Scholar Rock Holding Corporation (SRRK): Business Model Canvas [Dec-2025 Updated]

You're looking at Scholar Rock Holding Corporation (SRRK) right now, trying to map out exactly how they plan to turn their proprietary science into dollars, especially with apitegromab nearing a potential 2026 launch for Spinal Muscular Atrophy (SMA). Honestly, the Q3 2025 numbers tell a clear story: they are definitely in the investment phase, spending $50.5 million on Research and Development and $53.1 million on General and Administrative costs, all while sitting on $369.6 million in cash, before seeing a dime of product revenue. This canvas breaks down the nine critical blocks defining how Scholar Rock Holding Corporation is structuring its key activities, from late-stage trials to commercial prep, and where the value proposition for their muscle-targeted therapy truly sits against current standards of care. Dive in below to see the framework that maps their path from platform innovation to market reality.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Key Partnerships

You're looking at the essential external relationships Scholar Rock Holding Corporation uses to get its product pipeline, especially apitegromab, to market. These partnerships are critical, especially given the recent manufacturing hurdles and the company's pre-commercial status.

Third-party contract manufacturing organizations (CMOs) for drug supply represent a significant operational dependency. Scholar Rock Holding Corporation is actively managing supply chain risk by accelerating the transfer of commercial manufacturing capacity to a second facility, with commercial capacity reserved beginning in Q1 2026.

The relationship with Catalent Indiana LLC (now owned by Novo Nordisk) for fill-finish services has faced regulatory headwinds. Scholar Rock Holding Corporation reported that Catalent Indiana received an FDA warning letter following an inspection classification of "official action indicated" (OAI) on October 10, 2025, stemming from observations first cited in a Form 483 on July 14, 2025. Scholar Rock Holding Corporation is working closely with Novo Nordisk to address these issues, which previously led to a Complete Response Letter for apitegromab. The company still anticipates resubmitting its biologics license application (BLA) in 2026.

The financial commitment to manufacturing readiness is visible in the operating expenses. For the quarter ended September 30, 2025, Research and development expense was $50.5 million, which included $5.5 million in stock-based compensation, with increases driven partly by investment in commercial manufacturing and launch readiness for apitegromab.

The table below summarizes the key manufacturing/supply chain partner status as of late 2025:

The strategic alliance with Gilead Sciences for collaboration on fibrotic disease therapies provides non-dilutive funding and validation for Scholar Rock Holding Corporation's platform. The initial agreement terms included:

• Upfront Payment: $80 million total.
• Upfront Cash Component: $50 million.
• Upfront Stock Purchase Component: $30 million.
• Potential Milestone Payments: Up to an aggregate of $1,425 million across three programs upon achievement of research, development, regulatory, and commercialization milestones.

Scholar Rock Holding Corporation retains exclusive worldwide rights to discover, develop, and commercialize certain transforming growth factor beta (TGFβ) inhibitors for oncology and cancer immunotherapy within this partnership structure. This collaboration underpins a portion of the company's financial stability; as of September 30, 2025, Scholar Rock Holding Corporation had $369.6 million in cash, cash equivalents, and marketable securities, with an anticipated runway into 2027.

For Academic research institutions for early-stage discovery, Scholar Rock Holding Corporation leverages external scientific expertise to fuel its pipeline beyond apitegromab. While the company is advancing SRK-439, for which the Investigational New Drug (IND) application was cleared in Q4 2025, specific financial details regarding direct payments or grants to academic partners are not publicly itemized in the latest financial disclosures.

The commercial readiness team, a key operational partnership with the market, was planned to be fully staffed by mid-2025, comprising roughly 50 sales, reimbursement, and patient support personnel.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Key Activities

You're looking at the core engine driving Scholar Rock Holding Corporation right now-it's all about execution on their lead asset and advancing the next wave of pipeline candidates. Honestly, the numbers tell a story of heavy investment ahead of potential revenue.

Late-stage clinical development of apitegromab for SMA.

The focus here is on regulatory submission follow-up and expanding the patient base. While the Prescription Drug User Fee Act (PDUFA) target action date was set for September 22, 2025, the company is now planning to resubmit the Biologics License Application (BLA) following constructive discussions with the FDA, moving the potential U.S. launch to 2026. The European Medicines Agency (EMA) regulatory review is ongoing, with a decision anticipated by mid-2026. Operationally, Scholar Rock Holding Corporation initiated dosing in the Phase 2 OPAL clinical trial for infants and toddlers under two years old with SMA in Q3 2025. The financial burn is clear: the net loss for the third quarter ended September 30, 2025, was $102.22 million, resulting in a net loss per common share of $0.90 for that period.

Here's a quick look at the recent financial health supporting this development:

The company expects this cash position, plus anticipated warrant exercises of approximately $60 million expiring December 31, 2025, to fund operations into 2027.

Commercial launch preparation for apitegromab in the U.S. and Europe.

Preparation is a major cost driver right now. For the second quarter ended June 30, 2025, General & Administrative (G&A) expenses surged to $49.7 million, up from $17.1 million year-over-year, largely due to stock-based compensation and these commercial launch preparations. The company has hired and deployed U.S. customer-facing teams and is actively progressing launch preparedness in Germany for its first European market. They also anticipate monetizing a priority review voucher following approval.

Advancing SRK-439 for cardiometabolic disorders into the clinic.

Scholar Rock Holding Corporation is targeting an Investigational New Drug (IND) filing for SRK-439 in the second half of 2025, with specific plans pointing to Q3 2025. This novel anti-myostatin program is being developed to preserve lean muscle mass in patients undergoing weight loss treatments. Preclinical studies showed that SRK-439 protected against tirzepatide-induced muscle loss in diet-induced obese (DIO) mice, with lean mass preservation seen at doses as low as 0.3 mg/kg.

Research and development (R&D) expenses reflect this pipeline advancement; for Q2 2025, R&D was $62.4 million, an increase of $20.0 million year-over-year, driven partly by drug supply manufacturing costs.

Proprietary platform research on latent growth factor modulation.

The proprietary platform is the foundation, enabling the development of novel monoclonal antibodies designed to modulate protein growth factors with extraordinary selectivity. This technology is being applied across several areas, as evidenced by the pipeline:

• Apitegromab development is expanding into at least one additional neuromuscular indication, with clinical development expected to initiate by the end of 2025.
• The platform is also being used to develop SRK-439, which has shown potential in preclinical models to support healthier weight management by preserving lean mass.

The company did not record any revenue for the third quarter ended September 30, 2025.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Key Resources

You're looking at the core assets Scholar Rock Holding Corporation is counting on as it transitions toward a commercial focus. These aren't just ideas; they are tangible and intangible assets that underpin their entire strategy.

The most immediate resource is the capital base. As of the end of the third quarter of 2025, Scholar Rock Holding Corporation reported cash, cash equivalents, and marketable securities of approximately $369.6 million. This financial position is expected to be sufficient to fund operations into 2027.

The scientific foundation is the proprietary protein growth factor modulation platform. This platform is responsible for developing novel monoclonal antibodies designed to modulate protein growth factors with extraordinary selectivity. This forms the basis of their world-leading, highly selective anti-myostatin approach, which has successfully delivered apitegromab and is being expanded with assets like SRK-439.

The intellectual property portfolio protecting lead candidates is a direct output of this platform. The legal and executive team is tasked with managing legal risks and safeguarding this intellectual property. The company is focused on its ability to obtain, maintain, and protect this IP.

Clinical data from the Phase 3 SAPPHIRE trial for apitegromab represents a critical, validated resource. The trial enrolled 156 patients aged 2-12 in the main efficacy population, randomized 1:1:1 across two apitegromab doses and placebo. The data demonstrated a statistically significant 1.8-point improvement (p=0.0192) on the Hammersmith Functional Motor Scale-Expanded (HFMSE) at week 52 for the combined apitegromab dose versus placebo. This positions the company to target a Spinal Muscular Atrophy (SMA) market estimated at approximately $5 billion annually.

The specialized commercial and R&D executive team is actively engaged in scaling operations. For the quarter ended September 30, 2025, operating expenses totaled $103 million, reflecting significant investment in launch readiness infrastructure. The General and administrative expense for that quarter alone was $53.1 million.

Here is a summary of the key quantitative resources as of late 2025:

The company's internal capabilities also include the development of next-wave assets built on the same core science:

• SRK-439 IND filing targeted for the second half of 2025.
• Apitegromab is being advanced into additional rare neuromuscular disorders.
• Phase 2 EMBRAZE trial data in obesity was expected in mid-2025.
• The team includes leadership overseeing legal affairs and intellectual property.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Value Propositions

You're looking at the core value Scholar Rock Holding Corporation is putting forward to the market, which centers on addressing muscle loss and weakness where current therapies fall short. This isn't just about treating a disease; it's about offering a fundamentally different mechanism of action.

Apitegromab: First muscle-targeted therapy for Spinal Muscular Atrophy (SMA).

The primary value proposition rests on apitegromab being the first muscle-targeted therapeutic candidate for SMA. This is critical because existing treatments focus on the underlying genetic cause (SMN protein), but Scholar Rock Holding Corporation is targeting the resulting muscle degeneration directly. The SMA market in the U.S. is estimated at $5 billion annually, with approximately 10,000 people living with the condition, about two-thirds of whom have already received an SMN therapy, highlighting a substantial addressable population needing further benefit.

Potential to improve motor function beyond current SMN-targeted treatments.

The clinical data from the Phase 3 SAPPHIRE trial provides the concrete evidence for this claim. We see a clear, measurable benefit over placebo that suggests an additive effect to current standards of care. The drug demonstrated a 1.8-point improvement (p=0.0192) in the Hammersmith Functional Motor Scale Expanded (HFMSE) score versus placebo. Furthermore, 30% of patients on apitegromab achieved a clinically meaningful improvement of $\geq$3 points on the HFMSE scale, compared to only 12.5% of placebo patients. Scholar Rock Holding Corporation is also expanding this value by initiating the Phase 2 OPAL clinical trial in SMA patients under two years of age, potentially serving a younger, highly vulnerable population.

Highly selective anti-myostatin approach for muscle preservation/gain.

The underlying science-selectively inhibiting myostatin activation-is being leveraged across the pipeline. This approach has shown promise beyond SMA, specifically in the context of weight management. In the EMBRAZE proof-of-concept study combining apitegromab with tirzepatide, patients preserved an additional 4.2 pounds (1.9 kilograms) of lean mass, representing 54.9% (p=0.001) of the total weight loss over 24 weeks. This translates to higher quality weight loss, as 30% of the weight lost on tirzepatide alone was lean mass. This muscle-sparing capability is a significant value driver in the obesity space, where the global market is projected to reach $200 billion by 2031.

The pipeline momentum, supported by a cash balance of $369.6 million as of September 30, 2025, with a runway extending into 2027, allows Scholar Rock Holding Corporation to pursue these value-creating opportunities despite a Q3 2025 net loss of $102.2 million.

Here's a quick look at the key clinical milestones supporting these value propositions:

Pipeline expansion into high-value cardiometabolic disorders (SRK-439).

Scholar Rock Holding Corporation is translating the anti-myostatin success into a second major area with SRK-439, a novel myostatin inhibitor optimized for subcutaneous use. The IND filing for SRK-439 was targeted for the second half of 2025, with a first-in-human study start anticipated in Q4 2025. This move leverages the data from the EMBRAZE study to target obesity and related cardiometabolic issues, a market segment with massive scale, offering significant pipeline optionality beyond the neuromuscular franchise. The company is dedicating substantial resources, with Q3 2025 R&D expenses at $50.5 million.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Relationships

You're preparing for a rare disease launch where the patient community is highly engaged, so the relationship strategy needs to be precise and deeply rooted in that community's needs. Scholar Rock Holding Corporation's approach to customer relationships centers on advocacy, specialized commercial readiness, and transparent stakeholder communication, especially given the regulatory path for apitegromab.

High-touch engagement with patient advocacy groups (e.g., Cure SMA).

Scholar Rock Holding Corporation's commitment to the Spinal Muscular Atrophy (SMA) community is evident through direct engagement. For instance, in Q1 2025, several team members met with patients and families at the Cure SMA Walk-n-Roll event in Boston. Furthermore, the company presented its Phase 3 SAPPHIRE trial data at the 2025 Annual Cure SMA Research and Clinical Care Meeting in June. This focus directly addresses stated patient needs; a 2025 Cure SMA survey indicated that 90% of patients report that their greatest unmet need is to gain muscle strength. Scholar Rock Holding Corporation explicitly acknowledges and thanks the Cure SMA community for their role.

Direct sales force and medical affairs team for specialty providers.

To prepare for the anticipated U.S. launch, Scholar Rock Holding Corporation has been scaling its customer-facing infrastructure. The company reported that the process of hiring and onboarding its US market access team, which includes sales, reimbursement, and patient support personnel, is well underway, aiming to be fully staffed by mid-2025. This team is roughly 50 strong. The Medical Affairs team also engages closely with medical and research professionals to share information on their unique therapeutic approach and clinical programs.

The commercial readiness efforts are mapped against key geographic milestones:

Managed access programs for rare disease therapies post-approval.

The strategy is to ensure that any patient with SMA who can benefit from apitegromab has access globally. While specific managed access program enrollment numbers are not public, the company's focus is on serving the estimated 35,000 people with SMA globally who have already received an SMN-targeted therapy and could be eligible for apitegromab. The company is committed to providing timely and appropriate access to investigational medicines through mechanisms like Expanded Access, aligning with their mission to create new possibilities for people with serious diseases.

Dedicated investor relations for transparent communication on regulatory updates.

Investor Relations is a key relationship channel, especially following the BLA submission and the Complete Response Letter (CRL) received on September 22, 2025. Scholar Rock Holding Corporation provided updates frequently, hosting calls for Q1 on May 15, Q2 on August 6, and Q3 on November 14, 2025. The company ended Q3 2025 with $369.6 million in cash and cash equivalents. Operating expenses for Q3 2025 were $103 million, which included $18.3 million in noncash stock-based compensation. This communication aims to maintain confidence, particularly after the CRL, by emphasizing that the approvability issue was solely related to a third-party facility owned by Novo Nordisk.

Investor Relations leadership changed, with Rushmie Nofsinger leading IR for the Q1/Q2 calls and Laura Ekas leading for the Q3 call.

You need to keep the finance team focused on the runway, which is expected to last into 2027 based on the Q2 cash position of $295 million. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Channels

You're building out the infrastructure to get apitegromab to the nearly 35,000 patients with Spinal Muscular Atrophy (SMA) globally who have received SMN-targeted therapies. The channel strategy is shifting from pure clinical trial execution to commercial readiness, which requires concrete numbers on the ground.

Specialty pharmacies and distributors for drug delivery.

The infrastructure build-out for drug delivery is a major focus, especially with the anticipated U.S. launch now targeted for 2026 following regulatory updates. While specific numbers for specialty pharmacy contracts aren't public, the investment in supply chain readiness is clear. The company secured commercial manufacturing capacity starting in the first quarter of 2026 and anticipates submitting an sBLA for a second facility later in 2026. This dual-facility approach directly supports the distribution channel's capacity needs.

Direct commercial team for U.S. market access and sales.

Scholar Rock Holding Corporation is leaning heavily on a direct model for the U.S. market. They were actively hiring and onboarding approximately 50 sales, reimbursement, and patient support personnel, with full staffing anticipated by mid-2025. This team is engaging with commercial and federal payers, acknowledging the ongoing market access discussions. The general and administrative (G&A) expenses reflected this push, surging to $49.7 million in the second quarter of 2025, up from $17.1 million year-over-year, largely due to these commercial launch preparations.

Healthcare providers and specialized neuromuscular clinics.

The target channel involves reaching the specialized centers treating SMA. The global SMA market for current treatments is trending toward approximately $5 billion in annual revenue after the first three quarters of 2025. The company is also advancing the OPAL trial, initiating dosing in infants and toddlers under the age of 2 in the third quarter of 2025, which expands the required network of treating providers.

Potential future ex-U.S. partnerships for global commercialization.

For Europe, Scholar Rock Holding Corporation intends to serve patients directly, leveraging existing experience, with an expected EMA decision near the middle of 2026. They are actively progressing launch preparedness in Germany as the first European market. Beyond the U.S. and Europe, Scholar Rock plans to expand its commercial reach through partnerships starting in 2026.

Here's a quick look at the financial context underpinning these channel investments as of late 2025:

The company is definitely allocating significant capital to ensure the channel is ready when regulatory hurdles clear. The Q3 2025 operating expenses included $18.3 million in noncash stock-based compensation.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Segments

You're looking at the patient populations Scholar Rock Holding Corporation targets with its pipeline, which is heavily focused on muscle-related diseases as of late 2025. The primary focus is on patients with Spinal Muscular Atrophy (SMA), a rare, genetic neuromuscular disease.

For SMA, the estimated patient base is significant across key markets. Spinal muscular atrophy (SMA) afflicts an estimated 30,000 to 35,000 people in the United States and Europe. Breaking that down, approximately 9,000 people in the US live with SMA. Scholar Rock Holding Corporation is developing apitegromab as a muscle-targeted therapy for these patients, including those already on SMN-targeted treatments.

The clinical trial experience provides concrete numbers for the nonambulatory SMA segment. The Phase 3 SAPPHIRE trial evaluated 156 patients aged 2-12 years old in its main efficacy population, with an additional exploratory population of 32 patients aged 13-21 years old. Furthermore, the company is expanding into the youngest patients, with the Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA under two years of age.

Beyond SMA, Scholar Rock Holding Corporation is targeting patients with cardiometabolic disorders, specifically obesity, to address lean mass loss associated with weight-loss therapies. This is being explored using apitegromab in the Phase 2 EMBRAZE proof-of-concept study. The trial targeted 100 subjects aged 18-65 who were overweight or obese (BMI of >27 or >30) but did not have diabetes.

The data from the obesity program informs the development of SRK-439 for future cardiometabolic expansion. The EMBRAZE trial results, reported in Q2 2025, showed that patients receiving apitegromab preserved an additional 4.2 pounds (1.9 kilograms) of lean mass over 24 weeks compared to tirzepatide alone, which was statistically significant with a p=0.001. This preservation represented 54.9% of the lean mass preservation benefit. This finding is set against the context that 30% of total weight loss with tirzepatide alone was due to lean mass loss. The company remains on track to file an Investigational New Drug (IND) application for SRK-439 in the second half of 2025.

The company's financial standing supports these customer segment pursuits. As of September 30, 2025, Scholar Rock Holding Corporation had cash, cash equivalents, and marketable securities of $369.6 million, expected to fund operations into 2027.

Here's a quick math summary of the patient populations and trial sizes:

The customer segments Scholar Rock Holding Corporation is engaging with or planning to engage with include:

• Children and adults with Spinal Muscular Atrophy (SMA) receiving SMN-targeted treatment.
• Nonambulatory SMA patients aged 2-12 in the SAPPHIRE trial main population.
• SMA patients under two years of age targeted by the Phase 2 OPAL study.
• Adults with obesity/overweight receiving GLP-1 RA therapy, with 100 subjects targeted in the EMBRAZE trial.
• Patients with other rare, severe neuromuscular disorders, including preclinical work in a Duchenne muscular dystrophy (DMD) model.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Cost Structure

You're looking at the cost side of Scholar Rock Holding Corporation's business as they push toward a potential commercial launch. For a pre-revenue biotech, this structure is dominated by getting the science and the supply chain ready. Here's the quick math on what they spent in the third quarter of 2025.

The primary cost drivers reflect the intense, non-recurring investments needed to transition from clinical development to a commercial-ready entity, especially after the regulatory setback earlier in the year.

The total operating expenses for Scholar Rock Holding Corporation in Q3 2025 hit $103 million. Excluding the non-cash component, operating expenses were $85.3 million.

Here is a breakdown of the major expense categories for the quarter ended September 30, 2025:

You'll see that the R&D and G&A figures provided in the outline add up to more than the total operating expense because the SBC is double-counted in the line items but correctly accounted for in the total OpEx calculation when using the non-SBC figure. The total R&D and G&A spend for the quarter was $103.6 million ($50.5M + $53.1M), which aligns closely with the reported total operating expense of $103 million, accounting for rounding or minor classification differences in the reported figures.

Specifics on other major cost areas include:

• - Manufacturing and drug supply costs for commercial inventory build are a primary driver of the increased R&D expense year-over-year.
• - Clinical trial expenses support the ongoing OPAL Phase 2 study in infants and toddlers with Spinal Muscular Atrophy (SMA) and the planned start of Phase 1 for SRK-439 in Q4 2025.
• - Personnel costs are embedded within both R&D and G&A, with G&A specifically citing headcount and severance costs contributing to its rise.

The company ended Q3 2025 with $369.6 million in cash, cash equivalents, and marketable securities, which management expects will fund operations into 2027. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Scholar Rock Holding Corporation's business model as of late 2025. Honestly, for a company at this stage, the revenue streams are almost entirely potential, tied to clinical success and regulatory approvals. The current financial reality reflects a pre-commercial entity heavily focused on R&D investment.

The most immediate data point from the third quarter of 2025 shows a clear picture of their current revenue generation status.

• - Scholar Rock Holding Corporation did not record any product revenue for the quarter ended September 30, 2025, which is expected for a company in this phase. This translates to $0.0 million in product revenue for Q3 2025.

The primary near-term revenue driver is the anticipated commercialization of apitegromab for Spinal Muscular Atrophy (SMA). You know the Biologics License Application (BLA) faced a delay due to a third-party manufacturing site issue, but management is pushing hard now. They anticipate resubmitting the BLA and launching apitegromab in the U.S. in 2026, pending FDA reinspection results. The European Medicines Agency (EMA) decision is expected around the middle of 2026. This product targets the SMA market, which is estimated to be worth approximately $5 billion annually.

The structure of the strategic collaboration with Gilead Sciences provides a clear, albeit contingent, revenue framework. This deal, focused on TGF$\beta$ inhibitors for fibrotic diseases, is a classic biotech financing mechanism, securing non-dilutive cash and future upside. Here's a breakdown of the key financial components from that agreement:

Beyond apitegromab, Scholar Rock Holding Corporation is advancing other pipeline assets, which represent longer-term, more speculative revenue opportunities. The next concrete step in this area involves SRK-439, a next-generation myostatin inhibitor. The company cleared the Investigational New Drug (IND) application for this asset and plans to initiate first-in-human dosing in the fourth quarter of 2025. Also, Scholar Rock Holding Corporation expects to initiate clinical development activities in a second neuromuscular indication by the end of 2025.

To summarize the non-product revenue sources that underpin operations:

• - Milestone payments from the Gilead collaboration have already included a $25 million payment.
• - The potential for future milestone payments from Gilead remains significant, totaling up to $1,425 million across the programs.
• - Royalties are structured as high single-digit to low double-digit tiers on any future commercial products from the collaboration.
• - SRK-439 is set to enter Phase 1 clinical trials in Q4 2025, which will precede any revenue generation from that program.

Finance: draft 13-week cash view by Friday.