Full Press Release Details
Edgewise Therapeutics
Reports Second Quarter 2024 Financial Results and Recent Business Highlights
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
to advance GRAND CANYON global pivotal cohort of sevasemten in adults with Becker -
Phase 2 LYNX and FOX trials of sevasemten in children with Duchenne -
Boulder, Colo., (August 8,
2024) - 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."
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.
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
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
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
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
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.
Cautionary Note Regarding Forward-Looking
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,"