Metagenomi Reports Business Updates and Full Year 2024 Financial Results

Core Insights - Metagenomi, Inc. reported significant advancements in its hemophilia A program, demonstrating sustained Factor VIII (FVIII) activity in nonhuman primate studies for over 16 months, supporting the durability of its gene editing therapy [1][7] - The company is well-capitalized with $248.3 million in cash and equivalents as of December 31, 2024, providing a cash runway anticipated to support operations into 2027 [1][10] - Metagenomi plans to submit its first Investigational New Drug (IND) application for hemophilia A in 2026 and aims to nominate one to two development candidates from its Wave 1 Ionis collaboration programs in 2025 [2][14] Hemophilia A Program - The company has declared MGX-001 as a development candidate for hemophilia A, which includes a bioengineered FVIII construct with higher activity levels compared to the wild type [7] - An oral presentation at the American Society of Hematology (ASH) highlighted the sustained FVIII activity in nonhuman primate studies, reinforcing the potential of MGX-001 [7] - Plans are in place to release final FVIII durability and related preclinical study data in the first half of 2025 [14] Secreted Protein Deficiencies - Metagenomi has identified targets for wholly-owned therapeutic programs leveraging the gene integration approach used in MGX-001, achieving in vivo proof-of-concept in rodents across three secreted protein targets [4] - The company plans to demonstrate nonhuman primate proof-of-concept for its lead secreted protein deficiency target in 2025 and nominate a development candidate in 2026 [9] Cardiometabolic Indications - The company advanced four Wave 1 Ionis collaboration programs to lead optimization, including targets for transthyretin amyloidosis and refractory hypertension, achieving in vivo proof-of-concept in rodents [8] - Demonstrated a 95% protein knockdown in spontaneous hypertensive rats, showcasing the progress made in the collaboration [8] Financial Performance - For the full year ended December 31, 2024, Metagenomi reported research and development expenses of $109.2 million, an increase from $94.4 million in 2023 [10] - General and administrative expenses rose to $32.0 million in 2024 from $28.8 million in 2023 [11] - The net loss for 2024 was $78.1 million, compared to a net loss of $68.3 million in 2023, with a net loss per share of $2.36 [20]