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11/11/2025
Good day and thank you for standing by. Welcome to the MGX-001 program and business update conference call. At this time, all participants are in a listen-only mode. Please be advised that today's conference is being recorded. After the speaker's presentation, there will be a question and answer session. To ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. I would now like to hand the conference over to your speaker today. Steven Jasper, Managing Director at Gilmartin Group.
Ladies and gentlemen, welcome to the Metagenomic Conference Call to discuss the company's MGX-001 program and business updates. Before we begin, please note that this conference call will include forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Please refer to our most recent Form 10-K and 10-Q on file with the SEC for important risk factors that could cause our actual performance and results to differ materially from those expressed or implied in these forward-looking statements. Joining us on the call today with prepared remarks are the company's newly appointed Chief Executive Officer, Giann Irish, and the company's Senior Vice President of Research, Alan Brooks. Also joining on the call today is Dr. Glenn Pierce. an internationally recognized hemophilia physician-scientist. After the company's prepared remarks, we will open the line for a live Q&A session. I would now like to hand the call over to John Irish. John, please go ahead.
Welcome to today's update, our wholly-owned MGX-001 Hemophilia A program, and thank you for joining us. I'm extremely excited to share new data today from the recent dose range finding study in our hemophilia program. We believe the data presented to you today clearly supports our plan to advance MGX001 into clinical development. In this study, MGX001 demonstrated curative factor VIII activity in non-human primates. The data also revealed a clear dose-dependent efficacy across both the AAV and LMP components of MGX001, resulting in therapeutically relevant fact data activity in each animal treated in all but the lowest dose. The study informs the clinical dose regimen strategy for a therapy with best-in-class treatment potential. This demonstration of improved efficacy with reduced variability builds upon our previously announced MGX001 results. We previously demonstrated durable FACT-8 activity over an approximately 19-month study in NHPs and an encouraged safety profile with minimal steroid use. Additionally, MGX001 has also shown no identifiable off-target editing. Potential competitive advantages of MGX001 include enabling endogenous production of FACT8 for hemostatic regulation and restoring the body's own ability to produce FACT8 in contrast to bispecific FACT8 memetics and rebalancing therapies. And because of its mechanism of action, MCX001 has the potential to be an effective treatment for children as well as adults, with children and their caregivers having the most to benefit from a lifelong therapy. We believe the totality of our preclinical data for MGX001 is extremely encouraging and unique in the hemophilia A space. This gives us confidence that our novel approach may provide a one-time potentially curative therapy, allowing patients a hemophilia-free mindset. In light of the encouraging preclinical MGX001 hemophilia results, we reported today. We made a strategic decision to focus on our most compelling preclinical programs that have the highest probability of success and the potential to address unmet medical needs and create near-term value. This included the MGX001 program in hemophilia A, secreted protein disorders, leveraging the MGX001 approach, and cardiometabolic indications in collaboration with IMIS. In line with the strategic focus, we prioritized early discovery and platform research and reduced our workforce by 25%. As a result of these actions and our revised capital allocation strategy, we expected to extend our cash runway into the fourth quarter of 2027. I want to express my deepest gratitude to every member of the metagenomic team, including those who were impacted by the workforce reduction, for their invaluable contributions towards advancing our mission. Before I turn this presentation to Dr. Glenn Pierce, I would like to say a bit about Dr. Pierce's background. Dr. Pierce is an internationally recognized hemophilia physician scientist who has spent more than three decades in drug development, as well as patient advocacy. He has been actively involved in the approval of six hemophilia therapies. In addition to his previous leadership roles at various biopharma organizations, he also services as the vice president of medical for the World Federation of Hemophilia. He is currently our independent advisor. Importantly, Dr. Pierce was historically a hemophilia A patient and lived with the disease until being cured by a liver transplant. He can speak firsthand about the burden of the treatment for patients with hemophilia A. Glenn?
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