Avidity Biosciences(RNA) - 2025 Q1 - Quarterly Report

Financial Performance - The company reported net losses of $322.3 million and$212.2 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $1.0 billion as of March 31, 2025[81][82]. - Revenue for the three months ended March 31, 2025, was$1.573 million, a decrease of $1.970 million compared to the same period in 2024[96][97]. - The company has not generated any revenue from product sales since its inception in 2012 and plans to finance future needs through equity offerings and collaborations[80][83]. - Other income increased by$7.4 million for the three months ended March 31, 2025, due to higher interest income from marketable securities and cash[101]. - The company reported a net increase in cash, cash equivalents, and restricted cash of $34.3 million in Q1 2025, down from$286.3 million in Q1 2024, reflecting a decrease of $251.9 million[108]. Cash and Funding - The company has approximately$1.4 billion in cash, cash equivalents, and marketable securities as of March 31, 2025, expected to fund operations for at least 12 months[83]. - The company had cash, cash equivalents, and marketable securities of $1.4 billion as of March 31, 2025, sufficient to fund operations for at least 12 months[105]. - Net cash used in operating activities increased to$124.8 million for Q1 2025 from $70.4 million in Q1 2024, primarily due to higher research and development costs[109]. - Net cash provided by investing activities was$157.2 million in Q1 2025, driven by $330.2 million from maturities of marketable securities, offset by$169.3 million in purchases of marketable securities[110]. - Net cash provided by financing activities decreased significantly to $1.9 million in Q1 2025 from$389.4 million in Q1 2024, which included $244.2 million from common stock sales[111]. - The company terminated the 2022 Sales Agreement in August 2024 and entered into a new agreement allowing for the sale of up to$400 million in common stock[102][103]. Research and Development - Del-desiran is currently in Phase 3 development with the global HARBOR trial, involving approximately 150 participants aged 16 and older[70]. - The Phase 1/2 EXPLORE44 trial for del-zota demonstrated statistically significant improvements across key biomarkers, with a selected dose of 5 mg/kg every six weeks for future studies[72][74]. - Enrollment in the FORTITUDE trial for del-brax has been completed with a total of 51 participants, assessing safety and potential accelerated approval[76]. - The company plans to submit its first Biologics License Application (BLA) for del-zota by the end of 2025[74]. - Del-desiran has been granted Breakthrough Therapy designation by the FDA for the treatment of DM1, and all three programs have received Orphan Designation[67]. - The ongoing MARINA-OLE trial for del-desiran is expected to provide long-term safety data in Q4 2025[73]. - The company is advancing its AOC pipeline to develop treatments for rare diseases, including two candidates targeting rare genetic cardiomyopathies[78]. - The company anticipates ongoing increases in research and development expenses as it advances preclinical and clinical programs[89]. - Research and development expenses increased by $32.7 million to$99.490 million for the three months ended March 31, 2025, primarily due to increased external costs and higher personnel costs[99]. Expenses - General and administrative expenses rose by $19.7 million to$33.600 million for the three months ended March 31, 2025, mainly due to higher personnel costs and professional fees[100]. - The increase in cash used in operations is attributed to rising general and administrative expenses alongside research and development costs[109]. Collaborations and Agreements - The company entered into a collaboration with Bristol Myers Squibb, receiving approximately $100 million upfront, including$60 million in cash and $40 million from the purchase of common stock[84]. - The company is eligible for up to$1.35 billion in R&D milestone payments and $825 million in commercial milestone payments under the BMS Agreements[84]. Market and Risk - As of March 31, 2025, there have been no material changes in market risk compared to the previous year[115]. - The company continues to evaluate critical accounting estimates, with no material changes reported as of March 31, 2025[112]. - The company has not disclosed any new strategies or market expansions in the current report[115].