MaaT Pharma is set to advance its microbiome-based therapy, MaaT033, into Phase 2 trials for amyotrophic lateral sclerosis (ALS) following positive results from its Phase 1b study. The IASO trial (NCT05889572), an exploratory single-arm, open-label study, demonstrated that MaaT033 was safe and well-tolerated in ALS patients, meeting its primary endpoint.
The Phase 1b trial enrolled 15 participants across two centers in France: Hôpital de la Pitié-Salpêtrière and the University Hospital of Lille. The study evaluated the safety and tolerability of multiple doses of MaaT033 administered over two months.
Safety and Tolerability Profile
The Independent Data Safety and Monitoring Board (DSMB) validated the trial's findings, concluding that MaaT033 exhibited a favorable safety profile in ALS patients. Preliminary microbiome analysis also confirmed successful engraftment of MaaT033, indicating that the transplanted cells effectively integrated into the patients' gut microbiota.
Professor Gaëlle Bruneteau, a neurologist at Sorbonne University, commented on the results: "I am encouraged by these Phase 1b results, which underscore the strong safety and tolerability profile of MaaT033 in ALS. Preclinical and clinical evidence suggests a role of the gut microbiota in the pathogenesis and variability of ALS, and further studies are essential to fully explore the potential of the gut-brain axis in this disease."
Future Plans
MaaT Pharma plans to conduct a comprehensive analysis of the study's overall data, expected in early 2025, to determine the next steps in the clinical development of MaaT033. This may include initiating a larger, randomized, controlled efficacy study, contingent upon securing appropriate funding.
Hervé Affagard, CEO and co-founder of MaaT Pharma, stated, "The ALS trial represents a potentially transformative milestone in our mission to improve patient survival through innovative microbiome-based immune modulation therapies. These results demonstrate the potential versatility of our platform to address critical unmet medical needs across multiple therapeutic areas. As we look toward expanding the reach and impact of this innovation, we will explore collaboration opportunities to accelerate and broaden its application to benefit even more patients in need."
About MaaT033
MaaT033 is an oral Microbiome Ecosystem Therapy consisting of donor-derived, high-richness, high-diversity microbiota, containing anti-inflammatory Butycore species. It is designed as an adjunctive therapy to improve overall survival. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA).
ALS Context
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder affecting motor neurons in the brain and spinal cord, leading to muscle weakness, loss of voluntary movement, and eventually, paralysis. The disease typically leads to death within 3 to 5 years of diagnosis. ALS is estimated to affect up to 60,000 patients in the US and EU by 2040, and currently, there is no curative treatment available.
