Scholar Rock FY Conference Summary Company Overview - **Company**: Scholar Rock (NasdaqGS:SRRK) - **Industry**: Biotechnology - **Foundation**: Established in 2012, celebrating its fiftieth anniversary in 2026 - **Focus**: Development of antibody technology to inhibit difficult growth factors, particularly myostatin [2][3] Core Points and Arguments - **Myostatin Inhibition Success**: Scholar Rock has achieved a significant milestone by successfully inhibiting myostatin, leading to the first positive pivotal trial for treating spinal muscular atrophy (SMA) after decades of attempts by the industry [3] - **Regulatory Approvals**: The company is awaiting FDA and EMA approval for its lead asset, apitegromab, with plans for commercial launches in the U.S. and Europe in 2026 [5][49] - **Debt Instrument**: Scholar Rock announced a $550 million debt instrument to fund operations, with an additional $100 million expected to be drawn down by the end of the quarter [5][9] - **Clinical Trials**: The company is advancing its pipeline with key Phase 2 clinical trials, including: - OPAL study for apitegromab combined with gene therapy for infants and toddlers with SMA [6] - FORGE study for facioscapulohumeral muscular dystrophy (FSHD) involving 60 patients [59][61] Financial Position - **Cash Balance**: Ended the year with a cash balance of $368 million before the additional $100 million drawdown [9] - **Priority Review Voucher (PRV)**: Scholar Rock qualifies for a rare pediatric disease priority review voucher, expected to be monetized upon approval, with a value ranging between $150 million and $200 million [11][33] Market Strategy - **Global Expansion**: Plans to build a 50-country operating platform, targeting approximately 35,000 patients with SMA, with a focus on Germany as the first European market post-EMA approval [49][50] - **Patient Assistance Programs**: Launching "Scholar Rock Supports" and "Life Takes Muscle" disease education programs to assist patients and families [16][17] Regulatory Challenges - **Compliance Issues**: The delay in approval for apitegromab is due to compliance issues at the fill-finish facility owned by Novo Nordisk, which has affected the timeline for resubmission [13][27] - **Resubmission Timeline**: The company anticipates a Class 2 resubmission for FDA review, which could take up to six months, but is hopeful for a quicker turnaround based on previous experiences [29][32] Innovation and Future Directions - **Subcutaneous Formulation**: Development of a subcutaneous version of apitegromab is underway, with plans to stage discussions with regulators post-approval of the IV formulation [52][57] - **Focus on FSHD**: The rationale for targeting FSHD with apitegromab is based on the success in SMA and the potential to address muscle dysfunction in this patient population [61][64] Additional Insights - **KOL Engagement**: Ongoing discussions with key opinion leaders (KOLs) in the SMA community have been positive, highlighting the need for muscle-targeted therapies [17][19] - **Market Access in Germany**: Germany is expected to be the first country for launch in Europe due to established access and reimbursement processes [45][49] This summary encapsulates the key points discussed during the Scholar Rock FY Conference, highlighting the company's strategic direction, financial health, regulatory challenges, and innovative approaches in the biotechnology sector.

Scholar Rock (NasdaqGS:SRRK) FY Conference Summary Company Overview - Scholar Rock is focused on developing innovative therapies targeting muscle-related diseases, particularly Spinal Muscular Atrophy (SMA) through its anti-myostatin pipeline, including the drug apitegromab [3][4] Key Value Creation Milestones - The company anticipates a transformative year in 2026 with the expected regulatory approvals for apitegromab in the US and Europe, aiming to establish a commercial presence in 50 countries [4][19] - Plans to develop apitegromab and a second myostatin inhibitor, SRK-439, for various severe and rare neuromuscular disorders [5][6] Regulatory and Manufacturing Updates - The initial launch of apitegromab was expected in 2025 but faced delays due to compliance issues at the manufacturing facility in Indiana, owned by Novo Nordisk [12][13] - The FDA granted a priority review for the drug, with an action date of September 22, 2025, but a complete response letter was received due to manufacturing facility compliance issues [12][14] - Positive progress has been made in remediating the facility, with a successful Type A meeting with the FDA and ongoing inspections [16][17] - The company is also preparing a second fill-finish facility to ensure supply chain robustness, which could support a 2026 launch even if the Indiana facility inspection does not go as planned [18][45] Market Opportunity and Pricing Strategy - Scholar Rock sees a multi-billion dollar opportunity in the SMA market alone, with a patient population of approximately 35,000 globally [6][50] - Pricing strategy will consider the rarity and severity of SMA, the impact of the drug on motor function, and the competitive landscape of existing therapies [51][52] Launch Strategy and Patient Engagement - The company expects robust demand for apitegromab, particularly from patients currently on therapy, with over 90% of trial participants remaining in long-term studies [53][54] - Initial launch uptake may be tempered by prior authorization requirements and medical policies from payers, but the company is optimistic about converting enrollment forms into reimbursed patients over time [56][57] Future Developments - Scholar Rock is also exploring a subcutaneous formulation of apitegromab and a next-generation myostatin inhibitor, SRK-439, which shows promise in early trials [58][61] - The company has initiated a Phase 2 study for Facioscapulohumeral Muscular Dystrophy (FSHD), with plans to dose patients later this year [64][65] Conclusion - Scholar Rock is positioned to become a significant player in the biotech space with its innovative therapies targeting muscle diseases, particularly SMA, while navigating regulatory challenges and preparing for a robust market entry in 2026 [4][19][56]

Core Insights - Scholar Rock is preparing to resubmit the biologics license application (BLA) for apitegromab following a successful FDA reinspection of the Catalent Indiana facility, with a targeted U.S. launch in 2026 [2][6][12] Regulatory Updates - The FDA issued a warning letter to the Catalent Indiana facility after a Type A meeting in November, but subsequent meetings indicated no additional requests for the remediation plan [1] - The FDA plans to conduct a site reinspection after routine manufacturing activities resumed in late February [1] - The compliance status of the Catalent Indiana site is the sole issue affecting the approval of the BLA [2][6] Financial Position - Scholar Rock ended 2025 with $368 million in cash and secured a new debt facility of up to $550 million, with staged draws tied to milestones [4][20] - The company plans to monetize a Priority Review Voucher to strengthen its balance sheet further [4][20] Commercial Infrastructure - The company has developed a commercial infrastructure, including specialty pharmacy access and a home-infusion network with over 10,000 affiliated nurses [5][12] - Initial access and reimbursement challenges are anticipated, particularly related to J-codes and Medicaid processes [5][12] Pipeline Developments - Scholar Rock is advancing a second fill-finish facility to support future commercial demand, with engineering runs underway [8][9] - The company is also progressing in its anti-myostatin pipeline, including ongoing studies for apitegromab in infants and toddlers [15][19] European Market Strategy - The marketing authorization application (MAA) review for apitegromab is ongoing with the European Medicines Agency (EMA), and a decision is expected in mid-2026 [11][12] - Preparations for a European launch include establishing local leadership and progressing reimbursement planning [14]

Financial Data and Key Metrics Changes - For the fourth quarter of 2025, the company reported operating expenses of $91.9 million, which included $90.4 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $72.5 million [26] - For the year ended 2025, total operating expenses were $384.6 million, including $75.6 million in non-cash stock-based compensation, leading to adjusted operating expenses of $309 million [27] - The company ended 2025 with $368 million in cash and cash equivalents, bolstered by $60.4 million from the exercise of warrants [11][27] Business Line Data and Key Metrics Changes - The company is focused on the launch of apitegromab, the world's first muscle-targeted therapy for SMA, with preparations underway for a U.S. launch in 2026 [4][11] - The company is also advancing its anti-myostatin pipeline, including apitegromab and SRK-439, with ongoing clinical trials and promising data from phase 1 studies [10][20] Market Data and Key Metrics Changes - The global market for SMA therapies is currently valued at nearly $5 billion in annual sales, with a significant unmet need for muscle-targeted therapies [21] - The company is engaging with approximately 140 SMA treatment centers and 2,600 prescribing physicians in the U.S. to enhance disease education and awareness [22] Company Strategy and Development Direction - The company aims to deliver apitegromab to the SMA community as quickly as possible while also expanding its pipeline for other rare neuromuscular diseases [4][12] - The company is building a 50-country operating platform in Europe and enhancing its commercial capabilities to support the launch of apitegromab [12][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing engagement with the FDA and the urgency to bring apitegromab to market, reaffirming guidance for BLA resubmission and U.S. launch in 2026 [7][29] - The company remains focused on long-term shareholder value while navigating the complexities of regulatory approvals and market readiness [28][91] Other Important Information - The company secured a new debt facility for up to $550 million to support its commercial and R&D priorities, with immediate access to $100 million [11][28] - The company is preparing for a supplemental BLA submission for a second fill-finish facility later in 2026 [14][27] Q&A Session Summary Question: Is Novo now ready for re-inspection? - Management confirmed that the gating item is a successful re-inspection of the Catalent Indiana facility, which would trigger the BLA resubmission [33][34] Question: What happens if the backup facility is needed? - Management indicated that whether it is a supplemental BLA or a fallback to the backup facility, they do not expect significant delays in timelines [36][37] Question: What was the purpose of the FDA sending a field team? - Management noted that the field team's visit indicates the FDA's high priority on the remediation of the Catalent facility and the urgency to progress the apitegromab file [40][42] Question: What are the expectations for the label regarding age subgrouping? - Management anticipates a straightforward alignment with the FDA on the final label after BLA resubmission, considering the comprehensive data already presented [62] Question: What are the expectations for the launch trajectory? - Management expects robust demand for apitegromab but acknowledges potential access speed bumps due to payer reimbursement processes [69] Question: Will the MAA review be affected by the Catalent facility? - Management confirmed that progress with the FDA will positively influence the MAA review process with the EMA [81][82]

Financial Data and Key Metrics Changes - For Q4 2025, the company reported operating expenses of $91.9 million, including $90.4 million in non-cash stock-based compensation, while excluding stock-based compensation, operating expenses were $72.5 million [24] - For the full year 2025, operating expenses totaled $384.6 million, with $75.6 million in non-cash stock-based compensation, and $309 million when excluding stock-based compensation [25] - The company ended 2025 with $368 million in cash and cash equivalents, bolstered by $60.4 million from the exercise of warrants [9][25] Business Line Data and Key Metrics Changes - The company is focused on the commercial launch of apitegromab, the first muscle-targeted therapy for SMA, with preparations underway for a U.S. launch in 2026 [6][19] - The company is also advancing its anti-myostatin pipeline, including apitegromab and SRK-439, with ongoing clinical trials and promising data from phase 1 studies [8][18] Market Data and Key Metrics Changes - The global market for SMA therapies is currently valued at nearly $5 billion in annual sales, with a significant unmet need for muscle strength and motor function treatments [20] - The company is engaging with approximately 140 SMA treatment centers and 2,600 prescribing physicians in the U.S. to enhance disease education and awareness [21] Company Strategy and Development Direction - The company aims to deliver apitegromab to the SMA community as quickly as possible while also expanding its pipeline for other rare neuromuscular diseases [3][10] - The company is building a 50-country operating platform in Europe and preparing for a launch in Germany in the second half of 2026 [10][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing engagement with the FDA and the urgency to bring apitegromab to market, anticipating a successful BLA resubmission following a reinspection of the Catalent Indiana facility [5][11] - The company remains focused on its strategic priorities and is optimistic about the transformative potential of its therapies for patients with SMA and other neuromuscular diseases [10][28] Other Important Information - The company secured a new debt facility for up to $550 million to support its commercial and R&D activities, with $100 million immediately available for use [25][27] - The company is also planning to monetize a Priority Review Voucher to further strengthen its balance sheet [27] Q&A Session Summary Question: Is Novo now ready for re-inspection? - Management confirmed that the gating item is a successful re-inspection of the Catalent Indiana facility, which would trigger the BLA resubmission [31][32] Question: What would happen if the backup facility is needed? - Management indicated that whether it is a supplemental BLA or a primary filing, they do not expect significant delays in timelines [34][36] Question: What was the purpose of the FDA sending a field team? - Management noted that the field team's visit indicates the FDA's high priority on the remediation of the Catalent facility and the urgency to bring apitegromab to patients [38][40] Question: What are the expectations for the label regarding age subgrouping? - Management anticipates a straightforward alignment with the FDA on the final label after BLA resubmission, given the progress made during the original review process [60] Question: What are the expectations for time to profitability? - Management indicated that they expect a typical rare disease revenue trajectory, leading to profitability within two to three years from launch [91]

Core Insights - The Chief Scientific Officer of Scholar Rock Holding Corp, Mo Qatanani, sold 14,898 shares on January 22, 2026, for approximately $695,937, but this should not concern investors as it was part of a pre-scheduled trading plan [1][4]. Transaction Summary - The shares sold were part of a Rule 10b5-1 trading plan, indicating that the sale was pre-arranged and not a spontaneous decision [4]. - After the transaction, Qatanani retains direct ownership of 85,660 shares, which represents 21% of total holdings [5]. Company Overview - Scholar Rock Holding Corporation is a clinical-stage biotechnology company focused on developing therapies for severe diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and anemia [3]. - The company's current market capitalization is $4.52 billion, with a net income of -$353.43 million [2]. Future Plans - Scholar Rock plans to launch its key product candidate, apitegromab, in the U.S. and European markets, pending regulatory approvals [5]. - Apitegromab is positioned as the world's first and only muscle-targeted treatment for spinal muscular atrophy (SMA), a severe genetic neuromuscular disease [6].

44th Annual J.P. Morgan Healthcare Conference Scholar Rock Investor Presentation JANUARY 12, 2026 © 2026 Scholar Rock, Inc. All rights reserved. Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, "Scholar Rock"), including without limitation, Scholar Rock's expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK ...

Summary of Scholar Rock Holding FY Conference Call (December 04, 2025) Company Overview - **Company**: Scholar Rock Holding (NasdaqGS:SRRK) - **Focus**: Development of therapies for spinal muscular atrophy (SMA) and other neuromuscular disorders Key Points Industry and Company Developments - **Significant Transformation**: 2025 marked a year of transformation for Scholar Rock, particularly following the positive readout of the Sapphire study in October 2024, which was pivotal for the company's future activities [4][5] - **BLA Submission**: The company filed its Biologics License Application (BLA) with the FDA in January 2025 based on the Sapphire study results [4] - **FDA Priority Review**: In April 2025, the FDA accepted the BLA under priority review due to the high unmet need in SMA, marking the first muscle-targeted therapeutic for the condition [5] Leadership Changes - **CEO Transition**: Jay Backstrom stepped down as CEO in April 2025, with David Hallal taking over. A new executive team was assembled to focus on scaling and growth [6][7] Clinical Trials and Efficacy - **Sapphire Study Results**: The phase 3 trial demonstrated that patients receiving apitegromab showed a gain in motor function compared to those on placebo, achieving a statistically significant p-value of 0.019 [12] - **Response Rates**: Approximately one-third of patients on apitegromab achieved a three-point improvement on the Hammersmith Motor Function Scale, compared to 12% on placebo, indicating a significant therapeutic benefit [13] Regulatory Outlook - **Complete Response Letter (CRL)**: The company received a CRL in September 2025 due to compliance issues at the fill/finish facility, which is now owned by Novo Nordisk. The drug substance facility was not a concern [9][18] - **Type A Meeting with FDA**: A constructive Type A meeting was held in November 2025, where the FDA indicated a willingness to expedite reinspection once Novo Nordisk is ready [20] Future Plans - **Resubmission Timeline**: Scholar Rock expects to resubmit the BLA and launch in the U.S. in 2026, pending resolution of the facility issues [20] - **Second Indication**: The company plans to announce a second indication for apitegromab in a rare neuromuscular disorder in 2026 [9] Market and Payer Strategy - **Payer Discussions**: The company is confident in securing coverage for apitegromab, emphasizing its ability to stabilize and improve motor function in SMA patients compared to existing therapies [33] - **Market Opportunity**: There are approximately 7,000 patients in the U.S. who have received SMN-targeted therapies, with a global patient population of around 35,000, indicating a significant market opportunity for apitegromab [35] Community Engagement - **Building Relationships**: The company is focusing on strengthening relationships with the SMA community, including healthcare providers and patients, to enhance disease awareness and education [32] Additional Insights - **Operational Challenges**: The delay in the approval process has allowed the company to better prepare for launch, including establishing relationships with payers and refining its reimbursement processes [30] - **Importance of Muscle Health**: The company emphasizes that optimal outcomes for SMA patients require addressing both motor neuron survival and muscle health, which apitegromab aims to achieve [31]

Financial Data and Key Metrics Changes - The company reported a significant milestone with the successful readout of its phase III trial in October 2024, marking it as the first muscle-targeted treatment for SMA [2][5] - The company has a cash balance of $369 million, which is expected to sustain operations into 2027 [54] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned to serve patients globally with SMA, with plans for clinical development programs for additional myostatin inhibitors [5][21] - The phase III trial demonstrated a statistically significant improvement in motor function for patients receiving apitegromab alongside SMN-targeted therapies, with a p-value of 0.01 [18] Market Data and Key Metrics Changes - In the U.S., approximately 7,000 patients have received at least one SMN-targeted treatment, with a global total of about 35,000 patients [20] - The combined annual revenue from existing SMN-targeted therapies is approximately $5 billion, indicating a robust market for SMA treatments [21] Company Strategy and Development Direction - The company aims to build a global biotech powerhouse, focusing on rare neuromuscular diseases, starting with SMA and expanding into other indications [3][5] - Plans include a methodical expansion into Europe, Asia-Pacific, and Latin America, targeting a 50-country operating platform [5][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the unique approach to myostatin inhibition, which differentiates the company from competitors focusing solely on motor neuron survival [12][24] - The company anticipates a resubmission of its BLA and a potential launch in the U.S. in 2026, following a constructive meeting with the FDA [47][52] Other Important Information - The company is currently addressing manufacturing issues that led to a Complete Response Letter (CRL) from the FDA, with a focus on remediation plans at its drug product manufacturer [41][44] - The company is also advancing its pipeline with SRK-439, a next-generation myostatin inhibitor, expected to enter clinical trials soon [48][50] Q&A Session Summary Question: What is the state of FDA interactions post-CRL? - The company received a CRL due to manufacturing issues at its drug product manufacturer, but management remains optimistic about resolving these issues and expects to resubmit and launch in 2026 [41][47] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage its experience in rare diseases to identify and treat patients already diagnosed with SMA, ensuring a smooth market entry for apitegromab [26][28]

Financial Data and Key Metrics Changes - The company reported a significant milestone with the successful readout of its phase III trial in October 2024, marking it as the first muscle-targeted treatment for SMA [2][5] - The company has a cash balance of $369 million, which is expected to sustain operations into 2027 [54] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned to serve patients globally with SMA, with plans for clinical development programs for additional myostatin inhibitors [5][21] - The phase III trial demonstrated a statistically significant improvement in motor function, with a p-value of 0.01, indicating the drug's effectiveness when combined with existing SMN-targeted therapies [17][18] Market Data and Key Metrics Changes - In the U.S., approximately 7,000 patients have received at least one SMN-targeted treatment, while globally, the number is around 35,000 [20] - The combined annual revenue from existing SMN-targeted therapies is approximately $5 billion, indicating a robust market for SMA treatments [21] Company Strategy and Development Direction - The company aims to build a global biotech powerhouse, focusing on rare neuromuscular diseases, starting with SMA and expanding into other indications [3][32] - Plans include a methodical expansion into Europe, Asia-Pacific, and Latin America, targeting a multi-billion dollar business model [32][33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the approval process for apitegromab, viewing the recent CRL as a de-risking event that clarifies the path to approval [52] - The company anticipates launching apitegromab in the U.S. in 2026, with ongoing discussions with the FDA regarding manufacturing compliance [47][45] Other Important Information - The company is also developing SRK-439, a next-generation myostatin inhibitor, which is expected to enter clinical trials soon [48][49] - The management team has been strengthened with experienced leaders from previous successful biotech ventures, enhancing the company's operational capabilities [4][6] Q&A Session Summary Question: What is the state of FDA interactions post-CRL? - The company received a CRL due to manufacturing issues at the drug product facility, but they are working closely with Novo Nordisk to address these concerns and expect to resubmit in 2026 [41][45] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage existing patient networks and funding mechanisms for rare disease therapies to facilitate the launch of apitegromab [26][28]