Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Technical Presentation (April 2021)
33 slides from Scholar Rock's technical presentation, Apr 6, 2021
Slide 1: TOPAZ Phase 2 Trial Top-Line Results
TOPAZ Phase 2 Trial Top-Line Results April 6, 2021 Improvements in Motor Function with Apitegromab for Patients with Spinal Muscular Atrophy (SMA)
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 Next Steps for Apitegromab Program Tony Kingsley, President & CEO Tony Kingsley, President & CEOTed Myles, Chief Financial Officer Questions and Answers Trial Design and Baseline Characteristics Top-line Safety and Efficacy Results Yung Chyung M.D., Chief Medical Officer
Slide 4: Bringing a Revolutionary Approach to Highly Sought-After Growth Factors
4 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 5: Apitegromab Offers Potential to Pioneer a New Treatment Era in SMA
Apitegromab Offers Potential to Pioneer a New Treatment Era to Improve Motor Function in Patients with SMA 5 SMN = survival motor neuron. *Also referred to as SMN correctors. **Apitegromab (SRK-015) is an investigational product candidate under development. SMN Upregulator Therapies* + Muscle-Direct
Slide 6: TOPAZ 12-Month Data Further Support Potential of Apitegromab
6 TOPAZ 12-Month Data Further Support the Potential of Apitegromab in Patients with Type 2 and Type 3 SMA •Cohort 2: Mean change in HFMSE score from baseline was updated to +1.1-points (from +1.4-points). Proportion of patients with ≥3-point increases was updated to 2/14 (from 3/14) and updated to 1
Slide 7: Phase 2 Trial Design and 12-Month Top-Line Results
Phase 2 Trial Design and 12-Month Top-Line Results Yung Chyung, M.D. Chief Medical Officer
Slide 8: Apitegromab Phase 2 Trial Design
Apitegromab 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 apitegromab in improving motor function in patients with Type 2 and Type 3 SMA 8 Am
Slide 9: Considerations in the Conduct and Design of TOPAZ Proof-of-Concept Study
Considerations in the Conduct and Design of TOPAZ Proof-of-Concept Study 9 Main focus of TOPAZ was to assess the potential additive therapeutic benefit of apitegromab on top of background SMN upregulator therapy.*While the protocol allowed the use of any approved SMN upregulator as background therap
Slide 10: Baseline Characteristics
Baseline Characteristics 10 Ambulatory Patients Non-Ambulatory Patients Cohort 1 Cohort 2 Cohort 3 20 mg/kgmonotherapy 20 mg/kg +nusinersen Pooled 20 mg/kg +nusinersen 20 mg/kg +nusinersen 2 mg/kg +nusinersen Pooled N 11 12 23 15 10 10 20 Mean age (min, max) 12.1 (7, 19) 13.1 (7, 21) 12.6 (7, 21) 11
Slide 11: Safety Results from TOPAZ 12-Month Top-Line Analysis
Safety Results from TOPAZ 12-Month Top-Line Analysis Support Evaluation of Apitegromab in Phase 3 Trial 11 Treatment-emergent adverse events (TEAEs) Apitegromab 2 mg/kg (n=10) Apitegromab 20 mg/kg (n=48) Total(n=58) Any TEAE 9 (90.0%) 44 (91.7%) 53 (91.4%) Any Serious TEAE 1 (10.0%) 4 (8.3%) 5 (8.6%
Slide 12: Serious and Severe Treatment-Emergent Adverse Events
Serious and Severe Treatment-Emergent Adverse Events (TEAEs) 12 Treatment-emergent adverse events (TEAEs) are defined as AEs that start after the first dose of study drug or start prior to the administration of study drug and worsen in severity/grade or relationship to investigational medication aft
Slide 13: TOPAZ 12-Month Top-line Results Demonstrate Potential of Apitegromab
13 Cohort 1 data suggest potential clinical effect in certain patients in this patient populationMean decline in RHS from baselineMajority (57%) maintained or improved in RHS (≥0-point change from baseline) and 22% achieved ≥3-point increaseCohort 2 observed improvement of motor function from baseli
Slide 14: Cohort 1
Cohort 1 S$ ScholarRock.
Slide 15: Background Insights into Ambulatory Type 3 SMA Patients
Background Insights into Ambulatory Type 3 SMA Patients Source: Coratti, et, al. Annals of Neurology (2020; 88:1109-1117) DOI: 10.1002/ana.25900This third-party information is provided for background only and is not intended to convey or imply a comparison to the TOPAZ clinical trial results. 15 Cor
Slide 16: Cohort 1: Mean Decline in RHS at 12-Months but Majority Maintained or Improved
Cohort 1: Mean Decline in RHS at 12-Months but Majority of Patients Maintained or Improved in RHS Score from Baseline 16 Individual RHS responses Ambulatory Type 3 SMA (Intent-to-Treat Population) Apitegromab (20 mg/kg)monotherapy (n=11) Apitegromab (20 mg/kg) + nusinersen (n=12) Pooled (n=23) Mean
Slide 17: Cohort 2
Cohort 2 S$ ScholarRock.
Slide 18: Nusinersen CHERISH Trial in Later-Onset SMA
18 Nusinersen CHERISH Trial in Later-Onset SMA (15-month treatment period) *Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-635.**Mercuri E. et al. Patterns of disease progression in type 2 and 3 SMA: implications for clinical trial
Slide 19: Cohort 2: Improvement in Mean HFMSE at 12-Months
Cohort 2: Improvement in Mean HFMSE at 12-Months with Majority of Patients Achieving ≥1-point Increase Individual HFMSE responses Type 2 and Non-Ambulatory Type 3 SMA Apitegromab (20 mg/kg) + nusinersen Intent-to-Treat Population (n=14) Per Protocol Population* (n=13) Mean change from baseline in HF
Slide 20: Cohort 3
Cohort 3 S$ ScholarRock.
Slide 21: Background Insights Into Non-Ambulatory Later-Onset SMA with Early Nusinersen
Background Insights Into Non-Ambulatory Later-Onset SMA with Early Initiation of Nusinersen Therapy 21 Nusinersen SHINE Trial in Later-Onset SMA* Source: Darras, B., et.al. Nusinersen in later-onset spinal muscular atrophy. Neurology. May 2019; 92 (21) e2492-e2506.“Longer-term treatment with nusiner
Slide 22: Cohort 3: Sizeable Continued Improvements in Mean HFMSE
Cohort 3: Sizeable Continued Improvements in Mean HFMSE Observed Across 12 Months 22 Individual HFMSE responses Type 2 SMA (Intent-to-Treat Population) Apitegromab 20 mg/kg + nusinersen (n=8) Apitegromab 2 mg/kg + nusinersen (n=9) Pooled (n=17) Mean change from baseline in HFMSE (95% CI) +7.1 (1.8,
Slide 23: Pharmacokinetic and Pharmacodynamic Data Supportive of Clinical Effects
Dose-proportional and sustained drug exposure following chronic administration of apitegromab Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Effects Both 2 mg/kg and 20 mg/kg doses yielded high levels of target engagement (>100-fold increase from baseline)20 mg/kg dos
Slide 24: 12-Month Top-line Results Support Therapeutic Potential of Apitegromab
24 12-Month Top-line Results Support the Therapeutic Potential of Apitegromab and Further Development Data on file. Scholar Rock, Inc. Cambridge, MA 57 Patients* Completed 12-Month TOPAZ Trial All Elected to Opt Into Extension Period *Excludes one patient from Cohort 1 that discontinued from the tri
Slide 25: Hammersmith Functional Motor Scale Expanded for SMA (HFMSE)
Hammersmith Functional Motor Scale Expanded for SMA (HFMSE)Total achievable score of 6633 distinct measures of an individual’s ability to perform various activitiesQuality and execution of each movement is ranked on a scale of 0, 1, 2 http://columbiasma.org/docs/HFMSE_2019_Manual.pdf Plinth/chair si
Slide 26: Next Steps for Apitegromab Program
26 Next Steps for Apitegromab Program Tony Kingsley - President & CEOTed Myles - CFO & Head of Business Ops
Slide 27: Additional TOPAZ Data and Analyses Will Further Our Understanding
27 Additional TOPAZ Data and Analyses Will Further Our Understanding of Apitegromab’s Potential in SMA Exploratory analyses, including patient-level dataAdditional outcome measuresAdditional safety data Plan to present 12-month top-line data and additional analyses at upcoming medical congresses
Slide 28: Apitegromab Has Broad Potential in SMA
28 Apitegromab Has Broad Potential in SMA…Global Disease with Overall Prevalence of 30,000-35,000 in U.S. and Europe Alone High unmet medical need; benefits of SMN upregulators not well establishedTOPAZ data suggest potential clinical benefit; possibly more pronounced in subset of patientsOpportunit
Slide 29: Broad Potential Beyond SMA: Additional Indications
29 Spinal Muscular Atrophy Additional Indications Glucocorticoid induced myopathy Potential benefit for subset of patients unable to discontinue steroid therapy Post-cancer muscle recovery in pediatrics*** Some children may develop severe muscle wasting from chemotherapy Late-onset Pompe Disease** L
Slide 30: Potential for Apitegromab in Becker Muscular Dystrophy
30 Potential for Apitegromab in Becker Muscular Dystrophy (BMD); Aim to Initiate Clinical Trial in 2022 Strong fit for a selective inhibitor of latent myostatin… Key Scientific Question BMD Fit Genetic disorder present at birth, with majority of patients identified at young age (before 18) Is patien
Slide 31: Broad Patent Portfolio and Multiple FDA/EMA Designations
Rare Pediatric Disease for SMA granted by FDAOrphan Drug Designation for SMA granted by FDA Priority Medicines (PRIME) Designation for SMA granted by EMAOrphan Medicinal Product Designation for SMA granted by EMA Broad Patent Portfolio Protecting Apitegromab Into Late 2030s; Multiple Designations Gr
Slide 32: Potential for Another Transformative Year
Gilead fibrosis-focused TGFβ collaboration DRAGON Part A: dose escalation and continued follow-up ApitegromabSpinal Muscular Atrophy (SMA) SRK-181 Immuno-Oncology and Oncology Preclinical/ Platform TOPAZ 12M topline data Planned apitegromab Phase 3 program in SMA by year-end 2021: Potential for Anot
Slide 33: Scholar Rock
S$ ScholarRock.
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