In a significant milestone for both microbiome research and neurodegenerative diseases, MaaT Pharma, a leading biotech company specializing in Microbiome Ecosystem Therapies™, has announced positive Phase 1b trial results for MaaT033 in patients with Amyotrophic Lateral Sclerosis (ALS).
The trial, named IASO, achieved its primary endpoint, confirming the safety and tolerability of MaaT033 when administered over two months. These findings highlight the potential of microbiome-based therapies in addressing critical unmet needs in ALS, a devastating disease with limited treatment options.
Promising results in safety and tolerability
The IASO trial, conducted at two centers in France, enrolled 15 participants to evaluate MaaT033, an oral capsule developed to modulate the gut microbiome and its interaction with the immune system. The independent Data Safety and Monitoring Board (DSMB) validated the safety and tolerability of the therapy, while preliminary microbiome analysis demonstrated successful engraftment of the treatment.
Professor Gaëlle Bruneteau, a neurologist at Sorbonne University and consultant at Pitié-Salpêtrière Hospital, praised the findings: “These results underline the strong safety profile of MaaT033 in ALS. Growing evidence suggests a link between gut microbiota and ALS, making further studies into the gut-brain axis crucial in understanding this disease.”
Expanding the frontiers of microbiome research in ALS
The success of the IASO trial marks a potential breakthrough in ALS research, an area where therapeutic innovation is urgently needed. ALS, also known as Lou Gehrig’s disease, affects motor neurons, leading to progressive paralysis and death within 3–5 years for most patients. With an estimated 60,000 patients in the U.S. and Europe by 2040, the demand for effective treatments is critical.
Hervé Affagard, CEO and co-founder of MaaT Pharma, emphasized the transformative potential of MaaT033: “This trial represents a pivotal moment in our mission to harness the power of the microbiome to improve patient survival. These results reflect the versatility of our platform, and we are committed to exploring collaborations to expand its impact across therapeutic areas.”
What’s next for MaaT033?
Encouraged by these results, the DSMB has recommended advancing MaaT033 to a Phase 2 trial, pending a comprehensive analysis of the study’s data, expected in early 2025. The next steps could include a larger, randomized controlled trial to assess the therapy’s efficacy, subject to securing additional funding.
MaaT Pharma also plans to present key findings from the Phase 1b trial at the International Symposium on ALS/MND in Montreal this December, a testament to its leadership in this field.
Collaborations and a broader vision
The IASO trial is a collaborative effort involving leading clinicians, researchers, and patient advocacy groups, including FILSLAN/ACT4ALS-MND and Tous en Selles contre la SLA. This synergy reflects the broader commitment to advancing ALS research and addressing its multifaceted challenges.
In parallel, MaaT033 has shown promise in other indications, such as acute myeloid leukemia, and is being explored as an adjunctive therapy for patients undergoing stem cell transplants. Its donor-derived, high-diversity microbial composition is designed to optimize microbiota function and immune modulation, making it a versatile candidate across various diseases.
A bright future for ALS patients?
MaaT Pharma’s progress in ALS offers hope for patients and families affected by the disease. By leveraging its innovative microbiome ecosystem technology, MaaT Pharma is charting a path toward therapies that address the root causes of diseases rather than merely alleviating symptoms. As ALS remains a formidable challenge with no cure, MaaT033 may represent a new frontier in tackling the complex interplay between the gut and brain in neurodegenerative disorders.
