Scholar Rock

What Scholar Rock does

Scholar Rock is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, that develops monoclonal antibodies targeting latent forms of growth factors in the TGFβ superfamily. Its scientific premise is that selectively inhibiting growth factor activation before it occurs—rather than blocking activated forms after signaling has begun—enables higher specificity and fewer off-target effects. The company's lead asset, apitegromab, is a selective anti-latent myostatin antibody for spinal muscular atrophy that demonstrated statistically significant motor function improvement on top of standard-of-care SMN-targeted therapies in the Phase 3 SAPPHIRE trial, with a target FDA PDUFA date of late September 2026.

The pipeline extends the latent-growth-factor platform into five distinct programs: SRK-181 (linavonkibart) for checkpoint-resistant solid tumors, SRK-439 for rare neuromuscular diseases, SRK-373 for fibrotic diseases, and SRK-256 for iron-restricted anemia. Apitegromab is also being explored in cardiometabolic indications, where the EMBRAZE trial showed 54.9% preservation of lean mass during tirzepatide-induced weight loss. The company is pre-revenue from product sales; 2025 revenue of $112.5 million was derived entirely from collaboration milestones and grew 46% year over year.

Scholar Rock's commercial strategy targets rare-disease specialty channels: a home-infusion network of over 10,000 affiliated nurses, specialty pharmacy access, and direct engagement with neuromuscular specialists in the U.S. and Germany. The company plans U.S. launch in late 2026, European launch beginning in Germany in H2 2026, and stated expansion across 50+ countries. Capital structure includes a $300M public offering (October 2024) and a $550M non-dilutive debt facility from Blue Owl Capital (March 2026), with $367.6M–$403M in cash and securities at year-end 2025 supporting operations into 2028. The April 2025 executive refresh brought in leadership from Alexion, argenx, Bayer, and Acceleron, signaling the company's transition from development-stage to commercial-stage biopharmaceutical.

Name

Scholar Rock

Legal name

Scholar Rock, Inc. and Scholar Rock Holding Corporation

Website

https://scholarrock.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

251–500 employees

Short description

Scholar Rock is a clinical-stage biopharmaceutical company developing monoclonal antibodies that selectively target latent growth factors in the TGFβ superfamily. Its lead candidate apitegromab treats spinal muscular atrophy, with additional programs across neuromuscular, oncology, fibrosis, and hematology indications.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC)

akta.pro secondary industries

Rare Endocrine & Growth Disorder Therapies (HLAIAIAJ), In Vivo Discovery & Translational Models Platforms (humanized models, PK/PD, efficacy) (HLAAAIAK), Lead Optimization & Preclinical Candidate Selection (HLAIALAB)

Keywords

• Monoclonal antibody therapeutics
• Biopharmaceutical drug development
• Growth factor inhibitors
• Spinal muscular atrophy treatment
• Rare disease therapies

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Biopharmaceutical Product Revenue: Scholar Rock is a pre-revenue biopharmaceutical company developing apitegromab for spinal muscular atrophy and other pipeline candidates. The company expects to generate revenue from product sales following regulatory approval, with anticipated U.S. launch in late 2026 and European launch in H2 2026.
2. Collaboration and Milestone Revenue: Full-year 2025 revenue of $112.5 million was driven by milestone receipts and collaborations, representing 46% growth from $76.8 million in 2024. These are non-recurring revenue streams from partnership agreements.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels5 records

Core offering

Scholar Rock is a biopharmaceutical company developing monoclonal antibody therapies that selectively target the latent, inactive forms of protein growth factors in the TGFβ superfamily before they are activated. Its lead product candidate, apitegromab, is a selective anti-latent myostatin antibody for spinal muscular atrophy being prepared for U.S. commercial launch in late 2026 following FDA review, with additional pipeline candidates in immuno-oncology, cardiometabolic disorders, fibrosis, and iron-restricted anemia.

Product overview

Scholar Rock is a biopharmaceutical company with a proprietary platform that selectively targets latent growth factors before activation. The company's lead product is apitegromab (SRK-015), a selective anti-latent myostatin antibody for spinal muscular atrophy in Phase 3 with anticipated 2026 U.S. launch pending FDA approval. The pipeline spans five clinical and preclinical programs: SRK-181 (Linavonkibart) for immuno-oncology, SRK-439 for rare neuromuscular diseases, SRK-373 for fibrosis, and SRK-256 for iron-restricted anemia. The company is preparing commercial infrastructure including specialty pharmacy access and a home-infusion network of over 10,000 affiliated nurses for the anticipated apitegromab launch.

Differentiator

Problem solved

Functional benefit

Products and services

• Apitegromab (SRK-015) Selective inhibitor of latent myostatin activation designed to improve motor function in patients with spinal muscular atrophy (SMA). Administered as an intravenous infusion every 4 weeks as an adjunct to standard-of-care SMN-targeted therapies. The product is being prepared for patients with Type 2 and Type 3 SMA, including pediatric and adolescent/young adult populations, who continue to experience progressive muscle weakness on SMN-targeted standard of care.
• SRK-181 (Linavonkibart) Selective inhibitor of latent TGFβ1 activation being investigated for the treatment of locally advanced or metastatic solid tumors that have shown primary resistance to checkpoint inhibitor therapies. Studied in combination with anti-PD-(L)1 therapies to render tumors vulnerable to immune-mediated killing. Encouraging responses observed in heavily pretreated checkpoint inhibitor-resistant solid tumors in early clinical data.
• SRK-439 Novel anti-latent myostatin antibody in Phase 1 development for rare neuromuscular diseases. Represented in the pipeline as a follow-on myostatin-targeting program to apitegromab.
• SRK-373 Selective context-dependent anti-latent TGFβ1 antibody targeting LTBP1 and LTBP3 for the treatment of fibrosis. Designed to selectively inhibit TGFβ1 in fibrotic tissue while preserving immune-suppressing functions of TGFβ1 elsewhere in the body. IND-enabling studies underway for multiple fibrotic indications.
• SRK-256 Selective anti-RGMc/HJV (hemojuvelin) antibody in preclinical development for the treatment of iron-restricted anemia. Targets the BMP signaling pathway in the liver to reduce hepcidin expression and increase iron availability in the bloodstream, addressing anemia associated with chronic inflammatory conditions, chronic kidney disease, and myelofibrosis.

Quantifiable outcome

• Phase 3 SAPPHIRE trial achieved primary endpoint with statistically significant improvement in HFMSE motor function scores for apitegromab vs placebo in SMA patients on standard of care therapies
• +2 more outcomes

Named customers1 record

Segments5 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Novo Nordisk (ex-Catalent Indiana)coreTechnology or Integration · 10 October 2025Manufacturing partner for apitegromab fill-finish operations. The Catalent Indiana facility (owned by Novo Nordisk) experienced FDA scrutiny resulting in delays to the BLA resubmission. Scholar Rock added a second fill-finish facility as an alternative launch pathway. Engineering runs are underway at the second facility.
• Cure SMAcoreStrategic or Co-development PartnerPatient advocacy organization providing support to patients and families affected by spinal muscular atrophy and funding research toward a cure. Scholar Rock collaborates with Cure SMA to engage the patient community and advance clinical development.
• Muscular Dystrophy Association (MDA)coreStrategic or Co-development PartnerMDA accelerates research, advances care, and advocates for individuals affected by muscular dystrophy, ALS, and related neuromuscular diseases. Scholar Rock participates in MDA conferences and collaborates on patient education.
• SMA EuropeminorStrategic or Co-development PartnerEuropean patient organization supporting and advocating for SMA patients and families across European countries, collaborating on research and awareness initiatives.
• National Organization for Rare Disorders (NORD)minorStrategic or Co-development PartnerNORD provides support for individuals with rare diseases through advocacy, research funding, and networking among service providers.
• FSHD SocietycoreStrategic or Co-development PartnerScholar Rock collaborates with the FSHD Society on the FORGE clinical trial for facioscapulohumeral muscular dystrophy and participates in the FSHD Industry Collaborative to improve clinical trials.

Scale indicators10 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Biogen: Markets Spinraza, a leading SMN-targeted SMA therapy, and is the dominant incumbent in the SMA market where Scholar Rock's apitegromab would be used adjunctively. Comparable as a neuroscience/rare-disease commercial-stage biopharma.
• Novartis: Commercializes Zolgensma, the gene therapy for SMA. Directly comparable as another foundation SMA therapy provider where apitegromab would layer in as an adjunct.
• argenx: Commercial-stage rare-disease antibody biotech with a similar late-stage launch profile and home-infusion model. Highly comparable as a peer in antibody-based rare neuromuscular/autoimmune commercialization.
• Alnylam Pharmaceuticals: Late-stage RNAi-based biopharma with multiple approved rare-disease products and President of R&D background shared with Scholar Rock leadership. Comparable as a platform-based rare-disease commercial-stage biopharma.
• Krystal Biotech: Commercial-stage rare-disease gene therapy biotech with a similar growth profile and leadership pedigree (former CMO Jing Marantz was previously EVP/CBO at Krystal). Highly comparable as a peer rare-disease commercial-stage biotech.
• PTC Therapeutics: Rare-disease biopharma with SMA heritage (Evrysdi royalty and prior SMA programs) and a portfolio of neuromuscular and oncology assets. Comparable as a peer in rare neuromuscular drug development and commercialization.

Broad incumbents

• Roche (Genentech): Markets Evrysdi for SMA via Genentech and previously developed emugrobart in anti-myostatin. Highly comparable as a competitor in the SMA standard-of-care stack and a former direct competitor in anti-myostatin.
• BioMarin Pharmaceutical: Established rare-disease commercial biopharma with multiple approved enzyme-replacement and gene therapies. Comparable as a broader orphan-disease commercial benchmark and potential acquirer profile.

Emerging players

• Dyne Therapeutics: Clinical-stage muscle-targeted therapeutics company with programs in DM1, DMD and FSHD. Comparable as a direct competitor in muscle biology drug development, overlapping with Scholar Rock's FSHD and SMA programs.
• Ionis Pharmaceuticals: Antisense-based biopharma with SMA heritage (Spinraza royalty stream) and pipeline in rare neuromuscular diseases. Comparable as an SMA market participant and rare-disease platform biotech.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/ScholarRock
• LinkedInlinkedin.com/company/scholar-rock

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds9 records

Investors14 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Scholar Rock