MaaT Pharma has received a negative trend vote from the Committee for Medicinal Products for Human Use (CHMP) for MaaT013 (Xervyteg) following re-examination for its conditional marketing authorisation application. 

The committee’s concerns relate primarily to the attribution of clinical benefit and safety in the absence of a randomised control trial. 

“We are deeply disappointed by this outcome. Patients suffering from gastrointestinal-aGvHD continue to face a life-threatening condition with significant unmet medical need and limited treatment options,” said Hervé Affagard, Chief Executive Officer and Co-Founder of MaaT Pharma. “Together with the haematology community, we remain committed to these patients and will evaluate all available options to make this potentially important treatment accessible to patients worldwide.” 

MaaT Pharma has presented plans for PHOENIX, a global randomised controlled Phase III trial intended to generate the additional evidence requested by regulators to further characterise the benefit-risk profile of MaaT013. The trial will assess MaaT013 versus pre-specified BAT in patients with corticosteroid- and ruxolitinib-refractory aGvHD.

MaaT Pharma’s Microbiome Ecosystem Therapies (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximise clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases.  

MaaT013 is a full-ecosystem, off-the-shelf, standardised, pooled-donors, enema Microbiome Ecosystem Therapy for acute, hospital use. It is characterised by a consistently high diversity and richness of microbial species and the presence of Butycore (a group of bacterial species known to produce anti-inflammatory metabolites).  

The therapy aims to restore the symbiotic relationship between the patient’s functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)- acute Graft-versus-Host Disease (aGvHD). It has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).