A phase 3 trial of MaaT Pharma’s graft-versus-host disease (GvHD) drug candidate has hit its primary endpoint, positioning the biotech to seek approval of the microbiome therapy in Europe in mid-2025.
The single-arm, third-line trial enrolled 66 adults with acute GvHD with gastrointestinal involvement to receive MaaT013, a microbiota therapy made of fecal material provided by multiple healthy donors. Delivering MaaT013 via an enema is intended to restore microbiota diversity and production of functional metabolites, thereby controlling inflammation and restoring gut integrity.
MaaT reported a gastrointestinal overall response rate at Day 28 of 62%, achieving the primary goal of the trial. The French biotech said the expected response rate was 38% but didn’t explain how that figure was selected.
Digging deeper, MaaT reported that 38% of the gastrointestinal responses were complete, and a further 20% were very good. The biotech also shared an all-organ response rate of 64%, with 36% of patients having complete responses. MaaT framed the all-organ secondary endpoint data as evidence of the systemic effects that MaaT013 has beyond the gastrointestinal tract.
MaaT saw the response rates in patients who didn’t respond to ruxolitinib, the JAK inhibitor that Incyte markets as Jakafi. All the patients had also tried steroids and were either refractory to or dependent on them.
The top-line release lacked safety and tolerability data, with MaaT instead referring back to results on the first 30 patients that it shared in October. MaaT showed its confidence in the data by setting out plans to file for approval in Europe in mid-2025. The biotech is aiming (PDF) to win approval in the second half of next year and start commercializing MaaT013 in Europe by the end of 2026.
MaaT will need help to fund its plan. The biotech ended September with 27 million euros ($28 million), a sum it predicted would support operations into the second quarter of 2025. MaaT said it is looking at several funding options. The biotech is telling potential partners MaaT013 sales could peak at 250 million euros ($258 million) in GvHD, and a cancer candidate, MaaT033, could generate a further 500 million euros ($515 million).
A stock offering is another possibility. MaaT shares rose 23% in early trading in Paris, climbing toward 10 euros apiece. The stock last traded in that ballpark in May. That month, MaaT sold stock at 8 euros a pop and triggered a drop in the share price that persisted throughout the rest of the year.
However MaaT accesses funding, the money will need to support the European filing and plans to bring MaaT013 to the U.S. market. The FDA put a clinical hold on the candidate in August 2021 in response to MaaT’s request to run a phase 3 trial. MaaT eventually got the hold lifted in April 2023.
U.S. patients can now access MaaT013 under an early access program. MaaT plans to start a U.S. phase 3 trial this year but needs money to do so. The biotech said it is talking to the FDA about how to speed up MaaT013 development and will use the European phase 3 data to optimize the U.S. trial.