Amplia Therapeutics Advances Pancreatic Cancer Drug with Positive Trial Data and $27.5M Raise

Mature ACCENT Data Strengthens Narmafotinib Prospects

Amplia Therapeutics (ASX:ATX) has delivered a significant clinical update, with mature data from its ACCENT trial in metastatic pancreatic cancer showing promising improvements over chemotherapy alone. The Phase 1b/2a study combining Amplia’s lead drug candidate narmafotinib with gemcitabine and nab-paclitaxel achieved a confirmed objective response rate of 36%, well above the historical 23% benchmark from the MPACT trial. Median progression-free survival reached 7.7 months, and median overall survival extended to 11.1 months, representing an approximate two-month gain compared to chemotherapy alone.

These results, independently reviewed and presented at major oncology conferences including ASCO and AACR in 2026, reinforce the potential of focal adhesion kinase (FAK) inhibition to enhance treatment efficacy in a cancer type notorious for poor outcomes. Notably, the trial reported five complete responses (7.8%), an unprecedented rate in metastatic pancreatic cancer, highlighting the depth of tumor control achievable with narmafotinib combinations.

Importantly, the addition of narmafotinib did not increase toxicity beyond that expected from chemotherapy, supporting its favourable tolerability profile. The ACCENT dataset continues to mature, with several patients still on treatment as of March 2026.

Amplia’s CEO Dr Christopher Burns emphasised the significance of these findings in advancing narmafotinib towards registration-enabling studies. The company is actively engaging with the US FDA to finalise trial designs, with recent Type D meeting feedback supporting a two-dose comparison strategy for the upcoming Phase 2b/3 study. This regulatory alignment is a key step in Amplia’s clinical development roadmap.

The momentum from ACCENT builds on prior coverage of the company’s mature ACCENT trial data and FDA interactions, underscoring the program’s clinical and commercial promise.

AMPLICITY Trial Halted Amid Chemotherapy Toxicity

In contrast, Amplia discontinued recruitment in its AMPLICITY trial, which combined narmafotinib with the more aggressive FOLFIRINOX chemotherapy regimen. While FOLFIRINOX is commonly used in the US and Europe, significant toxicity observed early in the trial limited dose escalation of narmafotinib. Five patients remain on study, with data pending, but the company has shifted focus to alternative combination regimens with better tolerability profiles.

This development reflects the evolving pancreatic cancer treatment landscape, which is increasingly moving away from highly toxic chemotherapies towards targeted and combination therapies. Amplia is closely monitoring advances in kRAS inhibitors, a novel class of drugs showing encouraging efficacy, and is exploring clinical collaborations to combine narmafotinib with these agents to overcome resistance and improve outcomes.

The company’s preclinical data, presented at the AACR Special Conference on RAS Oncogenesis, demonstrated that narmafotinib enhances the activity of kRAS inhibitors across multiple cancer models, including pancreatic, lung, and ovarian cancers. This positions Amplia well to participate in the next generation of combination therapies targeting kRAS-driven tumors.

Manufacturing Scale-Up and Intellectual Property Milestones

Amplia has also made important strides in manufacturing readiness, completing its first large-scale GMP production campaign for narmafotinib, yielding approximately 13 kilograms of active pharmaceutical ingredient. This pilot-scale batch represents a commercial-ready manufacturing setting, sufficient to support ongoing and planned clinical trials.

The company’s Senior Product Development Manager, Dr Adrian Sulistio, highlighted the complexity of scaling production from laboratory to commercial standards, noting the critical role of strong global manufacturing partnerships in ensuring supply consistency and quality.

On the intellectual property front, Amplia secured a key patent covering the crystalline form of narmafotinib, extending protection to at least 2040 across major pharmaceutical markets including the US, Europe, Japan, and Australia. This patent strengthens the company’s commercial position as it advances clinical development.

Financial Position and Capital Raising

Amplia strengthened its balance sheet with a $27.5 million capital raise during the year, comprising a $25 million institutional placement and a $2.5 million share purchase plan. This funding supports clinical programs, manufacturing scale-up, and regulatory activities through 2027.

As of 31 March 2026, Amplia held cash and cash equivalents of $27.85 million, up from $10.86 million the prior year, with no borrowings. The company reported a loss after tax of $7.7 million for the year, reflecting increased investment in research and development, including clinical trial expenses and patent costs.

Amplia’s net tangible assets per share improved to 6.1 cents from 3.4 cents, reflecting the capital injection and operational progress. No dividends were declared, consistent with the company’s focus on reinvestment in development.

The company’s financials were audited with an unmodified opinion, though with a material uncertainty disclosed regarding going concern due to ongoing losses and cash burn, typical for clinical-stage biotech firms.

Leadership and Governance

The board and executive team, led by Non-Executive Chair Dr Warwick Tong and CEO Dr Christopher Burns, remain focused on advancing Amplia’s pipeline and preparing for commercialisation. The appointment of Dr Jason Lickliter as Chief Medical Officer in June 2025 added clinical trial expertise to the leadership team.

Directors and key management personnel hold significant shareholdings and options, aligning their interests with shareholders. The company maintains a disciplined approach to remuneration and governance, with regular board reviews to adapt to the evolving clinical and commercial environment.

Amplia also expanded its US presence with a listing on the OTCQB Venture Market, improving access for American investors and aligning with its global clinical development ambitions.

Next Steps in Clinical Development

Looking ahead, Amplia plans to complete the ACCENT trial by Q3 2026 and initiate a controlled registration-enabling Phase 2b/3 study comparing narmafotinib plus chemotherapy against chemotherapy alone. The company is also exploring new combination trials, including with kRAS inhibitors, and expanding into other solid tumor indications such as ovarian cancer.

Given the discontinuation of the AMPLICITY trial, the focus will be on combinations with less toxic chemotherapy backbones to optimise patient tolerability and therapeutic benefit.

Amplia’s recent Phase 2b daily dosing trial launch and the new ovarian cancer PRROSE trial with ANZGOG targeting resistant ovarian cancer demonstrate the company’s strategic expansion beyond pancreatic cancer.