Scholar Rock Holding Corp. (SRRK)

Key figures

The last twelve months reported, with the latest price and balance sheet.

Market value

$5.3B

Cash

$437.1M

Debt

$196.2M

Debt to equity

0.77

Shares outstanding

121.6M

Financial statements

Revenue, profit and cash flow by fiscal year, each year linked to its annual report.

By quarter

What the company said with its results

From the second quarter 2026 results, filed 6 August 2026.

• Net loss near prior-year level. Net loss was $109.9 million, or $0.84 per share, against $110.0 million, or $0.98 per share, in the second quarter of 2025.
• No revenue in either quarter. The company recorded no revenue in the second quarter of 2026 or the same quarter of 2025.
• Six-month loss per share higher. Six-month net loss per share was $1.67, against $1.65 for the first six months of 2025.
• Two fill-finish paths to approval. The BLA review is advancing through the Catalent Indiana facility and a second fill-finish facility, which the company describes as two independent paths.
• Catalent classification still pending. FDA classification of Catalent Indiana, following an April 2026 general site inspection, has not yet been issued.
• Second facility data under review. The data package for the second facility, agreed at the March 2026 Type C meeting, has been submitted and agency review is in progress.
• EMA review and second facility. Scholar Rock is discussing next steps with the EMA, including possibly adding the second fill-finish facility to the MAA.
• Launch ready on approval. The U.S. commercial team is prepared to launch apitegromab upon FDA approval.
• Ample supply on hand. Supply from the second fill-finish facility is available for commercialization upon FDA approval, which the company describes as ample.
• Presence at SMA meetings. The company had a significant presence at Cure SMA's annual conference and research meeting in Orlando, held June 23 to 28, 2026.
• FSHD Phase 2 trial started. The Phase 2 FORGE study of apitegromab in FSHD has been initiated.
• OPAL pediatric enrollment continues. Enrollment and dosing continue in the Phase 2 OPAL study in infants and toddlers with SMA under two years of age.
• Subcutaneous path after approvals. A high-concentration subcutaneous apitegromab formulation has been developed; the company plans FDA and EMA engagement after SMA approvals.
• G&A expense rose slightly. General and administrative expense was $50.7 million, against $49.7 million a year earlier, including $12.2 million of stock-based compensation.

Company events

The newest 8-K filings of 108 held.

Every filing

The newest filings with the SEC of 207 held.

Insider trades

Form 4 trades by officers, directors and 10% owners, last 12 months.

Planned insider sales

Form 144 notices, filed when the sell order goes to the broker.

Large holders

Schedule 13D and 13G filings by holders of more than 5% of the voting shares.

Short interest

Shares sold short and not yet bought back, on each FINRA settlement date.

Funds that hold it

From each fund's latest Form 13F.

328 funds held it at 30 Jun 2026: 178 added and 82 trimmed in the quarter. The largest:

Peers

Companies in the same industry.

• Amgen Inc. (AMGN): revenue $38.1B, market value $244.4B
• Gilead Sciences, Inc. (GILD): revenue $30.5B, market value $197.6B
• Moderna, Inc. (MRNA): revenue $2.2B, market value $85.4B
• argenx SE (ARGX): revenue $4.2B, market value $55.2B
• Biogen Inc. (BIIB): revenue $10.3B, market value $36.1B
• BioNTech SE (BNTX): revenue $3.2B, market value $22.1B
• Exelixis, Inc. (EXEL): revenue $2.4B, market value $16.5B
• Halozyme Therapeutics, Inc. (HALO): revenue $1.7B, market value $14.8B
• Neurocrine Biosciences Inc. (NBIX): revenue $3.4B, market value $14.8B
• Bio-Techne Corp. (TECH): revenue $1.2B, market value $11.3B

What the company does

Scholar Rock Holding Corp is a biopharmaceutical company focused on spinal muscular atrophy ("SMA") and other rare neuromuscular diseases. It works from a platform for making monoclonal antibodies that bind the latent, or precursor, forms of growth factors in the transforming growth factor beta ("TGFβ") superfamily. Its lead candidate is apitegromab, an antibody that inhibits the activation of myostatin in skeletal muscle. The company holds worldwide rights to its platform and to all of its product candidates.

How the technology works

Many growth factors are made by cells in an inactive, latent form. In this form a prodomain wraps around the growth factor like a cage and blocks signalling until an activation event releases it. Scholar Rock builds antibodies that bind the latent form and stop that activation. This is meant to act at the site of the disease rather than throughout the body.

The active forms of myostatin and a related factor, GDF11, are 90% identical, which makes it hard to block one without the other. Their prodomains are only 52% identical, and the company says this underpins the selectivity of its approach. The company was founded by Timothy A. Springer and Leonard I. Zon of Harvard Medical School and Boston Children's Hospital.

Apitegromab and SMA

Apitegromab is a fully human antibody given by intravenous infusion. Myostatin is a negative regulator of muscle mass, so blocking its activation is intended to increase muscle mass and strength. SMA affects an estimated 1 to 2 per 100,000 people globally. The filing says there are three approved SMN-targeted treatments and no approved muscle-targeted treatment. Apitegromab is being developed for people already taking an SMN-targeted therapy. The company estimates that 35,000 SMA patients globally have received such a treatment.

The Phase 3 SAPPHIRE trial enrolled 156 patients aged 2-12 in its main efficacy population and 32 patients aged 13-21 in an exploratory population. It was randomized, double-blind and placebo-controlled, and the company reports that it met its primary endpoint. Of the SAPPHIRE patients, 98% (185/188) went on to the ONYX extension study.

The company submitted a Biologics License Application to the FDA in January 2025 and received priority review. In September 2025 it received a Complete Response Letter tied to observations at a third-party fill-finish facility. The company says these observations were site-related and not specific to apitegromab. It says it plans to resubmit once the facility resolves the deficiencies. A marketing authorisation application to the European Medicines Agency was submitted and validated in March 2025.

Other programs

- OPAL: a Phase 2 study of apitegromab in infants and toddlers with SMA under two years of age. - FSHD: facioscapulohumeral muscular dystrophy has no approved treatments, and more than 30,000 patients are diagnosed in the U.S. and Europe. The company says it plans to start a Phase 2 trial called FORGE in mid-2026, enrolling approximately 60 patients. - Subcutaneous apitegromab: a Phase 1 study in 45 healthy volunteers is complete. - SRK-439: a subcutaneous anti-myostatin antibody in a Phase 1 study in healthy volunteers. - EMBRAZE: a Phase 2 trial of apitegromab with tirzepatide in people with obesity or overweight, run over 24 weeks. - SRK-181, SRK-373 and SRK-256: programs in solid tumors, fibrosis and iron-restricted anemias. The company says it is seeking partners for these.

How it makes and sells

Scholar Rock owns no facilities for clinical drug manufacturing, storage, distribution or quality testing, and all clinical manufacturing is outsourced. It has internal antibody discovery capabilities and also uses Adimab, LLC under a collaboration agreement. Under that agreement it has exercised options for exclusive licenses, including for the program that produced SRK-181. If it sells a product, it owes Adimab a low-to-mid single-digit percentage of annual worldwide net sales.

The company has built a U.S. customer-facing team of approximately 50 professionals. It has also started a commercial build-out in Europe, beginning with Germany. It describes a 50+ country platform for commercial readiness.

Patents

As of 31 December 2025 the company had 32 pending and/or granted patent families. Four granted European patents are the subject of opposition proceedings before the European Patent Office. Two of them were revoked by the opposition division, and those revocations are under appeal. The company also relies on trade secrets for parts of its platform.

Who it competes with

For apitegromab in SMA, the filing names Roche, Biogen and NMD Pharma as possible competitors. It also notes that Novartis, Roche and Biogen are developing other formulations or dosing regimens of their approved SMN treatments. For SRK-181, it lists AbbVie Inc, Roche, Bicara Therapeutics, Novartis, Bristol Myers Squibb and Merck KGaA, among others. The filing says many competitors have greater financial resources and expertise than the company.

*From Scholar Rock's annual report on Form 10-K for the year ended 31 December 2025. [The filing](https://www.sec.gov/Archives/edgar/data/1727196/000110465926022551/srrk-20251231x10k.htm)*

From the company's 10-K.