MaaT Pharma plans to advance a global Phase 3 trial of MaaT013 after European regulators maintained a negative opinion on its marketing application.

EMA calls for more evidence on MaaT013

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on MaaT Pharma’s application for conditional marketing authorisation for MaaT013 (Xervyteg) following a re-examination.

MaaT013 is being developed to treat acute graft-versus-host disease (aGvHD) with gastrointestinal involvement in adults whose disease has not responded to previous lines of treatment.

The CHMP said the available clinical data, which are based on a single-arm trial, do not allow sufficient characterisation of the treatment’s benefit-risk profile.

MaaT Pharma had proposed a randomised controlled Phase 3 trial, named Phoenix, during the regulatory review to generate additional evidence on MaaT013.

The European Commission is expected to make the final decision on the marketing authorisation application following the CHMP opinion.

Phoenix trial planned for US and Europe

MaaT Pharma now plans to focus on advancing Phoenix, subject to financing and regulatory clearance.

The global Phase 3 trial is designed to compare MaaT013 with pre-specified best available therapy (BAT) in patients with corticosteroid- and ruxolitinib-refractory aGvHD.

The company expects to enrol approximately 138 patients, randomised 1:1, with Day 28 all-organ overall response rate as the primary endpoint. Key secondary efficacy and safety measures will also be assessed.

MaaT Pharma said feedback received during a Type C interaction with the US Food and Drug Administration supports advancing Phoenix as a registrational Phase 3 trial.

The company is conducting a feasibility assessment across clinical trial sites in the US and other planned countries and is working towards finalising the protocol and activating US sites.

Subject to funding and regulatory clearance, the company expects the trial could begin with first-patient enrolment in the first half of 2027.

If successfully completed and positive results are obtained, MaaT Pharma said the study could support future regulatory submissions in the US, Europe and other territories.

Company reviews assets as cash horizon extends

The regulatory update comes as MaaT Pharma conducts a strategic review of its assets and introduces further measures to preserve cash.

The company said the measures are expected to extend its cash horizon to December 2026, compared with November 2026 under its previous operational assumptions.

MaaT Pharma said it is also exploring options for a US-focused development plan for MaaT013 with the aim of supporting potential global registration.

The next stage of development will therefore depend on the company’s ability to secure appropriate funding as well as regulatory clearance.

MaaT013 is a microbiome ecosystem therapy being developed by MaaT Pharma as an approach to immune modulation in patients with cancer and associated complications.