Metagenomi Reports Business Updates and Second Quarter 2025 Financial Results
Metagenomi (NASDAQ: MGX), a precision genetic medicines company, reported its Q2 2025 financial results and business updates. The company maintains a strong financial position with $205.0 million in cash and equivalents, expected to fund operations into 2027. Key highlights include progress on their MGX-001 hemophilia A program, where long-term nonhuman primate data showed durable FVIII activity levels through 19 months.
The company presented three abstracts at ASGCT demonstrating proof-of-concept data for compact nucleases and CRISPR-associated transposases (CAST). R&D expenses decreased to $22.5 million from $28.3 million year-over-year, while G&A expenses reduced to $7.0 million from $8.6 million. The company strengthened its leadership by appointing Dr. Laurence Reid to the Board and establishing an R&D Committee.
Metagenomi (NASDAQ: MGX), azienda di terapie genetiche di precisione, ha comunicato i risultati finanziari del secondo trimestre 2025 e gli aggiornamenti aziendali. La società mantiene una solida posizione finanziaria con $205.0 milioni in cassa e equivalenti, che dovrebbero finanziare le attività fino al 2027. Tra i principali risultati, si segnalano i progressi del programma MGX-001 per l'emofilia A, con dati a lungo termine su primati non umani che mostrano livelli duraturi di attività di FVIII fino a 19 mesi.
La società ha presentato tre abstract all'ASGCT che forniscono dati proof-of-concept su nucleasi compatte e trasposasi associate a CRISPR (CAST). Le spese di R&S sono diminuite a $22.5 milioni rispetto a $28.3 milioni anno su anno, mentre le spese generali e amministrative sono scese a $7.0 milioni da $8.6 milioni. La governance è stata rafforzata con la nomina del Dr. Laurence Reid nel Consiglio e l'istituzione di un Comitato R&S.
Metagenomi (NASDAQ: MGX), empresa de medicinas genéticas de precisión, anunció sus resultados financieros del 2T 2025 y actualizaciones de negocio. La compañía mantiene una posición financiera sólida con $205.0 millones en efectivo y equivalentes, suficientes para financiar las operaciones hasta 2027. Entre los puntos destacados están los avances en el programa MGX-001 para hemofilia A, donde datos a largo plazo en primates no humanos mostraron niveles sostenidos de actividad de FVIII durante 19 meses.
La empresa presentó tres resúmenes en ASGCT que demuestran datos proof-of-concept para nucleasas compactas y transposasas asociadas a CRISPR (CAST). Los gastos de I+D se redujeron a $22.5 millones desde $28.3 millones interanuales, mientras que los gastos generales y administrativos bajaron a $7.0 millones desde $8.6 millones. La compañía reforzó su liderazgo con el nombramiento del Dr. Laurence Reid en el consejo y la creación de un Comité de I+D.
Metagenomi (NASDAQ: MGX)는 정밀 유전치료제 회사로서 2025년 2분기 재무실적 및 사업 업데이트를 발표했습니다. 회사는 $205.0 million의 현금 및 현금성자산을 보유해 2027년까지 운영 자금을 확보한 강한 재무상태를 유지하고 있습니다. 주요 성과로는 MGX-001 혈우병 A 프로그램에서 비인간 영장류의 장기 데이터가 19개월까지 지속적인 FVIII 활성도를 보였다는 점이 있습니다.
회사는 ASGCT에서 컴팩트 뉴클레아제 및 CRISPR 연관 전이효소(CAST)에 대한 개념 검증(proof-of-concept) 데이터를 담은 3건의 초록을 발표했습니다. 연구개발비(R&D)는 전년 동기 대비 $28.3 million에서 $22.5 million으로 감소했고, 일반관리비(G&A)는 $8.6 million에서 $7.0 million으로 축소되었습니다. 또한 로런스 리드 박사(Dr. Laurence Reid)를 이사회에 선임하고 연구개발위원회(R&D Committee)를 신설해 리더십을 강화했습니다.
Metagenomi (NASDAQ: MGX), société de médicaments génétiques de précision, a publié ses résultats financiers du deuxième trimestre 2025 et des mises à jour opérationnelles. L'entreprise conserve une situation financière solide avec $205.0 millions en liquidités et équivalents, jugées suffisantes pour financer ses activités jusqu'en 2027. Parmi les points clés figurent les progrès du programme MGX-001 pour l'hémophilie A, des données à long terme chez des primates non humains montrant des niveaux durables d'activité de FVIII jusqu'à 19 mois.
La société a présenté trois résumés à l'ASGCT apportant des données proof-of-concept pour des nucléases compactes et des transposases associées à CRISPR (CAST). Les dépenses de R&D ont diminué à $22.5 millions contre $28.3 millions d'une année sur l'autre, tandis que les frais G&A ont été réduits à $7.0 millions contre $8.6 millions. La gouvernance a été renforcée par la nomination du Dr. Laurence Reid au conseil d'administration et la création d'un comité R&D.
Metagenomi (NASDAQ: MGX), ein Unternehmen für präzise gentherapeutische Medikamente, meldete seine Ergebnisse für das zweite Quartal 2025 und Geschäftsupdates. Das Unternehmen verfügt über eine starke Finanzlage mit $205.0 Millionen an Barmitteln und Zahlungsmitteln, die voraussichtlich bis 2027 den Betrieb finanzieren. Zu den Highlights zählt der Fortschritt im MGX-001-Programm gegen Hämophilie A, wo langfristige Daten an nicht-menschlichen Primaten über 19 Monate anhaltende FVIII-Aktivitätswerte zeigten.
Das Unternehmen stellte drei Abstracts auf der ASGCT vor, die Proof-of-Concept-Daten zu kompakten Nukleasen und CRISPR-assoziierten Transposasen (CAST) zeigen. Die F&E-Ausgaben sanken auf $22.5 Millionen von $28.3 Millionen im Jahresvergleich, während die Verwaltungs- und Gemeinkosten auf $7.0 Millionen von $8.6 Millionen reduziert wurden. Die Führung wurde durch die Ernennung von Dr. Laurence Reid in den Vorstand und die Einrichtung eines F&E-Ausschusses gestärkt.
- Strong cash position of $205.0 million with runway into 2027
- Promising long-term NHP data showing durable FVIII activity levels up to 19 months
- Successful demonstration of compact nucleases and CAST systems at ASGCT
- Reduced R&D and G&A expenses compared to previous year
- Strategic appointment of experienced biotech executive Dr. Laurence Reid to Board
- IND/CTA submission timeline pushed to 2026
- Potential need to revise bioengineered FVIII construct due to unexpected activity levels
Insights
Metagenomi shows mixed progress with stable financials but delayed timeline for lead hemophilia A program amid questions about its FVIII construct.
Metagenomi's Q2 update reveals a solid financial foundation with
The MGX-001 program shows encouraging long-term data with durable FVIII activity levels through 19 months in NHPs, with one animal maintaining
Their platform technology updates from ASGCT demonstrate incremental progress in compact nucleases for extrahepatic editing and CAST systems for large genetic cargo integration in hepatocytes – both technically significant achievements that strengthen their differentiated editing toolbox. The company appears to be leveraging AI effectively for system optimization, potentially accelerating discovery timelines.
The addition of Laurence Reid to the board brings valuable experience from Decibel Therapeutics and Warp Drive Bio, while the new R&D committee structure suggests increased focus on scientific governance. Overall, Metagenomi maintains financial discipline while navigating technical challenges in their lead program, with their broad platform capabilities providing multiple paths forward despite the hemophilia A program's uncertain trajectory.
Presented three abstracts at American Society of Gene & Cell Therapy (“ASGCT”) demonstrating early proof-of-concept data across multiple gene editing technologies
Well capitalized with $205.0 million in cash, cash equivalents and available-for-sale marketable securities as of June 30, 2025 with runway anticipated to support operations into 2027
EMERYVILLE, Calif., Aug. 12, 2025 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX) (the “Company”), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today reported financial results for the quarter ended June 30, 2025, and provided business updates.
“Entering the second half of the year, we continue to advance our MGX-001 wholly-owned program for hemophilia A towards pre-IND and ex-U.S. regulatory meetings in 2025, and our collaboration with Ionis in cardiometabolic indications towards nomination of development candidates in 2025,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “Our strong balance sheet and continued focus on operational efficiency are enabling investment in both our pipeline and AI-driven metagenomics platform. At ASGCT, we showcased key advancements of our platform, including compact nucleases for extrahepatic gene editing and CRISPR-associated transposases (“CAST”) for site-specific integration of large therapeutic genes.”
Second Quarter 2025 Updates
MGX-001 Hemophilia A Program
- In May 2025, the Company reported additional long-term data from its nonhuman primate (“NHP”) study that demonstrated durable and consistent FVIII activity levels through approximately 19 months.
- Mean FVIII activity of months 12-19 following lipid nanoparticle dosing was
75% ,8% and29% in animals 1001, 1002 and 1003, respectively. - Emerging data relating to MGX-001 using a bioengineered FVIII construct has, to date, demonstrated FVIII activity higher than expected; the Company has commenced additional studies and may consider alternatives to this construct.
- Based on the current information, we plan to submit an investigational new drug (“IND”) and clinical trial application (“CTA”) in 2026.
Platform Technology Updates
- Presented three abstracts at the ASGCT 28th Annual Meeting in May 2025 highlighting early proof-of-concept data demonstrating potentially best-in-class compact nucleases for extrahepatic gene editing; and the ability for novel CAST systems to integrate genetic cargo in primary human hepatocytes, a key milestone towards large (>10 kbp) targeted genomic integrations.
- In May 2025, the Company made an oral presentation at TIDES 2025 highlighting the use of AI to systematically identify and optimize efficient compact gene editing systems.
Corporate Updates
- In August 2025, the Company appointed Laurence Reid, Ph.D., to the Board of Directors. Dr. Reid is an entrepreneur, company builder, and biotech executive, leveraging his experience as an advisor to growing biotech companies. Previously, Dr. Reid served as Chief Executive Officer of Decibel Therapeutics, Inc. (acquired by Regeneron Pharmaceuticals) and as Chief Executive Officer of Warp Drive Bio, LLC (merged with Revolution Medicines).
- Established a Research and Development (“R&D”) Committee within the Board of Directors. The new committee, including Willard Dere, M.D., Jian Irish, Ph.D., MBA, Laurence Reid, Ph.D. and Brian Thomas, Ph.D., will provide the Board with scientific and clinical strategy insights related to the Company’s research and development initiatives.
Second Quarter 2025 Financial Results
Cash Position: Cash, cash equivalents, and available-for-sale marketable securities were
R&D Expenses: R&D expenses were
G&A Expenses: General and administrative (“G&A”) expenses were
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 gene editing company leveraging the power of artificial intelligence and machine learning to develop the next generation of potentially curative genome editing therapeutics. Its metagenomics-derived gene editing toolbox has analyzed over 7.4 billion proteins and has the potential to target any type of genetic mutation across the entire human genome. The Company’s innovative and comprehensive platform has full spectrum gene editing, with technologies ranging from ultra-small nucleases and base editors to large gene integrations using CRISPR-associated transposase (CAST) and RNA-Mediated Integration Systems (RIGS). The Company’s lead, wholly-owned development program in Hemophilia A is a potentially curative therapy designed to provide life-long protection from bleeding events and joint damage in adults and children. The Company has additional wholly-owned assets in its pipeline for secreted protein deficiencies and partnered assets for 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 conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, 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; 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 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 Contacts:
Stephen Jasper
Gilmartin Group
stephen@gilmartinir.com
or
Kiki Patel, PharmD
Gilmartin Group
kiki@gilmartinir.com
| Condensed Financial Statements Condensed Balance Sheet Data (Unaudited) | ||||||||
| June 30, | December 31, | |||||||
| (in thousands) | 2025 | 2024 | ||||||
| Cash, cash equivalents and available-for-sale marketable securities | $ | 204,993 | $ | 248,307 | ||||
| Total assets | $ | 272,284 | $ | 324,599 | ||||
| Total liabilities | $ | 76,453 | $ | 89,742 | ||||
| Redeemable convertible preferred stock | $ | — | $ | — | ||||
| Total stockholders’ equity | $ | 195,831 | $ | 234,857 | ||||
| Total liabilities and stockholders’ equity | $ | 272,284 | $ | 324,599 | ||||
| Condensed Statements of Operations (Unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| (In thousands, except share and per share data) | 2025 | 2024 | 2025 | 2024 | ||||||||||||
| Collaboration revenue | $ | 8,513 | $ | 20,008 | $ | 12,640 | $ | 31,167 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 22,507 | 28,320 | 47,649 | 59,759 | ||||||||||||
| General and administrative | 6,993 | 8,551 | 13,798 | 17,303 | ||||||||||||
| Total operating expenses | 29,500 | 36,871 | 61,447 | 77,062 | ||||||||||||
| Loss from operations | (20,987 | ) | (16,863 | ) | (48,807 | ) | (45,895 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 2,485 | 3,976 | 5,372 | 7,910 | ||||||||||||
| Change in fair value of long-term investments | (1,292 | ) | — | (1,292 | ) | — | ||||||||||
| Other expense, net | (70 | ) | (51 | ) | (78 | ) | (101 | ) | ||||||||
| Total other income (expense), net | 1,123 | 3,925 | 4,002 | 7,809 | ||||||||||||
| Net loss before provision for income taxes | (19,864 | ) | (12,938 | ) | (44,805 | ) | (38,086 | ) | ||||||||
| Benefit (provision) for income taxes | (44 | ) | 2,199 | (142 | ) | 2,199 | ||||||||||
| Net loss | $ | (19,908 | ) | $ | (10,739 | ) | $ | (44,947 | ) | $ | (35,887 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.54 | ) | $ | (0.29 | ) | $ | (1.21 | ) | $ | (1.24 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 37,156,979 | 36,625,291 | 37,088,383 | 28,901,399 | ||||||||||||
FAQ
What were Metagenomi's (MGX) key financial results for Q2 2025?
What progress has Metagenomi (MGX) made in its hemophilia A program?
When does Metagenomi (MGX) plan to submit IND/CTA for its hemophilia A treatment?
What technological advances did Metagenomi present at ASGCT 2025?
How long will Metagenomi's current cash position support operations?
