“This is the first time the Agency has authorized the Phase III clinical evaluation in the US of a microbiota-based live biotherapeutic using a pooling technology,” Hervé Affagard, director general and co-founder of MaaT Pharma, was pleased to say after it was announced the clinical hold would be lifted. Pooling is a patented technology, developed by MaaT Pharma, that consists of mixing together microbiota obtained by collecting stool from several carefully selected donors using strict safety criteria, in order to achieve a better standardized drug which is stronger and more diverse from a microbial point of view. “Pooling provides greater diversity compared with a mono-donor approach, maximizing the chances of clinical responses in patients,” the company spokesperson added. Thus far, the Agency had only authorized mono-donor clinical evaluation.
MaaT013, the French microbiota specialist’s most advanced product, is currently being assessed in Europe in two clinical trials for oncology. The first was a Phase III, open label, single arm clinical trial for the treatment of Acute Graft-versus-Host disease (hemato-oncology). The other study, Phase IIa, is a proof-of-concept sponsored by the AP-HP to improve patients’ responses to immunotherapies in metastatic melanoma (immuno-oncology). Currently, this is the only double-blind randomized clinical trial in the field evaluating a microbiome approach (MaaT013).
Before proceeding to Phase III on MaaT013 in the USA, the FDA had requested additional information on the “pooling” approach.The company therefore developed its safety, selection and donor monitoring procedures in order to fulfill the conditions requested by the health authority. “At the same time as our discussions with the FDA, we continued to generate data in Europe (through clinical trials and a compassionate access program), thus demonstrating the safety of the drug,” the MaaT Pharma spokesperson said. “These elements, coupled with fruitful discussions with the FDA, resulted in the lifting of the clinical hold.”
Several datasets were effectively shared with the US regulation authority, in particular the positive results from Phase 2 evaluating MaaT013 in GvH and the data obtained from the compassionate access program in Europe. These were also presented at world congresses, including the Annual Congress of the American Society of Hematology in the USA, in December 2022.
“We are grateful for the FDA’s continued engagement and are very pleased with the lift of the hold on MaaT013’s IND application (…) This major milestone is fundamental to the strategic decisions regarding the development of our portfolio outside Europe and to the company’s positioning in the US,” said Hervé Affagard, CEO and co-founder of MaaT Pharma.
The products obtained from “pooling” technology are standardized drug-candidates, the aim of which is to restore a functional intestinal microbiome in patients suffering from severe dysbiosis, often iatrogenic in origin (i.e. due to drug therapy).
MaaT Pharma was a pioneer and is now the leader in the development of microbiome drugs in oncology and in implementation of rigorous donor selection, strict quality standards and pharmaceutical current good manufacturing practices (cGMP) to develop and manufacture its MET-N line of products which today include MaaT013 and MaaT033.
MaaT033 is Maat Pharma’s second drug candidate. It will soon be tested in two clinical trials in Europe – one in a Phase 2 randomized placebo-controlled trial, the widest in hemato-oncology to date, targeting patients treated by stem cell transplants and suffering from blood cancers, and a Phase 1b trial for patients suffering from Charcot’s disease (amyotrophic lateral sclerosis – ALS). MaaT Pharma will also initiate discussions with the FDA regarding its clinical evaluation in the USA.