Scholar Rock Holding (SRRK) stock price is $42.81, up 3.49% on the day, as of Oct 21, 2026. It has a market cap of $5.58Bn, and is classified in the Biotechnology industry (Healthcare sector).
Enterprise Value
Margins
P/FCF
Key Stats
Market Cap5.58 Bn
P/E-13.66
Div. Yield0.00
Total Debt (Qtr)196.20 Mn
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About
Scholar Rock Holding Corp is a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy and additional rare severe and debilitating neuromuscular diseases. The company focuses on the biology of the transforming growth factor beta superfamily and has built a proprietary platform that enables the discovery of monoclonal antibodies which bind the latent precursor forms of growth factors. By preventing activation of…
Scholar Rock Holding Corp is a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy and additional rare severe and debilitating neuromuscular diseases. The company focuses on the biology of the transforming growth factor beta superfamily and has built a proprietary platform that enables the discovery of monoclonal antibodies which bind the latent precursor forms of growth factors. By preventing activation of these molecules in the disease microenvironment Scholar Rock aims to increase muscle mass and function while preserving normal physiological processes. Its lead product candidate apitegromab is an investigational fully human monoclonal antibody administered intravenously that selectively binds pro and latent myostatin to inhibit its catabolic activity. Scholar Rock is also developing a subcutaneous formulation of apitegromab to provide patients with a convenient self administered option. A second myostatin inhibitor SRK 439 is being evaluated as a subcutaneous agent with high affinity and selectivity for myostatin over related growth factors. Beyond myostatin the company leverages its platform to create inhibitors of latent TGFβ1 for oncology indications such as SRK 181 for tumors resistant to checkpoint blockade. Additional programs target fibrosis through SRK 373 and iron restricted anemia through SRK 256. The company’s scientific foundation rests on the pioneering work of its founders Dr Timothy A Springer and Dr Leonard I Zon whose insights into growth factor activation underlie the entire approach.
Scholar Rock Holding Corp does not yet generate revenue from the sale of approved products because its product candidates remain in clinical development. The company’s current revenue stems from collaboration agreements that provide upfront payments milestone payments and potential future royalties. In December 2018 Scholar Rock entered into a three year collaboration with Gilead to discover and develop therapeutics that target TGFβ driven signaling receiving an upfront cash payment of fifty million dollars and an equity investment of thirty million dollars. Under the same agreement the company achieved a preclinical milestone in December 2019 triggering an additional twenty five million dollar payment. Scholar Rock also maintains an amended and restated collaboration with Adimab LLC under which it pays a technology access fee and is obligated to make technical milestone payments and option exercise fees upon successful development of antibodies. The Adimab agreement further requires royalty payments on net sales of any product that emerges from the collaboration. Looking ahead Scholar Rock anticipates that upon regulatory approval of apitegromab or other candidates it will begin to record product sales revenue and will earn royalties from out licensed assets. Until such time all financial inflow is derived from these partnership arrangements.
Scholar Rock Holding Corp differentiates itself in the biotechnology sector by focusing on the inhibition of latent growth factors rather than their active forms. This strategy provides a mechanistic advantage because latent forms exhibit structural diversity that allows for selective targeting of individual family members such as myostatin without affecting closely related proteins like GDF11 or activins. In the spinal muscular atrophy arena the company competes with approved SMN directed therapies including nusinersen risdiplam and onasemnogene abeparvovec and with other myostatin antagonists being investigated by Roche Biogen and NMD Pharma. Scholar Rock believes its proprietary antibody apitegromab offers a first in human demonstration of clinically meaningful motor benefit in patients already receiving background SMN therapy. For its SRC 181 program the company faces competition from firms developing TGFβ pathway inhibitors for use alongside checkpoint inhibitors such as AbbVie Roche Bicara Therapeutics Novartis Bristol Myers Squibb and Merck KGaA. The company’s intellectual property portfolio is extensive with multiple patent families covering its platform product candidates and therapeutic methods. As of the end of 2025 Scholar Rock held thirty two pending or granted patent families with core platform patents projected to expire in 2034 and myostatin related patents projected to expire between 2035 and 2043. This long lived patent estate is intended to protect its innovations and to provide freedom to operate as its candidates advance toward commercialization. Furthermore the company’s deep expertise in growth factor biology and its internal antibody discovery capabilities reinforce its competitive stance.
Scholar Rock Holding Corp serves individuals living with rare and severe neuromuscular diseases that are characterized by muscle atrophy and weakness. The primary patient populations include children and adults with spinal muscular atrophy who are receiving baseline SMN directed therapy patients with facioscapulohumeral muscular dystrophy and individuals with other neuromuscular conditions where loss of muscle mass contributes to disease progression. In oncology the company targets patients with locally advanced or metastatic solid tumors that have become resistant to immune checkpoint inhibition and who may benefit from TGFβ1 blockade to restore antitumor immunity. In the fibrosis area Scholar Rock aims to treat patients with organ specific scarring such as chronic kidney disease lung fibrosis or liver cirrhosis where excess extracellular matrix drives morbidity. For iron restricted anemia the company focuses on patients with conditions such as chronic kidney disease inflammatory bowel disease or cancer where elevated hepcidin limits iron availability. While the filing does not list specific named customers the end users of Scholar Rock’s therapies are patients who receive care in hospitals specialty infusion centers and neurology or oncology clinics. Healthcare providers including neurologists oncologists pulmonologists and hepatologists are involved in prescribing and administering the treatments. Payers such as private insurers and government programs determine coverage and reimbursement. Collaboration partners such as Gilead and Adimab support research and development but are not considered customers in the commercial sense.
Sector:HealthcareSector rationaleScholar Rock is a biopharmaceutical company developing monoclonal antibodies for rare neuromuscular diseases, oncology, and fibrosis, with lead candidates like apitegromab. Its business model is centered on the discovery and clinical development of medical products for patients in hospitals and specialty clinics, which falls squarely within the Biotechnology and Pharmaceuticals industries of the Healthcare sector.Industry:BiotechnologyBiotechnologyPrimaryScholar Rock is a biopharmaceutical company that researches and develops therapies derived from biological science, specifically monoclonal antibodies like apitegromab and SRK 439. Its current revenue is derived from collaboration and milestone payments from partners such as Gilead, which is characteristic of clinical-stage biotechnology developers.Classified using BQ-MICSCIK: 0001727196
Bull & bear
Investment Thesis
▲ Bull case
Scholar Rock is positioned for a transformative 2026 driven by the near-certain approval of apitegromab for SMA, with dual fill-finish facilities eliminating single-point manufacturing risk and enabling commercial supply readiness well ahead of the September 30 PDUFA date. The company has filed the complete drug product required for FDA review at its second U.S.-based facility, ensuring ample commercial supply in early Q3 regardless of Catalent Indiana’s classification outcome, which reduces dependency on a single contractor and provides operational resilience. This dual-path strategy not only de-risks approval timing but also supports potential label expansion, as having two compliant facilities strengthens the case for broader patient inclusion beyond the pivotal study population. Management’s emphasis on being ready to launch at any time prior to September 30 reflects confidence in the review process and aligns with historical patterns where Class II resubmissions often see early action, suggesting upside to current consensus timelines. The upcoming CHMP opinion near midyear further validates European readiness, with EMA’s comfort with the FDA’s site classification process indicating minimal regulatory friction abroad, setting the stage for a rapid transatlantic launch. Scholar Rock’s $480 million cash balance, bolstered by a $100 million debt drawdown and $98 million in ATM proceeds, provides ample runway to fund commercial execution, pipeline advancement, and potential balance sheet optimization via a priority review voucher monetization post-approval, reducing near-term financing concerns. The company’s commercial infrastructure is already battle-tested, with established relationships across 140 SMA treatment centers, 2,600 prescribing physicians, and expanding home infusion and specialty pharmacy networks, ensuring seamless patient access from day one of launch. Beyond SMA, the Phase II OPAL trial in infants and toddlers and the impending FORGE study in FSHD represent significant pipeline catalysts, with apitegromab’s mechanism showing promise in diseases where muscle atrophy is central and no approved therapies exist, potentially expanding the addressable market to over 65,000 patients globally when combining SMA and FSHD populations. The subcutaneous formulation of apitegromab, demonstrating comparable bioavailability to IV in Phase I, offers a future differentiation that could enhance adherence and broaden use in outpatient settings, particularly relevant for pediatric and chronic dosing scenarios. Scholar Rock’s focus on the muscle as the principal organ impacted in SMA — a paradigm shift from a decade of motor neuron-centric therapies — addresses an unmet need affecting 95% of patients, creating a compelling value proposition that payers and physicians are increasingly receptive to, as evidenced by 75% of treating physicians endorsing dual-modality therapy as the future standard of care.
Scholar Rock is positioned for a transformative 2026 driven by the near-certain approval of apitegromab for SMA, with dual fill-finish facilities eliminating single-point manufacturing risk and enabling commercial supply readiness well ahead of the September 30 PDUFA date. The company has filed the complete drug product required for FDA review at its second U.S.-based facility, ensuring ample commercial supply in early Q3 regardless of Catalent Indiana’s classification outcome, which reduces dependency on a single contractor and provides operational resilience. This dual-path strategy not only de-risks approval timing but also supports potential label expansion, as having two compliant facilities strengthens the case for broader patient inclusion beyond the pivotal study population. Management’s emphasis on being ready to launch at any time prior to September 30 reflects confidence in the review process and aligns with historical patterns where Class II resubmissions often see early action, suggesting upside to current consensus timelines. The upcoming CHMP opinion near midyear further validates European readiness, with EMA’s comfort with the FDA’s site classification process indicating minimal regulatory friction abroad, setting the stage for a rapid transatlantic launch. Scholar Rock’s $480 million cash balance, bolstered by a $100 million debt drawdown and $98 million in ATM proceeds, provides ample runway to fund commercial execution, pipeline advancement, and potential balance sheet optimization via a priority review voucher monetization post-approval, reducing near-term financing concerns. The company’s commercial infrastructure is already battle-tested, with established relationships across 140 SMA treatment centers, 2,600 prescribing physicians, and expanding home infusion and specialty pharmacy networks, ensuring seamless patient access from day one of launch. Beyond SMA, the Phase II OPAL trial in infants and toddlers and the impending FORGE study in FSHD represent significant pipeline catalysts, with apitegromab’s mechanism showing promise in diseases where muscle atrophy is central and no approved therapies exist, potentially expanding the addressable market to over 65,000 patients globally when combining SMA and FSHD populations. The subcutaneous formulation of apitegromab, demonstrating comparable bioavailability to IV in Phase I, offers a future differentiation that could enhance adherence and broaden use in outpatient settings, particularly relevant for pediatric and chronic dosing scenarios. Scholar Rock’s focus on the muscle as the principal organ impacted in SMA — a paradigm shift from a decade of motor neuron-centric therapies — addresses an unmet need affecting 95% of patients, creating a compelling value proposition that payers and physicians are increasingly receptive to, as evidenced by 75% of treating physicians endorsing dual-modality therapy as the future standard of care.
Despite the optimistic narrative around dual fill-finish facilities, Scholar Rock remains vulnerable to delays stemming from the Catalent Indiana reinspection, as the FDA’s 90-day classification window introduces uncertainty that could push approval beyond the September 30 PDUFA date if the facility receives anything less than a clean bill of health, and management’s reluctance to disclose specific field notes or remediation details from Novo Nordisk suggests unresolved complexities that may not be fully appreciated by the market. The company’s repeated emphasis on having “ample supply” from the second facility in early Q3 does not guarantee that this site will be accepted as the sole basis for approval, as the FDA may still require data from both facilities or insist on Catalent Indiana’s compliance for labeling purposes, creating a scenario where approval is contingent on the slower of the two paths rather than the faster. While management highlights progress at the second fill-finish facility, they avoid detailing whether critical stability, sterility, or process validation studies — typically required for new manufacturing sites — have been completed to the FDA’s satisfaction, leaving open the risk of a CMC-related hold despite the filing of drug product. The commercial launch preparations, though extensive, are predicated on approval timing, and any delay would erode the cash runway advantage, as the $480 million balance includes non-recurring ATM proceeds and debt draws that are not sustainable long-term, with operating expenses of $84 million ex-stock-based compensation implying a burn rate that could deplete reserves faster than anticipated if launch slips into 2027. European readiness remains overstated, as the CHMP opinion timeline is explicitly tied to the FDA’s classification of Catalent Indiana, meaning EMA cannot proceed independently, and the mutual agreement to cancel the oral explanation meeting may reflect regulatory fatigue rather than confidence, particularly given that EMA has historically scrutinized myostatin inhibitors more closely than the FDA due to safety concerns observed in earlier trials. The pipeline expansion into FSHD, while scientifically plausible, carries significant execution risk, as the FORGE study’s reliance on a milder patient population — a departure from Roche’s failed trial — may limit the generalizability of results and fail to capture the progressive nature of the disease, increasing the likelihood of a negative or inconclusive Phase II outcome that would undermine the broader anti-myostatin platform’s credibility. Scholar Rock’s pricing strategy lacks clarity, with no discussion of rebate structures, outcomes-based contracts, or payer concessions despite the high expected cost of apitegromab, and the absence of any mention of Medicaid-specific negotiations is concerning given that over 50% of SMA patients rely on public payers, potentially creating access barriers that could slow adoption even post-approval. Finally, the company’s continued dependence on a single product — apitegromab — for near-term value creation means that any setback in SMA approval or label restriction would have a disproportionate impact on the stock, with limited diversification to buffer against clinical or regulatory failure in its core franchise.
Despite the optimistic narrative around dual fill-finish facilities, Scholar Rock remains vulnerable to delays stemming from the Catalent Indiana reinspection, as the FDA’s 90-day classification window introduces uncertainty that could push approval beyond the September 30 PDUFA date if the facility receives anything less than a clean bill of health, and management’s reluctance to disclose specific field notes or remediation details from Novo Nordisk suggests unresolved complexities that may not be fully appreciated by the market. The company’s repeated emphasis on having “ample supply” from the second facility in early Q3 does not guarantee that this site will be accepted as the sole basis for approval, as the FDA may still require data from both facilities or insist on Catalent Indiana’s compliance for labeling purposes, creating a scenario where approval is contingent on the slower of the two paths rather than the faster. While management highlights progress at the second fill-finish facility, they avoid detailing whether critical stability, sterility, or process validation studies — typically required for new manufacturing sites — have been completed to the FDA’s satisfaction, leaving open the risk of a CMC-related hold despite the filing of drug product. The commercial launch preparations, though extensive, are predicated on approval timing, and any delay would erode the cash runway advantage, as the $480 million balance includes non-recurring ATM proceeds and debt draws that are not sustainable long-term, with operating expenses of $84 million ex-stock-based compensation implying a burn rate that could deplete reserves faster than anticipated if launch slips into 2027. European readiness remains overstated, as the CHMP opinion timeline is explicitly tied to the FDA’s classification of Catalent Indiana, meaning EMA cannot proceed independently, and the mutual agreement to cancel the oral explanation meeting may reflect regulatory fatigue rather than confidence, particularly given that EMA has historically scrutinized myostatin inhibitors more closely than the FDA due to safety concerns observed in earlier trials. The pipeline expansion into FSHD, while scientifically plausible, carries significant execution risk, as the FORGE study’s reliance on a milder patient population — a departure from Roche’s failed trial — may limit the generalizability of results and fail to capture the progressive nature of the disease, increasing the likelihood of a negative or inconclusive Phase II outcome that would undermine the broader anti-myostatin platform’s credibility. Scholar Rock’s pricing strategy lacks clarity, with no discussion of rebate structures, outcomes-based contracts, or payer concessions despite the high expected cost of apitegromab, and the absence of any mention of Medicaid-specific negotiations is concerning given that over 50% of SMA patients rely on public payers, potentially creating access barriers that could slow adoption even post-approval. Finally, the company’s continued dependence on a single product — apitegromab — for near-term value creation means that any setback in SMA approval or label restriction would have a disproportionate impact on the stock, with limited diversification to buffer against clinical or regulatory failure in its core franchise.