MaaT Pharma, a clinical-stage biotechnology company specializing in microbiome-based therapies, has announced the completion of enrollment in its Phase 3 ARES clinical trial. The trial is evaluating the safety and efficacy of MaaT013 for the treatment of acute Graft-versus-Host Disease (aGvHD) in patients who have not responded to steroid and ruxolitinib treatments. Topline results from the study are expected in January 2025.
The ARES trial (NCT04769895) is a pivotal, multicenter, European, open-label, single-arm study. It focuses on patients with gastrointestinal (GI)-aGvHD who are refractory to both steroids and ruxolitinib, representing a significant unmet medical need. While the study anticipated recruiting a maximum of 75 patients to account for potential dropouts, MaaT Pharma reached its goal by enrolling 66 evaluable patients, the number required for an accurate assessment of the primary endpoint.
The primary endpoint of the ARES trial is the Gastrointestinal aGvHD Overall Response Rate. Secondary endpoints include one-year overall survival. MaaT013 is administered as an enema, delivering a full-ecosystem of microbial species to the patient's gut.
MaaT013: A Novel Microbiome Ecosystem Therapy
MaaT013 is an off-the-shelf, standardized, pooled-donor Microbiome Ecosystem Therapy designed for acute, hospital use. It is characterized by high diversity and richness of microbial species, including Butycore, a group of bacteria known to produce anti-inflammatory metabolites. The therapy aims to restore the symbiotic relationship between the patient’s gut microbiome and their immune system, correcting immune function and reducing steroid-resistant GI-aGvHD.
According to Hervé Affagard, CEO and co-founder of MaaT Pharma, "Completing the enrolment of this Phase 3 marks a major milestone in our mission to improve outcomes for blood cancer patients and underscores our continued commitment to the GvHD community. We are confident that MaaT013 could become the first-ever approved therapy harnessing microbiome-driven immune modulation in oncology, opening the door to the creation of a new therapeutic pillar across a broad range of cancer types."
Clinical Context and Previous Data
Acute Graft-versus-Host Disease occurs within 100 days of a stem cell or bone marrow transplant, where transplanted cells attack the recipient's tissues, causing inflammation, particularly in the skin, liver, and gastrointestinal tract. GI-aGvHD can lead to severe diarrhea and life-threatening complications. First-line treatment typically involves systemic steroids, with ruxolitinib approved for steroid-refractory cases.
MaaT013 has already been available through an Early Access Program in six European countries for the past five years, during which over 180 patients have been safely treated. An interim Data Safety Monitoring Board (DSMB) review of the Phase 3 ARES trial showed a favorable benefit/risk ratio, with reports of "high efficacy and low toxicity."
Looking Ahead
MaaT Pharma plans to host a Key Opinion Leader discussion in early December 2024 to present updated data from the Early Access Program. Following the topline results in January 2025, the company anticipates submitting for regulatory approval in Europe by the end of 2025.
