Industry: Biopharmaceuticals

About Company:

Edgewise Therapeutics is a clinical-stage biotech company focused on discovering and developing precision small-molecule therapies for skeletal and cardiac muscle disorders. Its lead candidate, sevasemten (EDG‑5506), is an oral fast-skeletal myosin inhibitor targeting Duchenne and Becker muscular dystrophy. Its cardiovascular pipeline includes EDG‑7500, a cardiac sarcomere modulator in Phase II trials for hypertrophic cardiomyopathy.

Company History:

• Incorporated in 2017, headquartered in Boulder, Colorado.
• Completed IPO on Nasdaq in March 2021 at $16/share.

Company Advantage Over Competitors:

• Targeted muscle‑focused drug discovery platform, with a deep emphasis on muscle biology and precision small molecules driving clinical candidates.
• First-in-class mechanism: sevasemten offers a novel approach for dystrophinopathies where no approved therapies exist for Becker muscular dystrophy.
• Strong balance sheet: recent $200M direct offering in April 2025 strengthened cash reserves to over $624M, giving runway to late‑stage development.

Risk Factors You May Want to Consider:

• Still unprofitable with substantial R&D outflows: reported net loss in Q1 2025 trailing 2024 net loss was ~$133.8M.
• Heavy reliance on future trials: success hinges on upcoming Phase II/III results in Becker, Duchenne (sevasemten) and HCM (EDG‑7500). Any trial delays or setbacks may significantly impact valuation.
• No commercial revenue yet: fully pre-commercial; value is based solely on pipeline potential and regulatory outcomes.

What Makes This Company Special or a Good Investment?

• Positive Phase II data delivered:
  • Sevasemten: CANYON trial in Becker showed meaningful functional stability. GRAND CANYON pivotal enrollment completed and data expected Q4 2026.
  • EDG‑7500: CIRRUS‑HCM Part A‑C showed rapid, clinically meaningful reductions in LVOT gradient and improved cardiac biomarkers and function. Part D data expected in H2 2025.
• Capital strength: Pro forma cash balance of ~$624M positions the company to finance key milestones, data readouts, and preparation for potential regulatory filings.
• Market opportunity in rare and underserved diseases: With Becker and Duchenne muscular dystrophy lacking approved treatments and HCM patients facing unmet needs, successful approvals could unlock first-mover commercial value.
• Upside potential supported by analyst and market sentiment: Director’s Talk Interviews calculates a ~175% upside to ~$39 fair value; median targets in the $39–$51 range represent significant upside from ~$14 current price.