Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (Duchenne)

Rhea-AI Summary

Edgewise Therapeutics, Inc. (Nasdaq: EWTX) receives FDA Fast Track designation for EDG-5506, an investigational treatment for Duchenne and Becker muscular dystrophy. The drug aims to prevent muscle damage in dystrophinopathies, highlighting the urgent need for new therapeutic options for rare muscle disorders.

Positive

• FDA grants Fast Track designation for EDG-5506 by Edgewise Therapeutics, Inc.
• EDG-5506 targets Duchenne and Becker muscular dystrophy to prevent muscle damage.
• The drug has received Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track designation for different indications.
• FDA Fast Track Program expedites the review of new therapeutics for serious conditions with unmet medical needs.
• Ongoing trials for Duchenne include the Phase 2 LYNX and FOX trials, with data expected in the second quarter of 2024.

Negative

• None.

Insights

Biopharmaceutical Industry Analyst

The FDA's Fast Track designation for EDG-5506, a treatment for Duchenne muscular dystrophy, is a significant milestone for Edgewise Therapeutics. This status not only enhances the company's credibility but also potentially shortens the time frame for EDG-5506 to reach the market, assuming clinical success. The designation indicates a recognition of the treatment's potential to address a significant unmet medical need in the area of rare muscle diseases.

From an investor's perspective, this development could be a positive indicator of the company's pipeline's strength and the market potential of its lead candidate. It's important to note, however, that the clinical success of EDG-5506 is not guaranteed and the regulatory pathway, while expedited, still requires comprehensive data to support efficacy and safety.

Medical Research Analyst

Duchenne muscular dystrophy is a severe, progressive disease with limited treatment options, making the Fast Track designation of EDG-5506 particularly noteworthy. The drug's mechanism, aimed at preventing contraction-induced muscle damage, represents a novel approach in the treatment of dystrophinopathies. Given the serious nature of Duchenne and Becker muscular dystrophy, treatments that can improve muscle function and slow disease progression have the potential to significantly impact patients' quality of life.

It is also crucial to consider the implications of the ongoing LYNX and FOX trials. The data expected in the second quarter of 2024 will be pivotal in determining the drug's efficacy and safety profile. For stakeholders, the results of these trials will be a major determinant of the drug's commercial viability and the company's future prospects.

Healthcare Economist

The Fast Track designation of EDG-5506 has broader implications beyond the immediate business impact for Edgewise Therapeutics. If the treatment proves to be effective, it could lead to significant healthcare savings by potentially reducing the long-term care costs associated with Duchenne and Becker muscular dystrophy. These conditions often require extensive medical care, including surgeries, respiratory support and cardiac care, which are financially burdensome on the healthcare system and patients' families.

Moreover, the designation reflects the FDA's commitment to addressing critical healthcare gaps through expedited review processes. This can encourage more biopharmaceutical companies to invest in research for rare diseases, ultimately fostering innovation and possibly leading to more treatment options for these underserved patient populations.

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of Duchenne. EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker muscular dystrophy (Becker). The FDA previously granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne and Becker, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designation for the treatment of Becker.

“Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted Fast Track designation,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Our roster of regulatory designations granted to EDG-5506 highlights the urgent and critical need for new and better therapeutic options for people living with these rare and life-threatening muscle disorders.”

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. The designation is granted to qualifying therapeutics based on factors such as potential to offer meaningful impact on survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress to a more serious condition. Therapeutics that receive this designation receive important benefits that include more frequent meetings with the FDA to discuss development of the drug candidate and rolling review of the marketing application for obtaining FDA approval.

Ongoing trials in Duchenne include the Phase 2 placebo-controlled LYNX trial in children aged 4 to 9 years with Duchenne (NCT05540860), and the Phase 2 placebo-controlled FOX trial in children and adolescents with Duchenne who have been previously treated with gene therapy (NCT06100887). The Company expects to report LYNX 3-month controlled dose-ranging data in the second quarter of 2024, once the Phase 3 dose is identified.

About Duchenne Muscular Dystrophy

Duchenne is a severe, degenerative muscle disorder with a median life expectancy of around 30 years old. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Duchenne is the most common type of muscular dystrophy, and 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. Currently, there is no cure for Duchenne; early, active multidisciplinary care from neuromuscular specialists, cardiologists, physical therapists, and other specialists is critical for optimized disease management. Current therapeutic options for Duchenne are inadequate to prevent significant morbidity and mortality; novel therapies in development for Duchenne, including muscle targeted interventions, aim to positively impact disease trajectory.

About EDG-5506 for Duchenne and Becker Muscular Dystrophies

EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Duchenne and Becker. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle damage that leads to functional impairment, EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders. Its unique mechanism of action provides the potential to establish EDG-5506 as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. The Company is advancing EDG-5506 through the clinic including completing enrollment of a Phase 2 trial cohort, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker, which has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON, which is currently enrolling. In Duchenne, the Company is advancing its Phase 2 clinical trials, LYNX, assessing safety, pharmacokinetics and biomarkers of muscle damage, and FOX, which includes children and adolescents previously treated with gene therapy. For more information on Edgewise’s clinical trials www.edgewisetx.com/clinical-trials.

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 first-in-class therapeutics. EDG-5506 is an orally administered skeletal myosin inhibitor in clinical trials in patients with Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other disorders of cardiac diastolic dysfunction. 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 (formerly Twitter), Facebook, Instagram and Threads.

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 EDG-5506; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the 3-month data for the Phase 2 LYNX trial); statements about the timeline for the development of EDG-5506; 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 the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete clinical trials for, obtain approvals for and commercialize any of its product candidates; Edgewise’s ability to take advantage of potential benefits associated with designations granted by FDA and/or to maintain qualifications for applicable designations over time; the timing, progress and results of clinical trials for EDG-5506; Edgewise’s ability to enroll and maintain patients in clinical trials; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240213462472/en/

Investors:

Michael Carruthers, Chief Financial Officer

ir@edgewisetx.com



Media:

Maureen Franco, VP Corporate Communications

media@edgewisetx.com

Source: Edgewise Therapeutics

FAQ

What is the ticker symbol for Edgewise Therapeutics, Inc.?

The ticker symbol for Edgewise Therapeutics, Inc. is EWTX.

What is the FDA Fast Track designation granted for?

The FDA granted Fast Track designation for EDG-5506 for the treatment of Duchenne and Becker muscular dystrophy.

What are the ongoing trials for Duchenne mentioned in the press release?

The ongoing trials for Duchenne include the Phase 2 LYNX trial in children aged 4 to 9 years and the Phase 2 FOX trial in children and adolescents who have been previously treated with gene therapy.

When does the company expect to report LYNX 3-month controlled dose-ranging data?

The company expects to report LYNX 3-month controlled dose-ranging data in the second quarter of 2024.

What benefits do therapeutics receiving Fast Track designation from the FDA get?

Therapeutics receiving Fast Track designation get benefits such as more frequent meetings with the FDA and rolling review of the marketing application for obtaining FDA approval.