The US Food and Drug Administration (FDA) has lifted the clinical hold and cleared MaaT Pharma’s investigational new drug (IND) application for MaaT013, thereby enabling initiation of a Phase III pivotal clinical trial to treat gastrointestinal acute Graft-versus-Host Disease (aGvHD) as a third line of treatment.

Safety and efficacy of MaaT013 will be evaluated in the open-label, single arm study, to be undertaken in the US.

MaaT Pharma CEO and co-founder Hervé Affagard said: “This is the first time the Agency has authorised the Phase III clinical evaluation in the US of a microbiota-based live biotherapeutic based on a pooling technology, which provides greater bacterial diversity, in a standardised and scalable approach, with the goal of safely improving patients’ outcomes.

“This major milestone is fundamental to the strategic decisions regarding the development of our portfolio outside Europe and to the Company’s outlook in the US.

“The US represents an important market for our therapeutics, and we have already benefited from our previous discussions with the FDA by adapting our pooling technology for our entire pipeline according to the guidelines received during this regulatory process.”

Besides the encouraging data from the Early Access Programme of MaaT013, the ongoing international multicentre open-label, single arm, pivotal Phase III trial (ARES) launched in March 2022 in Europe showed significant progression in the development of therapy.

 Affagard added: “It also confirms the robustness of our protocols for donor screening and selection which are now authorised for clinical evaluation in the U.S. as well as in Europe.

“We are now in a position to start discussions with the FDA for the clinical evaluation of MaaT033, our second drug-candidate, in the US.”