Metagenomi Reports Business Updates and Full Year 2024 Financial Results
Hemophilia A presentation at American Society of Hematology (ASH) 66th Annual Meeting demonstrated sustained Factor VIII (FVIII) activity in nonhuman primate (NHP) study for more than 16 months
Leveraged hemophilia A albumin platform to achieve in vivo proof-of-concept in multiple secreted protein deficiencies to support wholly-owned follow-on program
Progressed four Wave 1 Ionis targets to lead optimization with plans to declare one to two development candidates (DCs) in 2025
Well capitalized with $248.3 million in cash, cash equivalents and available-for-sale marketable securities at the end of Q4 2024; Cash runway anticipated to support operating plans into 2027
EMERYVILLE, Calif., March 17, 2025 (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 reported financial results for the full year ended December 31, 2024, and provided business updates.
"Our diverse and modular AI-driven metagenomics platform is designed to precisely target any site in the human genome, yielding the potential to address the full spectrum of genetic diseases," said Brian C. Thomas, PhD, CEO and founder of Metagenomi. "In 2024, we made significant progress toward our goal of developing curative genetic medicines for patients. We progressed MGX-001, our potentially transformative treatment for hemophilia A, and leveraged the MGX-001 platform to advance an additional wholly-owned program for an undisclosed secreted protein deficiency disorder. We advanced Wave 1 of our Ionis collaboration, progressing all four targets in cardiometabolic indications. We defined our goals for 2025 and 2026, and remain on track to submit our first IND in hemophilia A in 2026."
2024 Pipeline Achievements
Hemophilia A Program
Declared development candidate for wholly-owned program in hemophilia A, MGX-001
Oral presentation at ASH demonstrated sustained FVIII activity in an NHP study for more than 16 months, supporting the durability of our gene editing therapy
Nominated a development candidate MGX-001 that includes a bioengineered FVIII construct with higher FVIII activity levels vs wild type construct
Completed initial regulatory engagement with FDA and initiated GxP manufacturing activities
Secreted Protein Deficiencies
Identified targets for wholly-owned therapeutic programs leveraging the gene integration approach used in MGX-001 and achieved in vivo proof-of-concept in rodents across three secreted protein targets
Cardiometabolic Indications
Advanced four Wave 1 Ionis collaboration programs to lead optimization, including transthyretin (TTR) for transthyretin amyloidosis and angiotensinogen (AGT) for refractory hypertension, and achieved in vivo proof-of-concept in rodents across all four programs
Demonstrated 95% protein knockdown in spontaneous hypertensive rats, a widely used preclinical model for refractory hypertension, which represents an example of the progress across the collaboration
Key 2024 Technology Achievements
Used artificial intelligence (AI), ancestral state reconstruction, and structural biology to enhance our gene editing systems
Presented compact SMall Arginine-Rich sysTems (SMART) nucleases demonstrating robust in vitro genome editing activity at multiple therapeutically relevant loci
Presented novel adenine base editors (ABEs) achieving over 95% knockdown of three target proteins in primary T-cells via simultaneous triplex editing with high specificity and post-editing cell health
2025 - 2026 Anticipated Milestones
Hemophilia A
Plan to release final FVIII durability and related preclinical study data from NHP durability study in the first half of 2025
On track for Pre-IND and ex-US regulatory meetings in 2025
Plan to file IND/ CTA submissions in 2026 to advance MGX-001 into first-in-human studies
Secreted Protein Deficiencies
Plan to demonstrate NHP proof-of-concept for lead secreted protein deficiency target in 2025 and nominate DC for lead secreted protein deficiency in 2026
Cardiometabolic Indications
On track to nominate one to two DCs from the four Wave 1 Ionis collaboration development programs and disclose remaining therapeutic indications in large cardiometabolic indications in 2025
Plan to initiate IND-enabling activities for DCs nominated in 2025 and nominate additional DCs from the remaining Wave 1 targets in 2026
Other Business Updates
Eric Bjerkholt, CFO of Mirum Pharmaceuticals, Inc., joined Metagenomi's Board of Directors, serving on Metagenomi's Audit and Compensation committees
Publication in Nature Communications describing novel, compact CRISPR-associated transposases (CAST) demonstrated integration of a large, therapeutically relevant gene into the genome of human cells using CAST systems
Full Year 2024 Financial Results
Cash Position: Cash, cash equivalents, and available-for-sale marketable securities were $248.3 million as of December 31, 2024.
R&D Expenses: Research and development (R&D) expenses were $109.2 million for the full year ended December 31, 2024, compared to $94.4 million for the full year ended December 31, 2023.
G&A Expenses: General and administrative (G&A) expenses were $32.0 million for the full year ended December 31, 2024, compared to $28.8 million for the full year ended December 31, 2023.
About Hemophilia A
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. Intracranial bleeding is of greatest concern as this can lead to major morbidity and mortality. Bleeding into joints leads to cumulative joint damage and is a major cause of morbidity. Diagnosis of severe disease typically occurs in infancy due to exaggerated bleeding in response to minor injury or routine medical procedures. Prevalence is estimated to be up to 26,500 patients in the US and more than 500,000 patients globally according to the World Federation of Hemophilia, with the vast majority of patients being male.
About Metagenomi
Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its AI-driven metagenomics platform. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases (CAST)). Metagenomi believes its proprietary, modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://metagenomi.co.
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