Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Corporate Update (October 2020)
26 slides from Scholar Rock's corporate update, Oct 27, 2020
Slide 1: TOPAZ Interim Analysis: SRK-015 Demonstrates Clinical Proof-of-Concept in SMA
TOPAZ Interim Analysis: SRK-015 Demonstrates Clinical Proof-of-Concept in Spinal Muscular Atrophy October 27, 2020
Slide 2: Disclaimers
Disclaimers Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock, Inc. (“Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding its strategy, its product candidate selection and development timing, including timi
Slide 3: Agenda
Agenda 3 Opening Remarks Trial Design and Baseline Characteristics 6-month Interim Efficacy and Safety Results Summary and Next Steps Tony Kingsley, President & CEO Yung Chyung M.D., Chief Medical Officer Yung Chyung M.D., Chief Medical Officer Tony Kingsley, President & CEO Questions and Answers To
Slide 4: SRK-015 Has Potential to Pioneer a New Treatment Era in SMA
SRK-015 Has Potential to Pioneer a New Treatment Era to Improve Motor Function in Patients with SMA 4 SMN = survival motor neuron. *Also referred to as SMN correctors. **SRK-015 is an investigational therapyunder development. SMN Upregulator Therapies* + Muscle-Directed Therapy (SRK-015) Could Poten
Slide 5: SRK-015: Highly Selective Inhibitor of Activation of Pro and Latent Myostatin
Fully human monoclonal antibody (mAb)Half-life of ~23-33 daysAvoids related growth factors (e.g. GDF11, BMP9, Activin A)Rare Pediatric Disease Designation for SMA granted by FDA Orphan Drug Designation for SMA granted by the FDAOrphan Medicinal Product Designation for SMA granted by the European Com
Slide 6: Bringing a Revolutionary Approach to Highly Sought-After Growth Factors
6 Bringing a Revolutionary Approach to Highly Sought-After Growth Factors Implicated in Devastating Diseases Scholar Rock’s Target Growth Factor Precursor (Latent Form) Scholar Rock’s R&D PlatformTransform Medical Practice Pursue important targets with well-validated biology but are difficult to dru
Slide 7: TOPAZ Interim Analysis Results Demonstrate Proof-of-Concept
7 Mean improvements from baseline in HFMSE/RHS observed in each of the 3 cohorts67% of total patients achieved ≥1-point improvement in Hammersmith scoresSubstantial proportion of patients in each cohort attained ≥3-point improvement in HFMSE/RHSHigh bar and uncommon to observe in any given patient35
Slide 8: Phase 2 Trial Design and Baseline Characteristics
Phase 2 Trial Design and Baseline Characteristics Yung Chyung, M.D.Chief Medical Officer
Slide 9: Next Era of SMA Treatment: Muscle-Directed Therapy
9 Next Era of SMA Treatment: Muscle-Directed Therapy Overall Prevalence of 30,000-35,000 in U.S. and Europe Type 114% Type 251% Type 335% HFMSE = Hammersmith Functional Motor Scale Expanded Relative Prevalence Among Patients Living With SMA Unmet need would be served by a therapy that:Improves motor
Slide 10: SRK-015 Phase 2 Trial Design
SRK-015 Phase 2 Trial Design Design Patients Primary Objectives HFMSE=Hammersmith Functional Motor Scale Expanded; RHS=Revised Hammersmith ScaleData on file. Scholar Rock, Inc. Cambridge, MA Evaluate potential of SRK-015 in improving motor function 10 Ambulatory Patients(Revised Hammersmith Scale) N
Slide 11: Baseline Characteristics
Baseline Characteristics 11 Ambulatory Patients Non-Ambulatory Patients Cohort 1 Cohort 2 Cohort 3 20 mg/kgpooled 20 mg/kgmonotherapy 20 mg/kg +nusinersen 20 mg/kg +nusinersen 2 mg/kg +nusinersen 20 mg/kg +nusinersen N 23 11 12 15 10 10 Mean age (min, max) 12.6 (7, 21) 12.1 (7, 19) 13.1 (7, 21) 11.7
Slide 12: Key Strengths of TOPAZ Trial Design and Conduct
12 Key Strengths of TOPAZ Trial Design and Conduct Large and diverse group of study sites for a rare disease trialPatients enrolled across 16 study sites in the U.S. and EuropePatient enrollment was not skewed to any one site for any cohort or across the studyPrimary efficacy endpoints are well-vali
Slide 13: Six-Month Interim Analysis Results
13 Six-Month Interim Analysis Results Yung Chyung, M.D.Chief Medical Officer
Slide 14: TOPAZ Interim Analysis Results Demonstrate Proof-of-Concept
14 Mean improvements from baseline in HFMSE/RHS observed in each of the 3 cohorts67% of total patients achieved ≥1-point improvement in Hammersmith scoresSubstantial proportion of patients in each cohort attained ≥3-point improvement in HFMSE/RHSHigh bar and uncommon to observe in any given patient3
Slide 15: Cohort 1: Improvement in RHS with Monotherapy and Add-on to Nusinersen
Cohort 1: Improvement in RHS Observed with Both SRK-015 Monotherapy and as Add-on to Background Nusinersen 15 Individual RHS responses Ambulatory Type 3 SMA SRK-015 (20 mg/kg)pooled (n=23) SRK-015 (20 mg/kg)monotherapy (n=11) SRK-015 (20 mg/kg) +nusinersen (n=12) Mean change from baseline in RHS (95
Slide 16: Cohort 2: Consistent Improvements in HFMSE in Type 2 and Non-Ambulatory Type 3
Cohort 2: Meaningful and Consistent Improvements in HFMSE Observed in Type 2 and Non-Ambulatory Type 3 SMA Individual HFMSE responses Type 2 and Non-Ambulatory Type 3 SMA SRK-015 (20 mg/kg) + nusinersen (n=14) Mean change from baseline in HFMSE (95% CI) 1.4 (0.1, 2.7) # (%) patients achieving ≥1-pt
Slide 17: Cohort 3: High Dose Showed Greater HFMSE Improvements Than Low Dose
Cohort 3: SRK-015 High Dose Showed Substantially Greater Improvements in HFMSE scores Than Low Dose 17 Individual HFMSE responses Type 2 SMA SRK-015 low dose (2 mg/kg) +nusinersen (n=9) SRK-015 high dose (20 mg/kg) +nusinersen (n=9) Mean change from baseline in HFMSE (95% CI) 2.4 (-0.9, 5.8) 5.6 (2.
Slide 18: Cohort 3: Time Course Data Supportive of Dose Response
Cohort 3: Time Course Data Supportive of Dose Response 18 Mean (±SEM) change from baseline in HFMSE scores Greater improvements in HFMSE scores observed with high dose (20 mg/kg) across all timepoints High-dose arm outperformed low-dose arm numerically across the timepointsPlateau in improvement app
Slide 19: Pharmacokinetic and Pharmacodynamic Data Supportive of Dose Response
Well-behaved PK profile consistent with that commonly observed with monoclonal antibodiesDrug exposure was dose proportional Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Dose Response Pharmacokinetics* (PK) Pharmacodynamics (PD) Target engagement by SRK-015 was conf
Slide 20: No Safety Signals Identified from Interim Analysis
No Safety Signals Identified from Interim Analysis 20 Treatment-emergent adverse events (TEAEs) SRK-015 2 mg/kg dose (n=10) SRK-015 20 mg/kg dose (n=48) Total(n=58) Any TEAE 9 (90%) 40 (83.3%) 49 (84.5%) Any Serious TEAE 0 (0.0%) 1 (2.1%) 1 (1.7%) Any TEAE leading to study drug discontinuation 0 (0.
Slide 21: TOPAZ Interim Analysis Results Demonstrate Proof-of-Concept
21 TOPAZ Interim Analysis Results Demonstrate Proof-of-Concept Mean improvements in Hammersmith scores observed across all 3 cohortsMost patients experienced an improvement (≥1-pt increase in Hammersmith scores)Cohort 1: 52% (pooled)Cohort 2: 71% Cohort 3: 100% high dose, 67% low dosePotential for d
Slide 22: Summary and Next Steps
22 Summary and Next StepsTony KingsleyPresident & CEO
Slide 23: Beyond the TOPAZ Interim Analysis
23 Top-line data for 12-month treatment period expected 2Q21Longer-term evaluation of efficacy for potential durability of clinical effect and continued motor function gainsLonger-term safety, PK, PD, and anti-drug antibody39 of 39 patients who have completed 12-month study have opted into the exten
Slide 24: Unlocking the Potential of the Scholar Rock R&D Engine
24 Unlocking the Potential of the Scholar Rock R&D Engine Scholar Rock seeks to unlock the therapeutic potential of modulating growth factor biology Targeting the latent forms of growth factors Opportunities to Further Investigate Therapeutic Potential Broader exploration of SMA types, age range, an
Slide 25: Differentiated Pipeline with a Series of Anticipated Milestones
DRAGON Part B Initiation Gilead Fibrosis-focused TGFβ Collaboration DRAGON Clinical Response and Safety Data Across Multiple Tumor TypesPart A2 Combo Efficacy and Safety DataPart B – MelanomaPart B – NSCLCPart B – Urothelial CarcinomaPart B – Other Solid Tumor Types SRK-015SMA SRK-181 Immuno-Oncolog
Slide 26: S$ ScholarRock.
S$ ScholarRock.
Scholar Rock Holding — Corporate Update (October 2020) on 6ix