– No relationship observed between EDG-7500 exposure and LVEF; no LVEF reductions below 50%
–
– Consistent and clinically meaningful improvements observed in echocardiogram parameters,
biomarkers, symptoms and functional status across obstructive and nonobstructive HCM –
– EDG-7500 administration continued to be generally well tolerated –
– Company to host webcast today at 8:30 a.m. Eastern Time –
BOULDER, Colo., June 16, 2026
/PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical
company, today announced positive top-line results from the 12-week Phase 2 Part D CIRRUS-HCM trial of
EDG-7500 in obstructive (oHCM) and nonobstructive (nHCM) HCM. EDG-7500 is a novel, oral, selective cardiac
sarcomere modulator designed to slow early contraction velocity and improve impaired cardiac relaxation in
symptomatic HCM without compromising systolic function. To date, in over 700 echocardiograms performed in
healthy adults and patients with HCM, there was no relationship observed between multiple measures of
systolic function and concentration of EDG-7500.
CIRRUS-HCM is a multi-part (A-D), open label trial of EDG-7500 in patients with oHCM and nHCM. Part D is a
12-week cohort designed to inform a Phase 3 trial. In oHCM, dosing was guided by left ventricular outflow
tract gradient (LVOT-G), while in nHCM, dosing was guided by N-terminal pro-B-type natriuretic peptide
(NT-proBNP), a key biomarker of heart failure. Patients in both groups received doses ranging from 25 mg to
150 mg. A total of 53 patients (20 with oHCM and 33 with nHCM) completed Part D 12-week study.
Results in Patients with oHCM
Patients with oHCM receiving EDG-7500 demonstrated broad and
clinically meaningful responses across key measures of hemodynamics, biomarkers, patient-reported health
status and functional status. Consistent with previous findings, significant reductions in LVOT-G were
observed at rest and post Valsalva, with 90% of patients demonstrating improvement in hemodynamic measures.
In a key measure of heart failure severity, 74% of patients achieved either normalization of NT-proBNP
levels (defined as <150 pg/mL) or a ≥50% reduction from baseline. A 24-point mean increase was reported
on the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS), and 70% of patients
exhibited at least one class improvement in New York Heart Association (NYHA) functional class measures. In
addition, administration with EDG-7500 led to a ~20% mean increase in early diastolic mitral annular
velocity (e' lateral), an important marker of diastolic function. In a preliminary high frame rate
sub-study, E/e' improved by a mean of 5.3 points, suggestive of markedly improved diastolic function.
Results in Patients with nHCM
Patients with nHCM receiving EDG-7500 demonstrated robust diastolic
and symptomatic improvement at 12 weeks. Similar to oHCM, an approximately 65% mean reduction in NT-proBNP
was observed in nHCM patients, with 88% of patients achieving normalization or a ≥50% reduction from
baseline. A 13-point mean increase in KCCQ-OSS was reported in nHCM patients, and 64% of patients exhibited
at least one class improvement in NYHA. In addition, administration with EDG-7500 led to a 37% mean increase
in e' lateral. In the same high frame rate sub-study, E/e' improved by a mean of 6.1 points.
Overall Safety
EDG-7500 was generally well tolerated in Part D patients (n=53) and there were no
new safety signals identified. Nearly all AEs were considered mild to moderate in severity. There were no
meaningful changes in left ventricular ejection fraction (LVEF) or reductions in LVEF to below 50%. There
were 2 (3.8%) new onset atrial fibrillation events; both deemed unrelated to study drug by the
investigator.
"From the outset, our goal was to identify a cardiovascular therapy with a profile that avoids the liability
of the CMI class, with EDG-7500 emerging from that effort," said Kevin Koch, Ph.D., President and Chief
Executive Officer, Edgewise Therapeutics. "These Phase 2 data mark an important milestone for Edgewise and
the HCM community, reinforcing EDG-7500's unique approach with the potential to address diastolic
dysfunction across HCM without meaningful impact on LVEF."
"The totality of the Phase 2 efficacy and tolerability data continues to support a differentiated profile for
EDG-7500 across HCM," said Matthew Martinez, M.D., a leading HCM expert and President of the HCM Society.
"What is especially encouraging is the consistency of symptom and functional improvement across dose levels
without evidence of systolic liability or heart failure risk. The EDG-7500 profile is particularly relevant
in nonobstructive HCM, where preserving systolic function while improving relaxation may offer a meaningful
new approach to addressing a significant unmet need."
Phase 3 Preparation
Data from CIRRUS-HCM support advancing EDG-7500 into Phase 3 clinical development, with program initiation
targeted for the fourth quarter of 2026.
FIGURE: EDG-7500 Continues to Demonstrate No Meaningful Reductions in LVEF, Supporting a Differentiated Therapeutic Approach in Both oHCM and nHCM
EDG-7500 Top-line Data Webcast Event
Members of the Edgewise management team will hold a live webcast on Tuesday, June 16, 2026, at 8:30 a.m. ET
to discuss the top-line data, and will be joined by leading cardiology experts, Anjali T. Owens, M.D.,
Medical Director, Center for Inherited Cardiac Disease, Associate Professor of Medicine, in the Perelman
School of Medicine at the University of Pennsylvania. An accompanying slide presentation will also be
available. To register for the live webcast and replay, please visit the Edgewise events page.
About CIRRUS-HCM
CIRRUS-HCM is a multi-part, open label trial of EDG-7500 in patients with obstructive and nonobstructive HCM
at over 20 clinical sites in the U.S. Part A of the trial evaluated the safety and tolerability of a
single oral dose of EDG-7500 in patients with obstructive HCM (oHCM). Parts B and C evaluated fixed doses of
EDG-7500 over 28 days in oHCM and nonobstructive HCM (nHCM), respectively; the results can be found here. Part D is a 12-week study with an open-label extension
including patients with oHCM and nHCM designed to explore dose response and optimization. To learn more
about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159.
About 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. 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 nonobstructive. Despite advancements in
treatment options for some patients with HCM, 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 first-in-class fast
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 symptomatic hypertrophic
cardiomyopathy, currently in Phase 2 clinical development. EDG-15400 is a novel cardiac sarcomere modulator
for the treatment of heart failure, currently in Phase 1 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 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 benefits and potential of, and expectations
regarding, EDG-7500; statements regarding the market opportunity for EDG-7500; statements regarding
Edgewise's expectations relating to its clinical trials, including timing of the initiation of the Phase 3
trials of EDG-7500; clinical outcomes from trials of EDG-7500, which may materially change as more patient
data become available; and statements by Edgewise's President and Chief Executive Officer and the President
of the HCM Society. 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: the potential for the results of the ongoing or any future clinical trial of
EDG-7500 to differ from the results of the Phase 2 CIRRUS-HCM trial; the risk of delays in initiating
the planned Phase 3 trials of EDG-7500; 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 EDG-7500; Edgewise's ability to develop and commercialize EDG-7500; 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 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 production of
drugs by Edgewise's third-party manufacturers; risks related to changes in methods of product candidate
manufacturing or formulation; risks related to not achieving adequate market acceptance; risks related to
the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and
inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies;
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; risks related to 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.
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