Positive EMA Opinion on Orphan Drug Designation

Inside Information: Faron Receives Positive EMA Opinion on Orphan Drug Designation for Bexmarilimab for the treatment of myelodysplastic syndrome (MDS)

Company announcement, Inside Information, 27 February 2025 at 7:30 a.m. GMT / 9:30 a.m. EET

Key highlights

• Given strong efficacy data of bexmarilimab in the treatment of MDS patients, the EU's Committee for Orphan Medicinal Products (COMP) issued positive opinion on Orphan Drug Designation (ODD) for bexmarilimab
• Positive opinion on Orphan Drug Designation further strengthens bexmarilimab program by offering clinical development and commercialization benefits
• Faron is on track to report frontline and HMA-failed (r/r) MDS top-line efficacy results in April 2025

TURKU, Finland - Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company pursuing a CLEVER-1 receptor targeting approach to reprogramming myeloid cells to activate anti-tumor immunity in hematological and solid tumors, today announces that their lead candidate bexmarilimab has been issued positive opinion on Orphan Drug Designation (ODD) for the treatment of myelodysplastic syndrome (MDS) in combination with azacitidine by the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA).

The EMA's ODD program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases. The ODD qualifies the sponsor of the drug for certain development incentives, including protocol assistance, specific advice, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.

Previously, in August 2024, bexmarilimab was granted fast track designation by the FDA which aims to expedite the review process for promising medicines in areas of unmet medical needs.

"Receiving positive opinion on orphan drug designation for bexmarilimab for the treatment of myelodysplastic syndrome (MDS) in combination with azacitidine marks a significant milestone for Faron Pharmaceuticals as we continue to develop bexmarilimab for MDS and other cancers. The interim results of bexmarilimab's ongoing phase I/II BEXMAB MDS trial reviewed by the EMA included also a median Overall Survival estimate. That was 13.4 months which is significantly longer than the median Overall Survival of 5-6 months described in earlier published r/r MDS survival reports. We are excited by this EMA positive opinion which demonstrates our continued progress and reinforces our belief in the potential of bexmarilimab to become a new and effective therapy for MDS." says Dr. Bono, the CMO of Faron.

About Bexmarilimab

About Faron Pharmaceuticals Ltd.