Faron gets positive FDA feedback on bexmarilimab study plans

Faron Pharmaceuticals announced positive feedback from the FDA regarding the clinical development plan for its drug candidate bexmarilimab, aimed at treating myelodysplastic syndrome (MDS), on Thursday.

The AIM-traded firm said that in a formal meeting, the FDA acknowledged the challenges of conducting a randomised study with a comparator in the relapsed/refractory (r/r) setting, and instead proposed a confirmatory phase three study in frontline high-risk MDS (HR MDS).

That approach, Faron explained, would eliminate the need for a separate phase three study in r/r MDS.

It said the FDA’s guidance was part of Project FrontRunner, an initiative designed to expedite the availability of promising new cancer treatments to a broader patient population.

The proposed phase three study would target a significantly larger patient group, potentially leading to faster approval and accelerating the sales forecast for bexmarilimab.

Faron said it initially planned to advance to a randomised registrational phase three study for r/r MDS using a combination of bexmarilimab and azacitidine against the investigator's choice of a hypomethylating agent (HMA).

However, given the encouraging efficacy and established safety profile of bexmarilimab, the FDA suggested moving directly to a registrational blinded randomised study in frontline HR MDS, comparing bexmarilimab plus azacitidine against placebo plus azacitidine.

The FDA indicated that the frontline study might not need to be substantially larger than the proposed r/r study due to the modest efficacy of single-agent azacitidine and current response rates with bexmarilimab.

Faron said the FDA also noted that the frontline study could fit within the context of Project FrontRunner, which encourages the development and approval of promising new cancer drugs for advanced diseases in earlier clinical settings.

Such a strategy would support accelerated approval in r/r settings based on response rates from a frontline trial, with full approval contingent on survival outcomes from the same study.

Subject to continued positive results, the feedback meant that a separate phase three study in r/r MDS would not be necessary.

Faron said its ongoing BEXMAB phase two study could serve as the registrational trial for r/r MDS, with interim response rate read-outs from the phase three frontline HR MDS study supporting accelerated approval.

Full approval would be based on survival data from the single phase three study.

“Faron is now adjusting its development plan accordingly,” said chief executive officer Dr Juho Jalkanen.

“This is very positive feedback and exceeds our expectations.

“The FDA's proposal significantly reduces development costs and timelines to bring bexmarilimab therapy to all HR MDS patients.”

Dr Jalkanen said the feedback underlined that the FDA saw the high unmet need in HR MDS - a condition for which new treatment options were urgently needed.

“The FDA's proposal has provided Faron with clear guidance on the path to approval that will confirm the highly encouraging results bexmarilimab has already obtained in overcoming resistance to azacitidine.

“We are extremely grateful for this feedback and will work hard to deliver on this recommendation.”

At 1314 BST, shares in Faron Pharmaceuticals were up 29.29% at 128p.

Reporting by Josh White for Sharecast.com.