Group 1 - The article does not provide any relevant content regarding company or industry insights [1]

Company Overview - Scholar Rock is positioned as a leader in myostatin biology, focusing on treatments for rare neuromuscular disorders [3] - The company has developed apitegromab, a monoclonal antibody aimed at treating spinal muscular atrophy (SMA), which has shown significant clinical benefits [3] Product Development - Apitegromab is the first myostatin inhibitor to successfully complete a pivotal Phase III study, marking a milestone in muscle-targeted treatments for SMA [3] - The treatment has demonstrated clinically meaningful benefits for both children and adults living with SMA, highlighting its innovative approach in the field [3] Future Outlook - The year 2026 is anticipated to be transformative for Scholar Rock, indicating potential advancements and developments in their product pipeline and market presence [2]

Scholar Rock (NasdaqGS:SRRK) FY Conference Summary Company Overview - **Company**: Scholar Rock - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 12, 2026 - **Key Speaker**: David Hallal, Chairman and CEO Core Industry and Company Insights - Scholar Rock is positioned as a leader in myostatin biology, focusing on treatments for rare neuromuscular disorders, particularly Spinal Muscular Atrophy (SMA) [2][3] - The company has developed Apitegromab, the first myostatin inhibitor to successfully complete a pivotal phase III study, demonstrating clinically meaningful benefits for SMA patients [3][10] - Apitegromab is expected to receive U.S. and European approvals in 2026, with Germany being the initial launch country in Europe [3][5] Key Developments and Milestones - Scholar Rock plans to initiate dosing for a new indication of Apitegromab for Facioscapulohumeral Muscular Dystrophy (FSHD) later in 2026 [4][19] - The company has a strong cash position of approximately $365 million, providing a runway into 2027 to support commercial and R&D initiatives [25] - The phase III trial data showed that about one-third of patients receiving Apitegromab alongside SMN-targeted therapies had significant improvements in motor function, compared to only 12.5% in the placebo group [12] Market Opportunity - The global market for SMN-targeted therapies is projected to reach $5 billion in 2025, with Apitegromab representing a $2 billion-plus opportunity specifically for SMA [18][27] - The company aims to build a 50-country operating platform to serve SMA patients globally, with a focus on the U.S., Europe, and significant markets in Asia-Pacific and Latin America [6][7] Regulatory and Commercial Strategy - Scholar Rock is preparing for a BLA resubmission and U.S. launch following a complete response letter from the FDA, which was related to site inspection issues rather than clinical efficacy [13][15] - The company is actively engaging with national and regional payers to educate them on the unmet needs in SMA and the potential benefits of Apitegromab [16][17] - The U.S. commercial team is focused on disease education and building relationships with the SMA community to ensure successful market entry [16] Future Directions - Scholar Rock is committed to expanding the reach of Apitegromab to ensure no SMA patient is left behind, including those under two years of age [19][26] - The company is also exploring additional neuromuscular diseases for which Apitegromab may provide therapeutic benefits [22][23] - Ongoing development of SRK-439, a more potent myostatin inhibitor, is also a priority, with plans to share data from healthy volunteer studies in late 2026 [24] Financial Outlook - The company has maintained financial discipline while investing in high-value programs, with a focus on preparing for the commercial launch of Apitegromab [25][41] - There is an expectation of strong demand for Apitegromab upon approval, with plans for non-dilutive financing options to support operational needs [41] Conclusion - Scholar Rock is poised for a transformative year in 2026, with significant advancements in the treatment of SMA and a robust pipeline aimed at addressing various neuromuscular disorders [26]

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 ...

Financial Position - Scholar Rock has a cash position of $365 million as of December 31, 2025, providing a cash runway into 2027[4] - As of December 31, 2025, Scholar Rock has approximately $365 million in cash and equivalents, providing a cash runway to support operations into 2027[30] Product Development and Launch - The anticipated U.S. launch of apitegromab is expected in 2026, pending FDA approval, with an EMA decision expected in mid-2026[4][9] - The Biologics License Application (BLA) for Apitegromab was submitted to the FDA in January 2025, with acceptance under priority review in March 2025, and a U.S. launch anticipated in 2026[15] - Scholar Rock is preparing for a significant market opportunity in Europe, with a launch readiness for Apitegromab ahead of the mid-2026 EMA decision[16] - The company plans to commercialize apitegromab for both children and adults with SMA, following regulatory approvals[9] - Scholar Rock aims to expand the development of apitegromab for patients with SMA under 2 years of age and for additional rare neuromuscular diseases, with a Phase 2 FORGE study initiation planned for mid-2026[31] - The company is progressing the Phase 2 OPAL study for SMA patients under 2 years of age, with an EMA decision expected in mid-2026 and an initial launch planned in Germany[31] Market Opportunity - Approximately 35,000 patients have received SMN-targeted therapies, indicating a significant market opportunity for apitegromab, which targets muscle atrophy in SMA[6][12] - The global opportunity for apitegromab in SMA alone is estimated to be $2 billion[4] - The estimated global revenue potential for Apitegromab is over $2 billion, with a demand for the first and only muscle-targeted therapy in SMA valued at approximately $5 billion[18] Clinical Trials and Efficacy - Scholar Rock is initiating a Phase 2 study for apitegromab in patients with facioscapulohumeral muscular dystrophy (FSHD) in mid-2026[4][9] - Apitegromab demonstrated a 30.0% improvement in HFMSE for patients compared to 12.5% on SMN2-targeted treatment alone, with a statistically significant mean change of +1.8 HFMSE points (p=0.0192) in a Phase 3 study[14] - Ongoing Phase 2 OPAL study is evaluating Apitegromab in infants and toddlers with SMA, addressing the needs of patients under 2 years of age[20] - The Phase 1 study of subcutaneous Apitegromab showed favorable bioavailability, with pharmacodynamic profiles comparable to intravenous administration[26] - SRK-439, a novel myostatin inhibitor, is in Phase 1 trials, with dosing commenced in December 2025 and topline data expected in H2 2026[28] - Scholar Rock is advancing its anti-myostatin pipeline, with SRK-439 currently in a Phase 1 study[9] - Scholar Rock is advancing its anti-myostatin pipeline, with a completed Phase 1 study for subcutaneous apitegromab and a Phase 1 study for SRK-439, with topline data expected in H2 2026[31] Strategic Focus - The company aims to expand its market presence to 50 countries, focusing on rare neuromuscular diseases[7] - Scholar Rock emphasizes financial discipline and focused execution as key priorities for 2026[9] - The company has established a U.S. commercial field team focused on engagement and education across the SMA stakeholder landscape, with over 2,600 SMA prescribers identified[16] - Scholar Rock is building a world-class team and expanding its physical presence in Zug and Dublin to enhance its market access and engagement with the SMA community[16] - Scholar Rock is expanding its pipeline to include Apitegromab for additional neuromuscular diseases, starting with facioscapulohumeral muscular dystrophy (FSHD)[24]

Core Viewpoint - Scholar Rock Holding Corporation (SRRK) is a biopharmaceutical company focused on innovative treatments for serious diseases, currently receiving a consensus recommendation of "Buy" from analysts [1] Analyst Recommendations - Sixteen brokerages have provided recommendations for SRRK, including one sell, eleven buy, and four strong buy ratings [1] - Michael Yee from UBS has set a price target of $60 for SRRK, indicating a potential increase of approximately 45.24% from its current price of $41.31 [2][5] - The average 12-month price target among analysts is $48.9, with Barclays raising its target from $45 to $52 while maintaining an "overweight" rating [2][5] - Cantor Fitzgerald reiterated an "overweight" rating on December 11, while Wolfe Research began coverage with an "outperform" rating and a price target of $42 [3] Stock Performance - The stock has experienced fluctuations, with a recent low of $41.19 and a high of $43.06 [4] - Over the past year, SRRK has reached a high of $48.28 and a low of $22.71 [4] - The company's market capitalization is approximately $4.21 billion, with a trading volume of 707,076 shares, indicating active investor interest [4]

Core Viewpoint - The article emphasizes a focus on non-consensus long-short investment ideas within the biotechnology sector, particularly targeting small to mid-cap companies in the US and EU markets, with an interest in clinical catalysts and new drug launches [1]. Group 1: Investment Focus - The investment strategy centers on small to mid-cap biotechnology companies that are publicly traded [1]. - The analysis aims to identify clinical catalysts that could influence stock performance and earnings related to new drug launches [1]. Group 2: Analyst's Position - The analyst holds a beneficial long position in the shares of SRRK, indicating a personal investment interest in this specific stock [2]. - The article is authored by the analyst without external compensation, reflecting personal opinions rather than institutional views [2]. Group 3: Content Disclaimer - The content is intended for informational and educational purposes only, and should not be interpreted as financial or investment advice [3]. - The article acknowledges the possibility of errors or inaccuracies in the information provided, emphasizing the need for readers to conduct their own due diligence [3].

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]

Group 1 - Scholar Rock is positioned in the biopharma sector, focusing on innovative therapies and has attracted interest from investors [1][2] - The executives, David Hallal and Vikas Sinha, shared their backgrounds and motivations for joining Scholar Rock, emphasizing the company's potential [2] - The discussion included insights on the company's data and the strategic path towards commercial launch [1][2]

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]