FDA Orphan Drug Designation for Bexmarilimab

Faron Pharmaceuticals Oy

29 August 2023

 

Faron Pharmaceuticals Ltd.

("Faron" or "the Company")

 Inside Information:  Faron Receives FDA Orphan Drug Designation for Bexmarilimab in Acute Myeloid Leukemia

·    ODD further strengthens bexmarilimab program by offering clinical development and commercialization benefits

·    Expected completion of dose escalation, readout of enrichment cohorts, and Phase II initiation in Q4 2023

Company Announcement, August 29, 2023

Inside Information

TURKU, Finland / BOSTON, Massachusetts - Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers via novel immunotherapies, today announces the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its wholly owned asset bexmarilimab, for the treatment of acute myeloid leukemia (AML).

FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. ODD provides Faron with certain benefits, such as market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials.

"Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop bexmarilimab as a potential treatment for AML," said Chief Medical Officer Dr. Marie-Louise Fjällskog. "The designation represents a milestone in our development journey, one that we believe when combined with standard of care, will lead to better patient outcomes and improved quality of life."

Bexmarilimab is currently in Phase I/II BEXMAB study (ClinicalTrials.gov: NCT05428969) in combination with standard of care (SoC) for the treatment of aggressive hematological malignancies of relapsed/refractory AML and myelodysplastic syndromes (MDS). Last month, Faron reported updated, positive data from the Phase I portion of the trial. Three of five patients in the 6 mg/kg bexmarilimab + azacitidine doublet cohort achieved objective responses and eight (out of 15 patients) objective responses were observed in all three doublet dosing cohorts, with one patient remaining on treatment for 13 months.

The completion of dose escalation, readout of enrichment cohorts and initiation of the Phase II portion of the BEXMAB trial are expected in Q4 2023.

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 ("MAR").

For more information please contact:

Investor Contact

LifeSci Advisors

Daniel Ferry

Managing Director

[e-mail]

[phone]

Media Contact

Faron Pharmaceuticals

Jennifer C. Smith-Parker

Head of Communications

[e-mail]

Cairn Financial Advisers LLP, Nomad

Sandy Jamieson, Jo Turner

Phone: [phone]

Peel Hunt LLP, Broker

Christopher Golden, James Steel

Phone: [phone]

Sisu Partners Oy, Certified Adviser on Nasdaq First North

Juha Karttunen

Phone: [phone]

Jukka Järvelä

Phone: [phone]

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 BEXMAB

The BEXMAB study is a first-in-human, open-label Phase I/II 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 and to identify the recommended Phase II dose. 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 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 targeting 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

Certain statements in this announcement are, or may be deemed to be, forward-looking statements. Forward looking statements are identified by their use of terms and phrases such as ''believe'', ''could'', "should", "expect", "hope", "seek", ''envisage'', ''estimate'', ''intend'', ''may'', ''plan'', ''potentially'', ''will'' or the negative of those, variations or comparable expressions, including references to assumptions. These forward-looking statements are not based on historical facts but rather on the Directors' current expectations and assumptions regarding the Company's future growth, results of operations, performance, future capital and other expenditures (including the amount, nature and sources of funding thereof), competitive advantages, business prospects and opportunities. Such forward-looking statements reflect the Directors' current beliefs and assumptions and are based on information currently available to the Directors.

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.

 

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