Scholar Rock (SRRK -8.71%), a late-stage biopharmaceutical company specializing in therapies for serious neuromuscular and rare diseases, released its second quarter 2025 earnings on August 6, 2025. The key news was continued preparation for the potential 2025 U.S. launch of apitegromab, its lead muscle-targeted therapy for spinal muscular atrophy (SMA). The results showed a GAAP net loss per share of $0.98. No revenue was recorded, matching expectations. Overall, the period reflected Scholar Rock’s intense focus on launching its first commercial product, balanced by a rising cash burn and reliance on regulatory approvals.
| Metric | Q2 2025 | Q2 2025 Estimate | Q2 2024 | Y/Y Change |
|---|---|---|---|---|
| EPS (GAAP) | $(0.98) | $(0.66) | $(0.60) | (63.3%) |
| Revenue (GAAP) | $0.0 | $0.0 | $0.0 | – |
| Research and Development Expense | $62.4 million | $42.4 million | 47.2% increase | |
| General and Administrative Expense | $49.7 million | $17.1 million | 190.6% | |
| Cash, Cash Equivalents and Marketable Securities | $295 million | N/A |
Source: Analyst estimates for the quarter provided by FactSet.
Scholar Rock’s Business and Strategic Focus
The company is built around its proprietary platform that targets “latent” forms of growth factors, seeking to offer more selective therapies for neuromuscular and rare diseases. Its lead candidate, apitegromab, is designed as a muscle-targeted monoclonal antibody therapy for SMA, a genetic disorder that leads to muscle loss and weakness. Scholar Rock’s approach aims to improve motor function where current therapies leave treatment gaps.
Recent business priorities have centered on preparing apitegromab for commercial launch and broadening its possible uses. Success factors for the company include regulatory approvals, effective commercial deployment, and leveraging its technology into additional neuromuscular, cardiometabolic, and cancer indications. Managing its sizable investment in infrastructure and drug development is also crucial as the company transitions to a potential revenue-generating business.
Quarter Highlights: Financials, Pipeline, and Operations
During the quarter, Scholar Rock’s operating expenses escalated. Research and development expense was $62.4 million for the quarter ended June 30, 2025, compared to $42.4 million a year earlier, a 47% increase, primarily due to manufacturing the commercial supply of apitegromab and increased staffing. General and administrative expense (GAAP) soared to $49.7 million, nearly triple last year’s level, with significant increases linked to leadership transitions and commercial build-out efforts. The company stated that $8.6 million of G&A and $2.7 million of R&D expenses were tied to one-time costs for leadership changes.
The absence of any product or partnership revenue continued as anticipated. This reinforced Scholar Rock's position as a pipeline-driven company—its future hinges on the outcome of its pivotal clinical trial and regulatory reviews. The net loss (GAAP) widened to $110 million as operating expenses increased. Its cash, cash equivalents, and marketable securities balance was $295 million as of June 30, 2025, down from $437 million as of December 31, 2024, giving the company an estimated runway into 2027.
The quarter was marked by important regulatory advances for apitegromab. In the U.S, the Food and Drug Administration (FDA) accepted the company’s Biologics License Application (BLA) for priority review, setting a target action date of September 22, 2025. In Europe, the European Medicines Agency (EMA) validated apitegromab’s marketing application, positioning Scholar Rock for a possible 2026 launch in the region. The company also presented additional clinical data on apitegromab’s benefits for SMA at major conferences, supporting its case to providers, payers, and patients.
Scholar Rock continues to expand its product reach. The company will begin a Phase 2 OPAL trial in infants and toddlers with SMA in the third quarter of 2025, exploring apitegromab as a combination with existing therapies. Outside of SMA, results from the Phase 2 EMBRAZE trial in obesity showed that apitegromab, when added to tirzepatide (a GLP-1 receptor agonist), preserved an additional 4.2 pounds, or 54.9%, of lean mass compared to tirzepatide alone over 24 weeks. These findings could open new partnering and clinical opportunities, though Scholar Rock stated it intends to work with others for large-market cardiometabolic indications instead of pursuing them independently.
Pipeline diversification into oncology, once a stated focus, saw little attention in this period. No material updates were provided on SRK-181, a checkpoint inhibitor program for cancer patients, or on other preclinical oncology research. The company did mention plans to file an Investigational New Drug (IND) application for SRK-439, another anti-myostatin antibody, in the second half of 2025, expanding their neuromuscular and potential metabolic disease pipeline.
Looking Ahead: Financial Outlook and Key Watch Points
Management reaffirmed its intention to launch apitegromab in the U.S. in late 2025, pending FDA approval, and to follow with a European launch in 2026. The company projected that its cash reserves would support operations into 2027, including funding ongoing and planned clinical trials as well as launch-related activities, based on its cash, cash equivalents, and marketable securities as of June 30, 2025.
Investors and observers should watch for the outcome of the FDA review slated for September 22, 2025, which will determine the company’s first commercial launch. The company’s heavy spending on commercial infrastructure and pipeline expansion will continue to weigh on its cash position.
Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted.
