– In a Phase 1 in healthy subjects, EDG-7500
was well-tolerated without meaningful changes in left ventricle
ejection fraction (LVEF) –
– CIRRUS-HCM single-dose trial of EDG-7500 in
obstructive HCM demonstrated robust left ventricular outflow tract
(LVOT) gradient reductions without meaningful changes in LVEF –
– Company announced dosing of first patients in
CIRRUS-HCM 28-day trial –
– Edgewise to host webcast event on Thursday,
September 19 at 8:30 a.m. Eastern Time –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today announced top-line data of
EDG-7500 from the Phase 1 trial in healthy subjects and the
single-dose arm of the Phase 2 CIRRUS-HCM trial in patients with
obstructive HCM. EDG-7500 is a novel oral, selective, cardiac
sarcomere modulator, specifically designed to slow early
contraction velocity and address impaired cardiac relaxation
associated with HCM.
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CIRRUS-HCM Part A Single Dose
Observations with EDG-7500 in Obstructive HCM (Graphic: Business
Wire)
In the placebo-controlled Phase 1 single ascending dose (SAD)
trial (n=48), healthy subjects received single doses of EDG-7500,
ranging from 5 to 300 mg. In the multiple ascending dose (MAD)
portion of the trial (n=24), healthy subjects received 25 to 100 mg
once daily for 14 days. EDG-7500 was well tolerated in both the SAD
and MAD; there were no clinically meaningful changes or trends in
vital signs, clinical chemistry, hematology, or electrocardiograms.
There were no meaningful changes in LVEF for all SAD and MAD
subjects across a broad range of EDG-7500 exposures. In the MAD, a
half-life of approximately 30 hours was observed, and steady state
was achieved in approximately 4 days after the start of once-daily
dosing. Generally, dose proportional increases in exposure were
observed in both SAD and MAD.
In CIRRUS-HCM Part A, patients with obstructive HCM received a
single dose of 50, 100 or 200 mg of EDG-7500. A 67% mean reduction
in resting LVOT pressure gradient (LVOT-G) and a 55% mean reduction
in provokable (Valsalva) LVOT-G were observed in patients receiving
the 100 and 200 mg single doses. LVOT gradients less than 30 mmHg
at rest and less than 50 mmHg with Valsalva were observed in 60% of
patients receiving 100 or 200 mg of EDG-7500. Importantly, gradient
reduction was achieved without a meaningful change in LVEF.
Treatment with a single dose of EDG-7500 also led to a 64% mean
reduction in NT-proBNP, a key biomarker of heart failure, in the
200 mg cohort. This reduction highlights the potential of our
mechanism in the treatment of diseases of diastolic dysfunction,
including non-obstructive HCM.
Across the Phase 1 and CIRRUS-HCM trials, no subjects had a LVEF
reduction to below 50% across a broad range of EDG-7500
exposures.
“Based on the strength of clinical and preclinical data to-date,
we have initiated the 28-day part of CIRRUS-HCM in patients with
obstructive and non-obstructive HCM,” said Marc Semigran, M.D.,
Chief Development Officer, Edgewise Therapeutics. “Importantly, we
plan to continue the evaluation of tolerability, pharmacokinetics
and effects on LVOT-G, LVEF, biomarkers and measures of feel and
function in these patients.”
Anjali T. Owens, M.D., Medical Director, Center for Inherited
Cardiac Disease, Associate Professor of Medicine, University of
Pennsylvania and CIRRUS-HCM Investigator highlighted, “There
continues to be an unmet need for patients with obstructive and
non-obstructive HCM, and we are excited to be part of the ongoing
CIRRUS-HCM trial evaluating a novel treatment.”
Kevin Koch, Ph.D., President and Chief Executive Officer,
Edgewise Therapeutics added, “We believe our innovative approach,
where we have observed gradient relief without reductions in LVEF,
has the potential to be a valuable advancement in the treatment of
obstructive HCM. We expect to report initial 28-day data in the
first quarter of 2025.”
EDG-7500 Topline Data Webcast Event
Members of the Edgewise management team will hold a live webcast
on Thursday, September 19, 2024, at 8:30 am ET to discuss the
top-line data, and will be joined by leading cardiology expert,
Anjali T. Owens, M.D., Medical Director, Center for Inherited
Cardiac Disease, Associate Professor of Medicine, University of
Pennsylvania, who will share her perspective of EDG-7500 and HCM.
An accompanying slide presentation will also be available. To
register for the live webcast and replay, please visit the Edgewise
events page.
About 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. The Company is enrolling CIRRUS-HCM, a
three-part, multi-center, open-label trial, in approximately 55
patients with HCM at up to 20 clinical sites in the U.S. The
primary objective of Part A of the trial was to evaluate the safety
and tolerability of a single oral dose of EDG-7500. Other key
outcome measures included pharmacokinetics (PK), LVEF, and resting
and provocable LVOT-G. Parts B and C will evaluate multiple doses
of EDG-7500 over 28-days in patients with obstructive or
non-obstructive HCM.
To learn more about CIRRUS-HCM, visit clinicaltrials.gov,
NCT06347159 (Phase 2).
About Hypertrophic Cardiomyopathy
Hypertrophic Cardiomyopathy (HCM) is the most common form of
genetic heart disease, affecting approximately one in 500 people,
and is associated with reduced quality of life and an elevated risk
of heart failure, abnormal heart rhythms, and sudden cardiac death
(SCD). Individuals with HCM can become extremely limited in their
functional capacity and ability to perform the activities of daily
living. Commonly experienced symptoms include breathlessness,
irregular heartbeats, chest pain, tiredness, dizziness, or even
fainting. These symptoms are caused by excessive contraction and
thickening (hypertrophy) of the left ventricular wall of the heart.
Over time, the thickened muscle becomes stiff, making it difficult
for the heart to relax and fill with blood (diastolic dysfunction).
There are two major forms of HCM obstructive and non-obstructive.
The obstructive HCM pathology is observed in two thirds, while
non-obstructive HCM is present in one third of all individuals with
HCM. Despite advancements in treatment options for some HCM
patients, there remains a significant unmet need for additional
therapeutic approaches for patients.
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,
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 EDG-7500; statements regarding the
timing of reporting data (including the data from the CIRRUS-HCM
28-day trial); statements regarding Edgewise’s expectations
relating to its clinical trials (including the Phase 2 trial of
EDG-7500 in individuals with obstructive HCM, the CIRRUS-HCM 28-day
trial, and open-label extension trial of EDG-7500); statements
regarding the commencement of trials (including the open-label
extension trial of EDG-7500); and statements by Edgewise’s
president and chief executive officer and chief development
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240919115598/en/
Investors: Michael Carruthers, Chief Financial Officer
ir@edgewisetx.com
Media: Maureen Franco, VP Corporate Communications
media@edgewisetx.com
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