In early January MaaT Pharma made waves in the pharma world with the announcement of positive topline results for their phase 3 trial of MaaT013 in acute Graft-versus-Host Disease.
With robust efficacy and safety data, MaaT Pharma aims to submit a centralised marketing authorisation (MA) application to the European Medicines Agency (EMA) in mid-2025, which if granted, will make MaaT013 the first approved microbiome therapy worldwide for an indication beyond recurrent C Difficile.
In a sit-down interview, Maat Pharma’s CEO Hervé Affagard spoke to the Microbiome Times about the significance of the read-out for patients, how the results will impact the space, and plans to foster industry collaborations in his role as President of the European Microbiome Innovation for Health group.
MT: The ARES study Phase 3 positive topline results represent a potential new option for patients in need of effective treatments. Could you discuss the key findings and their implications for patients with acute Graft-versus-Host Disease?
The positive topline results from the ARES Phase 3 are unprecedented in aGvHD trial, thus very encouraging for patients. This is, of course, a major milestone for us and for the field of microbiome therapeutics. The study met its primary endpoint, demonstrating a 62% gastrointestinal overall response rate (GI-ORR) at day 28, significantly exceeding the expected 38% predefined in the protocol. Importantly, 38% of patients achieved a complete response, and another 20% had a very good partial response. Beyond the gut, the therapy showed systemic benefits, with a 64% overall response rate (ORR) across all evaluable organs. These results demonstrated strong and durable response rates for MaaT013, translating into prolonged survival for patients.
What stands out as well is the probability of survival—patients had a 54% one-year survival probability after being treated with MaaT013, which is a remarkable improvement in a population where survival rates at one-year are typically as low as 15% (Abedin et al. 2021). For patients with GI-aGvHD who are refractory to steroids and ruxolitinib, these results offer new hope to patients and clinicians, alike and have the potential to redefine disease management.
MaaT013 has the potential to be the first approved third-line treatment option and thereby transform the survival outcomes and redefine long-term prospects for approximately 3,000 third-line GI-aGvHD patients per year in the U.S., Canada and Europe.
MT: Your press release mentions plans to seek European market authorization for MaaT013 by mid-2025. How do you think this authorization would influence the broader microbiome therapeutics industry?
If approved, MaaT013 would become the first microbiome therapy in oncology to gain regulatory approval, setting a groundbreaking precedent for the field.
This would also confirm the potential of standardized microbiome therapies, extending their application beyond infectious diseases and into more complex areas such as oncology, therefore validating the scientific and clinical rigor behind standardized microbiome-based drug candidates and demonstrating their ability to address unmet medical needs in therapeutic areas.
Moreover, this would also highly contribute on making the industry more mature, strengthening its credibility and paving the way for large adoption of microbiome-based drugs in various therapeutic areas. This could also help to make more efficient regulatory pathways for future therapies to navigate the process in Europe.
MT: You’re opening day one of our upcoming Microbiome Times Partnering Forum, speaking from your role as President of the EMIH, could you explain briefly what the EMIH is, and what it’s goals for 2025 are?
The European Microbiome Innovation for Health (EMIH) is an independent, non-profit organization gathering key stakeholders from public and private sectors from the microbiome ecosystem. Members include companies specialized in biotechnology, research institutes, hospitals and patient associations representing the whole microbiome ecosystem. EMIH first started as a French-based, national association in 2022 and expanded its scope in 2024 to promote patients’ safety and timely access to microbiome innovation within the European Health Union.
We are committed to the advancement of microbiome-based therapeutics, diagnostics and medical bioproduction based on the highest quality standards while supporting the drafting of health policies to the greatest benefit of patients and industry.
Our goals for 2025 are:
- Represent members in key EU legislative discussions, including the Biotech Act, SoHO implementation acts, and Pharmaceutical legislation.
- Launch working groups that would impact the field in the short term: Therapeutics/Public Affairs and IVD
- Expand EMIH’s role as a key industry voice by growing membership, fostering community engagement, and ensuring long-term sustainability.
….and of course, participate in the Microbiome Times event (March 18-19) to raise awareness.
MT: As part of your role as a committee member for the MT Partnering Forum, you have organized a therapeutics regulatory panel. What guidance can you offer to microbiome therapeutic developers who are working to navigate the current regulatory landscape?
Four points of guidance immediately spring to mind:
- Engage early with regulators to understand as soon as possible their expectations for clinical trial design, manufacturing standards and safety assessments.
- Maintain ongoing communication with regulatory agencies and keep them up-to-date and educate them.
- Join forces with other industry players to advocate regulatory frameworks tailored to microbiome products and leverages existing guidelines.
- Prioritize safety and efficacy data for microbiome innovation as well as availability of products on a large basis.
MT: In this regulatory panel hosted by EMIH at the Microbiome Times Forum, the EMA will be making their first appearance at a microbiome dedicated event. What is the significance of this conversation for the attendees and for the field as a whole?
We are aware EMA is holding workshops to gather “from the ground” experience. This time it’s us having their feedback on regulating a new approach. I find this particularly relevant for all of us as the microbiome field is at an exciting turning point where we will see the next round of market approvals. Infant Bacterial Therapeutics and MaaT Pharma have both recently announced positive topline data. As such, I believe Staffan (CEO of Infant Bacterial Therapeutics) and I are in unique positions to share learnings on our respective journeys navigating microbiome regulation (US and Europe). The presence of the European Medicines Agency will, I’m sure, enrich our conversations and serve as a source of learning for other organizations.
MT: Thank you Hervé and we look forward to seeing you next month in Brussels!
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