Faron Pharmaceuticals Limited (AIM:FARN) rose 5% after securing approval from the US Food and Drug Administration to begin a registrational phase II/III trial of its lead drug, bexmarilimab, in higher-risk myelodysplastic syndrome (HR-MDS).
The FDA decision followed an end-of-phase II meeting, where regulators advised the Finnish biotech to focus development on newly diagnosed patients.
The upcoming trial will pair bexmarilimab with azacitidine, a standard treatment, starting with dose optimisation before advancing to the main study.
Importantly, the FDA confirmed that complete response and complete response equivalent, under the new International Working Group 2023 criteria, can serve as co-primary endpoints alongside overall survival. This clears a path for Faron to seek accelerated approval via an interim analysis.
Chief executive Juho Jalkanen called the guidance “a direct route towards accelerated approval,” while Yale’s Professor Amer Zeidan, a consultant to Faron, highlighted the novelty of the criteria in a registrational trial.
The shares rose 11.1p to 221.1p.