REG - Faron Pharma. Oy - Positive EMA Opinion on Orphan Drug Designation

Faron Pharmaceuticals OyAnnouncement

27/02/2025 07:30

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RNS Number : 6186Y  Faron Pharmaceuticals Oy  27 February 2025

Faron Pharmaceuticals Ltd.

 

("Faron" or the "Company")

 

 

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.

 

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 (mailto: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
(http://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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