Faron Pharmaceuticals
Ltd.
("Faron"
or the "Company")
Inside Information: FDA
Grants Orphan Drug Designation for Bexmarilimab in MDS
Company announcement, Inside Information, 3 March
2025
Key
highlights
- 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 granted Orphan
Drug Designation for the treatment of myelodysplastic syndromes
(MDS) by the USA Food and Drug Administration (the
FDA).
The FDA's Orphan Drug
Designation program provides orphan status to drugs defined as
those intended for the safe and effective treatment, diagnosis or
prevention of rare diseases that affect fewer than 200,000 people
in the United States. Orphan Drug Designation qualifies the
sponsor of the drug for certain development incentives, including
tax credits for qualified clinical testing, prescription drug user
fee exemptions and seven-year marketing exclusivity
upon FDA approval. The FDA previously granted Orphan Drug
Designation to bexmarilimab for the treatment of
acute myeloid leukemia (AML) in August 2023.
"Receiving FDA's orphan drug
designation for bexmarilimab for the treatment of
myelodysplastic syndrome marks a significant milestone for Faron
Pharmaceuticals as we continue to develop bexmarilimab for MDS and other
cancers. This FDA ODD along with the previously granted FDA fast
track designation highlights our continued progress and reinforces
our belief in the potential of bexmarilimab to address this
significant unmet need for treatment of MDS. These designations
allow us to receive important regulatory guidance for the
development of bexmarilimab and potential additional
market exclusivity upon approval" 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 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.
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
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.
