Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q3 2023 Earnings Presentation
24 slides from Scholar Rock's earnings presentation, Nov 7, 2023
Slide 1: Scholar Rock Q3 Business Update
Scholar Rock Q3 Business Update November 7, 2023
Slide 2: Forward-Looking Statements
© Scholar Rock, Inc. All rights reserved. 2023. Forward-Looking Statements 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 pro
Slide 3: Scholar Rock Call Participants
Jay Backstrom, MD, MPH Chief Executive Officer Ted Myles, MBA Chief Operating Officer & Chief Financial Officer Mo Qatanani, PhD SVP, Head of Research Scholar Rock Call Participants
Slide 4: Differentiated Benefits to Selective Targeting
Differentiated Benefits to Selective Targeting Scholar Rock’s TargetLatent Growth Factor Traditional Target“mature” active growth factor Our uniquely designed antibodies selectively target the latent form of growth factors Selectivity can mitigate unintended negative effects that have plagued tradit
Slide 5: Next Frontier in Antimyostatin
Next Frontier in Antimyostatin Leveraging our R&D platform to expand into cardiometabolic disorders Myostatin is a member of the TGFβ superfamily known to be a negative regulator of muscle mass and promotes muscle catabolism SRRK’s antibodies are engineered to only target pro- and latent forms of my
Slide 6: Pipeline and Anticipated Key Milestones
Potential to transform the lives of people living with a wide range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, oncology, and fibrosis TARGET DISCOVERY/PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ANTICIPATED KEY MILESTONES Latent Myostatin SPINAL MUSCULAR ATROPHYApiteg
Slide 7: Clinical Programs Update: SRK-181, Apitegromab
Clinical Programs UpdateSRK-181Apitegromab Jay Backstrom – Chief Executive Officer
Slide 8: SRK-181 | Immuno-Oncology
SRK-181 | Immuno-Oncology Transformative backbone for a new era of cancer immunotherapy Data from Phase 1 DRAGON Proof-of-Concept Trial Supports proof-of-concept for SRK-181 in heavily pre-treated patients with ccRCC resistant to anti-PD-1 Biomarker data from all cohorts in Part B supports proof-of-
Slide 9: SRK-181 Increases CD8+ T Cell Infiltration in Melanoma
SRK-181 in Combination with Anti-PD1 Increases Infiltration of CD8+ T Cells in Melanoma Paired biopsies from 2 melanoma patients were analyzed for CD8 content. An increase in CD8+ T cell infiltration was observed in both biopsy pairs, overcoming an initially excluded or desert phenotype and resultin
Slide 10: Continued Tolerability & Promising Anti-Tumor Activity in ccRCC
Duration of Treatment Best Response in Target Lesions Change in Tumor Volume Over Time Continued Tolerability & Promising Anti-Tumor Activity | ccRCC Patients SRK-181 + pembrolizumab in ccRCC Patients* ORR = 21.4% Disease control rate = 57% 6 PRs (4 remain on study) 33% to 93% best tumor reduction o
Slide 11: SRK-181 Differentiation and Summary
11 1.Sumanta Kumar Pal et al. Atezolizumab plus cabozantinib versus cabozantinib monotherapy for patients with renal cell carcinoma after progression with previous immune checkpoint inhibitor treatment (CONTACT-03): a multicentre, randomised, open-label, phase 3 trial. The Lancet, Volume 402, Issue
Slide 12: Apitegromab | Spinal Muscular Atrophy
Apitegromab | Spinal Muscular Atrophy First and only muscle-targeted investigational treatment to demonstrate clinical POC * Based on Animal Model Data; 1. Long KK, et al. Hum Mol Genet. 2019;28(7):1077-1088; 2. Pirruccello-Straub M, et al. Sci Reports. 2018;8(1):2292. doi:10.1038/s41598-018-20524-9
Slide 13: TOPAZ 36M Data: Improvements Across Motor Function and PRO Measures
Time (weeks) No. of observations Time (weeks) No. of observations Year 3 Year 2 Year 1 TOPAZ 36M Data: Robust and Consistent Improvements Observed across Motor Function and PRO Measures (Pooled Nonambulatory Patients | Age 2 – 21 | All Doses) IMPROVEMENT PEDI-CAT Daily Activities IMPROVEMENT IMPROVE
Slide 14: Executing on the Promise of Apitegromab
Executing on the Promise of Apitegromab 14 SMA=Spinal Muscular Atrophy *Subject to regulatory approval WHERE WE AREPhase 3 12-Month IN PROCESS Long-term EXTENSION 2023 enrollment completed Q4 2024 data 2025 launch* ANTICIPATED MILESTONES WHERE WE WERE Phase 2 12-Month COMPLETE 24-Month EXTENSION COM
Slide 15: Cardiometabolic Program
Cardiometabolic Program Mo Qatanani– SVP, Research
Slide 16: Our Antibodies Selectively Inhibit Activation of Myostatin
Our Antibodies Selectively Inhibit Activation of Myostatin Selective Targeting of Proforms of Myostatin 16 SRRK antibodies do not bind to mature myostatin or any form of GDF11, Activin A, or other TGF-β family members Selectivity is critical to avoid safety concerns Mature myostatin and mature GDF11
Slide 17: Loss of Lean Muscle Significant with GLP-1 RA Rx
Loss of Lean Muscle Significant with GLP-1 RA RxLean muscle is essential to healthy metabolic function & body composition Current weight loss strategies challenged by tolerability, lack of durability and significant muscle loss1,2,3 Recently approved GLP-1 receptor agonists (GLP-1 RAs) are highly ef
Slide 18: SRK-439: Novel Myostatin Inhibitor
SRK-439: Novel Myostatin Inhibitor Antibody/ Variant mTLL2 IC50 (nM) mAb KD (nM) SRK-439 0.145 0.579 SRK-439: A novel preclinical antimyostatin antibody with attractive properties and potential to address muscle loss associated with weight loss High in vitro affinity for pro- and latent myostatin Ma
Slide 19: SRK-439 Maintained Lean Mass When Combined with GLP-1 RA
SRK-439 Maintained Lean Mass When Combined with GLP-1 RA Therapy* 19 Improved fat mass loss vs GLP-1 RA alone Increased lean mass gain vs GLP-1 RA alone One-way ANOVA * P< 0.05 ** P< 0.005 *** P< 0.0005 **** P< 0.0001 *in Mouse Diet Induced Obesity (DIO) Model Figure shows the effects of increasing
Slide 20: SRK-439 Reversed Lean Mass Loss Induced by Semaglutide
SRK-439 Reversed Lean Mass Loss and Enhanced Fat Mass Loss Induced by Semaglutide Treatment* 20 One-way ANOVA * P< 0.05 ** P< 0.005 *** P< 0.0005 **** P< 0.0001 *in Mouse Diet Induced Obesity (DIO) Model Figure showed the effects of increasing doses of SRK-439 in combination with semaglutide on lean
Slide 21: Highly Selective Antimyostatin to Preserve Lean Muscle
Highly Selective Antimyostatin to Preserve Lean Muscle Opportunity for safe, durable weight loss Myostatin Inhibition Preclinical models demonstrated increased muscle mass and beneficial metabolic effects (insulin sensitivity, basal metabolic rate, reduction in fat mass)1 Exquisite Selectivity Stron
Slide 22: Expedite Cardiometabolic Program with Ph2 POC Study
Expedite Cardiometabolic Program with Ph2 Proof of Concept Study of Apitegromab in Obesity SRK-439 Rapid Proof of Concept Phase 2 Study APITEGROMAB + GLP-1 RA Advancing SRK-439 for cardiometabolic indication 2023 2024 2025 Apitegromab (SMA) Apitegromab POC (Cardiomet) Pipeline: SRK-439 Enrollment Co
Slide 23: Summary
Summary Ted Myles- COO & CFO S$ ScholarRock.
Slide 24: Key Investment Highlights
24 Key Investment Highlights Revolutionary Platform Overcome the Challenges by targeting the latent forms of growth factors Discover and Develop monoclonal antibodies with extraordinary selectivity Positioned For Success Cardiometabolic Initiate POC trial with apitegromab in combo with GLP-1 RA in 2