Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months

Twelve-month durability data from Metagenomi’s ongoing nonhuman primate (NHP) study 
in hemophilia A remains generally consistent with data previously released at 4.5 months

NHPs remain healthy and exhibit normal weight gain;
treatment is generally well tolerated

Program on track for IND filing in 2026

Company to host conference call with management
and Dr. Glenn Pierce, international thought leader in hemophilia A

EMERYVILLE, Calif., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.

“We are thrilled to achieve this preclinical milestone supporting our recent decision to declare MGX-001 as our development candidate for hemophilia A,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We conducted this NHP study in response to a competitive landscape where gene therapies have been unable to achieve long term persistence of FVIII activity levels in patients. Establishing proof-of-concept of site specific gene integration and durable activity levels of FVIII in NHPs over twelve months in hemophilia A represents an important validation of our platform. Our goal for MGX-001 is to provide a one-time, curative treatment for adults and children with hemophilia A. Furthermore, we intend to leverage the MGX-001 editing platform to pursue additional therapies for secreted protein disorders.”

The NHP study involved treating three NHPs with a single intravenous dose of an adeno associated virus (AAV) containing a FVIII donor template followed 35 days later by a single intravenous dose of a lipid nanoparticle (LNP) containing a novel Metagenomi nuclease and associated guide RNA targeting the first intron of the albumin gene. Each animal received only a single dose of dexamethasone prior to the AAV and LNP doses. The NHPs were then followed for safety and donor FVIII activity. The NHPs also underwent liver biopsy on Day 7 to evaluate editing and integration efficiency. The study remains ongoing.