MaaT Pharma announced today that leading hemato-oncological experts presented clinical data on the compassionate use of MaaT Pharma's lead full-ecosystem microbiome restoration biotherapeutic, MaaT013. The data included eight patients that developed gastrointestinal-predominant, acute Graft-versus-Host-Disease (GI aGvHD) after receiving an allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) to treat their hematologic malignancies. All patients were positively impacted by the MaaT013 treatment, with three patients achieving complete response. GvHD, a condition where the transplant donor's immune cells attack the patient's tissues, is one of the most serious complications of allo-HSCT, and its acute GI form is fatal in most cases. MaaT013 features a consistently high diversity and richness of microbial species in their natural environment. It aims to restore the symbiotic relationship between microbes in the gut and the immune system of the patient to correct the responsiveness and tolerance (homeostasis) of immune functions ? and thereby contain GI GvHD. The results were presented in a poster presentation on December 7, 2019 during the 61st American Society of Hematology (ASH) Annual Meeting and Exposition held in Orlando, Florida.
"The GI aGvHD patients who were treated with MaaT013 had a very poor prognosis with no other therapeutic options. The results following MaaT013 administration showed a positive impact on all patients," commented Professor Mohamad Mohty, MD, PhD, Head of the Hematology and Cellular Therapy Department at Sorbonne University, Saint Antoine Hospital in Paris. "The most impressive results were seen in those patients who achieved a complete response and who were able to taper and stop using steroids and other immunosuppressants without relapse of gastrointestinal symptoms."
In the presented evaluation, eight patients with a median age of 67 were treated for classical aGvHD, late-onset aGvHD or aGvHD with overlap syndrome that were either steroid-resistant or steroid-dependent following stem cell transplantation. These patients had previously been treated with and failed up to five lines of systemic therapy for aGvHD. Each patient received at least one and up to three doses of MaaT013 and treatment response was evaluated seven days after each administration and on day 28 after the first dose. Based on the best response to the treatment, all eight patients experienced at least a partial response with three patients achieving complete response, two patients with very good partial response and three patients with partial response. Overall, the data demonstrated that reintroduction of a full-ecosystem microbiota provided therapeutic effect and was tolerated in a satisfactory manner in these patients.
Hervé Affagard, Co-founder and CEO of MaaT Pharma added, "We provided our cGMP-manufactured lead biologic drug, MaaT013, to hospitals as part of a compassionate use program to give GI GvHD patients a therapeutic option where there are no other available treatments after steroids and additional lines of treatment. These findings indicate that reestablishing the gut microbiome improved outcomes in these patients."
Moreover, MaaT Pharma is currently conducting the HERACLES Phase II clinical trial (NCT03359980) to evaluate the safety and efficacy of MaaT013 in steroid-refractory, GI aGvHD patients, with more than half of the patients enrolled.
To date, a total of 46 patients with GI GvHD have been treated with MaaT013, including patients under compassionate use and patients enrolled in the Phase II clinical trial. MaaT Pharma is actively developing an oral formulation of MaaT013 (a capsule, MaaT033) to provide easier administration for patients while delivering a similar effect of regenerating the microbial ecosystem with the goal of restoring immune homeostasis in the gut.
The poster can be viewed on the company's website under "News".
The HERACLES study is a multi-center, single-arm, open-label study, enrolling 32 patients to evaluate the efficacy and safety of MaaT Pharma's lead microbiome restoration drug candidate, MaaT013, in steroid-resistant, gastrointestinal-predominant aGvHD patients. Acute GvHD is a serious, often fatal syndrome typically involving the gut, skin, and liver. Treatments up to now focused largely on suppressing the immune reaction that is induced by the donor cells derived from the hematopoietic stem cell graft reacting against the host. These strategies have remained clinically unsuccessful in most cases, with mortality rates around 80% after twelve months in steroid-resistant cases. Patients with hematological malignancies receive multiple courses of chemotherapy, antibiotics, and ultimately conditioning before HSCT, which are known to severely impact the gut microbial composition.
MaaT013 is the first full-ecosystem, off-the-shelf, reproducible, enema formulation manufactured using MaaT Pharma's integrated Microbiome Restoration Biotherapeutic (MMRB) platform. The product has a stability of up to 24 months and is characterized by a high diversity and consistent richness of microbial species derived from pooled healthy donors and manufactured at the company's centralized European cGMP production facility. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and is already being administered in compassionate use.
About MaaT Pharma
MaaT Pharma, a clinical stage company, has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating blood cancers and Graft-versus-Host-Disease, a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients. Supporting the further expansion of our pipeline into improving outcomes of immunotherapy in solid tumors, we have built a powerful discovery and analysis platform, GutPrint®, to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our biotherapeutics are produced under the strictest cGMP manufacturing and quality control process to safely deliver the full diversity and functionality of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spearhead microbiome treatment integration into clinical practice.
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