Edgewise Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights

Rhea-AI Summary

Edgewise Therapeutics (NASDAQ: EWTX) reported its Q4 and full year 2024 financial results, highlighting significant progress in its muscular dystrophy and cardiovascular portfolio. The company completed enrollment for the GRAND CANYON global pivotal study of sevasemten in Becker muscular dystrophy, with 175 adults across 51 sites in 12 countries.

Key financial metrics for Q4 2024 include:

• Cash position of $470.2 million
• R&D expenses of $36.4 million (up $4.2M from previous quarter)
• G&A expenses of $9.2 million
• Net loss of $39.7 million ($0.42 per share)

The company announced positive Phase 2 CANYON trial results and expects to report data from multiple ongoing trials in 2025, including: CIRRUS-HCM trial of EDG-7500 in Q1 2025, and Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy in H1 2025. GRAND CANYON study data is expected in Q4 2026.

Positive

• Strong cash position of $470.2M
• Completed enrollment of 175 adults in GRAND CANYON pivotal study
• Positive Phase 2 CANYON trial results in Becker muscular dystrophy
• 99% enrollment rate in MESA open-label extension trial

Negative

• Increased quarterly net loss to $39.7M from $34.1M in previous quarter
• R&D expenses increased by $4.2M quarter-over-quarter
• G&A expenses increased by $1.0M from previous quarter

Insights

Biotechnology Analyst

Edgewise Therapeutics has achieved significant clinical milestones across its muscular dystrophy and cardiovascular portfolios, positioning it for potential value inflection points in 2025. The completion of enrollment in the GRAND CANYON pivotal study for sevasemten in Becker muscular dystrophy (BMD) represents a critical advancement toward potential regulatory submission. With 175 adult patients enrolled across 51 sites globally, this reflects strong patient community engagement for a disease with no approved therapies.

The positive Phase 2 CANYON results announced in December 2024 strengthen sevasemten's clinical profile. As the company plans regulatory discussions in H1 2025 regarding marketing authorization filing strategies, this first-in-class fast skeletal myosin inhibitor could potentially address the fundamental pathology of contraction-induced muscle damage in BMD.

Their pipeline diversification is impressive, with multiple readouts expected in 2025: data from the Phase 2 CIRRUS-HCM trial for EDG-7500 in hypertrophic cardiomyopathy in Q1, and results from LYNX and FOX trials for sevasemten in Duchenne muscular dystrophy in H1. The 99% enrollment rate in the MESA open-label extension trial demonstrates remarkable patient retention and suggests confidence in sevasemten's safety profile.

The upcoming regulatory discussions and data readouts across multiple programs provide multiple shots on goal, potentially de-risking the company's development strategy while addressing serious unmet needs in both rare neuromuscular and cardiovascular diseases.

Financial Analyst

Edgewise Therapeutics maintains a strong financial foundation with $470.2 million in cash, cash equivalents, and marketable securities as of December 31, 2024. This substantial war chest provides runway to advance its diversified clinical pipeline through multiple upcoming value-creating milestones in 2025 and beyond.

The company's R&D expenses increased to $36.4 million in Q4 2024 from $32.2 million in the previous quarter, reflecting intensified investment in clinical development activities, particularly the CIRRUS-HCM trial and continued enrollment in the GRAND CANYON and MESA trials. Meanwhile, G&A expenses rose modestly to $9.2 million from $8.2 million.

The net loss widened to $39.7 million ($0.42 per share) in Q4 2024 compared to $34.1 million ($0.36 per share) in Q3 2024. This 16.4% increase in quarterly net loss reflects the company's strategic acceleration of clinical programs approaching critical inflection points.

With multiple potential catalysts in 2025—including data from the CIRRUS-HCM trial in Q1, results from the LYNX and FOX trials in H1, and regulatory discussions regarding sevasemten's path to market—Edgewise is strategically deploying its capital to maximize its chances of clinical and commercial success. The planned IND filing for a novel heart failure candidate and selection of a cardiometabolic drug candidate further demonstrates the company's commitment to building long-term value through pipeline expansion.

– Completed enrollment of the GRAND CANYON global pivotal study of sevasemten in Becker muscular dystrophy (Becker) –

– Announced positive top-line results from Phase 2 CANYON trial of sevasemten in Becker –

– On track to report data from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) in the first quarter of 2025 –

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

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2024 and recent business highlights.

“Closing out 2024 with positive Phase 2 CANYON results, completing enrollment in GRAND CANYON and advancing CIRRUS-HCM, we are well positioned in 2025 across our muscular dystrophy and cardiovascular portfolio,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise Therapeutics. “In the first half of this year, we look forward to announcing additional data from EDG-7500 in HCM as well as regulatory and clinical program updates on sevasemten in Becker and Duchenne.”

Recent Highlights

Muscular Dystrophy Program / Sevasemten

Sevasemten and Becker

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect muscle 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, and 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 that evaluated 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. Data from CANYON will be presented at the 2025 MDA Clinical and Scientific Conference. The Company plans to engage the U.S. FDA in the first half of 2025 to discuss 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 at 51 sites 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 work toward a therapeutic option. 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. To date, MESA has enrolled 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 first half 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 non-obstructive. Despite advancements in treatment options for some HCM patients, there remains a significant unmet need for additional therapeutic approaches for patients.

CIRRUS-HCM Phase 2 trial in adults with HCM: The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in individuals 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 topline data from Part A showing that treatment with EDG-7500 led to robust left ventricular outflow tract gradient reductions without meaningful changes in left ventricle ejection fraction. Parts B and C of CIRRUS-HCM are evaluating safety and efficacy of multiple doses of EDG-7500 over 28 days in individuals with either obstructive or non-obstructive HCM. The Company expects to report topline CIRRUS-HCM 28-day data in the first quarter of 2025. Further, the Company expects to report data from the 12-week CIRRUS-HCM trial in individuals with obstructive HCM and non-obstructive 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 select a proprietary cardiometabolic drug candidate based on preclinical proof-of-concept data.

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. Presentations were made at the International HCM Summit 8, American Heart Association Scientific Sessions, Global CVCT/Cardiovascular Clinical Trialists Forum, and the 29th International Annual Congress of the World Muscle Society. The team also connected with the community at the HCMA annual patient meeting and held a Becker community webinar hosted by Parent Project Muscular Dystrophy. The Company continues to sponsor and participate in numerous other clinician and patient-focused events.

Fourth Quarter Financial Results

Cash, cash equivalents and marketable securities were approximately $470.2 million as of December 31, 2024.

Research and development (R&D) expenses were $36.4 million for the fourth quarter of 2024, compared to $32.2 million for the immediately preceding quarter. The increase of $4.2 million was primarily driven by an additional $2.5 million higher personnel related costs, $2.0 million increase in clinical development activities related to the CIRRUS-HCM trial and continued enrollment in the GRAND CANYON and MESA trials, and a $0.3 million increase in professional fees and other research costs, offset by a $0.6 million decrease in manufacturing expenses.

General and Administrative (G&A) expenses were $9.2 million for the fourth quarter of 2024, compared to $8.2 million for the immediately preceding quarter. The increase of $1.0 million was primarily driven by increased personnel-related costs.

Net loss and net loss per share for the fourth quarter of 2024 was $39.7 million or $0.42 per share, compared to $34.1 million or $0.36 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 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 and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the presentation of data from the CANYON Phase 2 trial, 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;; Edgewise’s plans and timing for engaging the U.S. FDA to discuss marketing authorization filing strategies for sevasemten in Becker; the possibility of data from GRAND CANYON to support a marketing application; the timing of filing an investigational new drug application for a novel candidate and selecting a proprietary cardiometabolic drug candidate; statements regarding Edgewise’s pipeline of product candidates and programs; 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
		December 31, 2024			September 30, 2024
Operating expenses:
Research and development	$	36,370		$	32,222
General and administrative		9,170			8,210
Total operating expenses		45,540			40,432
Loss from operations		(45,540	)		(40,432	)
Interest income		5,878			6,303
Net loss	$	(39,662	)	$	(34,129	)
Net loss per share - basic and diluted	$	(0.42	)	$	(0.36	)
Weighted-average shares outstanding, basic and diluted		94,721,769			93,813,346
Edgewise Therapeutics, Inc.
Condensed Balance Sheet Data
(in thousands, unaudited)
		December 31,			December 31,
		2024			2023
Assets
Cash, cash equivalents and marketable securities	$	470,170		$	318,393
Other assets		16,647			21,642
Total assets	$	486,817		$	340,035
Liabilities and stockholders' equity
Liabilities		27,601			21,205
Stockholders' equity		459,216			318,830
Total liabilities and stockholders' equity	$	486,817		$	340,035

 

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Edgewise Contacts

Investors:

Michael Carruthers, Chief Financial Officer

ir@edgewisetx.com

Media:

Maureen Franco, VP Corporate Communications

media@edgewisetx.com

Source: Edgewise Therapeutics, Inc.

FAQ

What were the key financial results for Edgewise Therapeutics (EWTX) in Q4 2024?

EWTX reported Q4 2024 net loss of $39.7M ($0.42/share), R&D expenses of $36.4M, G&A expenses of $9.2M, and cash position of $470.2M.

When will EWTX report results from the GRAND CANYON pivotal study in Becker muscular dystrophy?

Edgewise expects to report data from the GRAND CANYON study in Q4 2026.

How many sites and countries are involved in EWTX's GRAND CANYON study?

The GRAND CANYON study is active at 51 sites across 12 countries, enrolling 175 adults.

What upcoming clinical trial results is EWTX expecting to report in 2025?

EWTX will report CIRRUS-HCM trial data in Q1 2025 and Phase 2 LYNX and FOX trials results in Duchenne muscular dystrophy in H1 2025.