Faron Pharmaceuticals Ltd. provided information on the result of its formal Type D Scientific Advice Meeting with the USA Food and Drug Administration (the FDA) regarding the registrational study plan for its drug candidate bexmarilimab in relapsed and refractory high risk MDS (r/r MDS). Given the previously reported promising results of treating r/r MDS using a combination of bexmarilimab + azacitidine to overcome primary or developed resistance to azacitidine, Faron had proposed to move into a randomized registrational Phase III study for the treatment of r/r MDS using bexmarilimab + azacitidine against the investigator's choice of a hypomethylating agent (HMA). Instead, given the encouraging efficacy already seen in both frontline and r/r HR MDS and the well-established safety profile of bexmarilimab, the FDA proposed that after the ongoing Phase II BEXMAB study in r/r MDS, Faron should move directly into a registrational blinded randomized frontline HR MDS study investigating bexmarilimab + azacitidine against placebo + azacitidine.

The FDA noted that given the relatively modest efficacy of single agent azacitidine and the current response rates with bexmarilimab that the size of such a frontline study may not have to be substantially larger than the proposed study in the r/r setting. Further, the FDA suggested such a frontline study could be seen in the context of FDA's Project Frontrunner. Project FrontRunner is an FDA Oncology Center of Excellence (OCE) initiative to encourage drug sponsors to develop and seek approval of promising new cancer drugs for advanced diseases in an earlier clinical setting, rather than the usual approach to develop and seek approval of a new drug for treatment of patients who have received numerous prior lines of therapies or have exhausted available treatment options.

The FDA guidelines give different possible approval strategies to sponsors, including the conduct of a frontline trial supporting an accelerated approval in the r/r settings.