Amplia Therapeutics reported mature ACCENT trial data showing improved survival and multiple complete responses with narmafotinib, alongside a GMP production milestone and CFO appointment.
- ACCENT trial median overall survival 11.1 months
- Five complete responses and 36% objective response rate
- 13 kg GMP production batch completed
- AMPLICITY trial recruitment halted due to chemotherapy toxicity
- Cash position $27.9 million with $3.5 million operating outflows
ACCENT Trial Delivers Encouraging Survival and Response Data
Amplia Therapeutics (ASX:ATX) has unveiled mature data from its ACCENT trial, highlighting a median overall survival of 11.1 months for patients treated with narmafotinib combined with gemcitabine and Abraxane chemotherapy. This marks an improvement of over two months compared to chemotherapy alone in the benchmark MPACT study. Notably, the trial recorded five confirmed complete responses, translating to a 7.8% complete response rate, and an updated objective response rate of 35.9% among 64 evaluated patients. The drug combination was well tolerated, without adding significant toxicity beyond standard chemotherapy effects. These results were presented at the American Association of Cancer Research annual meeting, underscoring Amplia’s growing clinical profile in pancreatic cancer treatment. This builds on earlier positive data showing a 35% response rate and FDA backing for dose optimisation in the ACCENT trial 35% response rate in ACCENT.
AMPLICITY Trial Faces Setback but Continues Monitoring
The company’s second clinical trial, AMPLICITY, which explores narmafotinib in combination with the FOLFIRINOX chemotherapy regimen, has encountered challenges. Recruitment was discontinued after observing three cases of dose-limiting toxicity attributed to chemotherapy rather than narmafotinib. Despite this, five patients remain on study at Australian sites, with further updates anticipated. This development introduces some clinical risk but does not diminish the potential of narmafotinib, which continues to be evaluated across different treatment combinations. The AMPLICITY trial's initial momentum followed promising interim data and a US IND opening at leading institutions including the University of California, Irvine and The Cleveland Clinic.
Manufacturing Milestone Achieved with GMP-Grade Narmafotinib
Amplia has completed a significant manufacturing milestone by producing approximately 13 kilograms of narmafotinib active pharmaceutical ingredient to Good Manufacturing Practice (GMP) standards. This large-scale batch, produced in partnership with a Contract Development and Manufacturing Organisation, marks a transition from R&D to commercial-scale production, enabling formulation into oral capsules for ongoing and future clinical trials. This step is critical for the company’s readiness to advance into Phase 3 studies and aligns with regulatory planning, including a targeted mid-year meeting with the US Food and Drug Administration to discuss the registrational trial design. The company’s regulatory engagement has been steadily progressing following positive FDA feedback on trial protocols and designs FDA backing for pivotal trial.
Preclinical Advances Highlight Synergy with kRAS Inhibitors
In preclinical research presented at a specialist AACR conference in Los Angeles, Amplia revealed that narmafotinib enhances the efficacy of emerging kRAS inhibitors by blocking resistance pathways in cancer models. This finding is significant given the widespread development of kRAS inhibitors for cancers including lung, colon, and pancreatic, where resistance and side effects remain hurdles. Amplia’s data suggest a potential for combination therapies that could improve outcomes and overcome current limitations in targeted cancer treatments.
Leadership and Financial Position
Operationally, Amplia appointed Hamish George as Chief Financial Officer in February, replacing Tim Luscombe. George brings extensive CFO experience within life sciences sectors, reinforcing the company’s leadership team as it advances clinical and regulatory milestones. Financially, Amplia closed the quarter with $27.9 million in cash, down from $31.5 million at the end of 2025, reflecting $3.5 million in net operating cash outflows primarily related to clinical trial activities and manufacturing costs. The company maintains an estimated cash runway of nearly eight quarters at the current burn rate, providing a solid foundation for continued development and business development efforts.
Bottom Line?
Amplia’s clinical progress and manufacturing scale-up position narmafotinib well, but the AMPLICITY trial’s toxicity issues and upcoming FDA interactions will be pivotal in defining next steps.
Questions in the middle?
- How will Amplia address the chemotherapy-related toxicities observed in the AMPLICITY trial?
- What insights will the mid-year FDA meeting provide regarding the registrational trial design?
- Can the synergy with kRAS inhibitors translate into new combination therapies in clinical settings?