LONDON (ShareCast) - (Sharecast News) - Faron Pharmaceuticals said on Monday that it has secured a clear regulatory pathway for the potential approval of its lead cancer therapy bexmarilimab, following positive feedback from the US Food and Drug Administration.
The AIM-traded Finnish biopharmaceutical company said the FDA advised it to prioritise development of the treatment in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients, enabling a possible accelerated approval in the frontline setting.

It said the guidance followed an end-of-phase two meeting held to align on the design of a planned registrational phase 2 and 3 trial combining bexmarilimab with azacitidine.

"We are extremely encouraged by the collaborative and highly productive dialogue with the FDA, which provided a clear and actionable path for the clinical development of bexmarilimab in frontline HR-MDS, an area of profound unmet medical need," said chief executive Dr Juho Jalkanen.

"The agency's guidance has endorsed a direct route towards accelerated approval using Complete Response (CR) + CR equivalent (CReq) per International Working Group (IWG) 2023 criteria as a co-primary endpoint with Overall Survival (OS).

"This is a significant step forward in our mission to provide a potentially transformative new treatment option to patients and represents a major regulatory de-risking milestone, as we are now only one study away from getting bexmarilimab approved for the benefit of HR MDS patients."The trial would begin with a dose optimisation stage comparing one milligram and three millligrams per kilogram regimens with placebo, before moving seamlessly into the pivotal phase.

An interim analysis of complete response (CR) and CR equivalent (CReq) rates, defined by the International Working Group's 2023 criteria, would support an accelerated approval application.

Overall survival woud be included as a co-primary endpoint, while composite complete remission will serve as a key secondary measure.

"I am thrilled that we have agreed with the regulators on a pathway to explore the full potential of bexmarilimab in the frontline setting for patients with HR-MDS," commented Dr Amer Zeidan, professor of medicine at Yale School of Medicine and chief of hematologic malignancies at Yale Cancer Center.

"I also would like to highlight the FDA accepting the use of CR+CReq IWG 2023 criteria as part of the primary endpoint, making this the first registrational clinical trial to do so.

"Many experts, including myself, strongly believe the IWG 2023 criteria to be more patient centric and reflect clinically meaningful benefits to patients in a more robust fashion than the old IWG 2006 criteria, which have been used for all past - and failed -phase 3 clinical trials for HR-MDS."

Faron said the FDA was also satisfied with its non-clinical and manufacturing plans to support the next stage of development and eventual approval.

At 1112 BST, shares in Faron Pharmaceuticals were up 6.9% at 224.5p.

Reporting by Josh White for Sharecast.com.

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