Metagenomi Prioritizes Hemophilia A Program, Lays Off 25% Workforce

Core Insights - Metagenomi, Inc. has shared new dose range finding data from its MGX-001 hemophilia A program, demonstrating curative factor VIII (FVIII) activity in non-human primates, which informs a clinical dose regimen strategy [1][2][3] Data Summary - In a preclinical study, a single dose of AAV containing a B-domain deleted human FVIII gene was administered to 24 non-human primates across six dose cohorts, with each animal receiving corticosteroids before treatment [4] - A functional cure is defined as FVIII levels of 50% to 150% of normal human levels, and therapeutically relevant levels of FVIII activity were achieved in the five highest AAV doses [4] - FVIII activity showed both AAV and LNP dose dependency, with no animal exceeding 150% of normal FVIII activity; the treatment was well tolerated, except for transient liver enzyme elevations in the highest LNP dose [5] Future Plans - The company anticipates a pre-investigational new drug regulatory meeting in Q4 2025, with submissions for investigational new drug and clinical trial applications expected in Q4 2026 [6] Strategic Focus - Metagenomi has shifted its pipeline to focus on advancing in vivo therapeutics, including the MGX-001 program for hemophilia A and other secreted protein disorders, while collaborating with Ionis Pharmaceuticals on cardiometabolic indications [7] - The company has deprioritized early discovery and platform research, resulting in a 25% workforce reduction, and expects to extend its cash runway into Q4 2027, with cash and equivalents totaling $184.1 million [8] Stock Performance - At the time of publication, Metagenomi shares were down 15.68% at $1.85 [8]