Faron Pharmaceuticals Limited (AIM:FARN) has received the green light from the US Food and Drug Administration to begin a registrational phase II/III trial of its cancer drug bexmarilimab in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS).
The decision follows an end-of-phase II meeting between the company and the regulator, during which the FDA advised Faron to focus on frontline development.
The study will test bexmarilimab in combination with azacitidine, beginning with a dose optimisation phase before moving into the main trial.
The agency confirmed that complete response plus complete response equivalent, as defined by International Working Group 2023 criteria, could be used as a co-primary endpoint alongside overall survival.
This allows Faron to seek accelerated approval on the basis of an interim analysis.
Chief executive Juho Jalkanen said the guidance “endorsed a direct route towards accelerated approval” and meant the company was “now only one study away from getting bexmarilimab approved for the benefit of HR-MDS patients.”
Professor Amer Zeidan of Yale School of Medicine, who has consulted for Faron, said the FDA’s acceptance of the IWG 2023 criteria marked the first time they had been used in a registrational trial. He argued that they better reflect meaningful benefit to patients than earlier measures.
Faron said the FDA was also satisfied with its manufacturing and non-clinical plans to support the transition to phase 2/3 testing and subsequent approval steps.