Immutep (ASX: IMM) has secured Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its eftilagimod alfa (efti) in soft tissue sarcoma (STS).
STS is a rare cancer that affects fewer than 200,000 people in the US.
ODD provides several benefits including regulatory support, potential tax credits, fee exemptions, and seven years of market exclusivity upon approval.
The designation is based on promising results from the Phase II EFTISARC-NEO investigator-initiated trial.
TACTI-004 Trial Discontinuation
This positive regulatory news follows the recent discontinuation of Immutep's TACTI-004 Phase III trial.
The trial, which evaluated eftilagimod alfa in first-line non-small cell lung cancer, was halted after an Independent Data Monitoring Committee recommended its discontinuation due to futility.
The company is now undertaking an orderly wind-down of the trial.
Immutep anticipates this discontinuation will lead to an extended cash runway, well beyond its previous guidance of Q2 CY2027.
Phase II Efficacy and Safety Data
The FDA's decision for ODD in soft tissue sarcoma is directly supported by the positive data from the Phase II EFTISARC-NEO trial.
This study evaluated efti in combination with radiotherapy and KEYTRUDA (pembrolizumab) in the neoadjuvant setting for resectable STS.
In 38 evaluable patients, the trial successfully met its primary endpoint.
It showed a median tumour hyalinization/fibrosis of 51.5%, significantly exceeding the pre-specified target of 35% and the historical benchmark of approximately 15% seen with radiotherapy alone.
Results were observed across multiple sarcoma subtypes. Translational data from the trial indicated immune activation, consistent with efti’s mechanism of action.
The safety profile was described as favourable, with no delays to planned surgery for patients.
Broader Pipeline and Financials
Immutep continues to advance a pipeline of five Lymphocyte Activation Gene-3 (LAG-3) programs.
This includes IMP761, currently in a Phase I study for autoimmune diseases.
Financially, the company reported a net loss of A$61.4 million for the 2025 financial year, compared to A$42.7 million in 2024.
As a pre-commercial entity, Immutep continues to require additional funding to support its ongoing research and development efforts.
The company maintains strategic collaborations with major pharmaceutical partners, including Merck (MSD), which are crucial for advancing its various programs.
