Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage
biopharmaceutical company focused on improving the lives of
patients with genetically defined rare diseases, today provided a
business update and reported financial results for the third
quarter of 2019.
“This past quarter, we made significant progress in advancing
our pipeline and executing on our goal of creating therapeutics
targeted at genetically defined rare diseases. We presented
encouraging Phase 1 clinical results from our lead program,
losmapimod in patients with FSHD, which support the design and dose
selection of our ongoing Phase 2 clinical trials. Additionally, we
announced our plans to advance our second program, FTX-6058 for the
potential treatment of sickle cell disease and beta-thalassemia,”
said Robert J. Gould, Ph.D., Fulcrum’s president and chief
executive officer. “We look forward to advancing these two programs
through our ongoing Phase 2 trials with losmapimod and our
IND-enabling studies with FTX-6058 and utilizing our product engine
to identify and validate drug targets to address diseases caused by
the mis-expression of certain genes.”
Third Quarter 2019 and Recent Business
Highlights
- In October 2019, Fulcrum presented Phase 1 data from its
clinical trial of losmapimod in facioscapulohumeral dystrophy
(FSHD) patients and related plans for using molecular biomarkers
indicative of the root cause of disease in the Company’s ongoing
Phase 2 clinical trials of losmapimod at the International Annual
Congress of the World Muscle Society in Copenhagen. The results
showed that losmapimod was well tolerated with no serious adverse
events (SAEs) reported, and that treatment with losmapimod
demonstrated dose-dependent pharmacokinetics (PK) and target
engagement (TE) in blood. These data support the selection of the
15 mg dose of losmapimod taken orally twice daily in the Company’s
ongoing Phase 2b placebo-controlled 24-week clinical trial,
referred to as ReDUX4, as well as its ongoing Phase 2 open
label-study, of losmapimod for the treatment of patients with
FSHD.
- In October 2019, Fulcrum announced progress in the Company’s
plan to advance the development program for FTX-6058 for the
potential treatment of sickle cell disease and beta-thalassemia.
Fulcrum has initiated IND-enabling studies for FTX-6058 and
anticipates filing an IND in mid-2020. In pre-clinical research,
treatment with FTX-6058 was shown to increase HbF levels to ~30% of
total hemoglobin as measured by HPLC and mass spectrometry methods
in human erythroid progenitor cells from multiple donors.
- In September 2019, Fulcrum announced the appointment of Pamela
Strode as senior vice president, regulatory affairs and quality
assurance. She previously served as senior vice president,
regulatory affairs and quality assurance at Epizyme, Inc.
Third Quarter 2019 Financial Results
- Cash Position: As of September 30, 2019,
cash and cash equivalents were $101.6 million, as compared to
$72.8 million as of December 31, 2018. Based on its
current plans, the Company expects that its existing cash and cash
equivalents, including the proceeds from its July 2019 initial
public offering, will be sufficient to enable it to fund its
operating expenses and capital expenditure requirements into the
third quarter of 2021.
- R&D Expenses: Research and development
expenses were $13.5 million for the third quarter of 2019, as
compared to $7.0 million for the third quarter of 2018. The
increase of $6.5 million was primarily due to a $2.5 million
increase associated with the achievement of a milestone due under
the right of reference and license agreement with GlaxoSmithKline
plc during the third quarter of 2019, increased costs related to
the advancement of losmapimod for the treatment of FSHD, including
increased external costs to support Fulcrum’s ongoing and planned
clinical trials, as well as increased personnel-related costs due
to additional headcount to support the growth of Fulcrum’s research
and development organization.
- G&A Expenses: General and administrative
expenses were $3.5 million for the third quarter of 2019, as
compared to $2.1 million for the third quarter of 2018. The
increase of $1.4 million was primarily due to increased
personnel-related costs due to additional headcount, as well as
increased consulting and professional fees.
- Net Loss: Net loss was $16.5 million for
the third quarter of 2019, as compared to a net loss of
$8.9 million for the third quarter of 2018.
About FSHD
FSHD is characterized by progressive skeletal muscle loss that
initially causes weakness in muscles in the face, shoulders, arms
and trunk, and progresses to weakness throughout the lower body.
Skeletal muscle weakness results in significant physical
limitations, including an inability to smile and difficulty using
arms for activities, with many patients ultimately becoming
dependent upon the use of a wheelchair for daily mobility.
FSHD is caused by mis-expression of DUX4 in skeletal muscle,
resulting in the presence of DUX4 proteins that are toxic to muscle
tissue. Normally, DUX4-driven gene expression is limited to early
embryonic development, after which time the DUX4 gene is silenced.
In people with FSHD, the DUX4 gene is turned “on” as a result of a
genetic mutation. The result is death of muscle and its replacement
by fat, leading to skeletal muscle weakness and progressive
disability. There are no approved therapies for FSHD, one of the
most common forms of muscular dystrophy, with an estimated patient
population of 16,000 to 38,000 in the United States alone.
About Losmapimod
Losmapimod is a selective p38α/β mitogen activated protein
kinase (MAPK) inhibitor that was exclusively in-licensed by Fulcrum
Therapeutics following Fulcrum’s discovery of the role of p38α/β
inhibitors in the reduction of DUX4 expression and an extensive
review of known compounds. Utilizing its internal product engine,
Fulcrum discovered that inhibition of p38α/β reduced expression of
the DUX4 gene in muscle cells derived from patients with FSHD.
Although losmapimod has never previously been explored in muscular
dystrophies, it has been evaluated in more than 3,500 subjects in
clinical trials across multiple other indications, including in
several Phase 2 trials and a Phase 3 trial. No safety signals were
attributed to losmapimod in any of these trials. Fulcrum is
currently conducting Phase 2 trials investigating the safety,
tolerability, and efficacy of losmapimod to treat the root cause of
FSHD.
About Sickle Cell Disease
Sickle cell disease (SCD) 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. SCD patients 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 Beta-thalassemia
Beta-thalassemia is a rare blood disorder caused by genetic
mutations in the HBB gene, which are associated with the absence or
reduced production of beta-globin – one of the two proteins that
comprise adult hemoglobin. This results in an abnormally low level
of hemoglobin as well as an excess of alpha-globin chains, causing
destruction of red blood cells.
Beta-thalassemia has been clinically characterized into three
forms, depending on disease severity: major, intermedia and minor.
The most severe form is generally diagnosed shortly after birth and
is characterized by life-threatening anemia. Pediatric patients do
not grow and gain weight at the typical rates, and often have
liver, heart and bone problems. Many patients with beta-thalassemia
major require chronic blood transfusions due to severe anemia that
results from low hemoglobin levels. Beta-thalassemia intermedia is
a less severe form of the disease that results in mild to moderate
anemia. These patients sometimes require blood transfusions
depending on the severity of their symptoms. Patients with
beta-thalassemia minor suffer from very mild anemia and generally
do not require treatment.
About Fulcrum Therapeutics
Fulcrum 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 proprietary product engine identifies drug targets which
can modulate gene expression to treat the known root cause of gene
mis-expression. Please visit www.fulcrumtx.com.
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, including
statements regarding the development status of the Company’s
product candidates, the timing of availability of clinical trial
data and the Company’s ability to fund its operations with cash on
hand. All statements, other than statements of historical facts,
contained in this press release, including statements regarding the
Company’s strategy, future operations, future financial position,
prospects, plans and objectives of management, are forward-looking
statements. 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 obtain and maintain necessary
approvals from the FDA and other regulatory authorities; continue
to advance its product candidates in clinical trials; replicate in
later clinical trials positive results found in preclinical studies
and early-stage clinical trials of losmapimod and its other product
candidates; advance the development of its product candidates under
the timelines it anticipates in current and future clinical trials;
obtain, maintain or protect intellectual property rights related to
its product candidates; manage expenses; and raise the substantial
additional capital needed to achieve its business objectives. For a
discussion of other risks and uncertainties, and other important
factors, any of which could cause the Company’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 the Company’s
most recent filings with the Securities and Exchange Commission. In
addition, the forward-looking statements included in this press
release represent the Company’s views as of the date hereof and
should not be relied upon as representing the Company’s views as of
any date subsequent to the date hereof. The Company anticipates
that subsequent events and developments will cause the Company’s
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so.
Fulcrum
Therapeutics, Inc.Selected Consolidated
Balance Sheet Data(In
thousands)(Unaudited)
|
September 30,2019 |
|
December 31,2018 |
Cash and cash equivalents |
$ |
101,597 |
|
|
$ |
72,797 |
|
Working capital(1) |
|
96,637 |
|
|
|
69,866 |
|
Total assets |
|
115,956 |
|
|
|
85,771 |
|
Convertible preferred stock |
|
— |
|
|
|
139,670 |
|
Total stockholders’ equity
(deficit) |
|
102,128 |
|
|
|
(63,670 |
) |
|
|
|
|
|
|
|
|
__________(1) We define working capital as current assets minus
current liabilities.
Fulcrum
Therapeutics, Inc.Consolidated Statements of
Operations and Comprehensive Loss(In thousands,
except per share data)(Unaudited)
|
Three Months
EndedSeptember 30, |
|
|
Nine Months
EndedSeptember 30, |
|
|
2019 |
|
|
2018 |
|
|
2019 |
|
|
2018 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
$ |
13,496 |
|
|
$ |
6,963 |
|
|
$ |
58,985 |
|
|
$ |
18,324 |
|
General and administrative |
|
3,510 |
|
|
|
2,125 |
|
|
|
8,742 |
|
|
|
5,923 |
|
Total operating expenses |
$ |
17,006 |
|
|
$ |
9,088 |
|
|
$ |
67,727 |
|
|
$ |
24,247 |
|
Loss from operations |
|
(17,006 |
) |
|
|
(9,088 |
) |
|
|
(67,727 |
) |
|
|
(24,247 |
) |
Other income, net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income, net |
|
457 |
|
|
|
138 |
|
|
|
1,151 |
|
|
|
133 |
|
Other income |
|
7 |
|
|
|
7 |
|
|
|
22 |
|
|
|
385 |
|
Net loss and comprehensive
loss |
$ |
(16,542 |
) |
|
$ |
(8,943 |
) |
|
$ |
(66,554 |
) |
|
$ |
(23,729 |
) |
Cumulative convertible preferred
stock dividends |
|
(796 |
) |
|
|
(1,858 |
) |
|
|
(7,128 |
) |
|
|
(3,736 |
) |
Net loss attributable to common
stockholders |
$ |
(17,338 |
) |
|
$ |
(10,801 |
) |
|
$ |
(73,682 |
) |
|
$ |
(27,465 |
) |
Net loss per share attributable
to common stockholders, basic and diluted |
$ |
(0.97 |
) |
|
$ |
(8.08 |
) |
|
$ |
(10.33 |
) |
|
$ |
(23.28 |
) |
Weighted average number of common
shares used in net loss per share attributable to common
stockholders, basic and diluted |
|
17,785 |
|
|
|
1,337 |
|
|
|
7,133 |
|
|
|
1,180 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Contact
Investors: Christina Tartaglia Stern IR, Inc.
christina.tartaglia@sternir.com 212-362-1200
Media: Lynn GranitoBerry & Company Public
Relationslgranito@berrypr.com212-253-8881
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