Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a
clinical-stage biopharmaceutical company focused on improving the
lives of patients with genetically defined rare diseases, today
outlined its recent accomplishments and expected 2023 milestones.
“We are entering 2023 with a tremendous amount of momentum and
expect it to be a productive year for our two clinical programs:
FTX-6058 for SCD, and losmapimod for FSHD,” said Robert J. Gould,
Ph.D., Fulcrum’s interim president and chief executive officer.
“FTX-6058 is a potential best-in-class oral HbF inducer candidate
that could address critical gaps in the SCD treatment landscape. We
are excited by the levels of HbF induction in our initial doses and
look forward to further broadening our understanding of its effect
at a higher dose. Meanwhile, the Phase 3 REACH trial with
losmapimod, a potential first-to-market therapy in FSHD, is
expected to complete enrollment in the second half of the
year.”
“We are encouraged by the new FTX-6058 data at 6 mg that show
clinically relevant HbF increases, up to 9.5% from baseline with
hemolysis and anemia improvement, suggesting its potential for
best-in-class therapy for people living with sickle cell disease,”
said Santiago Arroyo, M.D., Ph.D., Fulcrum’s chief medical
officer.
Key Business Updates and Upcoming
Milestones
FTX-6058
- Received Fast Track Designation from the U.S. Food and Drug
Administration (FDA) for the treatment of SCD in December 2022
- Phase 1b data from Cohort 1 subjects in the 6 mg cohort (n=10)
showed up to 9.5% absolute HbF increases from baseline; data
suggest no difference in response in subjects on (n=3) and off
(n=7) background hydroxyurea
- Improved biomarkers of hemolysis in evaluable patients dosed at
6 mg
- In the Phase 1b trial, FTX-6058 appears to have dose dependent
and clinically relevant increases in HbF; all subjects adherent to
dosing regimen showed a response
- Generally well tolerated with no drug-related treatment
emergent serious adverse events and no discontinuations due to
treatment emergent adverse events to date
- Enrolling 12 mg dose cohort of the Phase 1b trial
- Next data update planned during the fourth quarter of 2023
Losmapimod
- Enrollment ongoing in the REACH Phase 3 pivotal trial at sites
in the U.S., Canada, and Europe
- Plan to complete enrollment in the second half of 2023
Financial Guidance
- Fulcrum maintains its cash runway guidance and expects its
existing cash, cash equivalents, and marketable securities will be
sufficient to fund its currently planned operating expenses and
capital expenditure requirements into late 2024
Corporate
- Fulcrum announced CEO transition today; Robert J. Gould, Ph.D.,
former president and founding chief executive officer of Fulcrum
has been appointed as interim CEO as Bryan Stuart departs to pursue
other opportunities
J.P. Morgan Conference Webcast
- A live audio webcast of Fulcrum’s presentation at the 41st
Annual J.P. Morgan Healthcare Conference will be available through
the Investor Relations section of the Fulcrum website at
https://ir.fulcrumtx.com/events-and-presentations. An archived
replay will be available on Fulcrum’s website for 30 days.
About Fulcrum TherapeuticsFulcrum Therapeutics
is a clinical-stage biopharmaceutical company focused on improving
the lives of patients with genetically defined rare diseases in
areas of high unmet medical need. Fulcrum’s two lead programs in
clinical development are losmapimod, a small molecule for the
treatment of facioscapulohumeral muscular dystrophy (FSHD), and
FTX-6058, a small molecule designed to increase expression of fetal
hemoglobin for the treatment of sickle cell disease (SCD) and other
hemoglobinopathies, including beta-thalassemia. Fulcrum’s
proprietary product engine, FulcrumSeek™, identifies drug targets
that can modulate gene expression to treat the known root cause of
gene mis-expression. For more information, visit www.fulcrumtx.com
and follow us on Twitter @FulcrumTx and LinkedIn.
About FTX-6058FTX-6058 is an investigational
oral small-molecule inhibitor of Embryonic Ectoderm Development
(EED) that was discovered using FulcrumSeek™, Fulcrum’s proprietary
discovery engine. Inhibition of EED leads to potent downregulation
of key fetal globin repressors, including BCL11A, thereby causing
an increase in fetal hemoglobin (HbF). FTX-6058 is being developed
for the treatment of sickle cell disease (SCD) and other
hemoglobinopathies. FTX-6058 is currently being evaluated in a
Phase 1b multi-center open-label trial in people with SCD
(NCT05169580). Initial data demonstrated proof-of-concept and
achieved absolute levels of HbF increases associated with potential
overall patient benefit. To date, FTX-6058 has been generally
well-tolerated in people with SCD with up to three months of
exposure, with no serious treatment-emergent adverse events
reported. FTX-6058 has been granted U.S. Food and Drug
Administration (FDA) Fast Track designation and Orphan Drug
Designation for the treatment of SCD.
About Sickle Cell Disease Sickle cell disease
is a genetic disorder of the red blood cells caused by a mutation
in the HBB gene. This gene encodes a protein that is a key
component of hemoglobin, a protein complex whose function is to
transport oxygen in the body. The result of the mutation is less
efficient oxygen transport and the formation of red blood cells
that have a sickle shape. These sickle shaped cells are much less
flexible than healthy cells and can block blood vessels or rupture
cells. People with sickle cell disease typically suffer from
serious clinical consequences, which may include anemia, pain,
infections, stroke, heart disease, pulmonary hypertension, kidney
failure, liver disease, and reduced life expectancy.
About LosmapimodLosmapimod is a selective
p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum
exclusively in-licensed losmapimod from GSK following Fulcrum’s
discovery of the role of p38α/β inhibitors in the reduction of DUX4
expression and an extensive review of known compounds. Results
reported from the Phase 2b ReDUX4 trial demonstrated slowed disease
progression and improved function, including positive impacts on
upper extremity strength and functional measures supporting
losmapimod’s potential to be a transformative therapy for the
treatment of FSHD. Although losmapimod had never previously been
explored in muscular dystrophies, it had been evaluated in more
than 3,600 subjects in clinical trials across multiple other
indications, with no safety signals attributed to losmapimod.
Losmapimod has been granted U.S. Food and Drug Administration (FDA)
Fast Track designation and Orphan Drug Designation for the
treatment of FSHD. Losmapimod is currently being evaluated in a
Phase 3 multi-center randomized, double-blind, placebo-controlled,
48-week parallel-group study in people with FSHD (NCT05397470).
About FSHDFSHD is a serious, rare, progressive
and debilitating disease for which there are no approved
treatments. It is characterized by fat infiltration of skeletal
muscle leading to muscular atrophy involving primarily the face,
scapula and shoulders, upper arms, and abdomen. Impact on patients
includes profound decreases in the ability to perform activities of
daily living, loss of upper limb function, loss of mobility and
independence and chronic pain. FSHD is one of the most common forms
of muscular dystrophy and has an estimated patient population of
16,000 to 38,000 in the United States alone.
Forward-Looking Statements This press release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995 that involve
substantial risks and uncertainties. All statements, other than
statements of historical facts, contained in this press release are
forward-looking statements, including express or implied statements
regarding enrollment in Fulcrum’s ongoing clinical trials and
timing of completion; potential therapeutic benefit of FTX-6058 and
losmapimod; planned data announcements; and Fulcrum’s cash runway,
among others. The words “anticipate,” “believe,” “continue,”
“could,” “estimate,” “expect,” “intend,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “target,” “will,”
“would” and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Any forward-looking
statements are based on management’s current expectations of future
events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in, or implied by, such forward-looking statements.
These risks and uncertainties include, but are not limited to,
risks associated with Fulcrum’s ability to continue to advance its
product candidates in clinical trials; initiate and enroll clinical
trials on the timeline expected or at all; obtain and maintain
necessary approvals from the FDA and other regulatory authorities;
replicate in clinical trials positive results found in preclinical
studies and/or earlier-stage clinical trials of losmapimod,
FTX-6058 and any other product candidates; obtain, maintain or
protect intellectual property rights related to its product
candidates; manage expenses; realize the anticipated benefits of
the strategic realignment; manage executive and employee turnover;
and raise the substantial additional capital needed to achieve its
business objectives, among others. For a discussion of other risks
and uncertainties, and other important factors, any of which could
cause Fulcrum’s actual results to differ from those contained in
the forward-looking statements, see the “Risk Factors” section, as
well as discussions of potential risks, uncertainties, and other
important factors, in Fulcrum’s most recent filings with the
Securities and Exchange Commission. In addition, the
forward-looking statements included in this press release represent
Fulcrum’s views as of the date hereof and should not be relied upon
as representing Fulcrum’s views as of any date subsequent to the
date hereof. Fulcrum anticipates that subsequent events and
developments will cause Fulcrum’s views to change. However, while
Fulcrum may elect to update these forward-looking statements at
some point in the future, Fulcrum specifically disclaims any
obligation to do so.
Contact:
Investors:Stephanie AscherStern IR,
Inc.stephanie.ascher@sternir.com 212-362-1200
Media:Dee SmithExecutive Director, Corporate Communications
Fulcrum Therapeutics, Inc.dsmith@fulcrumtx.com202-746-1324
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