Edgewise Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights

Rhea-AI Summary

Edgewise Therapeutics (NASDAQ: EWTX) reported Q1 2025 financial results and business updates. The company announced positive top-line results from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy. They strengthened their financial position through a $200M public offering, resulting in pro-forma cash balance exceeding $624M. Key developments include: completed enrollment in GRAND CANYON trial for Becker muscular dystrophy with 175 adults; positive data from CANYON trial; and advancing CIRRUS-HCM trial showing promising results. Q1 2025 financials showed R&D expenses of $36.8M and net loss of $40.8M ($0.43 per share). The company expects to report data from Phase 2 LYNX and FOX trials in Duchenne muscular dystrophy in Q2 2025 and Part D of CIRRUS-HCM trial in H2 2025.

Positive

• Raised $188M net proceeds from April 2025 public offering, strengthening balance sheet to $624M pro-forma cash
• Positive top-line results from CIRRUS-HCM trial showing robust reductions in LVOT-G without meaningful LVEF changes
• High enrollment retention with 99% of eligible participants continuing in MESA trial
• Completed enrollment of 175 adults in GRAND CANYON global pivotal trial for Becker muscular dystrophy
• Strong pipeline progress across multiple indications (Becker, Duchenne, HCM)

Negative

• Increased net loss to $40.8M in Q1 2025 from $39.7M in previous quarter
• R&D expenses increased to $36.8M from $36.4M in previous quarter
• Reported adverse events in CIRRUS-HCM trial including dizziness, upper respiratory tract infection and atrial fibrillation

Insights

Biotech Financial Analyst

Edgewise strengthens balance sheet to $624M with strong clinical progress in HCM and muscular dystrophy programs, extending runway for multiple late-stage trials.

Edgewise Therapeutics has substantially strengthened its financial foundation with its recent $188 million capital raise, bringing their pro-forma cash balance to an impressive $624.4 million. This significant cash position provides approximately 3+ years of runway at current spending levels, giving the company considerable flexibility to advance its diverse pipeline without immediate financing pressure.

The Q1 financial metrics show a quarterly burn rate of approximately $46 million ($36.8 million R&D expenses plus $9.2 million G&A), representing only a slight increase from the previous quarter. This disciplined spend approach while advancing multiple clinical programs demonstrates effective capital management. The $0.13 increase in quarterly EPS loss ($0.43 vs $0.42) is marginal considering the development stage and multiple ongoing trials.

What's particularly compelling is the comprehensive clinical strategy targeting both skeletal muscle disorders (Becker and Duchenne) and cardiac conditions (HCM). This diversified approach reduces pipeline risk while addressing serious unmet medical needs with substantial market potential. The successful public offering at $20.13 per share demonstrates strong investor confidence in this strategy.

The company has positioned itself for multiple potential value-creating catalysts in 2025, including Phase 2 data readouts for both sevasemten (LYNX and FOX trials in Duchenne) and EDG-7500 (Part D CIRRUS-HCM trial). This fortified balance sheet provides the resources needed for potential commercial preparations for sevasemten in Becker, advancement to Phase 3 in Duchenne, and progression of the HCM program - critical development milestones that could significantly enhance enterprise value.

Clinical Cardiologist

Impressive Phase 2 CIRRUS-HCM data shows EDG-7500 reduces LVOT gradient without harming ejection fraction, addressing unmet need in both obstructive and non-obstructive HCM.

The positive Phase 2 CIRRUS-HCM results for EDG-7500 represent a significant advancement in hypertrophic cardiomyopathy treatment. The data demonstrates clinically meaningful reductions in left ventricular outflow tract gradient (LVOT-G) - a critical parameter in HCM - that were both rapid in onset and sustained throughout the four-week treatment period. What makes these results particularly compelling is that these hemodynamic improvements were achieved without compromising cardiac function, as evidenced by the preservation of left ventricular ejection fraction (no participant fell below 50% LVEF).

The improvements in patient-reported outcomes using validated instruments (Kansas City Cardiomyopathy Questionnaire and NYHA functional class) alongside reductions in NT-proBNP suggest that the pharmacological action translates to meaningful clinical benefit. The positive trends in diastolic function measures are especially noteworthy, as impaired relaxation is a fundamental pathophysiological mechanism in HCM.

EDG-7500's potential to address both obstructive and non-obstructive HCM variants is particularly significant from a clinical perspective. While there are approved treatments for obstructive HCM, there remains a complete absence of approved therapies for non-obstructive HCM patients, who represent approximately 30% of the HCM population. The safety profile appears manageable, with predominantly mild-to-moderate adverse events (dizziness, URI, and atrial fibrillation).

For the muscular dystrophy programs, the company is making methodical progress with sevasemten. The successful enrollment of 175 adults in the GRAND CANYON study for Becker muscular dystrophy reflects strong patient interest and investigator support. The upcoming data from the LYNX and FOX trials in Duchenne will be pivotal for dose selection and patient stratification for the planned Phase 3 program.

– Announced positive top-line results from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) –

– Strengthened balance sheet with net proceeds of approximately $188 million from the April 2025 public offering; pro-forma cash balance exceeds $624 million –

–  On track to report data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy (Duchenne) in the second quarter of 2025 –

– Expect to report data from Part D of the Phase 2 CIRRUS-HCM trial in the second half of 2025 –

BOULDER, Colo., May 8, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2025 and recent business highlights.

"We're seeing strong progress across our skeletal and cardiac muscle programs," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "Most recently, we announced positive top-line results from CIRRUS-HCM and completed a $200 million offering enabling the execution of our near and long-term goals.  Our goals include commercial readiness for a potential U.S. approval of sevasemten in Becker, advancement of a Phase 3 trial in Duchenne, a Phase 3 program in HCM and our ongoing research and development activities. We anticipate several key milestones this year in both our sevasemten and EDG-7500 programs." 

Recent Highlights

Strengthened Financial Position
On April 3, 2025, the Company closed an underwritten registered direct offering of 9,935,419 shares of common stock at an offering price of $20.13 per share. The aggregate gross proceeds from the offering were $200 million. The net proceeds after deducting underwriting discounts and commissions but before offering expenses were $188 million.

Muscular Dystrophy Program / Sevasemten 
Sevasemten and Becker
Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Becker is a rare, genetic, life-shortening, debilitating and degenerative neuromuscular disorder.  Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. The disease predominantly affects males, with functional decline beginning at any age.  Once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual's life. Currently, there are no approved therapies on the market to treat Becker.

CANYON Phase 2 placebo-controlled trial in adults with Becker: In December 2024, the Company announced positive topline results from CANYON, the largest interventional Becker trial to date, which evaluated 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. Data from CANYON was presented at the 2025 MDA Clinical and Scientific Conference. The Company is engaging with the U.S. FDA to discuss potential marketing authorization filing strategies for sevasemten in Becker. 

GRAND CANYON, a global pivotal placebo-controlled cohort in Becker: In February 2025, the Company completed enrollment in GRAND CANYON, an expansion of the CANYON placebo-controlled trial.  The 18-month GRAND CANYON study is active in 12 countries, including the United States, United Kingdom, Netherlands, Denmark, Belgium, France, Spain, Germany, Italy, Israel, New Zealand and Australia.  GRAND CANYON enrolled 175 adults, reflective of the Becker community's enthusiasm to have access to a therapy with the potential to stabilize their debilitating decline in function and ability to perform everyday activities.  Data from the study are expected in Q4 2026.  To learn more, go to clinicaltrials.gov (NCT05291091).

MESA Phase 2 open label extension trial in adults with Becker:  The Company is advancing MESA, an open-label extension trial to assess the long-term effect of sevasemten in individuals with Becker.  MESA provides continued access to sevasemten to participants who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE.  MESA has continuing enrollment of 99% of eligible participants completing these prior trials.

Duchenne
Duchenne, a severe degenerative muscle disorder, is the most common type of muscular dystrophy with a median life expectancy of around 30 years. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. While there are approved therapies on the market aimed to treat the disease, there remains a high unmet need for additional therapies.

LYNX and FOX Phase 2 trials in boys with Duchenne: LYNX is an ongoing multi-center, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in children with Duchenne treated with oral, once-daily sevasemten. 

FOX is a Phase 2 ongoing placebo-controlled trial to assess the effect of sevasemten on safety, biomarkers of muscle damage and function in children and adolescents with Duchenne who have been previously treated with gene therapy.

Based on collective dose finding observations from both LYNX and FOX, the Company will form its Phase 3 strategy for sevasemten in Duchenne including patient and dose selection.  The Company expects to report data from LYNX and FOX as well as its future clinical trial plans in the second quarter of 2025.

For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887). 

Cardiovascular and Cardiometabolic Programs 
EDG-7500 and 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 and other diseases of diastolic dysfunction.  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. There are two major forms of HCM: obstructive and nonobstructive. Despite advancements in treatment options for some HCM patients, there remains a significant unmet need for additional therapeutic approaches for patients, including nonobstructive HCM patients, for which there are no approved treatment options.

CIRRUS-HCM Phase 2 trial in adults with HCM:  The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in participants with HCM at up to 20 clinical sites in the U.S. Part A of the trial was designed to evaluate the safety and tolerability of a single oral dose of EDG-7500 in obstructive HCM. In September 2024, the Company announced positive top-line data from Part A of the trial showing that a single oral dose of EDG-7500 in participants with obstructive HCM demonstrated robust reductions in left ventricular outflow tract gradient (LVOT-G) without meaningful changes in left ventricular ejection fraction (LVEF).

Part B and Part C of the trial were designed to evaluate the safety and efficacy of once-daily doses of EDG-7500 for four weeks in participants with obstructive HCM (Part B) and in participants with nonobstructive HCM (Part C).  In April 2025, the Company announced positive top-line data from Parts B and C.  EDG-7500 over four weeks demonstrated rapid and sustained clinically meaningful reductions in LVOT-G.  Further, four-week treatment with EDG-7500 demonstrated substantial improvements in measures of feel and function (Kansas City Cardiomyopathy Questionnaire and/or New York Heart Association functional class scores), reductions in NT-proBNP, a key biomarker of heart failure, and positive trends in measures of diastolic function.  EDG-7500 was generally well-tolerated; clinical activity was observed without meaningful changes in LVEF, including no participant with a value below 50% at any measurement. The most frequently reported adverse events were dizziness, upper respiratory tract infection and atrial fibrillation (AF); nearly all were considered mild to moderate in severity.   

The Company expects to report data from the 12-week (Part D) CIRRUS-HCM trial in participants with obstructive HCM and nonobstructive HCM in the second half of 2025. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159 (Phase 2).

Preclinical programs: During 2025, the Company expects to file an investigational new drug application for a novel candidate for the treatment of heart failure and continue to advance a novel cardiometabolic program.

Strengthened Engagement with the Scientific and Patient Communities
The Company continued its education and outreach with the muscular dystrophy and HCM medical and patient communities.  The team participated in the Muscular Dystrophy Association annual conference and the American College of Cardiology Scientific Sessions. The Company continues to sponsor and participate in numerous other clinician and patient-focused events.

First Quarter Financial Results
Cash, cash equivalents and marketable securities were approximately $436.4 million as of March 31, 2025. Our cash and cash equivalents at March 31, 2025 do not include the $188 million in net proceeds after deducting underwriting discounts and commissions but before offering expenses from our underwritten registered direct offering that closed on April 3, 2025. Combining the actual balance at March 31, 2025 with the net proceeds of the offering provides for a proforma balance of $624.4 million.

Research and development (R&D) expenses were $36.8 million for the first quarter of 2025, compared to $36.4 million for the preceding quarter. The increase of $0.4 million was primarily driven by an additional $1.0 million higher personnel related costs, $0.9 million increase in clinical development activities, offset by a $1.2 million decrease in manufacturing expenses and a $0.4 million decrease in professional fees and other research costs. 

General and Administrative (G&A) expenses were $9.2 million for the first quarter of 2025, compared to $9.2 million for the preceding quarter. There were no significant changes between the first quarter of 2025 and the immediately preceding quarter. 

Net loss and net loss per share for the first quarter of 2025 was $40.8 million or $0.43 per share, compared to $39.7 million or $0.42 per share for the 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 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 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, Edgewise's product candidates and programs, including sevasemten, EDG-7500 and its novel cardiometabolic program; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including the presentation of data from the GRAND CANYON trial, the LYNX trial, the FOX trial, and the CIRRUS-HCM trial); timing of reporting Edgewise's future clinical trial plans; statements regarding the advancement of Edgewise's research and development programs; statements regarding Edgewise's ability to execute its near and long-term goals; statements regarding Edgewise's commercial readiness; statements regarding the outcome of Edgewise's discussions with the U.S. FDA on marketing authorization filing strategies for sevasemten in Becker; the timing of filing an investigational new drug application for a novel candidate; statements regarding Edgewise's anticipated milestones; 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 sevasemten and EDG-7500; 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 of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; 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 patient population for our product candidates having a small patient population; 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
		March 31, 2025		December 31, 2024
Operating expenses:
Research and development	$	36,757	$	36,370
General and administrative		9,202		9,170
Total operating expenses		45,959		45,540
Loss from operations		(45,959)		(45,540)
Interest income		5,161		5,878
Net loss	$	(40,798)	$	(39,662)
Net loss per share - basic and diluted	$	(0.43)	$	(0.42)
Weighted-average shares outstanding, basic and diluted		95,130,053		94,721,769
Edgewise Therapeutics, Inc.
Condensed Balance Sheet Data
(in thousands, unaudited)
		March 31		December 31,
		2025		2024
Assets
Cash, cash equivalents and marketable securities	$	436,387	$	470,170
Other assets		18,004		16,647
Total assets	$	454,391	$	486,817
Liabilities and stockholders' equity
Liabilities		24,672		27,601
Stockholders' equity		429,719		459,216
Total liabilities and stockholders' equity	$	454,391	$	486,817

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SOURCE Edgewise Therapeutics

FAQ

What were the key financial results for Edgewise Therapeutics (EWTX) in Q1 2025?

In Q1 2025, Edgewise reported a net loss of $40.8M ($0.43 per share), R&D expenses of $36.8M, and G&A expenses of $9.2M. The company strengthened its balance sheet with $188M net proceeds from a public offering, resulting in a pro-forma cash balance exceeding $624M.

What were the results of EWTX's CIRRUS-HCM trial for EDG-7500?

The CIRRUS-HCM trial showed positive results with EDG-7500 demonstrating rapid and sustained clinically meaningful reductions in LVOT-G, improvements in feel and function measures, and reductions in NT-proBNP. The drug was generally well-tolerated without meaningful changes in LVEF.

What is the status of Edgewise Therapeutics' Becker muscular dystrophy trials?

Edgewise completed enrollment in the GRAND CANYON pivotal trial with 175 adults across 12 countries, with data expected in Q4 2026. The MESA open-label extension trial maintains 99% enrollment retention from previous trials, and the company is discussing potential marketing authorization strategies with the FDA.

When will EWTX report key clinical trial results in 2025?

Edgewise expects to report data from the Phase 2 LYNX and FOX trials in Duchenne muscular dystrophy in Q2 2025, and data from Part D of the CIRRUS-HCM trial in H2 2025.

What are the main drug candidates in Edgewise Therapeutics' pipeline?

The main drug candidates are sevasemten for Becker and Duchenne muscular dystrophy, and EDG-7500 for Hypertrophic Cardiomyopathy (HCM). The company is also advancing preclinical programs for heart failure and a novel cardiometabolic program.