Drug intelligence / Profile preview

PFL-721

Development stage
Phase 2
Lead developer
Pierre Fabre
Modality
Small Molecules
Administration
Oral
01

Overview

PFL-721 is a highly mutant-specific small molecule inhibitor targeting both EGFR exon 20 and HER2 exon 20 mutations. It is being developed as a precision therapy for non-small cell lung cancer (NSCLC) patients whose tumors harbor these specific genetic alterations. The drug was originally discovered by Scorpion Therapeutics and further developed by Antares Therapeutics before Pierre Fabre Laboratories acquired worldwide rights in June 2025. As of mid-2025, PFL-721 is advancing to dose optimization within its first-in-human clinical trial for NSCLC. Its mechanism of action involves selective inhibition of mutant forms of the epidermal growth factor receptor (EGFR) and human epidermal growth factor receptor 2 (HER2), both implicated as oncogenic drivers in subsets of NSCLC[1][2][4][6].

Other names
STX-721STX721STX 721
02

Targets

EGFR ex20ins (Epidermal growth factor receptor exon 20 mutant)HER2 exon 20 ins (Human epidermal growth factor receptor 2 (HER2) exon 20 insertion mutants)

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