Immutep (ASX:IMM) announced that the United States Food and Drug Administration has granted Orphan Drug Designation to its lead product candidate, eftilagimod alfa, for the treatment of soft tissue sarcoma.
The regulatory achievement underscores the urgent need for innovative therapies to combat this rare and aggressive group of cancers, which affect fewer than 200,000 people in the U.S.
The FDA's decision was bolstered by compelling data from the Phase 2 EFTISARC-NEO trial. In this study, efti was evaluated in combination with radiotherapy and pembrolizumab as a neoadjuvant treatment for patients with resectable STS.
Results from 38 evaluable patients revealed a median tumour hyalinisation/fibrosis rate of 51.5%, vastly outperforming the pre-specified target of 35% and historical benchmarks of approximately 15% for radiotherapy alone.
Furthermore, translational data confirmed immune activation consistent with efti's unique mechanism of action, maintaining a favourable safety profile without delaying scheduled surgeries.
The ODD status provides Immutep with substantial development incentives, including tax credits for clinical testing, waived application fees, and seven years of market exclusivity upon potential approval.
CEO Marc Voigt noted that while the company continues a comprehensive analysis of its discontinued Phase 3 TACTI-004 trial, this designation provides a clear pathway towards late-stage studies.
By leveraging its expertise in lymphocyte activation gene-3 technology, Immutep aims to deliver new hope to patients facing limited treatment options.
At the time of reporting, Immutep's share price was $0.073.