Immutep Eyes Accelerated Approval Pathway for Eftilagimod Alfa in PD-L1 Low Patients

FDA Feedback Marks a Milestone for Immutep’s Head and Neck Cancer Program

Immutep Limited, a Sydney-based biotechnology company, has received positive and constructive feedback from the US Food and Drug Administration (FDA) regarding the clinical development of its novel immunotherapy, eftilagimod alfa (efti), for first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) patients with low PD-L1 expression (Combined Positive Score below 1).

This patient subgroup currently faces limited treatment options, as anti-PD-1 therapies like KEYTRUDA are only approved for those with PD-L1 expression above 1, leaving many reliant on chemotherapy. The FDA’s recognition of the potential for efti combined with KEYTRUDA to address this high unmet need is a significant endorsement for Immutep’s approach.

Clinical Development Pathways and Regulatory Opportunities

The FDA’s feedback outlines two possible paths forward, a randomized registrational trial comparing the combination therapy against standard of care, or a smaller single-arm study focusing on safety and response metrics, followed by a confirmatory randomized trial. This flexibility aligns with the FDA’s Project FrontRunner initiative, which encourages earlier development and approval of cancer drugs in less heavily treated patient populations.

Such regulatory pathways could accelerate the availability of new treatment options for patients with PD-L1 CPS <1, a group representing up to 20% of first-line HNSCC cases. Immutep’s ability to leverage this feedback may position it well to bring a novel, chemotherapy-free option to market.

Balancing Priorities, Head and Neck Cancer and Lung Cancer Trials

While the FDA’s support is a positive development, Immutep’s CEO Marc Voigt emphasised that the company’s primary focus remains on the pivotal Phase III TACTI-004 trial evaluating efti in non-small cell lung cancer. The company plans to carefully consider the head and neck cancer program’s future direction in consultation with stakeholders and potential strategic partners.

This dual focus highlights the challenges biotech firms face in managing multiple late-stage programs, balancing resource allocation, and maximising shareholder value while addressing critical patient needs.

Looking Ahead, Strategic Partnerships and Market Implications

Immutep’s collaboration with Merck & Co., through the combination with KEYTRUDA, remains a cornerstone of its clinical strategy. The FDA’s positive feedback may enhance Immutep’s attractiveness to potential partners and investors, particularly as it navigates the design and execution of future trials.

For patients with limited options, the prospect of a new immunotherapy that could improve outcomes without chemotherapy is a hopeful development. For the market, the next steps in trial design and regulatory engagement will be critical to watch.