Edgewise Therapeutics, Inc. · NASDAQ:EWTX
Edgewise Therapeutics — PPMD Connect Conference 2022
15 slides from Edgewise Therapeutics's conference presentation, 2022
Slide 1: EDG-5506: A Novel Approach to Protect Muscle in Duchenne Muscular Dystrophy
EDG-5506: A Novel Approach to Protect Muscle in Duchenne Muscular Dystrophy Joanne M Donovan, MD PhD Chief Medical Officer Edgewise Therapeutics PPMD Connect Conference 2022 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements that involve substantial risks and interim, preliminary, topline or expected results; Edgewise’s ability to enroll patients in its uncertainties of Edgewise Therapeutics, Inc. (“Edgewise” or the “Company”). All statemen
Slide 3: The Edgewise Approach: Protect susceptible muscle fibers
The Edgewise Approach: Protect susceptible muscle fibers Some muscle fibers are more susceptible to damage due to the lack of functional dystrophin We’ve made an investigational therapy, EDG- 5506 that is designed to protect these susceptible muscle fibers from damage In diseased animal models, EDG-
Slide 4: Dystrophin Protects Muscles from Stress During Contraction Rather than Powering Contraction
Dystrophin Protects Muscles from Stress During Contraction Rather than Powering Contraction The role of dystrophin is to connect parallel muscle fibers to distribute force across muscle during contraction. 4
Slide 5: Mechanical Stress Damages Dystrophic Muscle
Mechanical Stress Damages Dystrophic Muscle When dystrophin is lacking, the mechanical stress that occurs during muscle contraction damages the muscle, leading to replacement with fat and fibrotic tissue and ultimately, loss of function. Unchecked damage leads to fibrosis and functional decline 5 5
Slide 6: Myosin Modulation: An Approach to Prevent Mechanical Stress when Dystrophin is Absent
Myosin Modulation: An Approach to Prevent Mechanical Stress when Dystrophin is Absent EDG-5506 was designed to protect muscle by modulating myosin, a contractile protein in the fast muscle fibers that are damaged early and to a greater extent in Duchenne and Becker. Decreased stress allows muscle to
Slide 7: EDG-5506 Protects Dystrophic Mouse Muscle
EDG-5506 Protects Dystrophic Mouse Muscle Dystrophic mouse muscle is damaged EDG-5506 protects dystrophic mouse during contraction muscle during contraction With EDG-5506, contractions don’t cause Contraction leads to visible changes… these changes Claflin, Su and Brooks. U Michigan 7
Slide 8: Injured Muscles Release Muscle Protein Biomarkers
Injured Muscles Release Muscle Protein Biomarkers Creatine Kinase Myoglobin Fast Troponin Many other muscle proteins Multiple muscle proteins enter the bloodstream and can be measured as biomarkers 8
Slide 9: EDG-5506 Phase 1 Trial in Unaffected Adults and Those with Becker Muscular Dystrophy
EDG-5506 Phase 1 Trial in Unaffected Adults and Those with Becker Muscular Dystrophy What do we know about side effects? ▪ When dosed for two weeks, EDG-5506 was well-tolerated How is EDG-5506 given? ▪ EDG-5506 can be taken as an oral tablet once a day that is absorbed well with or without food Does
Slide 10: Rapid Decrease in Biomarkers of Muscle Damage in Participants with Becker Treated with EDG-5506
Rapid Decrease in Biomarkers of Muscle Damage in Participants with Becker Muscular Dystrophy Treated with EDG-5506 Creatine Kinase Fast Skeletal Muscle Troponin I 8 On 2 Weeks of 10 Before Treatment On 2 Weeks of Before Treatment Treatment Treatment 8 Plasma Concentration Plasma Concentration 6 (Nor
Slide 11: BMD and DMD are Related Dystrophinopathies
BMD and DMD are Related Dystrophinopathies Spectrum of Severity Across Dystrophinopathies Functional Dystrophin at Birth Unaffected Levels Severe BMD DMD BMD and DMD represent a continuum of the same disease. Edgewise’s approach aims to treat across the disease spectrum, regardless of dystrophin mut
Slide 12: EDG-5506 is Being Developed for Becker and Duchenne Muscular Dystrophy
EDG-5506 is Being Developed for Becker and Duchenne Muscular Dystrophy • Taken orally, intended to preserve and improve function in Becker and Duchenne patients with any mutation • Goal to prevent damage to muscle by protecting the most susceptible fast muscle fibers • Potential to be used alone or
Slide 13: EDG-5506 Clinical Trials Ongoing and in Planning
EDG-5506 Clinical Trials Ongoing and in Planning • A 12-month open-label study is ongoing, including adults with Becker who were in the Phase 1 trial (NCT05160415) • A natural history study of those with Becker has begun (ages 8 to 65) (NCT05257473) • A study in ambulatory adolescent and adult males
Slide 14: Initial Phase 2 Study in Boys with Duchenne
Initial Phase 2 Study in Boys with Duchenne • Planned to start in second half of 2022 • Population: —4 to 9 years old, inclusive —Confirmed mutation in dystrophin gene with characteristic phenotype —Ambulatory —On stable dose of corticosteroids • Design: 3-month placebo-controlled, followed by 9-mon
Slide 15: Thank You!
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