– Completed Phase 1 SAD and MAD trial of
EDG-7500 in healthy volunteers; Dosing patients in Phase 2
CIRRUS-HCM trial of EDG-7500 in individuals with oHCM –
– Continued to advance GRAND CANYON global
pivotal cohort of sevasemten in adults with Becker –
– Advanced Phase 2 LYNX and FOX trials of
sevasemten in children with Duchenne –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today reported financial results
for the second quarter of 2024 and recent business highlights.
“We continue to make strong progress on our cardiac and skeletal
muscle programs,” said Kevin Koch, Ph.D., President and Chief
Executive Officer of Edgewise. “We look forward to sharing
important updates on both our EDG-7500 and sevasemten programs over
the coming months.”
Recent Highlights
Cardiovascular Program /
EDG-7500
EDG-7500 is a novel oral, selective, cardiac sarcomere
modulator, specifically designed to slow early contraction velocity
and address impaired cardiac relaxation associated with
hypertrophic cardiomyopathy (HCM) and other diseases of diastolic
dysfunction. Preclinical data in models of both obstructive and
non-obstructive HCM suggest the ability to drive a beneficial
response at a low risk of decreasing left ventricular ejection
fraction below normal at all doses tested. Based on EDG-7500’s
self-limiting effect on systolic contraction observed in
preclinical models, the Company plans to investigate fixed-dose
regimens of EDG-7500, thus potentially avoiding intensive safety
monitoring of patients.
Phase 1 Trial of EDG-7500: During the quarter, the
Company completed the randomized, placebo-controlled, single and
multiple ascending dose Phase 1 trial evaluating safety,
tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in
healthy adults.
Phase 2 CIRRUS-HCM trial of EDG-7500: The Company
continues to enroll CIRRUS-HCM, a multi-center, two-part,
open-label trial of EDG-7500 in patients with obstructive HCM at up
to 20 clinical sites in the U.S. The trial will evaluate the
safety, tolerability, PK and PD of EDG-7500 in up to 55
participants. Participants enrolled in this trial will receive
EDG-7500 as a single oral dose (Part A) and have the option to
receive multiple oral doses for 28 days (Part B). Part B of the
trial will also enroll non-obstructive HCM patients who will be
studied over 28 days. The Company is on track to present topline
data of EDG-7500 from the Phase 1 trial in healthy volunteers and
from the single dose arm of CIRRUS-HCM (Part A) in patients with
obstructive HCM (three cohorts across different doses) in
September. To learn more about these trials visit
clinicaltrials.gov, NCT06011317 (Phase 1) and NCT06347159 (Phase
2).
Muscular Dystrophy Program /
sevasemten
Becker Muscular Dystrophy (Becker)
Sevasemten is an orally administered small molecule designed to
prevent contraction-induced muscle damage in muscular dystrophies
including Becker and Duchenne. There are currently no approved
therapies for individuals with Becker, a serious genetic,
progressive neuromuscular disorder with significant unmet need.
CANYON Phase 2 placebo-controlled trial in adults with
Becker: CANYON, the largest interventional Becker trial to
date, includes 40 adults and 29 adolescents with a sevasemten
treatment period of 12 months. The primary endpoint of CANYON is
change in creatine kinase (CK) over the treatment period with
additional measures collected, including North Star Ambulatory
Assessment (NSAA), 100-meter timed test, biomarkers of muscle
damage and MRI. The Company expects to report CANYON data in the
fourth quarter of 2024.
GRAND CANYON, a global pivotal cohort in Becker: GRAND
CANYON, an expansion of the CANYON placebo-controlled trial, is a
multi-center, randomized, double-blind, placebo-controlled cohort
to evaluate the safety and efficacy of sevasemten in adults with
Becker. The primary endpoint of GRAND CANYON is change in NSAA. In
addition, other functional assessments, biomarkers of muscle damage
and safety will be assessed. GRAND CANYON is an 18-month cohort
anticipated to recruit approximately 120 individuals with Becker.
Data from GRAND CANYON, if positive, could support a marketing
application. To learn more, go to clinicaltrials.gov (NCT05291091)
or the GRAND CANYON microsite: https://www.beckergcstudy.com.
Duchenne Muscular Dystrophy
LYNX Phase 2 trial in boys with Duchenne: LYNX is a
2-part multi-center, dose-finding Phase 2 trial to evaluate the
effect of sevasemten on safety, PK, and biomarkers of muscle damage
in children aged 4 to 9 years with Duchenne treated with oral,
once-daily sevasemten. The trial will also explore changes in
functional measures such as NSAA, stride velocity 95th centile
(SV95C) and self-reported/caregiver-reported outcomes.
The Company plans to report LYNX data, including safety, PK,
changes in biomarkers of muscle damage and functional changes in
NSAA and SV95C in the fourth quarter of 2024. The Company will rely
on LYNX data, along with data from the FOX trial of Duchenne
children previously treated with gene therapy, to guide the design
and powering of a Phase 3 trial in Duchenne, planned to be
initiated in the first half of 2025. For more information on LYNX
go to clinicaltrials.gov to learn more about this trial
(NCT05540860).
FOX Phase 2 trial in boys with Duchenne (previously treated
with gene therapy): The Company is advancing FOX, a Phase 2
placebo-controlled trial to assess the effect of sevasemten over 12
weeks on safety, PK and biomarkers of muscle damage in children and
adolescents aged 6 to 14 years with Duchenne who have been
previously treated with gene therapy. The trial will also explore
changes in functional measures such as NSAA, SV95C and
self-reported/caregiver-reported outcomes. There has been
exceptional enthusiasm from the Duchenne community for this trial,
evident in the Company’s ability to over-enroll the trial within
two months. Go to clinicaltrials.gov to learn more about this trial
(NCT06100887).
Strengthened Engagement with the
Scientific and Patient Communities
The Company continued its education and outreach in the HCM and
muscular dystrophy medical and patient communities. Presentations
were made at the American Heart Association’s Basic Cardiovascular
Sciences Scientific Sessions and Parent Project Muscular Dystrophy
Annual Conference, as well as additional patient-focused forums.
The Company continues to sponsor and participate in numerous other
clinician and patient-focused events.
Second Quarter Financial Results
Cash, cash equivalents and marketable securities were
$511.8 million as of June 30, 2024.
Research and development (R&D) expenses were $30.7
million for the second quarter of 2024, compared to $27.7 million
for the immediately preceding quarter. The increase of $3.0 million
was primarily driven by an additional $1.7 million in clinical
trial expenses and related development activities for the
sevasemten and EDG-7500 clinical programs from increasing clinical
trial enrollment, $0.9 million increase in drug discovery and
preclinical costs, and $0.4 million higher personnel-related
costs.
General and Administrative (G&A) expenses were $7.4
million for the second quarter of 2024, compared to $7.1 million
for the immediately preceding quarter. The increase of $0.3 million
was primarily driven by $0.2 million in increased personnel-related
costs and $0.1 million in professional and consulting costs.
Net loss and net loss per share for the second quarter of
2024 was $31.5 million or $0.34 per share, compared to $28.5
million or $0.33 per share for the immediately preceding
quarter.
About Edgewise Therapeutics
Edgewise Therapeutics is a leading muscle disease
biopharmaceutical company developing novel therapeutics for
muscular dystrophies and serious cardiac conditions. The Company’s
deep expertise in muscle physiology is driving a new generation of
novel therapeutics. Sevasemten is an orally administered skeletal
myosin inhibitor in late-stage clinical trials in Becker and
Duchenne muscular dystrophies. EDG-7500 is a novel cardiac
sarcomere modulator for the treatment of hypertrophic
cardiomyopathy and other diseases of diastolic dysfunction,
currently in Phase 2 clinical development. The entire team at
Edgewise is dedicated to our mission: changing the lives of
patients and families affected by serious muscle diseases. To learn
more, go to: www.edgewisetx.com or follow us on LinkedIn, X
(formerly Twitter), Facebook and Instagram.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as that
term is defined in Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. Statements in
this press release that are not purely historical are
forward-looking statements. Such forward-looking statements
include, among other things, statements regarding the potential of,
and expectations regarding, Edgewise’s product candidates and
programs, including sevasemten and EDG-7500; statements regarding
Edgewise’s expectations relating to its clinical trials, including
timing of reporting data (including the CANYON Phase 2 trial, LYNX
Phase 2 trial, the single dose arm of CIRRUS-HCM in patients with
obstructive HCM and the topline data of Phase 1 trial of EDG-7500
in healthy volunteers) and statements regarding Edgewise’s
expectations relating to its plans to investigate fixed-dose
regimens of EDG-7500; statements regarding the advancement of
Edgewise’s research and development programs; the timing of the
initiation of a Phase 3 trial of sevasemten in Duchenne, a 28-day
trial in patients with obstructive and non-obstructive HCM and an
open-label extension trial of EDG-7500; the possibility of data
from GRAND CANYON to support a marketing application; statements
regarding Edgewise’s pipeline of product candidates and programs;
statements regarding Edgewise’s anticipated milestones; statements
regarding potential over enrollment of the FOX Phase 2 trial; and
statements by Edgewise’s President and Chief Executive Officer.
Words such as “believes,” “anticipates,” “plans,” “expects,”
“intends,” “will,” “goal,” “potential” and similar expressions are
intended to identify forward-looking statements. The
forward-looking statements contained herein are based upon
Edgewise’s current expectations and involve assumptions that may
never materialize or may prove to be incorrect. Actual results
could differ materially from those projected in any forward-looking
statements due to numerous risks and uncertainties, including but
not limited to: risks associated with Edgewise’s limited operating
history, its products being early in development and not having
products approved for commercial sale; risks associated with
Edgewise not having generated any revenue to date; Edgewise’s
ability to achieve objectives relating to the discovery,
development and commercialization of its product candidates, if
approved; Edgewise’s need for substantial additional capital to
finance its operations; Edgewise’s substantial dependence on the
success of its sevasemten; Edgewise’s ability to develop and
commercialize sevasemten and EDG-7500 and discover, develop and
commercialize product candidates in future programs; risks related
to Edgewise’s clinical trials of its product candidates not
demonstrating safety and efficacy; risks related to Edgewise’s
product candidates causing serious adverse events, toxicities or
other undesirable side effects; the outcome of preclinical testing
and early clinical trials not being predictive of the success of
later clinical trials and the risks related to the results of
Edgewise’s clinical trials not satisfying the requirements of
regulatory authorities; delays or difficulties in the enrollment
and/or maintenance of patients in clinical trials; risks related to
failure to capitalize on other indications or product candidates;
risks related to competition; risks relating to interim, topline
and preliminary data from Edgewise’s clinical trials changing as
more patient data becomes available; risks related to the
regulatory approval processes being lengthy, time consuming and
inherently unpredictable; risks related to regulatory authorities
not accepting data from trials conducted in locations outside of
their jurisdiction; risks relating to Edgewise’s ability to attract
and retain highly skilled executive officers and employees;
Edgewise’s ability to obtain and maintain intellectual property
protection for its product candidates; Edgewise’s reliance on third
parties; general economic and market conditions; and other risks.
Information regarding the foregoing and additional risks may be
found in the section entitled “Risk Factors” in documents that
Edgewise files from time to time with the U.S. Securities and
Exchange Commission. These forward-looking statements are made as
of the date of this press release, and Edgewise assumes no
obligation to update the forward-looking statements, or to update
the reasons why actual results could differ from those projected in
the forward-looking statements, except as required by
law.
This press release contains hyperlinks to information that is
not deemed to be incorporated by reference into this press
release.
Edgewise Therapeutics, Inc. Condensed Statement of
Operations (in thousands except share and per share amounts,
unaudited) Three months ended June 30,
2024 March 31, 2024 Operating expenses: Research
and development $
30,680
$
27,694
General and administrative
7,427
7,059
Total operating expenses
38,107
34,753
Loss from operations
(38,107
)
(34,753
)
Interest income
6,610
6,228
Net loss $
(31,497
)
$
(28,525
)
Net loss per share - basic and diluted $
(0.34
)
$
(0.33
)
Weighted-average shares outstanding, basic and diluted
93,515,356
87,567,307
Edgewise Therapeutics, Inc. Condensed Balance Sheet
Data (in thousands, unaudited)
June 30,
December 31,
2024
2023
Assets Cash, cash equivalents and marketable securities $
511,751
$
318,393
Other assets
19,896
21,642
Total assets $
531,647
$
340,035
Liabilities and stockholders' equity Liabilities
21,173
21,205
Stockholders' equity
510,474
318,830
Total liabilities and stockholders' equity $
531,647
$
340,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240808684829/en/
Investors: Michael Carruthers, Chief Financial Officer
ir@edgewisetx.com
Media: Maureen Franco, VP Corporate Communications
media@edgewisetx.com
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