Edgewise Therapeutics, Inc. · NASDAQ:EWTX
Edgewise Therapeutics — PPMD Annual Conference 2023
22 slides from Edgewise Therapeutics's conference presentation, June 2023
Slide 1: EDG-5506: A Novel Approach Designed to Protect Muscle in Becker Muscular Dystrophy
EDG-5506: A Novel Approach Designed to Protect Muscle in Becker Muscular Dystrophy Joanne Donovan, MD, PhD Chief Medical Officer PPMD Annual Conference June 2023 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: Edgewise aims to improve lives of individuals living with rare muscle disorders
Edgewise aims to improve lives of individuals living with rare muscle disorders Founded in 2017, Edgewise is focused on developing novel therapies for muscular dystrophy, including Becker and Duchenne. We believe our investigational compound, a daily oral pill, could be used alone or in combination
Slide 4: The Becker Muscular Dystrophy Landscape
The Becker Muscular Dystrophy Landscape • Becker muscular dystrophy (BMD) is a serious dystrophinopathy with a variable clinical course. However, once declining, individuals irreversibly lose muscle and their disease progresses • Living with Becker poses multiple challenges. To develop treatments it
Slide 5: EDG-5506 Clinical Trials Ongoing
EDG-5506 Clinical Trials Ongoing • A 24-month open-label study is ongoing, including adults with Becker who were in the Phase 1 trial (NCT05160415) • A study in ambulatory adolescent and adult males with Becker is enrolling (ages 12+) (NCT05291091) • An initial study in ambulatory boys with Duchenne
Slide 6: 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, regardless of mutation In disea
Slide 7: How Does Dystrophic Muscle Break Down?
How Does Dystrophic Muscle Break Down? With dystrophin – fibers support each other Rest Contraction Without dystrophin – contraction causes injury Dystrophin connects contractile proteins to the membrane and surrounding matrix of fibers 7
Slide 8: A New Strategy to Rebalance Dystrophic Muscle
A New Strategy to Rebalance Dystrophic Muscle Protecting muscle is predicted to preserve function 8
Slide 9: 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 these Contraction leads to visible changes… changes Claflin, Su and Brooks. U Michigan 9
Slide 10: Injured Muscles Release Muscle Protein Biomarkers
Injured Muscles Release Muscle Protein Biomarkers Creatine Kinase Myoglobin Fast Skeletal Muscle Troponin Many other muscle proteins Multiple muscle proteins enter the bloodstream and can be measured, which we term biomarkers of muscle damage 10
Slide 11: BMD and Duchenne Muscular Dystrophy (DMD) are Related Dystrophinopathies
BMD and Duchenne Muscular Dystrophy (DMD) are Related Dystrophinopathies Spectrum Across Dystrophinopathies Functional Dystrophin No Dystrophin Unaffected Levels BMD DMD Severe Intensity of Muscle Damage Edgewise’s approach aims to prevent muscle breakdown due to lack of functional dystrophin, regar
Slide 12: North Star Ambulatory Assessment (NSAA)
North Star Ambulatory Assessment (NSAA) Snapshot of NSAA skills assessed • The NSAA includes 17 different activities that measure function in those who are able to walk • Each activity is scored on whether the activity can be complete the usual way, with an adjustment because of weakness, or not at
Slide 13: BMD: Once Muscle Loss Occurs, Function Progressively Declines
BMD: Once Muscle Loss Occurs, Function Progressively Declines Age doesn’t predict function, but function predicts future deterioration ~ % 3 0 Individuals with NSAA near maximal have low degree of replacement of the muscle by fat and fibrosis, otherwise known as fat infiltration Fat Infiltration Fun
Slide 14: 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 15: Biomarkers of Muscle Damage in Becker Participants Decreased after 12 Months of Treatment
Biomarkers of Muscle Damage in Becker Participants Decreased after 12 Months of Treatment with EDG-5506 Creatine Kinase Fast Skeletal Muscle Troponin I (TNNI2) 2000 50 40 TNNI2 Concentration (ng/ml) 1500 CK Activity (U/L) 30 1000 -37%** 20 500 10 -79%*** 0 0 Baseline 12 months Baseline 12 Months NCT
Slide 16: NSAA Shows Trend toward Improvement Relative to Natural History
North Star Ambulatory Assessment (NSAA) Shows Trend toward Improvement Relative to Natural History NSAA Change 10 mg 15 mg 20 mg 3 2 Change in NSAA 1 0 -1 Natural History BMD natural history average decline Dashed lines show 95% confidence intervals on the linear regression -2 0 2 4 6 8 10 12 EDG-55
Slide 17: CANYON: Phase 2 Trial in Becker
CANYON: Phase 2 Trial in Becker A 14-month long trial to evaluate the effect of EDG-5506 on safety, tolerability, biomarkers of muscle damage and functional measures in individuals living with BMD Population Assessments and Visits • Male, ages 12-50 • Approximately 10 site visits required • Genetic
Slide 18: Sites across the US will enroll for the CANYON trial
Sites across the US will enroll for the CANYON trial Sites actively recruiting • Little Rock, Arkansas • Sacramento, California • Aurora, Colorado • Iowa City, Iowa • Kansas City, Kansas • Baltimore, Maryland • Worcester, Massachusetts • St. Louis, Missouri • Cincinnati, Ohio • Columbus, Ohio • Dall
Slide 19: Edgewise is Committed to Reducing Trial Burden for Participants
Edgewise is Committed to Reducing Trial Burden for Participants Engages with the community to understand trial burden and barriers to support the design of patient-centric trials Provides support services for participants, i.e., travel liaison, pre-paid expenses, and resources to ease participation
Slide 20: 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 muscle fibers • Potential to be used alone or in co
Slide 21: Key takeaways
Key takeaways • Becker muscular dystrophy (BMD) is a serious dystrophinopathy with a variable clinical course. However, once declining, individuals irreversibly lose muscle and their disease progresses • Living with Becker poses multiple challenges. To develop treatments it is critical to listen to
Slide 22: THANK YOU!
THANK YOU! For questions or comments please email us! studies@edgewisetx.com Visit us at https://edgewisetx.com/ https://www.facebook.com/Edgewisetx/ https://twitter.com/EdgewiseTx 22