MaaT Pharma presented updated data on MaaT013 from its Early Access Program (EAP) in Europe, demonstrating promising results in patients with acute Graft-versus-Host Disease (aGvHD). The data, presented at the 66th American Society of Hematology (ASH) Annual Meeting, included 154 patients and highlighted the potential of MaaT013 as a transformative therapy for this life-threatening condition.
The study showcased sustained high response rates, with a gastrointestinal overall response rate (GI-ORR) of 51% at Day 28 and an overall response rate (ORR) of 49% for all organs. Long-term survival data indicated a 47% overall survival (OS) rate at 12 months and 42% at 24 months.
Significant Survival Benefit
Notably, the median OS among patients responding to MaaT013 within a subset resembling the Phase 3 ARES trial population was 444 days, compared to only 42 days in non-responders. This represents a substantial improvement compared to historical data with best available therapy following Ruxolitinib failure, which shows a median OS of 28 days.
"These findings underscore MaaT013’s potential as a transformative therapy for aGvHD, a condition with poor survival rates and limited treatment options," said Florent Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University. "The high response rates and long-term survival data further validate the critical role of the gut microbiome modulation in managing aGvHD."
ARES Trial and Future Outlook
The EAP data included a subset of 58 patients similar to the population enrolled in the Phase 3 ARES trial (receiving 2nd line ruxolitinib). This group exhibited even higher response rates, with a GI-ORR of 59% at Day 28 and 54% at Day 56. The ORR considering all organs was 55% at Day 28 and 56% at Day 56. The OS in this subset was 54% at 6 months, 49% at 12 months, and 40% at 24 months, compared to 15% at 12 months in published historical data.
MaaT Pharma anticipates topline results from the pivotal Phase 3 ARES trial in January 2025. This trial, completed in October 2024, is expected to further validate MaaT013’s potential in addressing the unmet medical need for patients with aGvHD who have limited therapeutic options.
About MaaT013
MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor Microbiome Ecosystem Therapy designed for acute, hospital use. It aims to restore the symbiotic relationship between the patient’s functional gut microbiome and their immune system, correcting immune function and reducing steroid-resistant, gastrointestinal-predominant aGvHD. MaaT013 has been granted Orphan Drug Designation by the FDA and EMA.
