Edgewise Therapeutics, Inc. · NASDAQ:EWTX
Edgewise Therapeutics — American Academy of Neurology (AAN) 2024
14 slides from Edgewise Therapeutics's conference presentation, Apr 16, 2024
Slide 1: Effects of Sevasemten (EDG-5506) on Function and Biomarkers in Adults with Becker
Effects of Sevasemten (EDG-5506), a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy (Becker) Han Phan1, Sam Collins2, Alan Russell2, Ben Barthel2, Liz Thaler2, Nicole Kilburn2, Maria Mancini2, James MacDougall2, Joanne Donovan2 1Rare Disea
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward-looking statements that involve substantial risks and uncertainties of Edgewise Therapeutics, Inc. (“Edgewise” or the “Company”). All statements other than statements of historical facts contained in this presentation, including statement
Slide 3: Becker is a severe, underappreciated condition with major unmet medical need
Becker is a severe, underappreciated condition with major unmet medical need and no standard of care Contraction-induced injury leads to replacement of muscle with fat and fibrosis ● Becker can lead to relentlessly progressive loss of motor function ● Individuals with Becker lose mobility, function
Slide 4: In Becker, fast muscle fibers are disproportionately injured by everyday contractions
In Becker, fast muscle fibers are disproportionately injured by normal, everyday contractions Healthy muscle contraction Becker muscle contraction mechanical stress fiber breakdown Dystrophin connects contractile proteins to the Contraction-induced muscle injuries occur in the membrane and surroundi
Slide 5: Sevasemten targets fast myosin to protect dystrophic muscle in mdx mouse models
Sevasemten targets fast myosin to protect dystrophic muscle against contraction-induced injury in mdx mouse models Contracting at 100% without sevasemten Contracting at 85% with sevasemten In mdx mouse muscle, even a few contractions By minimally decreasing contraction while cause visible injury pre
Slide 6: Open-label study of sevasemten safety and pharmacokinetics in adults with Becker
An open-label, single-center study to assess sevasemten safety and pharmacokinetics in adults with Becker ● Primary objective: Safety and tolerability at 24 months ● Key inclusion criteria: Ambulatory males aged 18 to 55 years with a dystrophin mutation and a Becker phenotype, not on corticosteroids
Slide 7: Participants had significant functional impairment and decreased muscle mass at baseline
Participants had significant functional impairment & decreased muscle mass at baseline BECKER AGE NORMATIVE CHARACTERISTIC PARTICIPANTS (n=12) VALUES Age (SD) 33 (8) years – Adults with similar Functional Measures (median) baseline 10-meter walk/run 8.4 sec < 4 sec NSAA scores Rise from floor 6/12 c
Slide 8: Sevasemten well-tolerated at all doses
Sevasemten well-tolerated at all doses Treatment Emergent AE After One Year After Two Years (seen in >1 subject) ● No dose reductions or COVID-19 4 5 adjustments Fall* 3 4 ● No treatment Dizziness 4 4 discontinuations due to AEs Arthralgia 4 4 Nasopharyngitis 3 3 ● No SAE URI 3 3 ● Withdrawals: Proc
Slide 9: NSAA stabilized, diverging from natural history
NSAA stabilized, diverging from natural history at 24 months NSAA responses NSAA change 30 +3 -0.2 mean Change in NSAA score +2 change in NSAA with +1 NSAA (0-34) 20 sevasemten 0 at 24 months -1 10 -2 -2.4 natural -3 history 95% confidence interval for slope 0 -4 average change 0 4 8 12 16 20 24 0 6
Slide 10: Biomarkers of muscle damage show rapid and sustained decreases with sevasemten
Biomarkers of muscle damage show rapid and sustained decreases with sevasemten Fast skeletal muscle Creatine Kinase (CK) Myoglobin troponin I (TNNI2) Myoglobin (ng/mL) TNNI2 (ng/mL) CK (U/mL) Months of Treatment Months of Treatment Months of Treatment All participants Mean ± SEM Reference: Data on F
Slide 11: No significant change in hand grip strength or measures of endurance
No significant change at 24 months in hand grip strength or measures of endurance 100-MeterTimed 100-Meter Timed Test Test Velocity Velocity Maximum Grip Maximum Grip Strength Strength 1.5 Dose: Dose: 10 15 20 10 mg 10 15 20 10 mg 30 (m/sec) (kg) Strength (kg) Speed (m/sec) 1.0 20 Strength Speed 0.5
Slide 12: Outcomes of the ARCH study
Outcomes of the ARCH study Pivotal dose Safety Biomarkers Function identified Maximal Demonstration of biomarker rapid, sustained Stabilization of response at 10 and significant functional mg dose Well-tolerated at decreases in assessments with all doses multiple trends toward PK/PD supportive bioma
Slide 13: Ongoing pivotal study in Becker
Ongoing pivotal study in Becker An 18-month long trial to evaluate the effect of sevasemten on efficacy and safety in individuals living with Becker NOW Key inclusion criteria: ENROLLING: Male, ages 18-50 Anticipated to enroll 120 adult Mutation in DMD gene with Becker males phenotype diagnosed
Slide 14: Acknowledgements
Acknowledgements The authors thank the patients and their families who participated in this study! Thank you to the following site personnel: Emily Murray, Alvin Nguyen, Lucy Reid, Kirsten Malerba, Lynette Slovenksy, Darwin Nguyen, and Trang Vo 14
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