Lyon, France-based MaaT Pharma has announced positive 18-month data for its lead candidate MaaT013 showing an overall survival advantage in acute graft versus host disease (aGvHD).

The company has evaluated data from 140 patients with steroid-refractory (SR) or steroid-dependant (SD) gastrointestinal aGvHD treated with MaaT013 as part of Europe’s expanded access programme (EAP).

EAP allows patients to have access to medicines prior to them being granted marketing authorisation, outside of clinical trials.

The data shows a gastrointestinal overall response rate (ORR) of 52% at day 28, with complete response (CR) observed in 28% of patients. ORR considering all organs was 52% with 24% CR. Overall survival (OS) was 54% at six months, 47% at 12 months, 42% at 18 months.

Professor of Haematology at Saint-Antoine Hospital and Sorbonne University in Paris, Dr. Florent Malard will be presenting data at the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in Glasgow, Scotland from 14-17 April 2024.

Dr Malard said: “MaaT013 shows remarkable efficacy at 18 months, yielding more complete responses in aGvHD patients who have shown resistance to current treatments, as compared to other available therapies. This effect is coupled with reduced toxicity compared to standard immunosuppressive drugs. Notably, these results are achieved with just three doses in less than two weeks of treatment initiation. This would not only improve patient outcomes but also significantly enhance their quality of life.”

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The ongoing Phase III ARES trial (NCT04769895) has enrolled 75 patients with corticosteroid and ruxolitinib-refractory GI-aGvHD. The company has previously shared positive data from the Data Safety Monitoring Board (DSMB) of a favourable benefit- to-risk ratio, with “high efficacy and low toxicity.”

The company added that a subset of patients who have received the candidate through the EAP scheme who are similar to those enrolled at Phase III have demonstrated stronger efficacy.

CEO of MaaT Pharma, Hervé Affagard, said the EAP data reinforces confidence in the Phase III trial outcome and in hopes a commercial launch of the candidate in 2026.

MaaT013 was awarded orphan drug designation by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

MaaT Pharma is also running a Phase IIb trial of MaaT033 as an adjunctive therapy to enhance OS in allogeneic haematopoietic stem cell transplantation. This international, multi-centre trial (NCT05762211) is running at 56 sites and aims to enrol 387 patients.

GvHD landscape

There are a number of unmet needs for patients with GvHD, especially those with steroid-refractory GvHD. These are patients who are resistant or develop resistance, to corticosteroid treatment. Therefore, there is a high unmet need to develop drugs that will treat these patients, as there are limited treatment options beyond corticosteroids.

A report by GlobalData predicts the GvHD treatment and prophylaxis market to be worth $819m in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK and Japan) in 2028.

GlobalData is the parent company of Clinical Trials Arena.