Summary of Scholar Rock Holding Conference Call Company Overview - **Company**: Scholar Rock Holding (NasdaqGS:SRRK) - **Event**: Jefferies London Healthcare Conference - **Date**: November 18, 2025 Key Points Industry and Product Focus - Scholar Rock is focused on developing therapies for spinal muscular atrophy (SMA) using their lead monoclonal antibody, ipilimumab, which targets myostatin [2][3] Regulatory Challenges - A positive Phase III study for ipilimumab led to a Biologics License Application (BLA) submission in January 2025, but a Complete Response Letter (CRL) was received on September 22, 2025, due to compliance issues at the GMP manufacturing facility [3][4] - The compliance issues were related to the Novo Catalent facility in Bloomington, Indiana, which received an Official Action Indicated (OAI) notice [4][10] - The FDA and Scholar Rock had a Type A meeting to discuss the path forward, with Novo presenting a robust remediation plan [4][10][12] Remediation and Inspection Timeline - Novo reported that approximately 90% of the remediation work has been completed, focusing on quality management systems [10][11][16] - The FDA is expected to communicate their impressions of the remediation progress soon, with a reinspection planned for early 2026 [12][21] - If the reinspection is successful, Scholar Rock anticipates resubmitting the BLA promptly, with hopes for approval in 2026 [13][30] Market Potential and Launch Strategy - There are over 11,000 SMA patients in the U.S., with two-thirds currently on SMN-targeted therapies [45] - Scholar Rock expects initial patients to be those already on SMN therapies, as ipilimumab is anticipated to be an add-on therapy [46][47] - The company plans to launch ipilimumab as the first monthly IV infusion for SMA, which may change the treatment paradigm [49] Pricing and Market Expansion - The market opportunity in SMA is significant, with Scholar Rock planning to price their product in the U.S. first before expanding to Europe, Japan, and South America [52][54] - The company is aware of the challenges in negotiating prices outside the U.S. but sees the necessity of entering these markets [53] Financial Position and Runway - Scholar Rock ended Q3 2025 with approximately $369.6 million in cash and raised an additional $91.7 million through an ATM and $50 million in loans [58] - The cash runway extends into 2027, assuming no revenue from ipilimumab and not accounting for potential monetization of a Priority Review Voucher (PRV) [58][59] Future Developments - Scholar Rock is working on a NextGen product targeting myostatin, with Phase I data expected next year [55] - There is significant interest in their anti-mystatin portfolio from companies in the obesity space, indicating potential for future collaborations [57] Additional Insights - The meeting highlighted the urgency and collaborative nature of the discussions with the FDA, emphasizing the importance of addressing the unmet need in SMA [23][25] - The company is committed to maintaining a high-quality manufacturing process and is optimistic about overcoming current challenges [18][19]

We recently published 10 Market Movers That Made Millionaires in a Week. Scholar Rock Holding Corp. (NASDAQ:SRRK) is one of the best-performing stocks of the past trading week. Scholar Rock saw its share prices climb by 29.2 percent on week-on-week as investors took heart from positive developments for its drug candidate for spinal muscular atrophy (SMA). In an announcement last week, Scholar Rock Holding Corp. (NASDAQ:SRRK) said that it sat down with the Food and Drug Administration and third-party manu ...

Scholar Rock Holding Corp. (SRRK) stock rose 25.67 percent to $37.79 on Friday after the company posted a wider net loss for the third quarter. The stock opened at $34.76 and traded between $34.25 and $39.50 on the Nasdaq. Shares have moved within a 52-week range of $22.71 to $46.98. Trading volume reached 3.44 million, above the average of 2.52 million.The biotech firm reported a net loss of $102.2 million, including $18.3 million in stock-based compensation, compared to $64.5 million, including $9.7 mill ...

Financial Data and Key Metrics Changes - The company ended Q3 2025 with $369.6 million in cash and cash equivalents, reporting operating expenses of $103 million, which includes $18.3 million in non-cash stock-based compensation [24][25] - Excluding stock-based compensation, operating expenses were $85.3 million, reflecting ongoing investments in infrastructure to support regulatory approval and commercial readiness [25] Business Line Data and Key Metrics Changes - The company is focused on advancing apitegromab for spinal muscular atrophy (SMA) and has initiated dosing in a phase two OPAL trial for infants and toddlers under the age of two [16][18] - The phase III SAPPHIRE study demonstrated apitegromab's potential to reverse the trajectory of SMA, showing significant benefits in motor function [13][20] Market Data and Key Metrics Changes - Annual revenue for current SMA treatments is trending to approximately $5 billion globally, indicating a growing demand for treatments [20][21] - There are an estimated 35,000 people with SMA who have received an SMN-targeted therapy and could be eligible for treatment with apitegromab [23] Company Strategy and Development Direction - The company aims to be a global biotech powerhouse, leveraging its expertise in myostatin biology to deliver transformative therapies for neuromuscular disorders [5][6] - The strategy includes strengthening the balance sheet, adding redundancy to the supply chain, and preparing for a commercial launch of apitegromab [11][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence that apitegromab will eventually be approved for SMA, emphasizing the urgency to serve the patient community [7][9] - The regulatory challenges faced are viewed as temporary, with significant opportunities ahead to serve patients [11][18] Other Important Information - The company has secured commercial capacity for a second fill-finish facility, expected to commence in Q1 2026, and anticipates submitting an SBLA for this facility later in 2026 [10][15] - The company is also advancing SRK-439 into clinical development, with an IND cleared by the FDA [17][18] Q&A Session Summary Question: Financing opportunities and operational risks during the transition to a new facility - The company plans to prioritize extending its loan facility for financing, with equity being a last resort [32] - Transitioning to a new facility is expected to be seamless operationally, minimizing risks [34] Question: Confidence in the remediation efforts at the Bloomington facility - Management highlighted the commitment from Novo Nordisk to quality and compliance, expressing optimism about the remediation efforts [40][41] Question: BLA submission strategy and EMA review status - The plan is to submit the BLA after the reinspection of the Bloomington facility, with a decision from the EMA expected by mid-next year [16][49] Question: Timelines for reinspection and potential pivot to a second fill-finish facility - A successful reinspection is crucial, but the company is prepared to pivot to the second facility if necessary [56][57] Question: Disclosure plans regarding the reinspection process - The company will maintain open communication regarding the reinspection timeline and outcomes, sharing material information as it becomes available [74][75]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with $369.6 million in cash and cash equivalents, reporting $103 million in operating expenses, which includes $18.3 million in non-cash stock-based compensation [24][25] - Excluding stock-based compensation, operating expenses were $85.3 million, reflecting ongoing investments in infrastructure to support regulatory approval and commercial readiness [25] - The company strengthened its balance sheet by adding $141.7 million during the quarter, which included net proceeds of $91.7 million from the sale of approximately 2.8 million shares and a $50 million drawdown from an existing debt facility [25][24] Business Line Data and Key Metrics Changes - The company is focused on advancing apitegromab for spinal muscular atrophy (SMA) and has initiated dosing in a phase two OPAL trial evaluating apitegromab in infants and toddlers under the age of two [16][17] - The company is also progressing SRK-439 into clinical development, with plans to initiate a phase one study in healthy volunteers before the end of the year [18] Market Data and Key Metrics Changes - The global annual revenue for current SMA treatments is trending to approximately $5 billion, indicating a growing demand for treatments in this market [20] - There are an estimated 35,000 people with SMA who have received an SMN-targeted therapy and could be eligible for treatment with apitegromab [23] Company Strategy and Development Direction - The company aims to be a global biotech powerhouse, leveraging its expertise in myostatin biology to deliver transformative therapies for neuromuscular disorders [5] - The strategy includes executing a strong commercial launch for apitegromab and advancing its pipeline, including a second indication for apitegromab and the development of SRK-439 [28][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the eventual approval of apitegromab, emphasizing the urgency to serve the SMA community and the commitment to quality and compliance in the remediation efforts at the Bloomington facility [7][8] - The company remains focused on executing its plans with financial discipline, aiming to bring apitegromab to market as rapidly as possible while managing its cash runway effectively [28][29] Other Important Information - The company has secured commercial capacity for a second fill-finish facility, expected to commence in Q1 2026, and anticipates submitting an SBLA for this facility later in 2026 [10][15] - The company is operating with a tight financial plan and has deferred investments in several areas to prioritize strategic initiatives [25] Q&A Session Summary Question: Financing opportunities and operational risks during facility transition - Management indicated that the first objective is to bridge financing until approval, with a preference for extending the loan facility before considering equity options [32] - Regarding operational risks, management assured that transitioning between facilities should be seamless, focusing on maintaining consistency in vial configuration and analytical testing [34][35] Question: Confidence in remediation efforts at the Bloomington facility - Management acknowledged the facility's history but expressed confidence in Novo Nordisk's commitment to quality and compliance, highlighting the collaborative efforts in the remediation plan [40][41] Question: BLA submission strategy and EMA review status - The plan is to submit the BLA after the reinspection of the Bloomington facility, with expectations for a decision from the EMA on the marketing authorization application by mid-next year [16][49] Question: Timelines for reinspection and potential pivot to second fill-finish facility - Management stated that a successful reinspection is the goal, and if issues arise, the second fill-finish facility could be pivoted to serve as the primary submission strategy [56][57] Question: Disclosure plans regarding inspection outcomes - Management committed to maintaining open communication regarding important developments, including the outcomes of inspections and any material information related to the facility [74][75]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with $369.6 million in cash and cash equivalents, reporting $103 million in operating expenses, which includes $18.3 million in non-cash stock-based compensation [21][22] - Operating expenses, excluding stock-based compensation, were $85.3 million, reflecting ongoing investments in infrastructure to support regulatory approval and commercial readiness [21][22] - The company strengthened its balance sheet by adding $141.7 million during the quarter, which included net proceeds of $91.7 million from the sale of approximately 2.8 million shares and a $50 million drawdown from an existing debt facility [22][23] Business Line Data and Key Metrics Changes - The company is focused on advancing apitegromab for spinal muscular atrophy (SMA) and has initiated dosing in a phase two OPAL trial evaluating apitegromab in infants and toddlers under the age of two [14][15] - The company is also progressing SRK-439 into clinical development, with plans to initiate a phase one study in healthy volunteers before the end of the year [15][16] Market Data and Key Metrics Changes - The global annual revenue for current SMA treatments is trending to approximately $5 billion, indicating a growing demand for treatments in this market [17] - There are an estimated 35,000 people with SMA who have received an SMN-targeted therapy and could be eligible for treatment with apitegromab [20] Company Strategy and Development Direction - The company aims to bring apitegromab to market as the world's first muscle-targeted therapy for SMA, with a focus on regulatory approval and commercial readiness [17][20] - The company is strategically investing in a second fill-finish facility to ensure redundancy in its supply chain and to support the anticipated launch of apitegromab [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence that apitegromab will eventually be approved in the U.S. for patients with SMA, emphasizing the commitment to the SMA community [6][10] - The regulatory challenges faced are viewed as temporary, with significant opportunities ahead to serve patients [10][21] Other Important Information - The company is working closely with Novo Nordisk to ensure the Bloomington facility is ready for reinspection by the end of the year, which is critical for the resubmission of the BLA [7][13] - The company has adjusted its operating plan to defer investments in certain areas while prioritizing key initiatives [22][23] Q&A Session Summary Question: How does the company view further financing opportunities? - The company aims to extend its loan facility as the first option for financing, with royalty financing as a secondary option, and equity financing as a last resort [29][30] Question: What operational risks are associated with transitioning between facilities? - The company believes that the transition should be seamless, focusing on maintaining consistency in vial configuration and analytical testing [31][33] Question: How confident is the company in the remediation efforts at the Bloomington facility? - Management expressed confidence in Novo Nordisk's commitment to quality and compliance, highlighting the collaborative efforts to ensure the facility is ready for reinspection [39][40] Question: What is the timeline for the reinspection and potential approval? - The company expects the reinspection to occur by the end of the year, with plans to resubmit the BLA following a successful inspection [52][55] Question: Will the company disclose information regarding the reinspection? - The company plans to maintain open communication and disclose important information as it becomes available, particularly regarding the reinspection timeline and outcomes [75][78]

Financial Performance - The company reported a net loss of $287.0 million for the nine months ended September 30, 2025, with an accumulated deficit of $1.2 billion[96]. - The net loss for the three months ended September 30, 2025, was $102.2 million, representing a 58.5% increase from a net loss of $64.5 million in 2024[108]. - The company reported a net loss of $287.0 million for the nine months ended September 30, 2025, which is a 59.6% increase from a net loss of $179.8 million in 2024[114]. - Net cash used in operating activities for the nine months ended September 30, 2025, was $224.5 million, compared to $151.4 million for the same period in 2024[127][129]. - Cash and cash equivalents increased to $286.8 million as of September 30, 2025, compared to $177.9 million at the end of 2024[122]. - The total cash, cash equivalents, and marketable securities decreased by $67.6 million during the nine months ended September 30, 2025, primarily due to operating expenses[122]. Research and Development - Apitegromab achieved its primary endpoint in the pivotal Phase 3 SAPPHIRE clinical trial for SMA, with positive top-line results announced in October 2024[88]. - Over 90% of patients in the Phase 2 TOPAZ trial for apitegromab remained on treatment at 48 months, showing sustained clinical benefit[89]. - The FDA granted priority review designation for the Biologics License Application (BLA) for apitegromab, indicating potential significant improvement in treatment for SMA[88]. - The company plans to initiate dosing of SRK-439 in healthy volunteers in Q4 2025, following FDA clearance of its investigational new drug application[93]. - In the Phase 2 EMBRAZE trial, patients receiving apitegromab with tirzepatide showed a 54.9% preservation of lean mass compared to tirzepatide alone (p=0.001)[93]. - Research and development expenses are expected to remain substantial as the company progresses its clinical development programs[101]. - The company expects continued substantial research and development expenses driven by employee compensation and clinical program development, particularly for apitegromab and SRK-439[111]. - The company expects substantial expenses related to the research and development of apitegromab and other product candidates[135]. Operating Expenses - For the three months ended September 30, 2025, total operating expenses increased by 59.8% to $103.6 million compared to $64.8 million in the same period of 2024[108]. - Research and development expenses for the three months ended September 30, 2025, were $50.5 million, up 3.6% from $48.7 million in 2024[109]. - General and administrative expenses surged by 230.4% to $53.1 million for the three months ended September 30, 2025, compared to $16.1 million in 2024[112]. - For the nine months ended September 30, 2025, research and development expenses totaled $161.6 million, an increase of 20.4% from $134.2 million in 2024[115]. - General and administrative expenses for the nine months ended September 30, 2025, were $131.2 million, up 170.4% from $48.5 million in 2024[118]. Future Outlook - The company continues to build its commercialization capabilities to support future product sales and marketing activities[96]. - The company expects to incur significant commercialization expenses related to the marketing approval and sales of apitegromab[132]. - The company anticipates that existing cash and marketable securities will fund operations into 2027, but additional capital will be required for clinical development and commercialization[133]. - The company will continue to rely on additional financing to achieve business objectives, with potential dilution of common stockholder ownership interests[136]. Capital and Financing - As of September 30, 2025, the company has sold 3,386,290 shares under the ATM program, generating net proceeds of $96.9 million[123]. - Net cash provided by investing activities was $179.7 million for the nine months ended September 30, 2025, up from $119.3 million in 2024[130]. - Net cash provided by financing activities was $155.1 million for the nine months ended September 30, 2025, significantly higher than $6.7 million in 2024[131]. - The company had 17,362,147 pre-funded warrants outstanding as of September 30, 2025, with none exercised during the nine months ended[125]. - The company had 8,220,620 common warrants outstanding with an exercise price of $7.35, which expire on December 31, 2025[126].

Financial Results Announcement - Scholar Rock Holding Corporation announced its financial results for the fiscal quarter ended September 30, 2025[4]. - A press release detailing the financial results has been issued and is available as Exhibit 99.1[4]. - The report includes a cover page interactive data file embedded within the Inline XBRL document as Exhibit 104[8]. Conference Call Information - The company will hold a conference call on November 14, 2025, at 8:00 am ET to discuss its financial and operating results[6]. - Presentation slides for the conference call are provided as Exhibit 99.2[6]. Company Information - The company is registered under the Securities Exchange Act of 1934 and trades on the Nasdaq Global Select Market under the symbol SRRK[3]. - The company is incorporated in Delaware and has a principal office located in Cambridge, MA[2]. - The company has not indicated if it is an emerging growth company[3]. Report Details - The report is intended to be furnished and not deemed "filed" under the Exchange Act[5]. - The report was signed by Junlin Ho, General Counsel & Corporate Secretary, on November 14, 2025[12].

Core Insights - Scholar Rock reported its financial results for Q3 2025, focusing on advancements in treatments for spinal muscular atrophy (SMA) and other neuromuscular diseases [1] Financial Performance - The company provided an update on its financial results for the third quarter ended September 30, 2025, indicating ongoing progress in its financial metrics [1] Company Developments - Scholar Rock emphasized its commitment to improving the lives of individuals with SMA and other rare neuromuscular diseases through its innovative platform in myostatin biology [1]

Core Insights - Short interest data provides insights into investor sentiment and potential risks in the stock market, helping gauge market confidence in a company's future [1] - A stock is deemed heavily shorted when a significant percentage of its available shares are borrowed and sold by investors anticipating a price drop [2] - Short squeezes can occur when short-sellers rush to cover their positions, leading to rapid price increases, as seen in recent meme stock manias [3] Short Interest Overview - The most heavily shorted stocks as of November 13 include Lucid Group, Inc. (48.99%), Choice Hotels International, Inc. (46.71%), and Avis Budget Group, Inc. (46.13%) [5] - Stocks are ranked by short interest, which is the total number of shares sold short and not yet covered, expressed as a percentage of shares available for public trading [4] Market Dynamics - Highly shorted stocks represent a battleground where negative fundamentals meet speculative trading [7] - Monitoring short interest can help identify potential short squeeze candidates, although timing such trades is challenging due to high volatility [7]