Faron Pharmaceuticals Oy has announced positive feedback from the FDA regarding the registrational clinical development plan for bexmarilimab in the treatment of myelodysplastic syndrome (MDS). The FDA acknowledged the challenges of conducting a randomized study with a comparator in the relapsed/refractory setting and proposed a confirmatory Phase III study in frontline high-risk MDS (HR MDS) instead. This guidance is part of Project Frontrunner, aiming to bring new cancer treatments to a broader patient population as early as possible.
The FDA's suggestion targets a significantly larger patient population with the potential for faster approval, accelerating and increasing the sales forecast for bexmarilimab. Faron had proposed a randomized Phase III study for the treatment of r/r MDS using bexmarilimab + azacitidine, but the FDA proposed moving directly into a registrational blinded randomized frontline HR MDS study investigating bexmarilimab + azacitidine against placebo + azacitidine. This approach aligns with the FDA's Project Frontrunner initiative.
Dr. Juho Jalkanen, CEO of Faron, expressed that the FDA's proposal significantly reduces development costs and timelines, underlining the urgent need for new treatment options in HR MDS. The feedback provides clear guidance on the path to approval and confirms the encouraging results bexmarilimab has shown in overcoming resistance to azacitidine. Faron will adjust its development plan accordingly and continue its ongoing activities and cash runway as planned.
The company will host a virtual webinar to discuss the FDA feedback and updated clinical development plans.