Metagenomi gets FDA clearance; trial planned H1 2027
Cash runway is anticipated to support operations into 2028 as clinical testing is planned for 2027.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Filing Summary
Metagenomi Therapeutics, Inc. (MGX) said the FDA cleared its Investigational New Drug application for MGX-001, enabling the planned Phase 1/2 INTEGR8 trial for hemophilia A. The company plans to start the trial in the first half of 2027 and provide initial clinical data in the second half of 2027. The trial is designed to assess safety and tolerability and identify a dose targeting normal physiologic Factor VIII activity.
Cash, cash equivalents and available-for-sale marketable securities totaled $101.7 million as of September 30, 2026; the company anticipates its runway will support operations into 2028. Third-quarter collaboration revenue was $2.184 million, compared with $8.659 million in 2025. R&D expense was $17.532 million versus $25.283 million; G&A expense was $6.878 million versus $6.215 million, including a $1.2 million one-time net litigation settlement expense. Net loss was $21.126 million, compared with $20.393 million.
MGX-001 is designed as a one-time treatment using site-specific FVIII gene integration at the albumin locus. Company-reported non-human-primate studies showed FVIII activity in the normal physiologic range and no detectable off-target editing; an earlier study reported activity over approximately 19 months.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Moderate point. Forward-looking: it has not happened yet and may not happen.IND clearance enables planned MGX-001 trial initiation in the first half of 2027.
Negative
- Moderate pointQuarterly collaboration revenue was $2.184 million, compared with $8.659 million in 2025.
Filing Explained
The filing reports cash, cash equivalents and available-for-sale securities of
8-K Event Classification
Key Figures
Key Terms
Investigational New Drug (IND) regulatory
endogenous FVIII expression medical
episomal FVIII expression medical
dose escalation medical
off-target editing technical
Earnings Snapshot
The company anticipates that the runway will support operations into 2028.
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What will MGX's INTEGR8 trial evaluate?
How long is MGX's cash expected to last?
What were MGX's Q3 2026 collaboration revenue and net loss?
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Item 2.02 Results of Operations and Financial Condition.
On November 10, 2026, Metagenomi Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended September 30, 2026 and additional business updates. A copy of the press release in connection with the announcement is being furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
The information in this Item 2.02 (including Exhibit 99.1 attached hereto) is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”), except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
Exhibit No. |
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Description |
99.1 |
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Earnings Press Release Issued by Metagenomi Therapeutics, Inc. on November 10, 2026 |
104 |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Metagenomi Therapeutics, Inc. |
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November 10, 2026 |
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/s/ Jian Irish |
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Jian Irish, Ph.D., M.B.A. |
Exhibit 99.1
Metagenomi Therapeutics Announces FDA Clearance of Investigational New Drug Application for MGX-001 and Reports Third Quarter 2026 Financial Results
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
Company plans to initiate the INTEGR8 clinical trial in the first half of 2027 and provide initial clinical data in the second half of 2027
$101.7 million in cash, cash equivalents, and available-for-sale marketable securities as of September 30, 2026, with runway anticipated to support operations into 2028
EMERYVILLE, Calif., November 10, 2026 (GLOBE NEWSWIRE) – Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the “Company”), an in vivo genome editing company capitalizing on its proprietary technologies to create potentially curative genetic medicines for patients, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for MGX-001, its lead, wholly owned program for the treatment of hemophilia A. The Company also reported financial results for the third quarter ended September 30, 2026, and provided business updates.
“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,” said Jian Irish, Ph.D., M.B.A., 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. This represents an exciting departure from prior approved gene therapy for hemophilia A that relied on episomal FVIII expression. 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. 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. 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.”
Third Quarter 2026 and Recent Updates
MGX-001 – Hemophilia A Program
Site-Specific Large Gene Integration for Other Protein Deficiency Diseases
Third Quarter 2026 Financial Results
Cash Position: Cash, cash equivalents and available-for-sale marketable securities were $101.7 million as of September 30, 2026. The Company anticipates that the runway will support operations into 2028.
R&D Expenses: Research and development (R&D) expenses were $17.5 million for the quarter ended September 30, 2026, compared to $25.3 million for the comparable period in 2025.
G&A Expenses: General and administrative (G&A) expenses were $6.9 million for the quarter ended September 30, 2026, including a $1.2 million one-time net litigation settlement expense, compared to $6.2 million for the comparable period in 2025.
About MGX-001
MGX-001 is an investigational, one-time genome editing treatment designed to provide life-long protection from bleeding events and joint damage in adults and children with hemophilia A. MGX-001 is designed to site-specifically integrate the FVIII gene in a “safe harbor” location within an intron of the albumin gene, leveraging the strong native albumin promoter to drive endogenous FVIII expression. MGX-001 consists of an adeno-associated virus (AAV) vector that delivers the promoterless FVIII gene cassette as donor DNA, and a lipid nanoparticle (LNP) that delivers mRNA encoding the Company’s proprietary Type V CRISPR nuclease, MG29-1, together
with a guide RNA targeting the albumin gene. This represents a departure from prior approved gene therapy for hemophilia A that relied on episomal FVIII expression.
In preclinical studies in non-human primates (NHPs), MGX-001 demonstrated FVIII activity in the normal physiologic range, with clear dose dependency across both the AAV and LNP components, and, in an earlier study, durable FVIII activity over approximately 19 months. MGX-001 has also shown no detectable off-target editing in a series of orthogonal assays.
About Hemophilia A
Hemophilia A is the most common X-linked inherited and de novo bleeding disorder, largely affecting males, and is caused by a variety of mutations in the FVIII gene that all lead to loss of functional FVIII protein. Hemophilia A affects approximately 25,000 patients in the United States and an estimated 500,000 worldwide. Current standard-of-care treatments, including FVIII replacement therapy and bispecific antibody therapy, require lifelong, repeated dosing, are not curative and carry a risk of breakthrough bleeding.
About Metagenomi Therapeutics
Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create potentially 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 CRISPR gene-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, insertion and correction.
MGX-001, the Company’s lead, wholly owned development program in hemophilia A, has demonstrated a preclinical profile with best-in-class treatment potential, including targeted genome editing and durable gene expression in a one-time treatment. The FDA has cleared the Company’s Investigational New Drug (IND) application for MGX-001, and the Company is advancing MGX-001 into the clinic in its global Phase 1/2 INTEGR8 trial. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children, potentially enabling a new standard of care for the treatment of hemophilia A. The Company is also currently pursuing indications leveraging the same site-specific large gene integration at the albumin locus as used in MGX-001 and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.co/.
Cautionary Note Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as “anticipate,” “believe,”
“could,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions include, but are not limited to, any statements relating to our product development programs, including the timing of and our ability to make regulatory filings such as INDs, the design and timing of the INTEGR8 trial and initial clinical data, expectations regarding MGX-001 including the preclinical profile with best-in-class treatment potential, statements regarding the Company’s plans to prioritize its preclinical pipeline and potential for value creation and sustainable growth, statements regarding upcoming milestones, statements concerning the potential of therapies and product candidates, statements concerning the impact of the organizational restructuring, statements concerning our anticipated cash runway, and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of IND submissions and starting and completing clinical trials; the risk that the FDA or other regulatory authorities may place a clinical hold on our clinical trials or that initiation of our clinical trials may be delayed; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation and the current regulatory environment; patent and intellectual property matters; competition; the volatility of capital markets and other adverse macroeconomic factors; as well as other risks described in “Risk Factors,” in our most recent Form 10-K and Form 10-Q and other risk factors set forth from time to time in our filings with the Securities and Exchange Commission made pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
Investor Contact:
Brian Ritchie
LifeSci Advisors
britchie@lifesciadvisors.com
Media Contact:
Michael Fitzhugh
LifeSci Communications
mfitzhugh@lifescicomms.com
Condensed Financial Statements
Condensed Balance Sheet Data
(Unaudited)
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September 30, |
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December 31, |
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(In thousands) |
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2026 |
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2025 |
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Cash, cash equivalents and available-for-sale marketable securities |
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$ |
101,674 |
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$ |
160,799 |
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Total assets |
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$ |
157,388 |
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$ |
221,103 |
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Total liabilities |
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$ |
64,200 |
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$ |
62,507 |
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Total stockholders’ equity |
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$ |
93,188 |
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$ |
158,596 |
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Total liabilities and stockholders’ equity |
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$ |
157,388 |
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$ |
221,103 |
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Condensed Statements of Operations
(Unaudited)
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Three Months Ended |
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Nine Months Ended |
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(In thousands, except share and per share data) |
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2026 |
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2025 |
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2026 |
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2025 |
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Collaboration revenue |
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$ |
2,184 |
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$ |
8,659 |
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$ |
3,175 |
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$ |
21,299 |
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Operating expenses: |
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Research and development |
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17,532 |
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25,283 |
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59,344 |
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72,932 |
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General and administrative |
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6,878 |
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6,215 |
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19,417 |
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20,013 |
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Total operating expenses |
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24,410 |
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31,498 |
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78,761 |
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92,945 |
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Loss from operations |
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(22,226 |
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(22,839 |
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(75,586 |
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(71,646 |
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Other income (expense): |
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Interest income |
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1,047 |
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2,214 |
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3,842 |
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7,586 |
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Change in fair value of long-term investments |
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— |
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— |
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— |
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(1,292 |
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Other income (expense), net |
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53 |
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(2 |
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87 |
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(80 |
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Total other income (expense), net |
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1,100 |
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2,212 |
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3,929 |
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6,214 |
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Net loss before provision for income taxes |
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(21,126 |
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(20,627 |
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(71,657 |
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(65,432 |
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Benefit from (provision for) income taxes |
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— |
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234 |
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(10 |
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92 |
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Net loss |
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$ |
(21,126 |
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$ |
(20,393 |
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$ |
(71,667 |
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$ |
(65,340 |
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Net loss per share attributable to common stockholders, basic and diluted |
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$ |
(0.56 |
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$ |
(0.55 |
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$ |
(1.90 |
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$ |
(1.76 |
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Weighted average common shares outstanding, basic and diluted |
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37,775,051 |
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37,349,449 |
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37,672,176 |
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37,176,361 |
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