Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Technical Presentation (June 2021)
13 slides from Scholar Rock's technical presentation, June 2021
Slide 1: Apitegromab for Treating SMA: Topline Phase 2 PK/PD Related to Efficacy
Apitegromab, a Novel, Investigational, High-Affinity Anti-pro Myostatin Monoclonal Antibody for Treating Spinal Muscular Atrophy (SMA): Topline Phase 2 PK/PD Related to Efficacy Amy Place, Ph.D., MBA, MS, RD, CLT On behalf of the TOPAZ Study Team and the Scholar Rock Development Team
Slide 2: Scholar Rock Disclosures and Disclaimers
Scholar Rock Disclosures and Disclaimers TOPAZ trial is sponsored by Scholar Rock, a biopharmaceutical company developing and investigating apitegromab for SMA Apitegromab is an investigational product candidate that is currently being evaluated in a clinical trial for the treatment of spinal mu
Slide 3: SMA Is a Chronic, Progressive Neuromuscular Disease With High Unmet Need
Spinal Muscular Atrophy (SMA) Is a Neuromuscular Disease of Chronic, Lifelong Progressive Pathology With a High Unmet Need Despite Current Foundational Therapy1-7 Overall Prevalence of SMA: 30,000-35,000 in US and EU1 Muscle Function in SMA2 Hammersmith Functional Motor Scale Expanded (HFMSE) 80 REL
Slide 4: Myostatin Is an Important Negative Regulator of Skeletal Muscle Growth
Myostatin Is An Important Negative Regulator Of Skeletal Muscle Growth Whose Inhibition Leads To Improved Muscle Function In Patients With SMA1,2 Selective Targeting of proMyostatin, the Myostatin Precursor: Apitegromab does not bind to mature myostatin or any form of GDF11, Activin A, or other TGF-
Slide 5: Apitegromab May Represent a Novel MOA for Neuromuscular Function
Apitegromab May Represent A Novel MOA to Possibly Improve and Maintain Neuromuscular Integrity and Function Throughout Life SMN upregulators directly address motor neuron Apitegromab is a potential muscle-directed degeneration but not muscle atrophy1 approach aimed at improving muscle atrophy and mo
Slide 6: TOPAZ Trial Design: Three pilot cohorts
TOPAZ Trial Design: Three pilot cohorts to identify therapeutic opportunities All SMA Types 2/3, groups defined by age and present ambulatory status Ambulatory Cohort • Ambulatory Type 3 (age 5-21) • Monotherapy or with nusinersen Apitegromab (20 mg/kg IV q4w) 57 Patients* Non-Ambulatory, Ages 5-21
Slide 7: Data Support Apitegromab Stabilizes and Improves Motor Function in Later-onset SMA
The Data Support That Apitegromab Stabilizes And Improves Motor Function In Patients With Later-onset SMA Ambulatory Cohort: Majority Maintained or Improved RHS Scores from Baseline1 Ambulatory Type 3 SMA Pooled (n=23) • TOPAZ results demonstrate functional stabilization or Apitegromab (20 mg/kg) +
Slide 8: Non-ambulatory >2 Years SMA Patients: Significant HFMSE Increases
Non-ambulatory > 2 years SMA Patients: Significant HFMSE Increases Attained with Apitegromab1 Non-ambulatory > 2 Years Type 2 SMA* Apitegromab 20 mg/kg + Apitegromab 2 mg/kg + Pooled (n=17) nusinersen (n=8) nusinersen (n=9) Mean change from baseline in HFMSE (95% CI) +7.1 (1.8, 12.5) +5.3 (-1.5, 12.
Slide 9: Dose Response Observed in Pharmacodynamic & Efficacy Data
Non-Ambulatory > 2 Years SMA Cohort: Dose Response Observed in Pharmacodynamic & Efficacy Data1 Target Engagement Mean (±SEM) change from baseline in HFMSE scores 10000 Mean (±SD) Latent Myostatin Concentration (ng/mL) 1000 100 10 1 0 28 56 84 112 140 168 196 224 252 280 308 336 364 Time (days) TOPA
Slide 10: Target Engagement Achieved With Durability Through 12 Months
Target Engagement Was Achieved With Durability Of Effect Observed Through 12-month Treatment Timeframe Across All Cohorts Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Dose Response Pharmacokinetics* (PK) Pharmacodynamics (PD) 10000 Mean (±SD) Latent Myostatin Concen
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 Trial1 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: TOPAZ Topline Results Demonstrate Apitegromab Improves Motor Function
TOPAZ Topline Results Demonstrate that Apitegromab Improves Motor Function in Patients with Later-Onset SMA1 ✔ Non-Ambulatory; > 2 Years, SMA Type 2 Cohort: HFMSE improvement in both high- and low-dose arms; dose response demonstrated • Mean +7.1-point increase in HFMSE on top of SOC with high dose
Slide 13: Thank You
THANK YOU!! Many thanks to all the patients who participate in these studies and their families, healthcare professionals and the support of patient groups Please Send an Email with Your Medical Questions to the Following Address: medicalinquiry@scholarrock.com
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