Scholar Rock(SRRK) - 2024 Q3 - Quarterly Results

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]