Type of information: Treatment of the first patient
Announcement: treatment of the first patient
Company: Maat Pharma (France)
Action mechanism: microbiome-based therapy. MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, Microbiome Ecosystem Therapy (MET). It is characterized by a consistently high diversity and richness of microbial species and the presence of ButycoreTM (group of bacterial species known to produce anti-inflammatory metabolites). MaaT013 aims to restore the symbiotic relationship between the patient’s functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal-predominant aGvHD. MaaT013 has been granted Orphan Drug Designation by the FDA and the EMA. MaaT013 is made of allogeneic, full-ecosystem pooled biotherapeutic intestinal microbiota manufactured by MaaT Pharma in Lyon, France, according to GMP requirements. The intestinal microbiota material in its natural environment is derived from healthy, strictly-vetted and selected donors, following the European consensus recommendations (Cammarota 2016) with the purpose of minimizing the risk associated with fecal material transplants (FMT) for clinical research. Thus, prior to donation, donors undergo a thorough medical evaluation and laboratory screening including SARS-CoV-2 detection, to avoid any known contamination risk. MaaT013 is administered as an enema.
Disease: steroid-resistant gastrointestinal acute graft-versus-host disease (SR GI-aGvHD)
Therapeutic area: Transplantation
Trial details: ARES is an open-label, single-arm Phase 3 study (NCT04769895),which evaluates the safety and efficacy of MaaT013, Maat Pharma’s high-richness, high-diversity lead Microbiome Ecosystem Therapy (MET) as a third-line, salvage therapy in GI-aGvHD patients. ARES is a Phase 3 multicenter, international, open-label, single-arm study assessing the safety and efficacy of MaaT013 in 75 patients with Grade II-IV GI-aGvHD who are refractory to steroids and are resistant or are intolerant to ruxolitinib. The clinical trial will be conducted in up to 40 different sites across the European Union. To date, the company has received regulatory approvals from France, Germany and Spain, and the clinical trial may also be expanded to sites in the United States subject to regulatory approval. Eligible patients in the ARES study will be administered a total of 3 doses of MaaT013 as an enema formulation over a period of 10 days. The primary endpoint of the study is the GI-overall response rate (GI-ORR) to MaaT013 treatment at day 28. Secondary endpoints include the safety and tolerability of MaaT013 up to 12 months, as well as overall survival after 12 months. A first data review is expected in Q1 2023 after enrollment of half the patients in the study.