Scholar Rock Holding Corp
SRRK evidence brief
What we can confirm
- BioSniper Score:
- 6.4 / 10
- Market Cap:
- $6.99B
- Lead Asset:
- apitegromab (Phase 3)
What it means
What to watch
Prepared research question
What matters most for SRRK—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · SRRK
Scholar Rock is a global biopharmaceutical company focused on developing monoclonal antibodies for spinal muscular atrophy (SMA) and other rare neuromuscular diseases, with its lead candidate apitegromab under FDA and EMA review [sec_mda · as of 2026-05-07].
Key risk: No approved products yet
BioSniper Score
Based on 4 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 5 pipeline candidates · 6 clinical trials for Scholar Rock Holding Corp.
Scholar Rock is a global biopharmaceutical company focused on developing monoclonal antibodies for spinal muscular atrophy (SMA) and other rare neuromuscular diseases, with its lead candidate apitegromab under FDA and EMA review [sec_mda · as of 2026-05-07].
- Lead candidate apitegromab (SRK-015) is a monoclonal antibody targeting myostatin activation for SMA; FDA PDUFA action date and EMA MAA validation have been received [sec_mda · as of 2026-05-07].
- Cash and equivalents were $437.1M as of 2026-06-30, with a net loss of $109.9M for the period and shareholders' equity of $253.8M [sec].
- Accumulated deficit reached $1.4 billion as of March 31, 2026, with net losses of $105.5M for the three months ended March 31, 2026 [sec_mda · as of 2026-05-07].
- Pipeline includes Phase 2-ready SRK-181 (TGFβ1 inhibitor for solid tumors) and preclinical candidates SRK-373 (fibrotic diseases) and SRK-256 (hemojuvelin inhibitor) [sec_mda · as of 2026-05-07].
- A Phase 2 study of apitegromab in patients with facioscapulohumeral dystrophy (FSHD) is expected to initiate in mid-2026 [sec_mda · as of 2026-05-07].
What works
- Apitegromab has achieved regulatory milestones with FDA and EMA submissions for SMA, indicating progress toward potential commercialization [sec_mda · as of 2026-05-07].
- The company has a strong cash position of $437.1M as of 2026-06-30, providing runway for ongoing development and commercialization activities [sec].
- Positive Phase 2 EMBRAZE study demonstrated proof-of-concept for apitegromab in preserving lean mass during tirzepatide-induced weight loss, expanding potential applications [sec_mda · as of 2026-03-03].
- Scholar Rock's proprietary platform targeting TGFβ superfamily activation enables a pipeline of candidates across multiple rare neuromuscular diseases [sec_mda · as of 2026-05-07].
What to weigh
- The company has incurred significant operating losses since inception, with an accumulated deficit of $1.4 billion as of March 31, 2026 [sec_mda · as of 2026-05-07].
- Net losses were $105.5M for the three months ended March 31, 2026, and the company expects to continue incurring significant expenses for the foreseeable future [sec_mda · as of 2026-05-07].
- Apitegromab has not yet received FDA or EMA approval, and there is uncertainty regarding the scope, outcome, and costs of clinical trials and commercialization [sec_mda · as of 2026-05-07].
- The company will require additional capital to complete clinical development and commercialization for its programs, and may use available resources sooner than expected [sec_mda · as of 2026-05-07].
From the filings
Quoted directly from source documents.
Apitegromab is under FDA and EMA review for SMA
“In March 2025 we submitted to the European Medicines Agency ("EMA") and received validation of our marketing authorisation application ("MAA") for apitegromab for the treatment of SMA.”
SEC MD&A · May 7, 2026
Accumulated deficit is $1.4 billion as of March 31, 2026
“As of March 31, 2026, we had an accumulated deficit of $1.4 billion.”
SEC MD&A · May 7, 2026
Phase 2 EMBRAZE study showed proof-of-concept for lean mass preservation
“our positive Phase 2 EMBRAZE study, demonstrating proof-of-concept in the ability of apitegromab to drive statistically significant preservation of lean mass during tirzepatide-induced weight loss.”
SEC MD&A · March 3, 2026
Catalyst timeline
- 2022-04-14 · Phase3_Start: Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy — Phase 3 trial for apitegromab in later-onset SMA began in April 2022.
- 2024-05-21 · Phase2_Start: A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apitegromab in Overweight and Obese Adult Subjects — Phase 2 trial for apitegromab in weight loss setting started in May 2024.
- 2025-09-15 · Phase2_Start: A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA) — Phase 2 OPAL trial for apitegromab in infants with SMA began in September 2025.
- 2025-11-12 · AdCom: apitegromab — Advisory Committee meeting for apitegromab occurred in November 2025.
- 2025-11-21 · Phase1_Start: A P — Phase 1 trial for an undisclosed candidate started in November 2025.
Risks & what to watch (4)›
Key risks
- No approved products yet — Apitegromab has not received FDA or EMA approval; regulatory and commercial success is uncertain [sec_mda · as of 2026-05-07].
- Significant accumulated deficit — Accumulated deficit of $1.4B as of March 31, 2026, with ongoing net losses [sec_mda · as of 2026-05-07].
- Future capital needs — Company expects to require additional capital to complete development and commercialization; may use resources sooner than expected [sec_mda · as of 2026-05-07].
- Clinical trial uncertainties — Risks include trial enrollment, safety/efficacy outcomes, and costs for multiple pipeline programs [sec_mda · as of 2026-05-07].
What to watch
- FDA PDUFA action date for apitegromab in SMA [sec_mda · as of 2026-05-07].
- EMA decision on marketing authorisation for apitegromab [sec_mda · as of 2026-05-07].
- Initiation of Phase 2 FORGE trial for apitegromab in FSHD, expected mid-2026 [sec_mda · as of 2026-05-07].
- Progress of Phase 2 OPAL trial for apitegromab in SMA patients under 2 years old [sec_mda · as of 2026-05-07].
Sources: Catalysts · Pipeline · SEC MD&A · Generated October 12, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
D/E 1.06 · 0% dilution
Lead: Phase 3
5 candidates
No upcoming catalysts tracked
0 buys · 13 sells (180d)
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
275
Total value held
$6.26B
| Holder | Shares | Value |
|---|---|---|
| fmr llc | 17,243,152 | $847.7M |
| siren, l.l.c. | 10,825,013 | $532.2M |
| redmile group, llc | 9,066,621 | $445.7M |
| blackrock, inc. | 7,469,293 | $367.2M |
| samsara biocapital, llc | 6,897,652 | $339.1M |
| wellington management group llp | 6,717,655 | $330.2M |
| orbis allan gray ltd | 4,179,983 | $205.5M |
| state street corp | 4,031,317 | $198.2M |
| vanguard capital management llc | 3,891,347 | $191.3M |
| bellevue group ag | 3,584,573 | $176.2M |
Showing top 10 of 275 institutional holders of Scholar Rock Holding Corp. Open the full 13F history after sign-in.
Short Interest
as of May 22, 2026- % of float
- 15.7%
- Days to cover
- 9.2
- Shares short
- 14,906,364
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
SRRK short interestGovernance
Frequently asked questions
What is the AI investment thesis for Scholar Rock Holding Corp?
Scholar Rock is a global biopharmaceutical company focused on developing monoclonal antibodies for spinal muscular atrophy (SMA) and other rare neuromuscular diseases, with its lead candidate apitegromab under FDA and EMA review [sec_mda · as of 2026-05-07].
What is Scholar Rock Holding Corp's lead drug candidate?
Scholar Rock Holding Corp's most advanced tracked candidate is apitegromab (Phase 3) for SMA; Spinal Muscular Atrophy; Facioscapulohumeral Muscular Dystrophy.
Who is the CEO of Scholar Rock Holding Corp?
Mr. David L. Hallal is the chief executive officer of Scholar Rock Holding Corp (SRRK).
Where is Scholar Rock Holding Corp headquartered?
Scholar Rock Holding Corp (SRRK) is headquartered in Cambridge, MA, United States.
What therapeutic areas does Scholar Rock Holding Corp focus on?
Scholar Rock Holding Corp develops therapies across Oncology, Rare Disease, Metabolic, Hematology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.