MaaT Pharma plans to talk to the FDA before starting clinical development in the U.S. Twenty months after being rocked by a clinical hold, MaaT Pharma has finally received the green light from the FDA to run a phase 3 trial of its pooled fecal therapy in gastrointestinal acute graft-versus-host disease (GvHD) patients in the U.S. The FDA issued the hold in August 2021 in response to MaaT’s request to run a late-stage trial of MaaT013. The biotech had answered the FDA’s clinical and manufacturing-related questions a year later but had yet to resolve sticking points related to its mixing of fecal donations from multiple donors. To MaaT, the approach is a way to improve diversity and standardization. To the FDA, it was a concern. The French company gave its most upbeat update yet in February of this year, revealing that the FDA had provided a list of conditions for studying MaaT013. Now, the biotech has met the conditions and the FDA has lifted the clinical hold. “This is the first time the agency has authorized the phase 3 clinical evaluation in the U.S of a microbiota-based live biotherapeutic based on a pooling technology, which provides greater bacterial diversity, in a standardized and scalable approach, with the goal of safely improving patients’ outcomes,” Hervé Affagard, CEO and co-founder of MaaT, said in a statement. Affagard added that the “major milestone is fundamental to the strategic decisions regarding the development of our portfolio outside Europe and to the company’s outlook in the U.S.” Exactly how the lifting of the clinical hold will shape MaaT’s plans remains unclear. Noting the progress of its European phase 3 trial, which began 13 months ago, and “encouraging data” from an early access program, MaaT said it plans to talk to the FDA before starting clinical development in the U.S. The talks are intended to identify “the most expeditious way” to bring MaaT013 to patients in the U.S. while MaaT continues late-stage clinical development in Europe. The European study has a primary completion data of the end of September, per ClinicalTrials.gov. MaaT expected its cash to fund operations into the second quarter of next year when it provided an update last month.

临床3期

2022-08-11

·BioSpace

FDA Wants More Answers Before Releasing Clinical Hold on MaaT's GvHD Drug

Sarah Silbiger/Getty Images French biotech MaaT Pharma announced Wednesday that the U.S. Food and Drug Administration has maintained the clinical hold on MaaT013, its candidate for patients with steroid-resistant acute graft-versus-host disease (aGvHD). MaaT first submitted an investigational new drug (IND) application for MaaT013 back in June 2021, seeking to start a Phase III, open-label, single-arm study of its candidate in the U.S. In August the same year, the federal agency replied with a clinical hold letter, along with questions about safety and efficacy. The clinical-stage company has since sent over detailed answers to these questions and a request for a type A meeting, which is intended to help companies with their stalled product. MaaT CEO and Co-founder Hervé Affagard attests that data from over 120 MaaT013-treated patients from an early access program in France and a completed Phase II trial in Europe show that the candidate is safe and effective for aGvHD. The FDA acknowledges that MaaT’s responses to several of its clinical and manufacturing-related concerns were satisfactory but has decided to maintain the clinical hold while it requests more information about how MaaT013 was developed. The regulatory body has also provided MaaT with a list of recommendations regarding trial design. “We value the FDA’s continued engagement as we seek to extend the Phase 3 clinical trial of MaaT013 to the U.S. and we intend to work with the Agency to resolve the issues raised in the communication while implementing a defined strategic plan to continue to deliver on our key milestones,” Affagard said in a statement. In particular, the FDA has raised concerns about the safety and efficacy of MaaT’s pooling approach to their candidate’s formulation. MaaT013 treats aGvHD by restoring the symbiosis between a patient’s immune system and the gut microbiome. The candidate is a full-ecosystem, off-the-shelf biotherapeutic enema that was developed by mixing samples from several donors. While the FDA has flagged this pooling approach, MaaT maintains that this is how their candidate achieves a high level of species diversity and richness while also making the end product better standardized for broader use. In response to the clinical hold, MaaT is engaging with the FDA in hopes of setting up a type A meeting to discuss these issues more thoroughly. “We remain focused on bringing innovative and safe microbiome therapies to patients with refractory aGvHD,” Affagard said. In the meantime, MaaT013 will continue clinical assessments in Europe. The candidate returned promising results in the Phase II HERACLES trial in March last year. Topline data showed that it could elicit a 33% combined complete and very good partial response rate in 21 heavily pre-treated, immunocompromised patients. Follow-up data, presented in November of the same year, confirmed that in patients who had failed a median of three prior lines of systemic therapy, MaaT013 was able to elicit a good objective gastro-intestinal response rate without inducing alarming safety signals. Earlier this year, MaaT was able to initiate a pivotal Phase III study for its candidate, dosing its first patient in March. This trial is set to enroll 75 patients across 19 clinical sites in France, Germany and Spain.

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