Faron Pharmaceuticals Ltd | Company announcement | February 27, 2025 at 09:30:00 EET
Inside Information: Faron Receives Positive EMA Opinion on Orphan Drug Designation for Bexmarilimab for the treatment of myelodysplastic syndrome (MDS)
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.
For more information please contact:
ICR Healthcare
Mary-Jane Elliott, David Daley, Lindsey Neville
Phone: +44 (0)20 3709 5700
E-mail: faron@icrhealthcare.com
Cairn Financial Advisers LLP, Nominated Adviser and Broker
Sandy Jamieson, Jo Turner
Phone: +44 (0) 207 213 0880
Sisu Partners Oy, Certified Adviser on Nasdaq First North
Juha Karttunen
Phone: +358 (0)40 555 4727
Jukka Järvelä
Phone: +358 (0)50 553 8990
About BEXMAB
The BEXMAB study is an open-label Phase 1/2 clinical trial investigating bexmarilimab in combination with standard of care (SoC) in the aggressive hematological malignancies of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The primary objective is to determine the safety and tolerability of bexmarilimab in combination with SoC (azacitidine) treatment. Directly targeting Clever-1 could limit the replication capacity of cancer cells, increase antigen presentation, ignite an immune response, and allow current treatments to be more effective. Clever-1 is highly expressed in both AML and MDS and associated with therapy resistance, limited T cell activation and poor outcomes.
About Bexmarilimab
Bexmarilimab is Faron's wholly owned, investigational immunotherapy designed to overcome resistance to existing treatments and optimize clinical outcomes, by targeting myeloid cell function and igniting the immune system. Bexmarilimab binds to Clever-1, an immunosuppressive receptor found on macrophages leading to tumor growth and metastases (i.e. helps cancer evade the immune system). By targeting the Clever-1 receptor on macrophages, bexmarilimab alters the tumor microenvironment, reprogramming macrophages from an immunosuppressive (M2) state to an immunostimulatory (M1) one, upregulating interferon production and priming the immune system to attack tumors and sensitizing cancer cells to standard of care.
About Faron Pharmaceuticals Ltd.
Faron (AIM: FARN, First North: FARON) is a global, clinical-stage biopharmaceutical company, focused on tackling cancers via novel immunotherapies. Its mission is to bring the promise of immunotherapy to a broader population by uncovering novel ways to control and harness the power of the immune system. The Company's lead asset is bexmarilimab, a novel anti-Clever-1 humanized antibody, with the potential to remove immunosuppression of cancers through reprogramming myeloid cell function. Bexmarilimab is being investigated in Phase I/II clinical trials as a potential therapy for patients with hematological cancers in combination with other standard treatments. Further information is available at www.faron.com.
Forward-Looking Statements
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A number of factors could cause actual results to differ materially from the results and expectations discussed in the forward-looking statements, many of which are beyond the control of the Company. In addition, other factors which could cause actual results to differ materially include the ability of the Company to successfully license its programs within the anticipated timeframe or at all, risks associated with vulnerability to general economic and business conditions, competition, environmental and other regulatory changes, actions by governmental authorities, the availability of capital markets or other sources of funding, reliance on key personnel, uninsured and underinsured losses and other factors. Although any forward-looking statements contained in this announcement are based upon what the Directors believe to be reasonable assumptions, the Company cannot assure investors that actual results will be consistent with such forward-looking statements. Accordingly, readers are cautioned not to place undue reliance on forward-looking statements. Subject to any continuing obligations under applicable law or any relevant AIM Rule requirements, in providing this information the Company does not undertake any obligation to publicly update or revise any of the forward-looking statements or to advise of any change in events, conditions or circumstances on which any such statement is based.
