Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Cure SMA 2020
11 slides from Scholar Rock's conference presentation, Jun 12, 2020
Slide 1: A Phase 2 Study to Evaluate SRK-015 in Later-Onset SMA (TOPAZ): An Introduction
A Phase 2 Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ): An Introduction Amy Place, PhD, MBA, MS, RD, CLT Global Medical Affairs Scholar Rock, Inc. Cure SMA June 12th, 2020 1 | CONFIDENTIAL AND PROPRIETARY
Slide 2: Disclaimer
Disclaimer • SRK-015 is an investigational product candidate that is currently being evaluated in a clinical trial • SRK-015 has not been approved by the U.S. Food and Drug Administration (FDA), the European Commission, or any other health authority, and the safety and effectiveness of this molecule
Slide 3: TOPAZ Study Schematic: Treatment Period
TOPAZ Study Schematic: Treatment Period www.clinicaltrials.gov 3 | CONFIDENTIAL AND PROPRIETARY NTC03921528
Slide 4: SRK-015 Phase 2 Trial (TOPAZ): Objectives and Design
SRK-015 Phase 2 Trial (TOPAZ): Objectives and Design Cohort 1 Cohort 2 Cohort 3 • N= 20; ages 5-21 • N= 20; ages ≥2 • N= 15; ages 5-21 • Double-blind, randomized (1:1) to 2 • Open-label, single-arm • Open-label, single-arm Design • 20 mg/kg SRK-015 IV Q4W • 20 mg/kg SRK-015 IV Q4W mg/kg or 20 mg/kg
Slide 5: TOPAZ Study Schematic: Extension Period
TOPAZ Study Schematic: Extension Period www.clinicaltrials.gov 5 | CONFIDENTIAL AND PROPRIETARY NTC03921528
Slide 6: Study Participant Baseline Demographics (1/2)
Study Participant Baseline Demographics (1/2) Age (Years) at Informed Consent by Cohort Age (Years) at Diagnosis by Cohort Cohort N Mean Std Min Med Max 1 23 12.6 4.53 7 13.0 21 2 15 11.7 3.94 8 10.0 19 3 20 4.0 1.23 2 4.0 6 Total 58 9.4 5.31 2 8.0 21 Scholar Rock. Data on File. March 2020 6 | CONFI
Slide 7: Study Participant Baseline Demographics (2/2)
Study Participant Baseline Demographics (2/2) Sex Distribution by Cohort Ethnicity Distribution by Cohort Race Distribution by Cohort Maintenance Dose of Nusinersen at Screening† †Excluded Cohort 1 patients who are not on Nusinersen 7 | CONFIDENTIAL AND PROPRIETARY Scholar Rock. Data on File. March
Slide 8: Study Participant Baseline Motor Function
Study Participant Baseline Motor Function Cohort 1: RHS* Score at Screening Cohorts 2 and 3: HFMSE** at Screening N Mean Std Min Med Max Cohort N Mean Std Min Med Max 15 22.3 8.98 12 19.0 37 RHS Score 23 49.0 11.00 25 49 63 2 19 25.0 9.58 12 22.0 44 3 Cohort 1: 6 Minute Walk at Screening‡ 34 23.8 9.
Slide 9: Preliminary TOPAZ Biomarker Data Provide First Demonstration of Target Engagement in SMA
Preliminary TOPAZ Biomarker Data Provide First Demonstration of Target Engagement in SMA 9 | CONFIDENTIAL AND PROPRIETARY
Slide 10: SRK-015 Phase 2 Trial (TOPAZ) Timelines
SRK-015 Phase 2 Trial (TOPAZ) Timelines • All 3 cohorts fully enrolled • Interim analysis: 6-month treatment period • Top-line results: 12-month treatment period • Patients eligible to continue treatment for an additional 12-month extension period • Results from TOPAZ trial may inform future studies
Slide 11: Acknowledgements
Acknowledgements • Phase 2 trial study investigators, site staff and participants • Medpace • Internal team Members: • Yung Chyung • Ryan Iarrobino • Doreen Barrett • Amy Place • Tiina Xu • Stephanie D’Eon • Mara Sadanowicz • Heather Faulds • Erin Treece • Shaun Cote • Ashish Kalra • Kimberly Long •