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Vertex Pharmaceuticals, but for rare muscle and heart diseases.

BioMarin Pharmaceutical, but primarily focused on rare muscle and heart conditions.

Sarepta Therapeutics, but targeting a broader range of rare muscle and heart disorders.

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• EDG-5506 (sevasemten): An investigational drug candidate currently in clinical trials for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
• EDG-7509: An investigational drug candidate in development for a broad range of sarcopenic conditions.
• Discovery Programs: Early-stage research and development programs aimed at identifying novel drug candidates for other neuromuscular diseases.

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Edgewise Therapeutics (EWTX) is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of new treatments for serious muscle disorders. As a clinical-stage company, Edgewise Therapeutics does not currently have approved products available for commercial sale.

Therefore, EWTX does not have traditional "customers" who purchase its products. Instead, its revenue is primarily derived from collaboration agreements and grants, which fund its ongoing research and development efforts.

Based on its public filings, a major source of its collaboration revenue has come from the following company:

• Sanofi (ADR Symbol: SNY) - Sanofi is a global biopharmaceutical company with whom Edgewise Therapeutics has entered into collaboration agreements for the development of certain therapies.

Additionally, Edgewise Therapeutics has received grant revenue from government agencies, such as the National Institutes of Health (NIH), which supports its research initiatives. However, the NIH is a government agency and not a public company customer.

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• Lonza Group AG (LONN)

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Kevin Koch, Ph.D. President and Chief Executive Officer

Dr. Koch has served as President, Chief Executive Officer, and a member of the board of directors of Edgewise Therapeutics since 2017. He is also a Venture Partner with OrbiMed Advisors Private Equity since 2016. Dr. Koch co-founded Array BioPharma Inc. in 1998, where he served as President, Chief Scientific Officer, and a board member until 2013, building a research and development team that oversaw the invention of over 20 clinical development candidates. Prior to Array, he held senior positions at Biogen, Amgen Inc., and Pfizer Central Research.

R. Michael Carruthers Chief Financial Officer

Mr. Carruthers has served as Chief Financial Officer of Edgewise Therapeutics since September 2020. He brings over 20 years of experience as CFO for publicly traded biopharmaceutical companies, with extensive experience in corporate finance, strategic planning, IPOs, secondary offerings, and M&A transactions. Notably, he served as CFO for Array BioPharma Inc. from 1998 to 2015, which was acquired by Pfizer Inc. He also served as Interim President and CFO of Nivalis Therapeutics, a publicly traded company acquired by Alpine Immune Sciences in 2017. Mr. Carruthers has also consulted as CFO for several private and public companies.

Alan Russell, Ph.D. Co-Founder and Chief Scientific Officer

Dr. Russell is a Co-Founder and Chief Scientific Officer of Edgewise Therapeutics, a role he has held since the company's inception in 2017. He has been active in the discovery and development of medicines to improve skeletal muscle health for over 20 years. Previously, Dr. Russell served as VP and Head of the Muscle Metabolism Discovery Performance Unit at GlaxoSmithKline. He also worked at Cytokinetics Inc., where he was a co-inventor of Tirasemtiv and Reldesemtiv, direct muscle sensitizers in clinical trials for Amyotrophic Lateral Sclerosis (ALS).

Behrad Derakhshan, Ph.D. Chief Operating Officer

Dr. Derakhshan has served as Chief Operating Officer of Edgewise Therapeutics since 2025, having previously joined in 2020 as Chief Business Officer. In his roles, he leads efforts in strategic planning, business development, commercial planning, capital formation, investor relations, and corporate communications. Prior to Edgewise, Dr. Derakhshan was Chief Business Officer at VectivBio and Head of Business Development at Therachon until its acquisition by Pfizer Inc. in 2019 for $810 million. He also held positions in business development and strategic evaluation at Alexion and worked in management consulting focusing on rare disease drug development.

Robert Blaustein, M.D., Ph.D. Chief Development Officer

Dr. Blaustein has served as Chief Development Officer of Edgewise Therapeutics since January 2025. He brings significant experience in cardiovascular drug development, most recently serving as Associate Vice President and Atherosclerosis Section Head at Merck. During his over 15 years at Merck, Dr. Blaustein contributed to clinical programs across various cardiovascular targets and led clinical development in atherosclerosis and heart failure, including leading the vericiguat development team through the completion of the Phase 3 VICTORIA heart failure trial and subsequent global approval.

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The key risks to Edgewise Therapeutics (EWTX) largely stem from its position as a clinical-stage biopharmaceutical company without a commercialized product. The most significant risks are as follows:

1. Dependence on Clinical Trial Outcomes: Edgewise Therapeutics' future and valuation are heavily reliant on the successful outcome of its ongoing clinical trials for lead product candidates, such as sevasemten for muscular dystrophies and EDG-7500 for hypertrophic cardiomyopathy. Any setbacks, delays in topline data readouts, or failures to meet primary endpoints in these pivotal trials would have a catastrophic impact on the company's prospects and stock.
2. Regulatory Approval Uncertainty: Even with positive clinical trial results, the company faces significant regulatory risks. The FDA's approval process is inherently unpredictable, lengthy, and time-consuming. There is no guarantee that trial designs will satisfy regulatory agencies, or that marketing approval will be granted for its drug candidates. Delays or denial of regulatory approval would severely hinder the company's ability to commercialize its products.
3. Lack of Revenue and Need for Additional Capital: As a clinical-stage company, Edgewise Therapeutics has not generated product revenue to date and incurs substantial operating losses, primarily due to high research and development expenses. While the company currently has a robust cash position that provides a multi-year operational runway, it remains a cash-consuming entity. Future operations are contingent upon successful fundraising or the commercial viability of its candidates. The inability to raise sufficient additional capital as needed could force the downscaling or discontinuation of its development programs.

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The clear emerging threat for Edgewise Therapeutics (EWTX), particularly concerning its lead candidate EDG-5506 for Duchenne Muscular Dystrophy (DMD), is the rapid advancement and increasing market penetration of gene therapies for DMD. Companies like Sarepta Therapeutics have already received accelerated approval for their gene therapy, Elevidys (SRP-9001), for certain ambulatory DMD patients, and other gene therapies are in late-stage development by various biopharmaceutical companies. These gene therapies aim to deliver a functional dystrophin gene, representing a potentially disease-modifying or curative approach that fundamentally shifts the treatment paradigm by addressing the genetic root cause of the disease. Should these gene therapies prove highly effective, safe, and broadly accessible, they could significantly diminish the long-term market opportunity and competitive positioning for symptomatic or disease-modifying therapies like EDG-5506, which focus on muscle stabilization rather than gene replacement.

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Edgewise Therapeutics (EWTX) is focused on developing therapies for rare muscle disorders and serious cardiac conditions. The addressable markets for their main products are as follows:

Sevasemten (EDG-5506)

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to preserve and protect unstable muscle against contraction-induced injury in individuals living with rare skeletal muscle disorders, including Becker and Duchenne muscular dystrophies.

• Duchenne Muscular Dystrophy (DMD):
  • The global DMD treatment market was valued at over USD 4.5 billion in 2024 and is projected to reach USD 5.1 billion in 2025.
  • The global Duchenne muscular dystrophy drugs market size was estimated at USD 3.47 billion in 2023 and is projected to reach USD 9.91 billion by 2030, growing at a CAGR of 16.8% from 2024 to 2030.
  • The global Duchenne muscular dystrophy drugs market size was valued at USD 4.1 billion in 2024 and is projected to reach USD 19.46 billion by 2034, expanding at a CAGR of 16.85% from 2025 to 2034.
  • The Duchenne muscular dystrophy market across the seven major markets (7MM) is set to grow from USD 2.3 billion in 2023 to USD 5.2 billion in 2033.
  • The U.S. Duchenne muscular dystrophy drugs market size was exhibited at USD 1.32 billion in 2024 and is projected to be worth around USD 6.38 billion by 2034, growing at a CAGR of 17.06% from 2025 to 2034.
• Becker Muscular Dystrophy (BMD):
  • The global BMD market was valued at USD 880.6 million in 2024 and is also anticipated to grow.

EDG-7500

EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, intended to slow the excessive rate of early contraction and improve impaired cardiac relaxation associated with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) and other diseases of diastolic dysfunction.

• Hypertrophic Cardiomyopathy (HCM):
  • The U.S. market for HCM is estimated to be worth USD 600 million today, with potential to rise to over USD 5 billion over time, based on approximately 900,000 HCM patients in the U.S. alone.
  • The global Hypertrophic Cardiomyopathy Therapeutics Market is predicted to reach USD 1.5 billion by 2033.

EDG-15400

EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 clinical development.

• Heart Failure:
  • Heart failure currently affects more than 64 million people worldwide, and dilated cardiomyopathy is a leading cause.
  • While a specific market size for EDG-15400 as a heart failure therapeutic is not available, the global cardiomyopathy market, which includes dilated cardiomyopathy (a major cause of heart failure), reached US$ 1.90 billion in 2023 and is expected to reach US$ 3.02 billion by 2031, growing at a CAGR of 6.1% during the forecast period 2024-2031.

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Edgewise Therapeutics (EWTX) anticipates several key drivers of future revenue growth over the next two to three years, primarily stemming from the advancement and potential commercialization of its pipeline of novel therapeutics for muscular dystrophies and cardiac conditions.

1. Potential Launch of Sevasemten for Becker Muscular Dystrophy (BMD): The company is actively building the commercial infrastructure to support a potential U.S. launch of sevasemten in Becker muscular dystrophy, with topline data from the pivotal GRAND CANYON trial expected in the fourth quarter of 2026. This orally administered fast skeletal myosin inhibitor is designed to protect against contraction-induced muscle damage, and its MESA open-label extension trial continues to gather long-term safety and efficacy data.
2. Advancement of Sevasemten for Duchenne Muscular Dystrophy (DMD): Edgewise plans to engage with the FDA in 2026 to discuss the design for a Phase 3 study of sevasemten in Duchenne muscular dystrophy, with the intention of initiating a pivotal study that same year. Initial results from the FOX study, evaluating sevasemten in Duchenne participants previously treated with gene therapy, suggest its potential to reduce the rate of functional decline.
3. Progress of EDG-7500 for Hypertrophic Cardiomyopathy (HCM): The Phase 2 CIRRUS-HCM trial for EDG-7500 is ongoing, with a program update expected in the fourth quarter of 2025 and more comprehensive data anticipated in the first half of 2026. Edgewise is actively developing Phase 3 trial designs for this cardiac sarcomere modulator, targeting an initiation of a Phase 3 study in the first half of 2026, to address the global hypertrophic cardiomyopathy market.
4. Development of EDG-15400 for Heart Failure: Edgewise initiated a Phase 1 healthy adult trial for EDG-15400 in the third quarter of 2025, with topline results expected in the first half of 2026. This novel cardiac sarcomere modulator is being developed for heart failure with preserved ejection fraction (HFpEF), a prevalent form of heart failure.

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Share Issuance

• On April 2, 2025, Edgewise Therapeutics announced the pricing of an underwritten public offering of 9,935,419 shares of common stock at $20.13 per share, expecting to generate approximately $200 million in gross proceeds.
• The net proceeds from this April 2025 offering, after deducting underwriting discounts and commissions, were approximately $188 million.
• In February 2024, Edgewise Therapeutics completed an underwritten public offering, raising gross proceeds of $75 million.

Inbound Investments

• Edgewise Therapeutics raised approximately $200 million in gross proceeds from an underwritten public offering of common stock, priced on April 2, 2025, with net proceeds of about $188 million.
• The proceeds from the April 2025 offering are intended to support the potential commercial launch of sevasemten for Becker muscular dystrophy, advance Phase 3 trials for sevasemten in Duchenne muscular dystrophy, and finance Phase 3 trials of EDG-7500 in hypertrophic cardiomyopathy.
• In February 2024, the company secured $75 million in gross proceeds from an underwritten public offering.

Capital Expenditures

• Edgewise Therapeutics reported capital expenditures of -$203.00K in 2024, -$668.00K in 2023, and -$5.55M in 2022.
• The company's short-term material cash requirements as of June 30, 2025, primarily fund research and development expenses, and to a lesser extent, general and administrative expenses.
• Research and development expenses, reflecting increased investment in clinical programs, rose to $52.4 million in 2024 from $41.3 million in 2023.