MaaT Pharma announced last week that the first patient has been dosed in its Pivotal Phase 3 study. The French biotechnology company has established a complete approach to restoring patient-microbiome symbiosis in oncology.
The goal of the Phase 3 clinical trial is to investigate the efficacy of lead Microbiome Ecosystem Therapy MaaT013 as third-line treatment in patients with steroid-resistant gastrointestinal acute Graft-versus-Host Disease (aGvHD). aGvHD is a serious complication of allogeneic stem cell transplantation and each year, approximately 10,000 patients are diagnosed globally. Acute Graft-versus-Host Disease occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant. The transplanted cells “attack” the recipient, causing inflammation of the skin, liver and gastro-intestinal tract.
The ARES clinical study
ARES is the name of the first open-label, single-arm Phase 3 global study for a microbiome-based therapy in haemato-oncology. It evaluates the safety and efficacy of MaaT013, the company’s high-richness, high-diversity lead Microbiome Ecosystem Therapy (MET) as a third-line, salvage therapy in 75 patients with Grade II-IV GI-aGvHD, who are refractory to steroids and are resistant or are intolerant to ruxolitinib.
The Phase 3 ARES trial builds on previously shared positive results from the company’s Phase 2 HERACLES study of MaaT013 (n= 24) and from data of 52 patients benefiting from an ongoing early access program with MaaT013 in France. Both clinical datasets demonstrated that MaaT013 was generally well tolerated in this heavily immunocompromised patient population and demonstrated promising clinical benefit, which translated into a positive impact on overall survival rates in patients responding to the treatment. To date, more than 100 patients with aGvHD have been safely treated with MaaT013.
Hervé Affagard, co-founder and CEO of MaaT Pharma stated: “Initiating the pivotal Phase 3 trial with our lead candidate MaaT013 is an important milestone for our company and for the microbiome therapeutics field worldwide. If ARES is successful, we believe this study could serve as the basis for MaaT013’s approval in this severe indication with a very high unmet need.”
ARES will be conducted in more than 40 different sites across the European Union. Until now, the company has received regulatory approvals from France, Germany and Spain, with the opportunity to extend the clinical trial to sites in the US, subject to regulatory approval. The clinical trial involves the administration of three doses of MaaT013 to eligible patients in the ARES study, as an enema formulation over a 10-day period. The primary endpoint of the study is the GI-overall response rate (GI-ORR) to treatment with MaaT013 at day 28. Secondary endpoints include the safety and tolerability of MaaT013 for up to 12 months, as well as overall survival after 12 months. A first review of the data is planned for Q1 2023 after enrolment of half of the patients in the study.
According to the Prof. Mohamad Mohty, Professor and Head of the Hematology and Cellular Therapy Department at the Saint-Antoine Hospital and Sorbonne University and past-president of the European Society for Blood and Marrow Transplantation (EBMT), there is an extremely high medical need for GI-aGvHD patients who are refractory to both steroids and ruxolitinib, as they face an 80% mortality rate within the first few months. “By restoring the gut microbiome ecosystem, MaaT013 opens a brand-new therapeutic modality and provides a strongly differentiated, immuno-restorative approach that may complement standard immunosuppressive drugs and may provide a breakthrough for patients with no other therapeutic options.”