Scholar Rock Holding (SRRK) Q1 2025 Earnings Call May 14, 2025 08:15 AM ET Company Participants Rushmie Nofsinger - Vice President of Corporate Affairs & Investor RelationsDavid Hallal - CEO & ChairmanAkshay Vaishnaw - President, R&DKeith Woods - COOVikas Sinha - CFOMichael Yee - Managing DirectorDavid Nierengarten - Managing Director - Equity ResearchGary Nachman - Managing Director - Equity ResearchMalcolm Hoffman - Senior BioPharma Equity Research AssociateAndres Maldonado - Vice President Conference Cal ...

Scholar Rock Holding (SRRK) Q1 2025 Earnings Call May 14, 2025 08:15 AM ET Speaker0 Ladies and gentlemen, thank you for standing by and welcome to ScholarRock's First Quarter Financial Results and Business Update Call. At this time, all participants are in a listen only mode. Following the presentation, we will conduct a question and answer session. To ask a question during the session, you will need to press 11 on your telephone. You would then hear an automated message advising your hand is raised. To wit ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM _ TO _ COMMISSION FILE NUMBER 001-38501 ______________________________________________ SCHOLAR ROCK HOLDING CORPORATION (Exact name of registrant as sp ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT (857) 259-3860 (Registrant's telephone number, including area code) Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event Reported): May 14, 2025 Scholar Rock Holding Corporation (Exact Name of Registrant as Specified in Charter) (State or Other Jurisdiction of Incorporation) Delaware 001-38501 82-3750435 (Commission File Number) (I.R.S. Employer Identificat ...

Although there are many examples throughout history of positive changes in top company management teams, important personnel moves tend to unsettle investors. They get worried that significant changes could reflect instability, indecision, or both in a company's decision-making. That seemed to be very much the case Monday with biotech Scholar Rock (SRRK -3.35%). Its stock closed almost 4% lower in price on a day when the S&P 500 index basically traded flat. A quartet of new executive appointments Scholar Ro ...

Scholar Rock Holding Corporation. (NASDAQ:SRRK) Q4 2024 Earnings Conference Call February 27, 2025 8:15 AM ET Company Participants Jay Backstrom - President & CEO Ted Myles - COO & CFO Tracey Sacco - Chief Commercial Officer Conference Call Participants Allison Bratzel - Piper Sandler Michael Yee - Jefferies Tessa Romero - JPMorgan Gary Nachman - Raymond James Operator Good morning and welcome to Scholar Rock's Fourth Quarter Financial Results and Business Update Call. All participants will be in listen-onl ...

Regulatory Approval and Compliance - The company is preparing for the potential commercialization of apitegromab in the U.S. and Europe, investing significant resources in compliance, medical affairs, and commercial organizations[339]. - The company anticipates submitting a Biologics License Application (BLA) for apitegromab in January 2025, which requires extensive preclinical and clinical data[336]. - The company is seeking priority review for apitegromab, which could shorten the FDA's review time to six months if granted[346][349]. - The regulatory approval process for product candidates is lengthy and unpredictable, with potential delays impacting commercialization[330][336]. - Changes or disruptions at the FDA and other regulatory agencies could negatively impact the timely review and approval of the company's applications[343][344]. - The company must comply with Good Clinical Practice (GCP) requirements, and any non-compliance could delay regulatory approval[372]. - Regulatory approvals may come with limitations on approved uses and requirements for costly post-market testing, including Phase 4 clinical trials[395]. - Regulatory approval in one jurisdiction does not guarantee approval in others, and failures in one region may negatively impact the approval process elsewhere[389]. - The company is subject to ongoing regulatory obligations and continued regulatory review, which may result in significant additional expenses and penalties for non-compliance[393]. - The company may not be able to realize the benefits associated with Orphan Drug designation if it fails to meet regulatory requirements or if the designation is revoked[379]. Financial Performance and Capital Needs - The company has incurred net losses every year since inception and expects to continue incurring losses, necessitating additional capital for operations[335]. - The company expects to rely on third-party manufacturers for commercial supplies of apitegromab, with no long-term supply agreements in place, which may lead to delays in clinical trials or commercialization if new manufacturers are needed[414]. - The company may face substantial civil and criminal penalties for failing to comply with international business laws, which could hinder government contracting opportunities[457]. - The company may struggle to obtain adequate product liability insurance, which could inhibit the commercialization of developed products[462]. Clinical Development and Trials - The Phase 2 TOPAZ trial for apitegromab showed sustained clinical benefit over 48 months, with a retention rate of over 90% in nonambulatory Types 2 and 3 SMA patients[362]. - The Phase 3 SAPPHIRE trial for apitegromab announced positive top-line data in October 2024, indicating efficacy and safety[362]. - The company initiated the Phase 2 EMBRAZE trial of apitegromab in obesity in May 2024, with top-line results expected in Q2 2025[362]. - Delays in clinical trials may arise from various factors, including regulatory agreements and site negotiations, potentially leading to denial of regulatory approval[352]. - Access to approved therapies is critical for ongoing trials; any limitations could necessitate pausing or stopping clinical studies[359]. - The company anticipates that clinical trial delays could shorten exclusive commercialization periods, allowing competitors to enter the market sooner[358]. - The company may face difficulties in patient enrollment for clinical trials due to the small size of the SMA patient population and competition with other trials[367]. - Delays in patient enrollment could result in increased costs and affect the timing or outcome of future clinical trials, potentially hindering the development of product candidates[368]. - The company relies on third parties for conducting clinical trials, and any failure by these parties to meet contractual obligations could materially harm the business[369]. Market and Competitive Landscape - Market acceptance of product candidates is uncertain and depends on factors such as efficacy, safety, and competitive pricing[400]. - The biopharmaceutical industry is highly competitive, with many companies potentially developing similar products, which could affect market share[405]. - The company may seek collaborations for product candidates like apitegromab, SRK-181, and SRK-439, but failure to secure successful collaborations could adversely affect business[477]. - Collaborations pose risks such as collaborators not performing obligations satisfactorily or failing to pursue development of product candidates[480]. - The company faces significant competition in seeking appropriate partners for product candidates, and the negotiation process is complex and time-consuming[484]. Intellectual Property and Legal Risks - The ability to protect intellectual property is crucial for commercial success, and challenges in securing patent protection could adversely affect the commercialization of product candidates[486]. - The patenting process is expensive and time-consuming, and failure to maintain compliance with patent requirements could result in loss of patent rights[492]. - The company depends on intellectual property licensed from third parties, and failure to comply with obligations under these licenses could result in significant rights loss[498]. - The company may have limited control over the maintenance and prosecution of in-licensed patents and patent applications, which could affect the validity and enforceability of these rights[499]. - Disputes may arise regarding intellectual property subject to license agreements, potentially impairing the ability to develop and commercialize product candidates[500]. - Changes in patent law in the U.S. and other jurisdictions could diminish the value of patents, impairing the ability to protect products[508]. - Recent U.S. Supreme Court rulings have narrowed the scope of patent protection, creating uncertainty regarding the value of patents once obtained[510]. - Third-party claims of intellectual property infringement may delay product discovery and development efforts, leading to potential litigation costs[514]. Operational and Management Challenges - Future growth will require hiring additional personnel across various functions, which may pose challenges in managing this growth effectively[422]. - The company is dependent on third parties for critical services, and any failure to manage these relationships could delay regulatory approvals and hinder business advancement[423]. - Attracting and retaining qualified personnel in the competitive biopharmaceutical space is crucial, and failure to do so may impede the company's ability to develop and commercialize its product candidates[424]. - Management transitions or loss of key personnel could disrupt the company's strategy and operational execution, negatively affecting business performance[425]. - Cybersecurity risks and potential breaches of IT systems could disrupt operations and delay development programs, impacting the company's overall business[429]. Compliance and Regulatory Costs - The EU General Data Protection Regulation (GDPR) imposes fines of up to €20 million or 4% of total worldwide annual turnover for serious offenses, highlighting the financial risks associated with non-compliance[438]. - The UK GDPR allows for monetary penalties of up to £17.5 million or 4% of worldwide revenue, whichever is higher, indicating significant compliance costs[439]. - The company is subject to various healthcare laws and regulations that could expose it to civil penalties and reputational harm, affecting future earnings[434]. - The introduction of the EU's Artificial Intelligence Act may require the company to implement costly compliance measures, impacting research and development budgets[450]. - Compliance with state privacy laws, such as California's CCPA, may necessitate operational changes and increase overall compliance costs[445]. - The adequacy decision for the EU-U.S. Data Privacy Framework allows for safe data transfers but carries risks of future challenges, increasing regulatory complexity[442]. - The evolving regulatory landscape for artificial intelligence may require substantial resources to ensure compliance and mitigate risks associated with new technologies[450]. Product Liability and Market Risks - Product liability lawsuits pose a significant risk, potentially resulting in substantial liabilities and limiting the commercialization of product candidates[460]. - Global events, such as health crises and geopolitical instability, could adversely impact business operations and financial results[466]. - Coverage and reimbursement from third-party payors are critical for the success of product candidates, with significant uncertainty regarding future approvals[468]. - Changes in healthcare legislation may limit reimbursement amounts, resulting in reduced demand for pharmaceutical products[471]. - Increasing scrutiny on drug pricing practices may lead to pricing pressures for the company's product candidates[472]. - The company's disaster recovery and business continuity plans may prove inadequate in the event of significant disruptions, impacting operations[467]. - In the EU, the proposed pricing for a drug must be approved before lawful marketing, and pricing negotiations can take considerable time after receiving marketing approval[476]. - Historical data indicates that products launched in the EU generally have significantly lower prices compared to the U.S., which may negatively affect revenue and profitability[476].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT (Former name or former address, if changed since last report) Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions: Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event Reported): February 27, 2025 Scholar Rock Holding Corporation (Exact Nam ...

Core Insights - Scholar Rock Holding Corporation is focusing on the development of apitegromab for spinal muscular atrophy (SMA), with discussions at the J.P. Morgan Healthcare Conference highlighting its potential market and adoption factors [1][4]. Group 1: Product Development and Trials - The company plans to submit U.S. and European marketing applications for apitegromab in Q1 2025, targeting a U.S. launch in Q4 2025 and a subsequent European launch [7]. - Upcoming results from the Phase 2 EMBRAZE trial, evaluating apitegromab in combination with tirzepatide or semaglutide for overweight or obese adults, are expected in Q2 2025 [3]. - Detailed results from the Phase 3 SAPPHIRE trial, involving non-ambulatory type 2/3 SMA patients, will be presented at medical meetings in 2025 [7]. Group 2: Market Potential and Financial Projections - The current SMA market is estimated at approximately $4.5 billion across three SMN-targeted therapies, primarily for the 2-21 age group [7]. - Scholar Rock expects apitegromab to generate over $2 billion in global revenue, an increase from previous estimates of $1 billion, due to strong data and a favorable competitive landscape [7]. - JP Morgan maintains a positive outlook on Scholar Rock, anticipating a strong product launch and further value if cardiometabolic data is favorable, with initial results expected in Q2 2025 [4]. Group 3: Stock Performance - Scholar Rock's stock (SRRK) has seen an increase of 4.89%, reaching $43.56 [5].

Financial Data and Key Metrics Changes - Scholar Rock reported a successful quarter with a cash balance of approximately $463 million following a $345 million follow-on offering, enabling the company to focus on key priorities [27][28] - The SAPPHIRE study met its primary endpoint with a statistically significant improvement of 1.8 points in the Hammersmith Functional Motor Scale for patients receiving apitegromab compared to placebo [11][39] Business Line Data and Key Metrics Changes - The apitegromab program is on track for regulatory submissions in Q1 2025, with a commercial launch anticipated in the U.S. by Q4 2025 [7][21] - The EMBRAZE Phase 2 proof-of-concept study for apitegromab in obesity completed enrollment ahead of schedule, with top-line results expected in Q2 2025 [8][29] Market Data and Key Metrics Changes - The current treatment landscape for spinal muscular atrophy (SMA) is valued at $4.5 billion and is growing, with apitegromab positioned to address the muscle component of the disease [35] - The company anticipates a total revenue potential of greater than $1 billion for apitegromab, assuming a competitive market for muscle-targeted therapies in SMA [36] Company Strategy and Development Direction - Scholar Rock aims to expand its anti-myostatin platform and prepare for the commercialization of apitegromab, focusing on building relationships with healthcare providers and patient communities [30][31] - The company is also working on the IND submission for SRK-439, targeting mid-2025, to preserve lean muscle mass in obesity treatments [23][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of apitegromab to transform the standard of care in SMA, highlighting the favorable safety profile and efficacy data [39][40] - The company is committed to ensuring an excellent treatment experience for patients and is actively preparing for a successful launch of apitegromab [33][34] Other Important Information - The addition of Beth Shafer as Chief Business Officer is expected to enhance investment decisions and partnering opportunities [9] - The company launched the "Life Takes Muscle" campaign to raise awareness about SMA and the need for more effective treatments [32] Q&A Session Summary Question: Differences in trial design and context against apitegromab - Management discussed the EMBRAZE trial's focus on lean muscle mass as the primary endpoint, contrasting it with Lilly's trial design [42][43] Question: OPAL study considerations and obesity data parameters - Management emphasized the importance of PK/PD data in guiding dosing for the OPAL study and the significance of preserving muscle mass in obesity treatments [47][48] Question: Approval for different doses and age range - The company plans to seek approval for the 10 mg/kg dose only, confident in the data supporting its efficacy across the entire age range [52][53] Question: Initial payer discussions and pricing considerations - Early payer discussions have been positive, with management optimistic about the value proposition of apitegromab based on the SAPPHIRE data [57][58] Question: Broader labeling chances and regulatory interactions - Management anticipates a broad label for apitegromab, similar to existing SMA treatments, based on the strength of the data [62][63]