MaaT Pharma Reports Positive Phase 1b Results for MaaT033 in ALS

LYON, France--(BUSINESS WIRE)--Regulatory News: MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company specializing in Microbiome Ecosystem Therapies™ (MET) aimed at improving cancer patient survival through immune modulation, announced that their exploratory Phase 1b clinical trial named IASO (NCT05889572) assessing MaaT033 in ALS has achieved its primary goal of evaluating the safety and tolerability of MaaT033 with multiple doses. The independent Data Safety and Monitoring Board (DSMB) has confirmed that MaaT033 was well-tolerated and safe for ALS patients over a two-month period. Preliminary microbiome analysis has indicated the successful engraftment of MaaT033, underscoring its safety and tolerability.

Prof. Gaëlle Bruneteau, a Neurology Professor at Sorbonne University and consultant neurologist at the Paris ALS expert center of the Pitié-Salpêtrière Hospital in Paris, France, expressed optimism regarding these Phase 1b results. She stated, "I am encouraged by these findings that demonstrate the good safety and tolerability profile of MaaT033 in ALS. Both preclinical and clinical evidence point to the gut microbiota's role in ALS pathogenesis and variability, necessitating further research to fully understand the gut-brain axis's potential in this disease."

Further study endpoints are anticipated to be analyzed in the coming months. Based on the Phase 1b IASO study results, the DSMB has recommended proceeding to Phase 2. MaaT Pharma plans to determine the next steps based on a detailed analysis of the study’s overall data, expected in early 2025. These steps may include initiating a larger randomized controlled efficacy study, depending on securing appropriate funding.

Hervé Affagard, CEO and co-founder of MaaT Pharma, expressed his gratitude to the study participants, acknowledging their contribution to advancing ALS treatment. He remarked, "The ALS trial marks a potentially transformative milestone in our mission to enhance patient survival through innovative microbiome-based immune modulation therapies. These results showcase the versatility of our platform to address critical unmet medical needs across multiple therapeutic areas. As we aim to expand the reach of this innovation, we will pursue collaboration opportunities to accelerate and broaden its application, benefiting more patients in need."

Fifteen participants across two centers in France were enrolled in the Phase 1 trial. This study was a collaborative effort involving leading researchers and clinicians from Hôpital de la Pitié-Salpêtrière – AP-HP and University Hospital of Lille, along with experts from the French academic FILSLAN/ACT4ALS-MND and the French patients’ association Tous en Selles contre la SLA. These results, together with previous data from the Phase 1b CIMON trial in Acute Myeloid Leukemia and the latest DSMB for the ongoing Phase 2b trial PHOEBUS in Europe, reinforce confidence in MaaT033's safety profile for continued use.

Key safety and tolerability data will be presented through a poster at the 35th International Symposium on ALS/MND, scheduled for December 6-8, 2024, in Montreal, Canada.

MaaT Pharma, founded in 2014 and based in Lyon, France, is a leading late-stage clinical company developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Utilizing its proprietary pooling and co-cultivation technologies, MaaT Pharma creates high diversity, standardized drug candidates aimed at extending the life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021.

MaaT033 is a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem Therapy™ currently being developed as an adjunctive therapy to improve overall survival in patients undergoing HSCT and other cellular therapies. Developed with the "pooling" technology, MaaT033 aims to ensure optimal microbiota function for a larger patient population in a chronic setting. MaaT033 has received Orphan Drug Designation from the European Medicines Agency (EMA).

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease in the US and Charcot’s disease in Europe, is a progressive neurodegenerative disorder that affects motor neurons in the brain and spinal cord, leading to muscle weakness, loss of voluntary movement, and eventual paralysis. ALS affects an estimated 60,000 patients in the US and EU by 2040 and currently lacks any curative treatment with only a few symptomatic treatments available.