Overview
- Immutep, which received the FDA designation Wednesday, saw its shares jump about 101% on the ASX to A$0.079.
- Orphan Drug Designation supports drugs for diseases affecting fewer than 200,000 people in the U.S. and can bring tax credits, fee waivers, regulatory guidance, and seven years of market exclusivity if approved.
- The FDA move draws on Phase II EFTISARC-NEO results that met the primary goal in 38 patients, with a median tumor hyalinization and fibrosis of 51.5% versus a 35% target and roughly 15% seen with radiotherapy alone.
- The investigator-led study tested efti with radiotherapy and pembrolizumab before surgery in resectable soft tissue sarcoma and reported consistent effects across subtypes with no surgery delays and a favorable safety profile.
- CEO Marc Voigt said the designation could enable a direct step into a late-stage neoadjuvant STS trial as the company winds down the TACTI-004 lung-cancer study for futility, which it says should extend its cash runway beyond Q2 2027.