Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q4 2024 Earnings Presentation
23 slides from Scholar Rock's earnings presentation, Feb 27, 2025
Slide 1: Fourth Quarter 2024 Business Update
Fourth Quarter 2024 Business Update February 27, 2025 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 2: Forward-Looking Statements
Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, “Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding its strategy, its
Slide 3: Agenda
TO P I C SPEAKER Apitegromab in SMA Agenda SAPPHIRE Phase 3 Pivotal Jay Backstrom, Trial Results and President & Chief Executive Officer Regulatory Milestones Apitegromab in SMA Tracey Sacco, Planning for a 2025 Launch Chief Commercial Officer to Serve Patients Scholar Rock Innovative Myostatin Plat
Slide 4: Strong Momentum in 2024 Sets the Stage for a Transformative Year Ahead
Strong Momentum in 2024 Sets the Stage for a Transformative Year Ahead RECENT ACCOMPLISHMENTS Positive Phase 3 BLA Submitted SAPPHIRE data SAPPHIRE data for apitegromab and DMD poster in SMA accepted for presentation at MDA 2025 ANTICIPATED MILESTONES 2025 Q1 Q2 Q3 Q3 Q4 MAA OPAL US launch SMA apite
Slide 5: Positive Phase 3 Trial Using HFMSE, Gold Standard SMA Scale
SELECTIVITY DRIVES SUCCESS Positive Phase 3 Trial Using HFMSE, Gold Standard SMA Scale The Only Muscle-Targeted Therapy with Over 4 Years of Clinical Experience in >200 Patients 1.8 CONSISTENT clinically meaningful POINT IMPROVEMENT benefit observed across (p=0.0192) all age groups (2‒21) in HFMSE*
Slide 6: Apitegromab in SMA: Planning for a 2025 Launch to Serve Patients
Apitegromab in SMA: Planning for a 2025 Launch to Serve Patients Tracey Sacco, MBA Chief Commercial Officer 6 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 7: Progressive, Debilitating Muscle Atrophy Remains a Critical Unmet Need in SMA
COMMERCIAL PREPAREDNESS Progressive, Debilitating Muscle Atrophy Remains a Critical Unmet Need in SMA Despite SMN-Targeted Therapies Personal hygiene, using the toilet and the shower on my own would be huge. My four-year-old can do it on her own. It’s degrading. US Patient 97% >80% of of patients ne
Slide 8: The Future of SMA is in Treating the Muscle and the Motor Neuron
COMMERCIAL PREPAREDNESS The Future of SMA is in Treating the Muscle and the Motor Neuron SMA Today IN THE US WORLDWIDE 74% of neurologists agree that in the future, multiple modalities ~10,000 2/3 ~35,000 are necessary living SMA patients patients have received an to treat SMA5 with SMA1 received an
Slide 9: Preparing the Market for a Potential New Treatment Paradigm in SMA
COMMERCIAL PREPAREDNESS Preparing the Market for a Potential New Treatment Paradigm in SMA Disease Patient Advocacy HCP Engagement Education Partnership • Amplifying the • MSL team engaged 100% of • Long-tenured relationship patient voice in first Cure SMA-identified sites and strong partnerships mu
Slide 10: Significant Rare Disease Experience to Drive Successful Apitegromab Launch
COMMERCIAL PREPAREDNESS Significant Rare Disease Experience to Drive Successful Apitegromab Launch INTERNAL SEASONED RARE DISEASE ATTRACTING COMMERCIAL COMMERCIAL LAUNCH TOP TALENT EXPERTISE LEADERSHIP EXPERIENCE Across market access, Commercial leadership Among Commercial, To build world-class mark
Slide 11: Scholar Rock is Positioned for a Successful Launch in SMA
COMMERCIAL PREPAREDNESS Scholar Rock is Positioned for a Successful Launch in SMA Preparing patient services and home infusion offering for a Q4 launch* Building a customer-facing team of sales, reimbursement, and patient support personnel in Q2/Q3 Initiating payer outreach to top US commercial and
Slide 12: Scholar Rock Innovative Myostatin Platform: Ambition to Transform Current GLP-1 Treatment Paradigm
Scholar Rock Innovative Myostatin Platform: Ambition to Transform Current GLP-1 Treatment Paradigm Jay Backstrom, M.D., MPH 12 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 13: GLP-1 RAs are Transforming Weight Loss for Millions of People
CARDIOMETABOLIC PROGRAM GLP-1 RAs are Transforming Weight Loss for Millions of People RECENTLY APPROVED GLP-1 RAs are highly effective in weight loss & experiencing rapid uptake BY 40 million people on GLP-1 RAs 2029... $126 billion in sales* Source: UBS Bank, GLP-1: A medication worth $126 billion
Slide 14: However, Patients Want Healthier Weight Loss
CARDIOMETABOLIC PROGRAM However, Patients Want Healthier Weight Loss* Patients feel good about the number on the scale, but there are issues with muscle loss WEAKNESS Improved Significant Weight … they complain of is a Concern LEAN MASS REGAIN weakness or reduced Patients complain of Patients hope f
Slide 15: Significant Muscle Loss Accompanies Tirzepatide-Induced Weight Loss
CARDIOMETABOLIC PROGRAM Significant Muscle Loss Accompanies Tirzepatide-Induced Weight Loss Percent Change in Body Weight by Week (efficacy estimand) Tirzepatide, 5 mg Tirzepatide, 10 mg Tirzepatide, 15 mg Placebo Tirzepatide Time period of EMBRAZE Overall mean baseline weight = 104.8 kg 0 DECREASE
Slide 16: Every Kilogram Matters: Significant Cost of Losing Lean Mass
CARDIOMETABOLIC PROGRAM Every Kilogram Matters: Significant Cost of Losing Lean Mass Loss of ~1 kg lean mass resulted in decreased leg strength and increased insulin resistance* ~7% decrease in leg strength LE A N MA SS STRENGTH L o s s o f ~1% Loss of 10‒15% ~6% reduction per year^ per decade^ in e
Slide 17: Goals of the EMBRAZE Proof-of-Concept Study
CARDIOMETABOLIC PROGRAM Goals of the EMBRAZE Proof-of-Concept Study PRESERVE LEAN MASS INFORMING THE DEVELOPMENT OF SRK-439 COMPARABLE WEIGHT LOSS Highly selective muscle- REDUCE FAT REGAIN targeted therapy in obesity COMPARABLE SAFETY (no added toxicities) Exploratory: Potential to improve METABOLI
Slide 18: SRK-439: Best in Class Potential
CARDIOMETABOLIC PROGRAM SRK-439: Best in Class Potential SRK-439 ActRII Ab Ligand Trap Adnectin Selectivity for myostatin Action limited to muscle Combination GLP-1 RA data in obesity preclinical models1-3 Low efficacious dose in preclinical obesity models1-3 Potential lower risk of undesirable effe
Slide 19: Industry-Leading Anti-Myostatin Platform: Leveraging Apitegromab's Success
CARDIOMETABOLIC PROGRAM Industry-Leading Anti-Myostatin Platform: Leveraging Apitegromab’s Success to Advance the Obesity Program 2025 Testing hypothesis of selective POC data readout Q2* anti-myostatin APITEGROMAB + tirzepatide antibody in obese Ph 2 proof-of-concept trial population Cardiometaboli
Slide 20: Conclusions
Conclusions 20 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 21: Scholar Rock is Moving with a Sense of Urgency: Commercialize, Expand, Advance
T O D AY ’ S F O C U S Scholar Rock is Moving with a Sense of Urgency To Bring Transformative Medicines to Patients 1 COMMERCIALIZE 2 EXPAND 3 ADVANCE Execute a Successful Apitegromab Development Anti-myostatin Program Commercial Launch* Program: Building a Pipeline into Cardiometabolic in a Product
Slide 22: Q&A
Q&A © 2025 Scholar Rock, Inc. All rights reserved. 22
Slide 23: Q&A: Scholar Rock Management
Q&A: Scholar Rock Management Jay Backstrom, M.D., MPH Ted Myles, MBA Mo Qatanani, PhD Tracey Sacco, MBA President & Chief Chief Operating Officer and Chief Scientific Officer Chief Commercial Officer Executive Officer Chief Financial Officer 23 © 2025 Scholar Rock, Inc. All rights reserved.