Metagenomi Announces Q2 2024 Financial Results and Business Updates
Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company, recently provided a business update and announced its second-quarter financial results for 2024. With a mission to develop curative therapeutics, Metagenomi leverages a unique gene-editing toolbox derived from metagenomics, which is powered by artificial intelligence (AI).
CEO and Founder Brian C. Thomas, PhD, emphasized the company's progress, particularly in their hemophilia A program. The regulatory interactions with the Food and Drug Administration (FDA) have been positive, and the company has designated MGX-001 as its leading development candidate. Data from a 12-month nonhuman primate (NHP) durability study will be presented earlier than expected, in September 2024, rather than late 2024. This study is essential to demonstrate the therapeutic potential and longevity of MGX-001 in treating hemophilia A.
Metagenomi's collaboration with Ionis Pharmaceuticals has also shown promise. The partnership now includes two high-value targets related to cardiometabolic diseases, with all programs advancing into the lead optimization phase. The company plans to nominate multiple development candidates by 2025. Additionally, Metagenomi achieved a milestone with Affini-T, contributing to regulatory filings and establishing current good manufacturing practices (cGMP) for cell therapy.
Financially, Metagenomi is robust, with $299.9 million in cash and marketable securities as of June 30, 2024. This financial health is projected to sustain its operations into 2027.
Second Quarter 2024 Business Updates and Key Milestones
Metagenomi’s pipeline is making significant strides. The hemophilia A program, designed as a one-time curative treatment for both adults and children, has met critical milestones. The company engaged with the FDA to discuss key aspects of their planned Investigational New Drug (IND) submission. MGX-001 has been declared the lead development candidate, with data from the 12-month NHP durability study expedited to September 2024. The study aims to show durable expression of Factor VIII, a major challenge in other genetic medicine programs for hemophilia A. Metagenomi plans to initiate cGMP manufacturing and IND enabling activities in 2024 and 2025, with an IND filing expected in 2026.
Building on the hemophilia A program's success, the company plans to advance additional therapeutic candidates targeting secreted protein disorders, leveraging the MGX-001 editing system. This system employs a highly efficient nuclease and an AAV vector delivered DNA template, facilitating precise gene edits.
In the Ionis collaboration, all four Wave 1 therapeutic programs have moved into the lead optimization phase. These programs focus on cardiometabolic diseases, with refractory hypertension as the initial target. Metagenomi aims to demonstrate in vivo proof-of-concept in 2024 and advance NHP studies to support development candidate (DC) nominations, aiming to nominate one to two DCs in 2025.
A milestone was also achieved with Affini-T, establishing cGMP gene editing reagents for cell therapy, and filing related Drug Master Files with FDA to support an IND for Affini-T’s T-cell receptor-based therapy. Metagenomi received 933,650 shares of Affini-T common stock upon achieving this milestone.
Technology Platform Updates
Metagenomi has made considerable progress in its technology platforms. The company achieved in vitro proof-of-concept for an undisclosed neuromuscular target using its ultra-small editing system, opening therapeutic potential for extrahepatic disease targets. They also demonstrated in vitro proof-of-concept using their RNA-mediated integration system (RIGS) for liver targets and achieved multiplex base editing proof-of-concept, with plans to present this data later in 2024. Furthermore, Metagenomi demonstrated in vitro proof-of-concept in human cells for large gene integration using their CRISPR-associated transposase (CAST) technology, with a publication planned.
Other Business Updates
Metagenomi continues to exercise fiscal responsibility, prioritizing development programs and seeking collaboration partners for other opportunities. The company is looking for a partner for its primary hyperoxaluria type 1 (PH1) program, which has shown preclinical proof-of-concept. While focusing on in vivo gene editing approaches, they are also out-licensing technology for ex vivo cell therapy. The company has decided not to pursue amyotrophic lateral sclerosis (ALS) based on recent clinical data from peer companies.
As part of its pipeline strategy, Metagenomi is providing guidance only through year-end 2025, including DC nominations. The company maintains its planned IND filing for hemophilia A in 2026.
Financial Results
For the second quarter of 2024, Metagenomi reported a strong cash position with $299.9 million in cash and marketable securities. Research and development (R&D) expenses increased to $28.3 million from $22.7 million in the same period of 2023. General and administrative (G&A) expenses also rose to $8.6 million from $6.6 million.
Metagenomi remains focused on advancing its genetic medicines pipeline, leveraging its innovative gene-editing technologies, and maintaining its strong financial position to support its strategic initiatives.
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