Metagenomi Highlights Progress Across Therapeutic Portfolio and Outlines Anticipated Milestones
Metagenomi (Nasdaq: MGX) has provided updates on its therapeutic development programs and outlined key milestones for 2025-2026. The company reported significant progress in its MGX-001 hemophilia A program, achieving sustained Factor VIII activity in nonhuman primate studies over 16+ months. The company plans regulatory interactions in 2025 and IND/CTA submissions in 2026.
The company is advancing its secreted protein deficiencies program, expecting NHP proof-of-concept in 2025 and development candidate nomination in 2026. In collaboration with Ionis, Metagenomi is progressing on cardiometabolic programs, targeting one to two development candidate nominations in 2025.
Metagenomi has also advanced its technology platforms, including compact SMART nucleases and novel Adenine Base Editors. The company's cash runway is expected to support operations into 2027.
Metagenomi (Nasdaq: MGX) ha fornito aggiornamenti sui suoi programmi di sviluppo terapeutico e ha delineato i traguardi chiave per il 2025-2026. L'azienda ha riportato progressi significativi nel suo programma per l'emofilia A MGX-001, ottenendo un'attività sostenuta del Fattore VIII in studi su primati non umani per oltre 16 mesi. L'azienda prevede interazioni regolatorie nel 2025 e sottomissioni IND/CTA nel 2026.
L'azienda sta avanzando il suo programma per le carenze di proteine secreti, prevedendo una prova di concetto in primati non umani nel 2025 e la nomina di un candidato per lo sviluppo nel 2026. In collaborazione con Ionis, Metagenomi sta progredendo nei programmi cardiometabolici, mirando a una o due nomine di candidati per lo sviluppo nel 2025.
Metagenomi ha anche sviluppato le sue piattaforme tecnologiche, comprese le nucleasi SMART compatte e i nuovi Editor di Base Adenina. Si prevede che il capitale dell'azienda supporti le operazioni fino al 2027.
Metagenomi (Nasdaq: MGX) ha proporcionado actualizaciones sobre sus programas de desarrollo terapéutico y ha esbozado hitos clave para 2025-2026. La compañía reportó avances significativos en su programa MGX-001 para la hemofilia A, logrando una actividad sostenida del Factor VIII en estudios con primates no humanos durante más de 16 meses. La empresa planea interacciones regulatorias en 2025 y presentaciones IND/CTA en 2026.
La empresa está avanzando en su programa de deficiencias de proteínas secretadas, esperando una prueba de concepto en primates no humanos en 2025 y la nominación de un candidato para el desarrollo en 2026. En colaboración con Ionis, Metagenomi está avanzando en programas cardiometabólicos, con el objetivo de una o dos nominaciones de candidatos para el desarrollo en 2025.
Metagenomi también ha avanzado en sus plataformas tecnológicas, incluidas nucleasas SMART compactas y nuevos Editores de Base de Adenina. Se espera que la disponibilidad de efectivo de la empresa apoye las operaciones hasta 2027.
메타지노미 (Nasdaq: MGX)는 치료 개발 프로그램에 대한 업데이트를 제공하고 2025-2026년 주요 이정표를 설명했습니다. 이 회사는 MGX-001 혈우병 A 프로그램에서 비인간 영장류 연구에서 16개월 이상 지속적인 VIII 인자 활성을 달성하여 상당한 진행을 보고했습니다. 회사는 2025년에 규제 상호작용을 계획하고 2026년에 IND/CTA 제출을 예정하고 있습니다.
회사는 분비 단백질 결핍 프로그램을 발전시키고 있으며, 2025년에 NHP 개념 증명을 기대하고 2026년에 개발 후보 지명을 할 예정입니다. 이오니스와 협력하여 메타지노미는 심혈관 대사 프로그램을 진행하고 있으며, 2025년에 1~2개의 개발 후보 지명을 목표로 하고 있습니다.
메타지노미는 또한 컴팩트한 SMART 뉴클레이즈 및 새로운 아데닌 엑스펙터를 포함한 기술 플랫폼을 발전시켰습니다. 회사의 현금 흐름은 2027년까지 운영을 지원할 것으로 예상됩니다.
Metagenomi (Nasdaq: MGX) a fourni des mises à jour sur ses programmes de développement thérapeutique et a tracé les jalons clés pour 2025-2026. L'entreprise a rapporté des progrès significatifs dans son programme MGX-001 pour l'hémophilie A, atteignant une activité soutenue du facteur VIII dans des études sur des primates non humains pendant plus de 16 mois. La société prévoit des interactions réglementaires en 2025 et des soumissions IND/CTA en 2026.
L'entreprise fait avancer son programme de déficiences en protéines sécrétées, s'attendant à une preuve de concept chez les primates non humains en 2025 et à la nomination d'un candidat au développement en 2026. En collaboration avec Ionis, Metagenomi progresse dans des programmes cardiométaboliques, visant une à deux nominations de candidats au développement en 2025.
Metagenomi a également fait progresser ses plateformes technologiques, y compris des nucléases SMART compactes et de nouveaux éditeurs de base d'adénine. La trésorerie de l'entreprise devrait soutenir ses opérations jusqu'en 2027.
Metagenomi (Nasdaq: MGX) hat Updates zu seinen therapeutischen Entwicklungsprogrammen bereitgestellt und wesentliche Meilensteine für 2025-2026 skizziert. Das Unternehmen berichtete über erhebliche Fortschritte in seinem MGX-001-Programm zur Behandlung der Hämophilie A und erzielte über 16 Monate hinweg eine nachhaltige Faktor-VIII-Aktivität in Studien mit nichtmenschlichen Primaten. Das Unternehmen plant regulatorische Interaktionen im Jahr 2025 und IND/CTA-Einreichungen im Jahr 2026.
Das Unternehmen entwickelt sein Programm für sekretierte Proteindefizite weiter und erwartet 2025 den Nachweis des Konzepts bei nichtmenschlichen Primaten sowie die Nominierung eines Entwicklungs kandidaten im Jahr 2026. In Zusammenarbeit mit Ionis kommt Metagenomi in den kardiometabolischen Programmen voran, wobei ein bis zwei Nominierungen für Entwicklungskandidaten im Jahr 2025 angestrebt werden.
Metagenomi hat außerdem seine Technologieplattformen weiterentwickelt, einschließlich kompakter SMART-Nukleasen und neuer Adenin-Baseneditoren. Die Liquidität des Unternehmens wird voraussichtlich die Betriebe bis 2027 unterstützen.
- Sustained Factor VIII activity achieved in NHP study over 16+ months for MGX-001
- Cash runway extends into 2027
- All four Wave 1 Ionis collaboration programs achieved in vivo proof-of-concept
- Advanced technology platforms with SMART nucleases and Adenine Base Editors showing 95% genome targeting potential
- None.
Insights
The pipeline update showcases MGX-001's promising development in Hemophilia A, with sustained Factor VIII activity over 16 months in NHP studies. The bioengineered construct demonstrates superior FVIII activity compared to wild type, suggesting enhanced therapeutic potential. The clear regulatory pathway with planned FDA interactions and IND submission timeline provides concrete development visibility.
The expansion into secreted protein deficiencies leveraging the MGX-001 platform technology indicates efficient R&D resource utilization. The Ionis collaboration's progression with 4 Wave 1 programs achieving in vivo proof-of-concept demonstrates platform validation across multiple targets. The focus on large cardiometabolic indications, including TTR amyloidosis and refractory hypertension, addresses substantial market opportunities.
The
The technical achievements are particularly noteworthy. The compact SMART nucleases' compatibility with AAV delivery systems significantly expands therapeutic applications. The novel Adenine Base Editors reaching
The sustained FVIII activity in NHP studies over 16 months is especially promising, as durability has been a key challenge in gene therapy. The higher FVIII activity at similar integration rates with the bioengineered construct suggests optimal protein engineering that could translate to improved clinical outcomes.
The platform's expansion into secreted protein deficiencies using the albumin-based approach is scientifically sound, leveraging validated biology while minimizing development risk. The technology development pipeline, particularly the RNA-mediated integration systems, positions the company well for addressing diverse genetic disorders.
MGX-001 in Hemophilia A Advances Towards US and Ex-US Regulatory Interactions in 2025
NHP Proof-of-Concept Anticipated for Secreted Protein Deficiency Platform in 2025 Leveraging MGX-001 Editing Approach
On Track for One to Two Development Candidate Nominations in 2025 from Wave 1 Ionis Collaboration Programs Focusing on Cardiometabolic Indications
Cash Runway Anticipated to Support Operating Plans into 2027
EMERYVILLE, Calif., Jan. 16, 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 provided updates on the successful achievement of critical milestones across its therapeutic development programs and technology platforms in 2024 and anticipated milestones for 2025 and 2026.
“Our vision at Metagenomi is to create curative genetic medicines for patients by harnessing the power of our metagenomics platform,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We made tremendous progress toward this goal in 2024, with accomplishments including substantially advancing MGX-001, our potentially curative development candidate for hemophilia A, as well as leveraging the MGX-001 gene editing system to drive our wholly-owned programs in secreted protein deficiencies. Our partnership with Ionis in large cardiometabolic indications remains on track. We also announced advancements across our gene editing toolbox, including next-generation base editors, ultra-small nucleases and large gene integration systems. Building on this momentum, we look forward to significant additional milestones for Metagenomi in 2025 as we progress our wholly-owned and partnered programs toward the clinic.”
Recent Pipeline Advancement and Corporate Updates
MGX-001 - Hemophilia A Program:
2024 Achievements
- Declared wholly-owned Development Candidate MGX-001, engaged with FDA in initial regulatory discussions and initiated GxP manufacturing activities
- Presented data in an oral presentation at the American Society of Hematology (ASH) 66th Annual Meeting and Exposition in December 2024
- Achieved sustained Factor VIII (FVIII) activity in an ongoing nonhuman primate (NHP) study over more than 16 months of follow-up
- Achieved higher FVIII activity at similar integration rates with bioengineered MGX-001 FVIII construct compared to wild type FVIII construct in rodent studies
2025 and 2026 Anticipated Milestones
- 2025: Finalize ongoing NHP durability study, continue Investigational New Drug (IND) enabling efforts, and Pre-IND/ ex-US regulatory meetings to support IND/ Clinical Trial Application (CTA) submissions
- 2026: Submit IND/CTA to advance MGX-001 into first-in-human studies
Secreted Protein Deficiencies:
2024 Achievements
- Identified targets for wholly-owned therapeutic programs leveraging the albumin approach used in MGX-001 to achieve in vivo proof-of-concept in rodents
2025 and 2026 Anticipated Milestones
- 2025: Demonstrate NHP proof-of-concept for lead secreted protein deficiency target
- 2026: Nominate Development Candidate for lead secreted protein deficiency target
Cardiometabolic Programs:
2024 Achievements
- Advanced all four Wave 1 Ionis collaboration programs to lead optimization and achieved in vivo proof-of-concept in rodents across all programs
- Programs include transthyretin (TTR) for transthyretin amyloidosis and angiotensinogen (AGT) for refractory hypertension as well as two undisclosed programs in significant cardiometabolic indications
2025 and 2026 Anticipated Milestones
- 2025: Nominate one to two Development Candidates from the Wave 1 Ionis collaboration development programs and disclose remaining therapeutic indications in large cardiometabolic indications for the remaining development programs
- 2026: Initiate IND-enabling activities for the Development Candidates nominated in 2025 and nominate additional Development Candidates from the remaining Wave 1 targets
Technology Development:
2024 Achievements
- Presented compact SMART nucleases demonstrating robust in vitro genome editing activity at multiple therapeutically relevant loci; Metagenomi continues to use AI, ancestral state reconstruction, and structural biology to enhance our gene editing systems, as highlighted with our recent publication in Nature Communications. These compact SMART genome editing tools are also small enough to fit within an adeno-associated virus (AAV), expanding our delivery options.
- Presented novel Adenine Base Editors (ABEs) demonstrating potential targeting to over
95% of the human genome; Simultaneous ABE triplex editing resulted in over95% knockdown of all three target proteins in primary T-cells, and demonstrated highly specific on-target deamination with no detectable translocations and no adverse effects on cell viability, expansion, or other measures of cell health. - Continued to advance our CRISPR-associated transposases (CASTs), including testing these systems in new human cell types with therapeutically-relevant targets and cargo; Demonstrated improvements to RNA-mediated integration-based systems for correction of multiple mutations known to cause disease.
2025 and 2026 Anticipated Milestones
- Continue to advance early-stage pipeline for multiple future IND filings
Other Business Updates
- Eric Bjerkholt, CFO of Mirum Pharmaceuticals, Inc., will join Metagenomi’s Board of Directors, serving on Metagenomi’s Audit and Compensation committees
About Metagenomi
Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. 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 diverse and 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.
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 growth strategy and 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, 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 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; patent and intellectual property matters; competition; as well as other risks described in “Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. 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.
Contact:
Simon Harnest - CIO, SVP Investor Relations
IR@metagenomi.co
Ashlye Hodge - Manager, Communications
Ashlye@metagenomi.co
FAQ
When will Metagenomi (MGX) submit IND/CTA for MGX-001 hemophilia A treatment?
What are the key milestones for MGX's secreted protein deficiencies program in 2025-2026?
How many development candidates does MGX expect to nominate from Ionis collaboration in 2025?
What was the duration of Factor VIII activity in MGX-001's NHP study?
How long will MGX's current cash runway last?
