Faron Pharmaceuticals
Ltd.
("Faron"
or the "Company")
Inside Information: Faron
Announces Positive FDA Feedback
Company announcement, Inside Information, 11 July 2024 at 7:00
a.m. BST / 9:00 a.m. EEST
Key
highlights
- Faron had a formal meeting with the FDA to discuss the
registrational clinical development plan for bexmarilimab in the treatment
myelodysplastic syndrome (MDS).
- The FDA acknowledged the difficulties of running a randomized
study with a comparator in the relapsed / refractory setting (r/r)
and instead proposed that Faron conduct a confirmatory Phase III
study in frontline high-risk MDS (HR MDS), that would not require a
separate Phase III in r/r MDS.
- This
FDA guidance is part of Project Frontrunner, an initiative intended
to bring promising new cancer treatments as early as possible to a
broader patient population.
- The Phase III suggested by the FDA targets a significantly
larger patient population with potential for faster approval
earlier than anticipated, speeding up and increasing our sales
forecast for bexmarilimab.
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 tumor
microenvironments, today provides information on the result of its
formal Type D Scientific Advice Meeting with the USA Food and Drug
Administration (the FDA) regarding the registrational study plan
for its drug candidate bexmarilimab in relapsed and
refractory high risk MDS (r/r MDS).
Given the previously reported
promising results of treating r/r MDS using a combination of
bexmarilimab + azacitidine
to overcome primary or developed resistance to azacitidine, Faron
had proposed to move into a randomized registrational Phase III
study for the treatment of r/r MDS using bexmarilimab + azacitidine against the
investigator's choice of a hypomethylating agent (HMA). Instead,
given the encouraging efficacy already seen in both frontline and
r/r HR MDS and the well-established safety profile of bexmarilimab, the FDA proposed that
after the ongoing Phase II BEXMAB study in r/r MDS, Faron should
move directly into a registrational blinded randomized frontline HR
MDS study investigating bexmarilimab + azacitidine against
placebo + azacitidine. The FDA noted that given the relatively
modest efficacy of single agent azacitidine and the current
response rates with bexmarilimab that the size of such a
frontline study may not have to be substantially larger than the
proposed study in the r/r setting.
Further, the FDA suggested such a
frontline study could be seen in the context of FDA's Project
Frontrunner. Project FrontRunner is an FDA
Oncology Center of Excellence (OCE) initiative to encourage drug
sponsors to develop and seek approval of promising new cancer drugs
for advanced diseases in an earlier clinical setting, rather than
the usual approach to develop and seek approval of a new drug for
treatment of patients who have received numerous prior lines of
therapies or have exhausted available treatment options. The
FDA guidelines give different possible approval strategies to
sponsors, including the conduct of a frontline trial supporting an
accelerated approval in the r/r settings.
Project FrontRunner | FDA
Subject to continued positive
results, the FDA's feedback means that a separate Phase III in r/r
MDS would not be required and Faron's ongoing BEXMAB Phase II study
could be the registrational trial for patients with r/r MDS, given
that the benefit of bexmarilimab + azacitidine against
azacitidine alone will be confirmed in an interim read-out of the
response rate from a Phase III in frontline HR MDS study.
Accelerated approval for frontline HR MDS would come from the
response rate of this single Phase III study and the full approval
from the survival read-out of the same study.
"Faron is now adjusting its
development plan accordingly", says Dr. Juho Jalkanen, Chief
Executive Officer of Faron. "This is very
positive feedback and exceeds our expectations. The FDA's proposal
significantly reduces development costs and timelines to bring
bexmarilimab therapy to
all HR MDS patients. This feedback underlines that the FDA sees the
high unmet need in HR MDS, a condition for which new treatment
options are urgently needed. The FDA's proposal has provided Faron
with clear guidance on the path to approval that will confirm the
highly encouraging results bexmarilimab has already obtained in
overcoming resistance to azacitidine. We are extremely grateful for
this feedback and will work hard to deliver on this
recommendation."
"The suggested Phase III targets a
significantly bigger patient population sooner than anticipated,
speeding up and increasing our sales forecast for bexmarilimab. This does not
significantly impact our ongoing activities and cash runway, as the
Phase II in r/r MDS continues as planned. In addition, we will
enroll more frontline HR MDS patients into the Phase I part of
BEXMAB to better understand the effect size, which will enable us
to successfully power and design the proposed frontline Phase III
study. We believe we can offset this additional clinical investment
through other savings, so that it will not have a significant
impact on our cash runway. The only deviation from the original
plan is that instead of a Phase III in r/r MDS, we will start
preparations for a Phase III in frontline HR MDS, which is a
remarkable achievement.", continues Dr. Jalkanen.
Faron will be hosting a virtual
webinar to discuss the FDA feedback and updated clinical
development plans July 15th, at 15.00 EEST / 13.00
BST.
To register for the event
visit:
https://faron.videosync.fi/fda-feedback-update
or contact the IR team for more information
at investor.relations@faron.com.
For
the purposes of MAR and UK MAR, the person responsible for
arranging for the release of this announcement on behalf of Faron
is Juho Jalkanen, Chief Executive Officer.
For
more information please contact:
Investor Contact
Faron Pharmaceuticals
E-mail:
investor.relations@faron.com
Media Contact
ICR
Consilium
Mary-Jane Elliott, David Daley,
Lindsey Neville
Phone: +44 (0)20 3709
5700
E-mail: faron@consilium-comms.com
Cairn Financial Advisers LLP, Nomad
Sandy Jamieson, Jo Turner
Phone: +44 (0) 207 213
0880
Peel Hunt LLP, Broker
Christopher Golden, James
Steel
Phone: +44 (0) 20 7418
8900
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
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
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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.