Financial Performance - The company reported a net loss of $179.8 million for the nine months ended September 30, 2024, with an accumulated deficit of $856.3 million[84]. - The net loss for Q3 2024 was $64.48 million, reflecting a 52.2% increase from a net loss of $42.36 million in Q3 2023[109]. - The company reported a net loss of $179.8 million for the nine months ended September 30, 2024, which is a $60.2 million increase or 50.3% from a net loss of $119.7 million in the same period of 2023[117]. - Total operating expenses for Q3 2024 were $64.78 million, a 48.3% increase from $43.67 million in Q3 2023[109]. - Total operating expenses for the nine months ended September 30, 2024, were $182.7 million, an increase of $59.4 million or 48.2% compared to $123.3 million in the same period of 2023[117]. - Cash, cash equivalents, and marketable securities decreased by $140.9 million during the nine months ended September 30, 2024, primarily due to operating expenses[126]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $151.4 million, compared to $107.9 million for the same period in 2023, reflecting an increase in net loss from $119.7 million to $179.8 million[137][140]. - The net decrease in cash, cash equivalents, and restricted cash for the nine months ended September 30, 2024, was $25.5 million, an improvement from a decrease of $35.6 million in the same period of 2023[138]. Research and Development - The Phase 3 SAPPHIRE trial for apitegromab enrolled 156 patients aged 2-12, achieving a statistically significant improvement in motor function with a mean difference of 1.8 points in HFMSE compared to placebo (p=0.0192)[88]. - 30.4% of patients receiving apitegromab in the main efficacy population had a ≥3 point improvement in HFMSE versus 12.5% of patients on placebo[91]. - The company plans to submit a U.S. Biologics License Application and a European Union marketing authorization application for apitegromab in Q1 2025, with a potential commercial launch in 2025[76]. - SRK-439, a novel anti-myostatin monoclonal antibody, is being developed for cardiometabolic disorders, with an IND submission targeted for 2025[78]. - The DRAGON trial for SRK-181, an inhibitor for cancers resistant to anti-PD-(L)1 therapies, completed enrollment in December 2023 and is ongoing[80]. - The company is advancing multiple antibody profiles toward product candidate selection, including those targeting latent TGFβ1 for fibrotic diseases[75]. - The company expects substantial ongoing research and development expenses driven by clinical stage programs, particularly for apitegromab and SRK-439[121]. - The external costs associated with apitegromab increased by $12.5 million in Q3 2024, primarily due to clinical trial costs[110]. - The company completed enrollment of the Phase 1 DRAGON clinical trial for SRK-181 in December 2023, with expected costs for this program to decrease[121]. Regulatory and Designations - The company has received multiple designations for apitegromab, including Fast Track and Orphan Drug designations from the FDA[77]. - The FDA has granted multiple designations for apitegromab, with plans to submit a Biologics License Application in Q1 2025 and a commercial launch expected in Q4 2025[100]. Adverse Events - The most common adverse events in the main efficacy population were pyrexia (29.2%), nasopharyngitis (24.5%), and cough (24.5%)[97]. - Serious adverse events were consistent with the underlying disease, with no SAEs assessed as related to apitegromab[97]. Capital and Financing - The company raised approximately $324.4 million from a follow-on offering, including the exercise of an option for additional shares[101]. - Approximately $324.4 million in net proceeds from a follow-on offering completed in October 2024 is expected to fund operating expenses and capital expenditures into Q4 2026[144]. - The company anticipates requiring additional capital to complete clinical development and commercialization for its current programs, with future capital requirements dependent on various factors including clinical trial costs and regulatory reviews[144][145]. - Net cash provided by financing activities was $6.7 million for the nine months ended September 30, 2024, compared to $6.0 million for the same period in 2023, primarily from warrant exercises[142]. - The company continues to rely on additional financing to achieve its business objectives, with potential dilution of common stockholder ownership interests if additional capital is raised through equity offerings[146][147].

Clinical Trials and Data - Reported positive topline data from the pivotal Phase 3 SAPPHIRE trial, achieving a statistically significant mean difference of 1.8 points in HFMSE for apitegromab versus placebo in patients aged 2-12[4] - Completed enrollment of the Phase 2 EMBRAZE trial for apitegromab in obesity, with topline data expected in 2Q 2025[6] - The ONYX open-label extension study is ongoing, with over 90% of patients from the TOPAZ trial completing 4 years of treatment[4] - New data from the Phase 1 DRAGON trial of SRK-181 showed a 23.3% overall response rate in the clear cell renal cell cohort[8] - SRK-439, a novel myostatin inhibitor for obesity, is in preclinical development, with an IND filing planned for 2025[5] Financial Performance - Reported a net loss of $64.5 million or $0.66 per share for Q3 2024, compared to a net loss of $42.4 million or $0.53 per share for Q3 2023[11] - Total operating expenses for the three months ended September 30, 2023, were $43,672,000, compared to $123,263,000 for the nine months ended September 30, 2023[20] - Net loss for the three months ended September 30, 2023, was $42,359,000, resulting in a net loss per share of $0.53[20] - Research and development expenses increased to $48.7 million in Q3 2024 from $30.3 million in Q3 2023, primarily due to costs associated with ONYX and EMBRAZE trials[12] - Research and development expenses for the three months ended September 30, 2023, were $30,337,000[20] - General and administrative expenses for the three months ended September 30, 2023, were $13,335,000[20] Cash and Assets - As of September 30, 2024, cash, cash equivalents, and marketable securities totaled approximately $139.1 million, with a proforma amount of $463.5 million including the recent equity offering[12] - Total assets decreased from $311,035,000 as of December 31, 2023, to $179,133,000 as of September 30, 2024[21] - Cash, cash equivalents, and marketable securities decreased from $279,938,000 to $139,065,000[21] - Total stockholders' equity decreased from $225,218,000 to $79,196,000[21] Liabilities - Total liabilities increased from $85,817,000 to $99,937,000 during the same period[21] Future Plans - Plans to submit a U.S. Biologics License Application (BLA) and European Union marketing authorisation application (MAA) in 1Q 2025[2] - Raised $345 million in an upsized public offering to fund the commercial launch of apitegromab and advance priority programs[10]

Core Insights - Scholar Rock Holding Corporation (SRRK) is showing potential as a technical investment due to a recent "golden cross" formation, where the 50-day simple moving average has crossed above the 200-day simple moving average, indicating a possible bullish breakout [1] - Over the past four weeks, SRRK has experienced a significant price increase of 225.4%, and currently holds a 3 (Hold) rating on the Zacks Rank, suggesting further breakout potential [2] - The positive earnings outlook for SRRK, with no downward revisions in earnings estimates and two upward revisions in the past two months, strengthens the bullish case for the stock [2] Technical Analysis - The golden cross pattern consists of three stages: a downtrend that bottoms out, a crossover of the shorter moving average above the longer moving average, and a continuation of upward momentum [1] - As of October 18, 2024, SRRK's 50-day simple moving average is at 12.789, while the 200-day simple moving average is at 12.743, indicating a strong technical position [2] - The volume of trading for SRRK is reported at 146.97, which may reflect increased investor interest [2]

Core Viewpoint - Scholar Rock's stock surged 364% following positive clinical trial results for its lead candidate, apitegromab, which is being tested for spinal muscular atrophy (SMA) treatment [1][2]. Company Overview - Scholar Rock is a clinical-stage drugmaker focusing on therapies for muscle-wasting diseases, with apitegromab as its primary candidate [3][4]. Clinical Trial Results - Apitegromab is an antibody targeting myostatin, tested in the phase 3 Sapphire trial involving 156 nonambulatory SMA patients aged 2 to 12 [3][4]. - The trial showed that adding apitegromab to standard care improved patient scores on the expanded Hammersmith functional motor scale (HFMSE) by an average of 1.8 points [5]. - After 12 months, 30.4% of patients improved by three or more points compared to 12.5% in the placebo group, indicating significant efficacy [5]. Market Potential - Apitegromab could generate over $1 billion in annual sales at peak, as it serves as a companion therapy for SMA alongside existing treatments like Spinraza and Evrysdi [7]. - Analysts are optimistic about apitegromab's potential beyond SMA, particularly in combination with anti-obesity medications like tirzepatide, which has seen rapid sales growth [8][9]. Future Developments - Scholar Rock plans to submit marketing authorization applications for apitegromab to the FDA and European Medicines Agency in Q1 2025 [10]. - If accepted, the fast-track designation could reduce the review period from 10 months to 6, potentially allowing sales to begin by the end of next year [11]. - The company expects to report top-line data from the phase 2 Embraze trial in Q2 2025, which could further influence stock performance [12][13]. Valuation - Scholar Rock currently has a market cap of $2.5 billion, reflecting its potential for SMA treatment approval [14].

Core Viewpoint - Scholar Rock's shares surged 362% following the announcement of positive results from the late-stage study of apitegromab for treating spinal muscular atrophy (SMA) [1] Company Overview - Scholar Rock is focused on developing apitegromab, a fully human monoclonal antibody that inhibits myostatin activation, which is crucial for muscle growth regulation [2] - The company has seen an 82.3% increase in shares year-to-date, contrasting with a 2.8% decline in the industry [7] Clinical Study Results - The phase III SAPPHIRE study met its primary endpoint, showing a statistically significant improvement in motor function in SMA patients treated with apitegromab compared to placebo at week 52, measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) [3] - In the main efficacy group (ages 2-12), 30% of patients on apitegromab achieved a 3-point or more improvement in HFMSE, compared to 12.5% in the placebo group [4] - Early motor function gains were observed as soon as eight weeks into treatment, with continued improvement at 52 weeks [5] Safety Profile - Apitegromab was well-tolerated across all age groups, with no significant differences in adverse events between the two dosage strengths (10 mg/kg and 20 mg/kg) [7] - Serious adverse events were not attributed to apitegromab, and no patients discontinued the study due to adverse events [7] Regulatory and Future Plans - Scholar Rock plans to submit regulatory applications for apitegromab in the U.S. and EU in Q1 2025, with existing designations such as Fast Track and Orphan Drug in the U.S. and Priority Medicines in the EU [8] - Detailed data from the SAPPHIRE study is expected to be presented at a medical conference in early 2025 [9] Other Developmental Programs - Scholar Rock is also evaluating apitegromab for cardiometabolic disorders, with a phase II EMBRAZE study completed for preserving lean muscle mass in individuals on GLP-1 receptor agonist therapy [10] - The company is advancing SRK-439, a selective myostatin inhibitor, for treating cardiometabolic disorders [11] - Another candidate, SRK-181, is in a phase I study for advanced cancer treatment [12] Market Impact - Following Scholar Rock's positive data, Biohaven's stock rose 10.8%, as investors anticipate favorable results from its own SMA study [13] - Biohaven is evaluating taldefgrobep alfa (BHV-2000) in a phase III study for SMA, with top-line data expected by the end of 2024 [14]

Group 1 - Scholar Rock Holding Corp. (SRRK) shares increased by 335% following positive Phase 3 trial results for their drug apitegromab, which targets spinal muscular atrophy (SMA) and significantly improves motor function in patients [1] - In the SAPPHIRE study, patients receiving apitegromab experienced an average increase of over 3 points in the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52, while only 12.5% of placebo patients achieved similar improvements [2] - Patients on apitegromab began to see enhancements in motor function as early as week 8, with these improvements sustained throughout the 52-week study [2] Group 2 - Scholar Rock plans to submit regulatory applications for apitegromab to the U.S. FDA and the European Medicines Agency (EMA) in Q1 2025, potentially providing new treatment options for SMA patients and fostering growth in the biopharmaceutical sector [3]

Core Insights - Scholar Rock Holding Corporation (NASDAQ: SRRK) reported positive results from its phase 3 SAPPHIRE study for apitegromab, aimed at treating spinal muscular atrophy (SMA) [2] Group 1 - The SAPPHIRE study results indicate a significant advancement in the treatment options available for SMA patients [2] - The article highlights the importance of the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities [2] Group 2 - The author of the article is affiliated with Biotech Analysis Central, which offers a library of over 600 biotech investing articles and a model portfolio of small and mid-cap stocks [2]

Market Overview - U.S. stocks traded lower with the Dow Jones index falling more than 200 points, down 0.49% to 42,146.78, NASDAQ down 0.36% to 18,073.37, and S&P 500 down 0.32% to 5,732.84 [1] - Energy shares increased by 0.5%, while consumer discretionary shares decreased by 1.3% [1] Company News - AstraZeneca entered an exclusive license agreement with CSPC Pharmaceutical Group to develop a novel small-molecule Lp(a) disruptor for dyslipidemia patients [2] - Scholar Rock Holding Corporation's shares surged 310% to $30.44 after positive Phase 3 SAPPHIRE trial results for apitegromab in SMA patients [2] - Arcadium Lithium PLC shares rose 41% to $4.34 following acquisition interest from Rio Tinto [2] - Duckhorn Portfolio Inc shares increased by 102% to $10.88 after better-than-expected Q4 results and an acquisition agreement with Butterfly Equity [2] Declining Stocks - Trevena, Inc. shares dropped 48% to $2.4192 due to a Nasdaq delisting notification [3] - Heritage Insurance Holdings, Inc. shares fell 21% to $9.65 [3] - Universal Insurance Holdings, Inc. shares decreased 18% to $17.12 [3] Commodities - Oil prices increased by 3.4% to $76.99, while gold prices decreased by 0.2% to $2,662.50 [4] - Silver fell 1.1% to $32.025, and copper decreased by 0.5% to $4.5535 [4] Economic Indicators - Eurozone retail sales rose 0.2% month-over-month in August, while the Halifax House Price Index in the UK increased by 4.7% year-over-year in September [5] - Germany's factory orders declined by 5.8% month-over-month in August [5] Asia Pacific Markets - Asian markets closed mixed with Japan's Nikkei 225 gaining 1.80% and Hong Kong's Hang Seng Index gaining 1.60%, while India's BSE Sensex dipped 0.78% [6] Foreign Exchange Reserves - Singapore's foreign exchange reserves fell to SGD 499.7 billion in September from SGD 501.3 billion [7] - Japan's leading economic indicators index fell to 106.7 in August, and the coincident economic indicators index declined to 113.5 [7]

Scholar Rock Holding Corporation (NASDAQ:SRRK) Q2 2024 Results Conference Call August 8, 2024 8:15 AM ET Company Participants Jing Marantz - Chief Medical Officer Mo Qatanani - Chief Scientific Officer Jay Backstrom - Chief Executive Officer Conference Call Participants Michael Yee - Jefferies David Nierengarten - Wedbush Securities Etzer Darout - BMO Capital Markets Tessa Romero - JPMorgan Gary Nachman - Raymond James Srikripa Devarakonda - Truist Securities Jay Backstrom Thank you, Sandra. Good morning a ...

Exhibit 99.1 Scholar Rock Reports Second Quarter 2024 Financial Results and Highlights Business Progress - Remains on track to report topline data from pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy (SMA) in 4Q 2024 - New data from Phase 2 TOPAZ extension study in patients with nonambulatory SMA showed sustained clinical benefit over 48 months, a continued favorable safety profile with no new safety findings; patient retention rate of over 90% - Phase 2 EMBRAZE proof-of-concept tria ...