(News Bulletin 247) – The Lyon biotech announced positive results for MaaT013, its most advanced drug candidate at the annual conference of the American Society of Hematology.

Two years after its IPO on Euronext Paris, MaaT Pharma announces an important milestone for its most advanced drug candidate, MaaT013.

The Lyon-based company, whose acronym stands for “Microbiote As A Therapy”, presented positive efficacy results at the annual conference of the American Society of Hematology (ASH), for MaaT013 in acute graft-versus-graft disease. the host (aGvH). This is a very serious complication occurring after a stem cell transplant.

Remember that MaaT013 is a complete ecosystem microbiotherapy, ready to use, standardized and derived from a combination of microbiota from healthy donors. The product is characterized by a high and standardized diversity and richness of the microbial species it contains and the presence of Butycore (set of bacteria producing anti-inflammatory metabolites).

The compound, today the most advanced from the Lyon company, aims to restore the symbiotic relationship between the patient’s intestinal microbiota and their immune system, in order to correct reactivity and restore tolerance of immune functions and thus reduce steroid-resistant gastrointestinal aGvHD.

This acute graft-versus-host disease occurs in patients usually within 100 days after a stem cell or bone marrow transplant as part of cancer treatment. In aGvH the transplanted cells “attack” those of the recipient, causing inflammation of the skin, liver and/or gastrointestinal tract.

“Promising” results

Concerning the results published this Monday, they come from the compassionate access program (a program which allows experimental treatments to be administered to patients without a therapeutic solution, but which does not strictly correspond to a clinical study) in Europe relating to 111 patients with acute graft-versus-host disease aGvHD and steroid-refractory or steroid-dependent treated with MaaT013.

And as part of this program, Maat Pharma reveals a gastrointestinal overall response rate (GI-ORR) of 53% at day 28, with a positive and significant impact on overall survival in responding patients. The overall survival results were even “more pronounced” (81% for responders and 8% for non-responders), adds MaaT Pharma for the population of patients with a profile similar to those treated in the ARES clinical trial (GI- ORR of 61% on day 28).

As a reminder, ARES is a multicenter, international, open-label, single-arm pivotal Phase III trial evaluating the safety and efficacy of MaaT013 in 75 patients with grade II-IV gastrointestinal acute GvHD, refractory to steroids. and resistant or intolerant to ruxolitinib, an anticancer drug which is marketed in France under the name Jakavi by the Novartis laboratory.

“These promising results mark a significant advance in the treatment of aGvHD, thanks to innovations derived from the microbiota, especially when they allow complete responses to be achieved,” underlined Hervé Affagard, general director and co-founder of MaaT Pharma.

The company specifies that a Phase III trial (last phase of clinical trials before possible marketing) is underway in Europe to confirm these results in patients refractory to ruxolitinib.

The overall response rate is expected by mid-2024, specifies Maat Pharma. The Lyon-based company also has an Investigational New Drug application, granted by the US Food and Drug Administration, and active discussions with potential clinical investigational sites.

A Phase IIb trial for MaaT033

MaaT Pharma also presented at this conference the design of the ongoing Phase IIb trial evaluating MaaT033, its second drug candidate in patients receiving an allogeneic hematopoietic stem cell transplant.

“Developed as an adjuvant treatment dedicated to improving overall survival in patients receiving HSCT and other cellular therapies, MaaT033 aims to optimize microbiota function and is aimed at a broader patient population, in chronic use,” explains MaaT Pharma.

“This international, multicenter trial is the largest randomized control arm study to date of a microbiota therapy in oncology, covering up to 56 sites and recruiting 387 patients,” the company continues.

On the Paris Stock Exchange, MaaT Pharma shares jumped by more than 7%, around 2:00 p.m. after peaking at 12.35% at the very start of the session, in reaction to the announcement of these results.