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Metagenomi, Inc. (NASDAQ:MGX) on Tuesday shared new dose range finding data from its MGX-001 hemophilia A program.
The data demonstrated curative factor VIII (FVIII) activity in non-human primates (NHPs) and informs a clinical dose regimen strategy.
Hemophilia A is the most common X-linked inherited bleeding disorder, caused by a large variety of mutations in the FVIII gene, leading to a loss of functional FVIII protein.
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Factor VIII activity refers to a blood test that measures the function of Factor VIII, a protein essential for blood clotting.
Metagenomi intends to advance MGX-001 into clinical development.
In this preclinical dose range finding study, a single dose of AAV containing a B-domain deleted human FVIII gene was administered to 24 NHPs in six dose cohorts.
Each animal received a single dose of corticosteroids before both AAV and LNP doses.
A functional cure is generally defined as FVIII levels of 50% to 150% of normal human levels.
Therapeutically relevant levels of FVIII activity were achieved in the five highest AAV doses of the six dose cohorts.
FVIII activity exhibited both AAV and LNP dose dependency, with no animal exceeding 150% of normal, the maximum acceptable level of human FVIII activity.
The treatment was well tolerated in all animals without significant elevation of liver enzymes, except in the highest dose of LNP, where transient elevations in liver enzymes were observed.
At a proposed clinical dose of AAV at 5e12 vg/kg and LNP at 0.6 mg/kg, MGX-001 achieved average FVIII activity of 49% within a range of 29.3% – 59.5%.
The company expects a pre-investigational new drug regulatory meeting in the fourth quarter of 2025, with investigational new drug and clinical trial application submissions expected in the fourth quarter of 2026.
The company has evolved its pipeline and discovery efforts to focus on advancing its leading in vivo therapeutics, including the MGX-001 program in hemophilia A, other secreted protein disorders leveraged by the MGX-001 approach, and cardiometabolic indications in collaboration with Ionis Pharmaceuticals Inc. (NASDAQ:IONS).
In line with this strategic focus, the company deprioritized early discovery and platform research and reduced its workforce by 25%. With the update, Metagenomi expects to extend its cash runway into the fourth quarter of 2027.
Cash, cash equivalents, and available-for-sale marketable securities were $184.1 million.
MGX Price Action: Metagenomi shares were down 15.68% at $1.85 at the time of publication on Wednesday, according to Benzinga Pro data.
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