Edgewise Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights
– 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 –
“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
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
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
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
Research and development (R&D) expenses were
General and Administrative (G&A) expenses were
Net loss and net loss per share for the fourth quarter of 2024 was
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
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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.