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]

Financial Data and Key Metrics Changes - The company reported a significant transformation over the past 12 to 14 months, particularly following the positive 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, with plans to expand its loan facility to support the upcoming launch [50] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned as a muscle-targeted treatment for SMA, with clinical trials demonstrating statistically significant improvements in motor function when used alongside existing SMN-targeted therapies [16][20] - The company anticipates a multi-billion dollar opportunity for apitegromab, estimating over $2 billion in annual revenue from SMA alone [21] 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 [19] - The existing SMN-targeted therapies generate approximately $5 billion in annual revenues, indicating a robust market for SMA treatments [20] Company Strategy and Development Direction - The company aims to build a global operating platform across 50 countries, focusing on expanding its reach in Europe, Asia-Pacific, and Latin America [5] - Future plans include entering clinical trials for other rare neuromuscular disorders, with indications such as FSHD and DMD being assessed [30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in overcoming the recent CRL issued by the FDA, emphasizing that the remaining issue is solvable and that approval is a matter of when, not if [48][49] - The company is committed to a collaborative approach with the FDA and its manufacturing partners to expedite the approval process for apitegromab [42] Other Important Information - The company has established a world-class executive team to drive its strategic vision, with leadership experienced in building successful biotech companies [3][4] - The company is also developing SRK-439, a next-generation myostatin inhibitor, which is expected to enhance its rare neuromuscular franchise [45][46] 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 [32][44] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage its existing knowledge of the rare disease market, focusing on patients already diagnosed and receiving treatment, to effectively launch apitegromab [26][27]

Core Insights - Commodore Capital has fully exited its position in Scholar Rock Holding Corporation, selling approximately 1.5 million shares in Q3, which represented a net position change of about $51 million [1][2][10] Company Overview - Scholar Rock Holding Corporation is a biotechnology company focused on developing therapeutics for serious diseases driven by protein growth factor signaling, with a pipeline that includes neuromuscular, oncology, and fibrotic indications [5][8] - As of the latest data, Scholar Rock's share price is $41.08, with a market capitalization of $4.2 billion and a net income of -$353.4 million over the trailing twelve months [4][8] Financial Performance - The company reported a significant increase in Q3 operating expenses, rising to $103.6 million from $64.8 million the previous year, leading to a quarterly net loss of $102.2 million [11] - Scholar Rock has $369.6 million in cash, providing a runway into 2027 to support its programs through key catalysts expected in 2026 [11] Regulatory and Market Position - Scholar Rock is preparing to resubmit its apitegromab biologics license application following a constructive FDA meeting on November 12, which indicated progress in remediation efforts [10] - The company is entering a critical phase with potential regulatory milestones that could significantly impact its market position and investor sentiment [9][12]

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]

Core Insights - Scholar Rock Holding Corp. (NASDAQ:SRRK) experienced a significant share price increase of 29.2% week-on-week, driven by positive developments regarding its drug candidate for spinal muscular atrophy (SMA) [1][4] Company Developments - The company recently met with the Food and Drug Administration (FDA) and its manufacturing contractor, Catalent Indiana, to discuss the biologics license application (BLA) for apitegromab, following a previous rejection due to inspection issues [2][3] - Catalent reported progress in its remediation plan, with plans for the facility to be ready for reinspection by the end of the year [3] - Scholar Rock aims to resubmit the BLA application, targeting an official launch of apitegromab next year and seeking European Medicines Agency (EMA) approval by mid-2026 [4] Financial Performance - In the third quarter, Scholar Rock widened its net loss by 58% to $102 million, compared to $64.5 million in the same period last year, primarily due to a 60% increase in operating loss to $103.5 million from $64.78 million year-on-year [5]

Core Points - Scholar Rock Holding Corp. (SRRK) stock increased by 25.67 percent to $37.79 following the announcement of a wider net loss for the third quarter [1][2] - The stock opened at $34.76 and fluctuated between $34.25 and $39.50 on the Nasdaq, with a trading volume of 3.44 million shares, surpassing the average of 2.52 million [1] Financial Performance - The company reported a net loss of $102.2 million for the third quarter, which included $18.3 million in stock-based compensation, compared to a net loss of $64.5 million with $9.7 million in stock-based compensation in the same quarter of the previous year [2] - Net loss per share rose to $0.90 from $0.66 year-over-year [2]

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].