Edgewise Therapeutics (EWTX) Piper Sandler 36th Annual Healthcare Conference summary

Key clinical trial updates

• CANYON trial data is expected imminently, with a quiet period starting after the conference and data readout planned before JPMorgan in December.

• CANYON is a 40-patient, placebo-controlled trial with a 3-to-1 randomization, focusing on a 10 mg oral dose over one year; primary endpoint is creatine kinase, with secondary endpoints including North Star, NSAD, 10-meter walk, MRI fat fraction, T2, and PROMIS-57.

• Statistical significance on the primary endpoint is the goal, with a one-point change in North Star considered clinically meaningful; a two-point change would likely achieve statistical significance and support approval.

• Biomarker expectations are 40% placebo-adjusted change in CK, 80% in TNNI2, and 40% in myoglobin, consistent with prior DUNE data.

• GRAND CANYON, a pivotal 120-patient study, is fully enrolled and will provide confirmatory safety and efficacy data.

Regulatory and market outlook

• Regulatory discussions have led to a pivotal strategy with two discrete placebo-controlled studies (CANYON and GRAND CANYON), both using North Star as the primary endpoint.

• CK is not considered a surrogate endpoint by regulators due to variability and disease progression effects, but a true drug effect on muscle damage biomarkers is being pursued.

• The U.S. Becker market is estimated at 5,000–6,000 diagnosed patients, with 70% ambulatory; peak market potential is $2–$3 billion.

• Adolescents are included in CANYON, and positive biomarker responses in this group could support a broad ambulatory label.

• Patients report feeling better on the drug, with anecdotal evidence of improved well-being and reduced pain.

HCM program and future plans

• The HCM MAD study is enrolling both obstructive and non-obstructive patients, with a fixed-dose approach expected to treat 70–80% of patients and dose adjustments for efficacy.

• The product profile aims for minimal echo monitoring, with no expected significant impact on ejection fraction; standard of care echoes will be used for dose adjustments.

• Single-dose data showed rapid patient-reported benefits, supporting compliance and physician confidence.

• MAD data targets a 60–70% change in gradient, deepening proBNP response, and demonstration of diastolic effects, which are key for strategic interest.

• Both obstructive and non-obstructive MAD data are planned for disclosure, with the timing and format to be decided based on data maturity.

• Strategic interest is high for both programs, with CANYON readout seen as a major inflection point and MAD data in 1Q expected to further de-risk the cardiac program.

• Second-generation molecule for heart failure is entering GLP tox studies, with IND planned for Q2 2025.

• Pivotal studies for 7500 are planned for the first half of 2026, with increased recruitment in CIRRUS-HCM and potential for parallel phase III trials in both patient populations.