The results, presented at the 2023 American Society of Hematology (ASH) Annual Meeting, demonstrated good overall tolerability and safety profile from 111 patients with acute graft-versus-host disease (aGvHD) in the early access program.
At the event, the company also presented its ongoing phase 2b trial design for MaaT033, its second candidate.
Developed as an adjunctive therapy to enhance overall survival (OS) in HSCT (hematopoietic stem cell transplantation) and cellular therapy recipients, MaaT033 targets optimal microbiota function for a broader patient population in a chronic setting.
“These promising findings pave the way for advancing the treatment landscape for aGvHD, with microbiome-based innovations, especially when they achieve complete responses,” said Hervé Affagard, CEO and co-founder of MaaT Pharma.
“It reinforces our approach based on restoring patients’ immune systems through gut microbiome ecosystem therapies. The efficacy and safety results underscore the strong favourable benefit-risk profile for MaaT013, and we look forward to continuing to investigate MaaT013 with the aim of having it accessible globally for patients in need as soon as possible”.
A gastrointestinal overall response rate of 53% positively and significantly impacted OS in responder patients
MaaT Pharma reported OS as 56% at 6 months and 47% at 12 months. OS was found to be significantly higher in patients who responded to MaaT013 compared to non-responders, 67% versus 24% at 12 months.
In addition, a separate phase 3 trial is currently ongoing in Europe to confirm these results in ruxolitinib-refractory patients with results expected in mid-2024.
The company also has an open-IND granted by the U.S. Food and Drug Administration (FDA) with discussions ongoing with potential clinical investigation sites.
This international, multi-centre trial is the largest randomized controlled study to date of a microbiome-based therapy in oncology, spanning up to 56 sites and enrolling 387 patients.
Mohamad Mohty, professor of hematology at Sorbonne University, and head of the clinical hematology and cellular therapy department at Saint-Antoine Hospital, Paris, commented: “GvHD remains a significant challenge and source of mortality following allogeneic HSCT. The clear connection between treatment response and overall survival demonstrates Microbiome Therapies as a potentially life-saving approach, particularly in patients who have failed earlier treatment lines, corticosteroids and ruxolitinib.”
“MaaT013 introduces a novel therapeutic approach by restoring the gut microbiome ecosystem, offering a distinct immuno-restorative option that could complement standard immunosuppressive drugs, and could become a potential breakthrough for patients with limited options.”
