Faron Pharmaceuticals Limited (AIM:FARN) has won fast-track approval from the US Food and Drug Administration (FDA) for its lead candidate, bexmarilimab, which is designed to treat relapsed or refractory myelodysplastic syndrome (r/r MDS) in combination with azacitidine.
Fast-track designation is granted by the FDA for products that are intended for the treatment of serious or life-threatening disease or conditions, which demonstrate the potential to address an unmet medical need.
It will allow Faron to engage more closely with the FDA throughout the drug-development process and expedite the path to approval.
"This news highlights the urgency for new treatment options besides HMAs (hypomethylating agents) for the treatment of higher-risk MDS and solidifies our case that bexmarilimab can overcome resistance to HMAs," said Juho Jalkanen, chief executive of Faron.
Chief medical officer Petri Bono added: "r/r MDS is a serious life-threatening condition with limited treatment options and therefore highly significant unmet medical need.
“Our trial results to date in hypomethylating agent-failed MDS have shown bexmarilimab's efficacy to induce deep, clinically meaningful responses for these patients.
“This FDA fast track designation significantly strengthens bexmarilimab's position to become a new cornerstone treatment of r/r MDS and will facilitate the development of bexmarilimab for full market approval in r/r MDS.
“It also represents a significant additional recognition of the potential of myeloid cell reprogramming in overcoming resistance and activating immune system in the treatment of various haematological and solid tumours.”
In a first-half trading update, Faron noted that preliminary phase II data from the bexmarilimab trial confirmed the positive findings from Phase I in treating MDS patients who had failed HMAs.
Out of 14 patients treated with the combination of bexmarilimab and azacitidine, an overall response rate of 79% was observed.
Faron's cash position was significantly strengthened in the first half, with €35.5 million (£30 million) raised through a convertible loan issuance and two share placements, resulting in a cash balance of €30 million as of June 30.