Metagenomi Therapeutics Announces FDA Clearance of Investigational New Drug Application for MGX-001 and Reports Third Quarter 2026 Financial Results
Globe Newswire•10/11/2026•16:05 ET
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Key Highlights
- ➤FDA clears MGX-001 IND for first-in-human hemophilia A trial
- ➤INTEGR8 Phase 1/2 trial planned to begin in first half of 2027
- ➤Initial MGX-001 clinical data expected in second half of 2027
- ➤Cash, equivalents and securities totaled $101.7 million at September 30
- ➤Cash runway anticipated to support operations into 2028
Expert Statements
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“We are thrilled to reach this pivotal milestone with the FDA clearance of our IND for MGX-001, our lead, wholly owned program in hemophilia A”
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“MGX-001 is designed to leverage our proprietary genome editing platform to integrate a Factor VIII (FVIII) gene site-specifically into the genome, harnessing the power of the body’s native albumin promoter to drive durable, endogenous FVIII production.”
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“This represents an exciting departure from prior approved gene therapy for hemophilia A that relied on episomal FVIII expression.”
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“The MGX-001 preclinical data demonstrated FVIII activity in the normal physiologic range and durable expression in non-human primates and no detectable off-target editing.”
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“In a landscape with no cure for hemophilia A, we believe MGX-001 has the potential to provide long-term benefit to both adult and pediatric patients.”
Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics
“We plan to initiate the INTEGR8 global clinical trial in the first half of 2027 and provide initial clinical data, including safety, tolerability, and FVIII functional activity data, in the second half of 2027.”
FDA clearance enables initiation of a first-in-human Phase 1/2 trial of MGX-001 in patients with hemophilia A
MGX-001 is designed to deliver durable, endogenous Factor VIII (FVIII) activity from a one-time treatment for adults and children
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