Metagenomi Therapeutics Reports Business Updates and Full Year 2025 Financial Results

Completed pre-IND meeting following MGX-001 preclinical data demonstrating curative FVIII activity in non-human primates (NHPs) and remains on track for global regulatory submission including investigational new drug application (“IND”) in 4Q 2026

Announced corporate name change to Metagenomi Therapeutics, Inc. to reflect Company’s strategic evolution

$160.8 million in cash, cash equivalents and available-for-sale marketable securities as of December 31, 2025 with runway anticipated to support operations through 4Q 2027

EMERYVILLE, Calif., March 05, 2026 (GLOBE NEWSWIRE) -- Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the “Company”), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today reported financial results for the full year ended December 31, 2025, and provided business updates.

“2025 was a pivotal year for Metagenomi as we sharpened our strategic focus, advanced our core genome-editing technologies, and continued to demonstrate the breadth and durability of our technology across multiple therapeutic areas,” said Jian Irish, Ph.D., M.B.A., President and Chief Executive Officer of the Company. “Over the year, we made meaningful progress across our pipeline and collaborations, which reinforce the versatility of our signature technologies and our ability to engineer differentiated genome-editing solutions tailored to specific disease contexts. With our corporate rebranding, we are entering 2026 with a clearer identity and mission centered on unlocking the full potential of precision genetic medicines.”

Fourth Quarter 2025 Updates

MGX-001 - Hemophilia A Program

• Announced preclinical data from MGX-001 hemophilia A program that demonstrated curative FVIII activity in NHPs for a therapy with best-in-class treatment potential supporting advancement into clinical development.
• Completed a pre-IND meeting for MGX-001 and remain on track for regulatory submission to advance global clinical program, including an IND in the fourth quarter of 2026, and subject to regulatory clearance, initiate clinical trials in 2027.

Secreted Protein Deficiencies

• Demonstrated in vivo proof-of-concept in NHPs for Antithrombin (AT-III) Deficiency evidencing the potential to expand the MGX-001 site-specific genome integration system into additional curative therapies for secreted protein disorders.
  

Cardiometabolic Indications

• Presented preclinical data supporting APOC3 as a new collaboration target with Ionis Pharmaceuticals (“Ionis”) at the Nature Conference, “Cracking the Code: Nucleic Acid Medicines Coming of Age.”
  • APOC3 is a part of Wave 1 of the Ionis collaboration, which includes four targets in significant cardiometabolic indications.
• Completed corporate name change to Metagenomi Therapeutics, Inc. to reflect the Company’s strategic evolution focused on advancing its lead program in hemophilia A and other compelling programs and technologies that have the highest probability of success.
  

Full Year 2025 Financial Results

Cash Position: Cash, cash equivalents, and available-for-sale marketable securities were $160.8 million as of December 31, 2025.

R&D Expenses: Research and development (R&D) expenses were $94.4 million for the full year ended December 31, 2025, compared to $109.2 million for the full year ended December 31, 2024.

G&A Expenses: General and administrative (G&A) expenses were $26.8 million for the full year ended December 31, 2025, compared to $32.0 million for the full year ended December 31, 2024.

About Metagenomi Therapeutics

Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, integration and correction.

MGX-001, the Company’s lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile potentially competitive with best-in-class treatment options, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children with hemophilia A. The Company is also currently pursuing other secreted protein deficiencies leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.co.

Cautionary Note Regarding Forward‐​Looking Statements

Condensed Financial Statements Condensed Balance Sheet Data (Unaudited)
	December 31,				December 31,
(in thousands)	2025				2024
Cash, cash equivalents and available-for-sale marketable securities	$	160,799			$	248,307
Total assets	$	221,103			$	324,599
Total liabilities	$	62,507			$	89,742
Total stockholders’ equity	$	158,596			$	234,857
Total liabilities and stockholders’ equity	$	221,103			$	324,599

Condensed Statements of Operations (Unaudited)
	Years Ended December 31,
(in thousands, except share and per share data)	2025				2024
Collaboration revenue	$	25,210			$	52,295
Operating expenses:
Research and development		94,433				109,179
General and administrative		26,790				32,017
Total operating expenses		121,223				141,196
Loss from operations		(96,013	)			(88,901	)
Other income (expense):
Interest income		9,470				14,722
Change in fair value of long-term investments		(1,292	)			(9,185	)
Other expense, net		(91	)			(207	)
Total other income, net		8,087				5,330
Net loss before benefit from income taxes		(87,926	)			(83,571	)
Benefit from income taxes		58				5,513
Net loss	$	(87,868	)		$	(78,058	)
Net loss per share attributable to common stockholders, basic and diluted	$	(2.36	)		$	(2.36	)
Weighted average common shares outstanding, basic and diluted		37,251,050				33,027,889