Scholar Rock (NasdaqGS:SRRK) FY Conference January 12, 2026 10:30 AM ET Company ParticipantsAkshay Vaishnaw - President of R&DKeith Woods - COODavid Hallal - Chairman and CEOVikas Sinha - CFOConference Call ParticipantsTess Romero - Senior Biotech AnalystTess RomeroWelcome, everyone, to the 44th Annual J.P. Morgan Healthcare Conference. My name is Tess Romero, and I'm one of the Senior Biotech Analysts here at J.P. Morgan. We're very pleased to be kicking off the conference with Scholar Rock, and presenting ...

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].

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]

Core Viewpoint - Scholar Rock's stock price surged by 6% following a bullish initiation of coverage by Bank of America Securities, despite a slight decline in the S&P 500 index [1][2]. Group 1: Analyst Coverage - Tazeen Ahmad of Bank of America Securities initiated coverage on Scholar Rock, setting a buy rating with a price target of $53 per share, which is nearly 30% higher than the company's recent closing price [2]. - The positive market reaction was attributed to the analyst's favorable outlook on the company's potential [2]. Group 2: Pipeline Drug - Scholar Rock's investigational drug apitegromab, aimed at treating spinal muscular atrophy (SMA), is highlighted as significantly more effective than existing treatments [3]. - The company focuses on developing therapies for rare diseases that currently lack effective treatments [4]. - Apitegromab is the most advanced drug in Scholar Rock's pipeline and has been submitted for FDA approval under a biologics license application (BLA) [5]. - The FDA issued a complete response letter (CRL) last month, indicating that the application is not approved in its current form, and Scholar Rock is addressing the issues raised [5].

Group 1 - The article discusses Scholar Rock Holding Corporation (NASDAQ: SRRK) and its potential beyond its current focus on Spinal Muscular Atrophy (SMA) treatments, particularly highlighting upcoming data related to obesity treatment expected in Q2 2025 [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a model portfolio of small and mid-cap stocks [2] - The service offers a subscription model with a promotional two-week free trial and a discounted annual plan [1] Group 2 - The article does not provide specific financial data or performance metrics related to Scholar Rock or the broader biotech industry [4]

Group 1 - The article discusses Scholar Rock Holding Corporation (NASDAQ: SRRK) and its potential beyond its current focus on Spinal Muscular Atrophy (SMA) treatments, particularly highlighting upcoming data related to obesity treatment expected in Q2 2025 [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a model portfolio of small and mid-cap stocks [2] - The service offers a subscription model with a promotional two-week free trial and a discounted annual plan [1] Group 2 - The article does not provide specific financial data or performance metrics related to Scholar Rock or the broader biotech industry [3][4]

Apitegromab in SMA - Positive Phase 3 trial data showed a 18 point improvement versus placebo in Hammersmith Functional Motor Scale-Expanded (HFMSE) in SMA patients[23] - 30% of apitegromab patients achieved ≥3-point improvement in HFMSE compared to 125% of placebo patients[23] - FDA accepted BLA under Priority Review with a target action date of September 22, 2025[25] - MAA Validated with anticipated EU approval in 2026[14] - The company is initiating Phase 2 OPAL Trial in Q3 to study apitegromab in patients under 2 years old[14, 27] Pipeline Expansion - EMBRAZE trial achieved primary endpoint with positive Phase 2 proof-of-concept in obesity[14] - Apitegromab combined with tirzepatide resulted in a 549% reduction in lean mass loss versus tirzepatide alone[36] - The company is on track to file IND for SRK-439 in 2H 2025[14, 39, 43] Financial Status - The company had $295 million cash as of June 30, 2025[14]

Business Overview - Scholar Rock's strategy focuses on selectivity in targeting latent growth factors to develop therapeutics across multiple disease areas[12, 15, 17, 19] - The company is advancing a pipeline of product candidates in neuromuscular disorders, cardiometabolic disorders, immuno-oncology, fibrosis, and anemia[20, 23] - Scholar Rock anticipates transformative execution in the next 12-24 months, driven by upcoming milestones and commercialization efforts[12] Apitegromab (Spinal Muscular Atrophy) - Topline readout from the Phase 3 SAPPHIRE trial in SMA is expected in Q4 2024, with a potential SMA launch in Q4 2025 if successful and approved[30] - 48-month data from the TOPAZ clinical trial showed substantial and sustained improvements in motor function outcomes in pooled nonambulatory SMA patients[39, 46, 52] - In the TOPAZ trial, pooled nonambulatory patients showed mean change from baseline in HFMSE of 2.8 at 6 months, 3.6 at 12 months, 4.2 at 24 months, 4.0 at 36 months, and 5.3 at 48 months[40] - In the TOPAZ trial, pooled nonambulatory patients showed mean change from baseline in RULM of 0.6 at 6 months, 1.3 at 12 months, 2.3 at 24 months, 2.4 at 36 months, and 3.6 at 48 months[47] Apitegromab (Obesity) & SRK-439 - A Phase 2 proof-of-concept EMBRAZE study of apitegromab in obesity is enrolling, with topline data expected in Q2 2025[23, 30, 60, 63] - SRK-439, a novel anti-myostatin antibody, is being advanced to IND submission, with preclinical data showing potential for healthier weight loss in combination with GLP-1 receptor agonists[23, 30, 75] - Preclinical data showed that SRK-439 increased lean mass and attenuated regain of fat mass after GLP-1 RA withdrawal in an obesity mouse model[78] - In preclinical studies, SRK-439 demonstrated greater potency than an anti-ActRII antibody at maintaining lean mass during GLP-1 RA-induced weight loss[77, 88]

Investment Rating - The industry investment rating is "Positive" (maintained) [1] Core Insights - The report highlights recent positive developments in the high-quality weight loss sector, particularly focusing on myostatin pathway clinical trial results from Regeneron and Scholar Rock, which demonstrate significant reductions in lean body mass loss when combined with GLP-1 medications [4][12] - The pharmaceutical and biotechnology sector experienced a decline of 4.35% in the third week of June 2025, underperforming the CSI 300 index by 3.9 percentage points, ranking 29th among 31 sub-industries [5][16] - The report emphasizes the potential of Activin receptor drugs, such as Eli Lilly's Bimagrumab and Lai Kai's LAE-102, to retain lean body mass and even promote muscle gain, with promising safety and pharmacokinetic data [4][12] Summary by Sections 1. Recent Developments in Weight Loss Sector - Regeneron's myostatin antibody Trevogrumab combined with semaglutide showed a 50.8% reduction in lean body mass loss over 24 weeks, compared to a 7.9 lbs loss with semaglutide alone [4][12] - Scholar Rock's myostatin antibody Apitegromab combined with Tirzepatide demonstrated a 54.9% reduction in lean body mass loss [4][12] 2. Market Performance - The pharmaceutical and biotechnology sector's decline of 4.35% in June's third week was the largest among all sectors, with the vaccine sector experiencing the smallest drop of 0.4% [5][21] - Other sub-sectors, such as blood products and medical consumables, also faced declines, with the largest drop seen in other biological products at 6.7% [5][21] 3. Recommended Stocks - Recommended stocks include major pharmaceutical and biotechnology companies such as Heng Rui Medicine, East China Medicine, and Innovent Biologics, among others [6]

Core Insights - Scholar Rock's Phase 2 EMBRAZE trial results indicate that apitegromab combined with tirzepatide can significantly preserve lean mass during weight loss, with a notable reduction in lean mass loss compared to tirzepatide alone [1][2][3] Group 1: Trial Results - The trial showed that 30% of the weight loss from tirzepatide was attributed to lean mass loss, while the addition of apitegromab reduced this to 14.6% [3][4] - Patients receiving the apitegromab-tirzepatide combination lost 12.3% of their body weight, compared to 13.4% for those on tirzepatide with placebo [4] - The combination therapy resulted in a higher quality of weight loss, with patients losing 85% fat and 15% lean mass, compared to 70% fat and 30% lean mass in the placebo group [4] Group 2: Market Reaction - Following the positive trial results, Scholar Rock's stock price increased by 18%, reaching $36.77 [6]