Xervyteg (MaaT013) / MaaT Pharma, Clinigen - LARVOL DELTA

MaaT013 for ruxolitinib-refractory acute graft-versus-host disease with gastrointestinal involvement: Results from the ARES phase III trial (ASH 2025) - P3 | "The treatment period included apreparatory course of 2-day oral vancomycin [250 mg 4 times a day (D)] followed by rectal administrationof 3 MaaT013 doses (on D1, D5 and D10). Out of 26 fatal AEs, one was deemed related toMaaT013.ConclusionResults from the ARES study demonstrate that MaaT013 induces frequent and deep responsestranslating into prolonged survival and an acceptable safety profile in patients with aGvHD whopreviously failed both systemic steroids and ruxolitinib, a population characterized by poor clinicaloutcomes with limited therapeutic options. These positive primary analysis results support MaaT013 asthe first microbiome-based therapy for the treatment of refractory aGvHD."

P3 data • Acute Graft versus Host Disease • Gastrointestinal Disorder • Graft versus Host Disease • Immunology

A Multicentre, Randomized, Double-Blinded, Phase 2b Study Evaluating the Efficacy and Safety of MaaT033, an Oral, Pooled Microbiome Ecosystem Therapy in Patients Undergoing Allogenic Hematopoietic Cell Transplantation to Improve Overall Survival: The Phoebus Trial (ASH 2023) - P2 | "Exclusion criteria comprise non-myeloablative conditioning regimen, conventional myeloablative conditioning regimen, in-vitro T-cell depletion, alloHCT with cord blood cells, alloHCT from an unrelated donor with ≥ 3/10 HLA-mismatches, use of alemtuzumab, vedolizumab or abatacept for GvHD prophylaxis, history of chronic digestive disease...Exploratory endpoints will describe the impact of MaaT013 on immune recovery, the nutritional status of the patients, and resource utilization...The study is approved in France and Germany; expansion to other countries is planned for 2024. Study start is expected in September 2023."

Clinical • P2b data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

MaaT Pharma Presents Pivotal ARES Phase 3 Results for MaaT013 (Xervyteg) in Acute GvHD at ASH 2025 Annual Congress and Announces 54% 1-Year Overall Survival (Businesswire) - "GI-ORR at Day 28 occurred in 41/66 patients (62%) and prevalently consisted of complete response (CR) (38%, 25/66 patients), and very good partial response (VGPR) (20%, 13/66 patients); All-organ ORR at Day 28 occurred in 42/66 patients (64%) patients and was similarly driven by high rates of CR (36%, 24/66 patients) and VGPR (18%, 12/66 patients); GI-ORR at Day 56 was maintained at 47% (31/ 66 patients) and prevalently consisted of CR (35%, 23/66 patients)...Overall survival (OS) at 12 months was 54% (median survival not reached)...The OS was significantly higher in patients who had a GI response at Day 28 than those who did not respond: 68% vs 28% respectively (p <0.0001), indicating a strong association between early GI response and improved survival in refractory GI-aGvHD."

P3 data • Acute Graft versus Host Disease

Pooled Fecal Allogenic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease : Results from Early Access Program in Europe (ASH 2024) - P3 | "Interestingly, the response to MaaT013 correlates with increased OS, suggesting a strong favorable benefit-risk profile for MaaT013. A Phase 3 trial is currently ongoing to confirm these results in ruxolitinib-refractory patients (NCT04769895)."

Acute Graft versus Host Disease • Acute Respiratory Distress Syndrome • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Novel Coronavirus Disease • Pediatrics • Septic Shock

Pooled Fecal Allogenic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease : Results from Early Access Program in Europe (ASH 2023) - P3 | "Interestingly, response of GI-aGvHD correlates with increased overall survival, suggesting a strong favorable benefit-risk profile for MaaT013. A Phase 3 trial is currently ongoing to confirm these results in ruxolitinib-refractory patients (NCT04769895)."

Acute Graft versus Host Disease • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease • Osteoarthritis • Pediatrics • Rheumatology • Septic Shock

MaaT Pharma Announces Positive Phase 3 Results Evaluating Xervyteg (MaaT013) in Acute Graft-versus-Host Disease Selected for Oral Presentation at ASH Congress 2025 (Businesswire) - "Xervyteg (MaaT013) is currently under regulatory review by the European Medicines Agency (EMA), with a decision anticipated in the second half of 2026...Final results, including 1-year overall survival, are expected by the end of 2025...Efficacy data to be presented at the ASH annual meeting is summarized below...(up to the data cut-off of November 11, 2024): GI-Overall Response Rate at Day 28 occurred in 41/66 patients (62%) and prevalently consisted of complete response (CR) (25/66 patients, 38%) and very good partial response (VGPR) (13/66 patients, 20%); Overall Response Rate (all organs) at Day 28 occurred in 42/66 patients (64%) patients and was similarly driven by high rates of CR (24/66 patients, 36%) and VGPR (12/66 patients, 18%)....The estimated OS was 54% with a median follow-up of 140.5 days (median survival not reached)."

EMA approval • P3 data • Acute Graft versus Host Disease

MaaT Pharma Launches a Capital Increase of Approximately €9 Million (Businesswire) - "Preparation of the commercialization of Xervyteg and related regulatory activities in Europe during the second half of 2026, in collaboration with our commercial partner, contingent upon Xervyteg approval (potential marketing authorization (MA) could be delivered around mid-2026). Advancement of MaaT033 clinical development in Europe, currently being evaluated in an ongoing Phase 2b trial, designed to be pivotal, aimed at improving survival in patients undergoing allogeneic hematopoietic stem cell transplantation."

Financing • Acute Graft versus Host Disease • Transplantation

MaaT Pharma Reports Financial Results for the Third Quarter 2025… (Businesswire) - "MaaT Pharma reported revenues from France from its Early Access Program of EUR 1.0 million for the quarter ended September 30, 2025, a 56% increase over the third quarter of 2024. Total revenues for the first nine months of 2025 amounted to EUR 3.4 million compared with EUR 2.3 million for the same period of 2024, a year-over-year increase of 45%1, reflecting the continued demand from the medical community for MaaT Pharma’s drug candidate Xervyteg(MaaT013)."

Commercial • Acute Graft versus Host Disease

Acute Graft-versus-Host Disease (aGvHD) – Xervyteg (MaaT013) (MaaT Pharma Press Release) - "Final results from the pivotal ARES study, including 12-month overall survival data, are expected before the end of 2025 and will be incorporated into the filing dossier. Data are also being submitted in a peer reviewed journal and upcoming scientific congress."

P3 data • Acute Graft versus Host Disease

Xervyteg – Exploratory trials using the MET-N platform (donor derived conducted as Investigator-Sponsored Trials (ISTs) to inform further developments (MaaT Pharma Press Release) - "In March 2024, the Company completed patient recruitment for the Phase 2a randomized clinical trial (NCT04988841)...evaluating Xervyteg in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients....Data readout is expected in H2 2025 as previously announced."

P2a data • Melanoma

MaaT Pharma…Provides a Business Update (MaaT Pharma Press Release) - "The potential marketing authorization could be delivered around mid-2026 (if approved), then enabling the start of the commercialization of Xervyteg in Europe in the second half of 2026."

EMA approval • Launch Europe • Acute Graft versus Host Disease

PICASSO: Assessing the Tolerance and Clinical Benefit of feCAl tranSplantation in patientS With melanOma (clinicaltrials.gov) - P2 | N=70 | Completed | Sponsor: Assistance Publique - Hôpitaux de Paris | Recruiting ➔ Completed | Trial completion date: Jun 2024 ➔ Apr 2025 | Trial primary completion date: Jan 2024 ➔ Aug 2024

Trial completion • Trial completion date • Trial primary completion date • Melanoma • Oncology • Solid Tumor • Transplantation

MaaT Pharma Secures €37.5 Million Loan From European Investment Bank (EIB) Marking a New Step in Advancing its Clinical Program in Hemato-Oncology (Businesswire) - "MaaT Pharma...announced that it has secured a €37.5 million, 4-tranche financing from the European Investment Bank (EIB). The financing will support the advancement of its late-stage hemato-oncology clinical programs including the lead-asset Xervyteg, recently partnered with Clinigen in Europe, and currently under regulatory review by the European Medicines Agency (EMA) for the treatment of acute Graft-versus-Host Disease (aGvHD) and the second drug candidate, MaaT033, currently being evaluated in a Phase 2b randomized controlled trial in improving survival for patients receiving allogeneic stem cell transplants."

Financing • Acute Graft versus Host Disease • Transplantation

MaaT Pharma Announces Exclusive Commercialization Partnership With Clinigen for Xervyteg in acute Graft-versus Host Disease in Europe (Businesswire) - "MaaT Pharma...announces the signature of a license and commercial agreement with Clinigen, a global specialty pharmaceutical services group and a leading European player in hospital distribution and market access, to streamline the pathway for ensuring access to this medicine across Europe....Under the terms of the agreement, MaaT Pharma will grant Clinigen exclusive European rights to distribute this medicine for the treatment of patients with aGvHD, if approved by the EMA. MaaT Pharma will receive an upfront payment of €10.5 million and additional eligible payments of up to €18 million depending on the achievement of pre-specified regulatory and sales milestones. MaaT Pharma will also be eligible to receive royalty payments on net sales of a percentage in the mid-thirties and regular cash flow as per the supply agreement."

Licensing / partnership • Acute Graft versus Host Disease

POOLED FECAL ALLOGENIC MICROBIOTHERAPY FOR REFRACTORY GASTROINTESTINAL ACUTE GRAFT-VERSUS-HOST DISEASE : RESULTS FROM THE EARLY ACCESS PROGRAM IN EUROPE (EHA 2025) - P3 | "EAP data shows that MaaT013 is a safe and effective treatment of refractory GI-GvHD especially in patients treated with ruxolitinib. Response to MaaT013 correlates with increased OS, suggesting a strong favorable benefit-risk profile. Positive toplines results from the pivotal ARES Phase 3 trial confirm these results in ruxolitinib-refractory patients (NCT04769895)."

Acute Graft versus Host Disease • Acute Respiratory Distress Syndrome • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease • Oncology • Pediatrics • Septic Shock

Acute Graft-versus-Host Disease (aGvHD) – Xervyteg (MaaT013) (Businesswire) - "As part of a strategic focus on Xervyteg’s registration activities in Europe, the Phase 3 dedicated trial launch in the U.S is now expected in 2026....In parallel, the Company continues discussions with the FDA to optimize a dedicated pivotal study in the U.S., with the objective of enabling the earliest possible access to Xervyteg for U.S. patients. Such a study could be initiated in 2026 (instead of Q4 2025), subject to regulatory confirmation as MaaT Pharma continues watching the evolving regulatory policies and process in the United States."

New P3 trial • Acute Graft versus Host Disease

Pooled Faecal Microbiotherapy Can Curb Refractory Gastrointestinal Acute Graft-versus-Host Disease Likely Through DP8a Treg Induction/Activation (FOCIS 2025) - P | "Remarkably, DP8a Tregs expanded only in MaaT013-responding patients, suggesting that MaaT013 could stimulate DP8a Tregs to curb GI-aGvHD.Additional investigations revealed that DP8a Tregs can cross-react with Akkermansia muciniphila, another bacterium contained in MaaT013. These cross-reactions, and potentially others, could explain the DP8a Treg-mediated anti-inflammatory effect observed both in vitro and in vivo.We further demonstrated that MaaT013 induces anti-inflammatory and Treg-polarizing molecules on dendritic cells, likely contributing to the induction/activation of DP8a Tregs.Altogether, these data suggest the key role of DP8a Tregs to alleviate GI-aGvHD in response to MaaT013 faecal microbiotherapy."

Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

Pooled Faecal Microbiotherapy Can Curb Refractory Gastrointestinal Acute Graft-versus-Host Disease Likely Through DP8a Treg Induction/Activation (FOCIS 2025) - P | "Remarkably, DP8a Tregs expanded only in MaaT013-responding patients, suggesting that MaaT013 could stimulate DP8a Tregs to curb GI-aGvHD.Additional investigations revealed that DP8a Tregs can cross-react with Akkermansia muciniphila, another bacterium contained in MaaT013. These cross-reactions, and potentially others, could explain the DP8a Treg-mediated anti-inflammatory effect observed both in vitro and in vivo.We further demonstrated that MaaT013 induces anti-inflammatory and Treg-polarizing molecules on dendritic cells, likely contributing to the induction/activation of DP8a Tregs.Altogether, these data suggest the key role of DP8a Tregs to alleviate GI-aGvHD in response to MaaT013 faecal microbiotherapy."

Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

MaaT Pharma Presents Updated Positive Data in Early Access Program for Xervyteg at the EHA Congress Validating High Efficacy Observed in Pivotal ARES Study in Acute Graft-versus-Host Disease (Businesswire) - P3 | N=66 | ARES (NCT04769895) | Sponsor: MaaT Pharma | "Gastrointestinal Overall Response Rate (GI-ORR) of 53% at D28, with Complete Response (CR) observed in 30% of patients; all-organ Overall Response Rate (ORR) was 50% with 26% CR. Response is maintained at D56 indicating a long-term disease control with a GI-ORR of 47% and an ORR considering all organs of 46%. Overall Survival (OS) in all patients was 55% at 6 months, 48% at 12 months, 44% at 24 months. Xervyteg displayed a good overall safety profile in the EAP population. OS was significantly higher in patients who responded to Xervyteg (MaaT013) compared to non-responders (69% versus 25% at 12 months, and 61% versus 25% at 24 months)."

P3 data • Acute Graft versus Host Disease

MaaT Pharma Advances Toward Commercialization And Submits Marketing Authorization Application to the European Medicines Agency (EMA) for Xervyteg (MaaT013) in Acute Graft-versus-Host Disease (Businesswire) - "MaaT Pharma...announced the submission of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead drug candidate MaaT013, under the registered brand name of Xervyteg. If approved, the Marketing Authorization would establish Xervyteg as the first microbiota therapeutic approved by the EMA, and the first one globally for a hematology indication. Xervyteg would also be the first approved therapy for the treatment of acute Graft-versus-Host Disease including gastro-intestinal involvement (GI-aGvHD) following 2 prior lines of systemic therapy...The MAA is supported by positive results from the Pivotal ARES study, a single-arm, open-label, multicenter European study evaluating the efficacy and safety of Xervyteg in GI-aGvHD as third-line therapy in 66 patients....The EMA will review the application under the centralized marketing authorization procedure and potentially a marketing authorization could be granted in H2 2026."

EMA approval • EMA filing • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

MaaT Pharma To Present Updated Data for MaaT013 in Early Access Program at the European Hematology Association (EHA) Annual Congress (Businesswire) - "Oral presentation will highlight updated data in Early Access Program (EAP) for 173 patients with acute Graft-vs-Host Disease (aGvHD) treated with MaaT013. Poster presentation selected for ongoing Phase 2b trial of MaaT033 in patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT)"

Clinical data • Clinical protocol • Acute Graft versus Host Disease

MaaT013– Proof-of-Concept trials with donor derived drugs (MET-N platform) (MaaT Pharma Press Release) - "MaaT013 is currently being evaluated in a Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients. The Company provided its MaaT013 drug candidate and placebo and contributes to the microbiome profiling of patients using its proprietary gutPrint AI research engine, while the trial investigator sponsor handled recruitment, treatment and oversee data collection and analysis. Data readout is expected in H2 2025."

P2a data • Melanoma

The Capital Increase will enable the Company to pursue its developments plan and confirm its potential for being a key player in hemato-oncology. (Businesswire) - "...the Company is targeting to submit its Market Authorization Application for MaaT013 in aGvHD with the European agency in June 2025, with a potential approval expected in H2 2026; the Company is also preparing for a commercial launch in Europe, based on compelling Phase 3 data released in January 2026...in the US, the Company is preparing for the launch of a dedicated US Phase 3 trial in aGvHD...in view of a late 2026 commercial launch, if approved...the Company will continue the ongoing allo-HSCT Phase 2b study 'PHOEBUS' (1yr-OS read out expected in late 2027)."

EMA approval • EMA filing • Launch Europe • Launch US • P2b data • Acute Graft versus Host Disease • Transplantation

MaaT Pharma Completes a Capital Increase of €13 Million with Historical Shareholders… (Businesswire) - "MaaT Pharma...announces the completion of a private placement of €13 million at market price through the issuance of 2,131,148 new ordinary shares of the Company....The funds will support the Company’s progress towards key value milestones, including market submission for EU approval of MaaT013 in aGvHD, its US expansion strategy, and the conclusion of a potential partnership for its hemato-oncology products in Europe."

Financing • Acute Graft versus Host Disease • Hematological Malignancies

MaaT Pharma Announces Positive Outcomes from Final DSMB Meeting for Pivotal Phase 3 Clinical Trial Evaluating MaaT013 in Acute Graft-versus-Host Disease (Businesswire) - "MaaT Pharma...announced, after recently receiving the approval of its Pediatric Investigation Plan (PIP) by the EMA Pediatric Committee, that the DSMB completed its final safety assessment of the pivotal Phase 3 trial ARES, a single-arm, open-label, multicenter European study evaluating the efficacy and safety of MaaT013 in acute Graft-versus-Host Disease patients with gastrointestinal involvement (GI-aGvHD) in third-line treatment, refractory to steroids and refractory or intolerant to ruxolitinib...The DSMB confirmed that 'given the remarkable efficacy results, the study results show an acceptable safety profile and a favorable benefit /risk ratio'. The DSMB members will continue to review safety on an ongoing basis until the 1-year follow-up....Evaluation of the One-year Overall Survival (secondary endpoint evaluation) is expected in Q4 2025; The Company is actively engaging with investors and potential partners to advance toward the regulatory phase and commercialization."

DSMB • Trial status • Acute Graft versus Host Disease