancreatic cancer patients in the U.S. By designing a trial tailored to American treatment protocols, Amplia Therapeutics is strategically aligning its drug development to meet regulatory expectations and potential market needs in its largest commercial target.

The new trial will commence with a dose-exploration phase (Part A), followed by a dose-comparison phase (Part B). Up to six sites in the U.S. and two in Australia are expected to participate. Initial recruitment will target 60–70 patients, depending on how many dosing arms are deemed necessary.

Preclinical studies have shown that narmafotinib added to FOLFIRINOX improves survival outcomes in animal models, providing a scientific rationale for this investigational combination.

What does institutional sentiment suggest about Amplia Therapeutics’ strategy and clinical momentum?

Analysts and institutional investors have begun to express cautious optimism about Amplia Therapeutics’ dual-pronged clinical strategy. The biotech firm’s continued updates, robust trial design, and alignment with U.S. regulatory pathways have signaled execution discipline and scientific credibility—two attributes often lacking in early-stage oncology platforms.

While Amplia Therapeutics has not yet reported median overall survival data from the ACCENT trial, the strong partial response rate and continued patient retention (20 patients still enrolled) indicate a potentially durable therapeutic effect. This may be critical for eventual partnering or licensing discussions, particularly if the Phase 2 U.S. trial validates a distinct dose-response benefit in FOLFIRINOX-treated patients.

Institutional attention is also being drawn to the fact that narmafotinib is one of the few FAK inhibitors advancing in solid tumors with both preclinical synergy and clinical proof-of-concept. The oncology field has previously seen limited success in FAK-targeting agents due to either toxicity or lack of efficacy in monotherapy settings. Amplia Therapeutics’ approach—positioning narmafotinib exclusively as a combination partner—is considered a more pragmatic and commercially viable path.

What are the future milestones for Amplia Therapeutics’ clinical programs in cancer?

Looking ahead, Amplia Therapeutics is expected to begin patient dosing for the U.S.–Australia FOLFIRINOX trial within the second half of calendar year 2025, pending final site-level approvals. Recruitment will proceed in two stages to identify the optimal daily dose of narmafotinib when paired with bi-weekly intravenous chemotherapy.

In parallel, additional interim results from the ACCENT trial are anticipated over the coming months. With 20 patients still undergoing treatment, the total number of confirmed partial or complete responses could rise further—potentially strengthening Amplia Therapeutics’ clinical case heading into conference season or investor roadshows.

Should the new trial yield safety and efficacy data that mirror or exceed the ACCENT outcomes, Amplia Therapeutics could emerge as a serious candidate for late-stage development partnerships. Potential acquirers or collaborators may include mid- to large-cap oncology players seeking assets in pancreatic cancer or fibrosis-related solid tumors.

Amplia Therapeutics has also previously indicated a long-term interest in expanding its FAK platform into other fibrotic cancers like ovarian carcinoma, as well as chronic diseases such as idiopathic pulmonary fibrosis. However, pancreatic cancer remains the lead indication for narmafotinib as of mid-2025.

Is Amplia Therapeutics becoming a serious oncology contender?

With a 31% response rate in a notoriously difficult-to-treat cancer and active expansion into the U.S. clinical landscape, Amplia Therapeutics appears to be entering a phase of strategic credibility. The dual-trial strategy—balancing Australia-Korea-based data from gemcitabine-Abraxane with a U.S.-anchored FOLFIRINOX cohort—demonstrates both scientific rigor and market awareness.

While the Australian biotech company is still in early clinical phases, its alignment with U.S. regulatory frameworks, solid safety data, and consistent partial responses are beginning to generate institutional confidence. The road to commercialization remains long, but Amplia Therapeutics is increasingly being recognized as more than a speculative microcap—especially if future data reinforce the hypothesis that narmafotinib can enhance frontline chemotherapy outcomes in metastatic pancreatic cancer.