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].
Scholar Rock(SRRK) - 2024 Q3 - Quarterly Report