Xervyteg (MaaT013) / MaaT Pharma - LARVOL DELTA

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 | "At both Day 28 and Day 56, Xervyteg demonstrated durable efficacy in SR/RR aGvHD patients, with GI-ORRs of 57% and Complete Response (CR) observed in 44% of patients at D28 and 51% at D56. All-organs ORR was 54% with 41% CR at D28, and 55% with 48% CR at D56. OS was 55% at 6 months, 51% at 12 months, 40% at 24 months. OS was significantly higher in patients who responded to Xervyteg compared to non-responders (77% versus 14% at 12 months, and 59% versus 14% at 24 months). Median survival in all 70 patients was 445 days. In responder patients, median survival was 834 days vs 53 days in non-responders."

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

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

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 • Hematological Disorders • Immunology • Infectious Disease • Novel Coronavirus Disease • Oncology • Pediatrics • Septic Shock

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

MaaT Pharma Receives Positive Opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for MaaT013 (Businesswire) - "...MaaT013 is on track towards a marketing authorization submission to the EMA in June 2025...MaaT Pharma...announced today that the European Medicines Agency (EMA) Pediatric Committee (PDCO) has approved the Pediatric Investigation Plan (PIP) for MaaT013 for the treatment of acute Graft-versus-Host Disease (aGvHD)...The EMA PDCO approved the clinical program to evaluate the safety and efficacy of MaaT013 in patients from 6 years old to less than 18 years old, with the initiation, in 2026, of a single-arm trial in third-line treatment for 18 patients with aGvHD and in line with the Company’s cash projections....Based on this positive opinion, MaaT013 would be eligible for up to an additional two years of marketing exclusivity in Europe, on top of the ten-year European market exclusivity as an orphan drug if the Marketing Authorization is granted by the EMA. This also confirms the Company’s ability to reach the full patient population."

EMA filing • New trial • Acute Graft versus Host Disease

MaaT013: EMA approval in EU for acute GvHD (based on ARES trial) in H2 2026 (MaaT Pharma) - Corporate Presentation: Launch in EU for acute GvHD in Q4 2026

EMA approval • Launch Europe • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

MaaT013: “62% GI-ORR with high CR and VGPR rates”; Graft versus host disease (MaaT Pharma) - Corporate Presentation

P3 data: top line • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

MaaT Pharma Announces Positive Topline Results from the Pivotal Phase 3 ARES Study Evaluating MaaT013 in acute Graft-versus-Host Disease (Businesswire) - P3 | N=66 | ARES (NCT04769895) | Sponsor: MaaT Pharma | "The study met its primary endpoint of GI-ORR at Day 28 of treatment with MaaT013 (p <0.0001), as assessed by the Independent Review Committee (IRC). Frequent, strong and durable response rates translating into prolonged survival; GI-ORR 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%). ORR in all evaluable organs 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%)....With robust data supporting efficacy and safety, MaaT Pharma is pursuing the regulatory submission of MaaT013, in Europe, as a treatment for GI-aGvHD in third-line, aiming for a Centralized Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in mid-2025, earlier than previously expected."

EMA filing • P3 data: top line • Acute Graft versus Host Disease

MaaT013: “MaaT013 is highly effective therapy for SR- and SD-GI- a GVHD”; Graft versus host disease (MaaT Pharma) - ASH 2024 Annual Meeting: “D28 GI ORR 51% and ORR 49%”

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

MaaT013: Final OS data from P3 ARES trial (NCT04769895) for acute GvHD in H2 2025 (MaaT Pharma) - ASH 2024 Annual Meeting: Completion of P3 ARES trial for acute GvHD in Nov 2025

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

MaaT013: EMA filing in EU for acute GvHD (based on ARES trial) in H2 2025 (MaaT Pharma) - ASH 2024 Annual Meeting: EMA approval in EU for acute GvHD in 2026

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

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 • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Novel Coronavirus Disease • Oncology • Pediatrics • Septic Shock

MaaT Pharma Presented Positive Updated Data on MaaT013 in the Early Access Program at ASH 2024 Annual Meeting (Businesswire) - "Topline results for the ARES pivotal Phase 3 trial anticipated in January 2025....For the full cohort (154 patients) in the EAP; Durable response: 51% GI-ORR at Day 28 and a 44% GI-ORR at Day 56. ORR for all organs was 49% at D28 and 42% at D56. Overall survival (OS): 53% at 6 months, 47% at 12 months, and 42% at 24 months....Higher response rate than the full cohort: GI-ORR was 59% at Day 28 and 54% at D56. ORR considering all organ was 55% at D28, and 56% at D56."

Clinical data • P3 data • Acute Graft versus Host Disease

MaaT Pharma Announces First U.S. Patient Treated at City of Hope Under Single Patient Expanded Access for MaaT013 in Acute Graft-versus-Host Disease (Businesswire) - "MaaT Pharma...announced the first treatment in the United States of a patient with acute Graft-versus-Host Disease (aGvHD) under the US Food and Drug Administration (FDA) Single Patient Expanded Access....'We believe that microbiome-based immune modulation may play a crucial role in the treatment of aGvHD and look forward to exploring the potential of MaaT013 to enhance GvHD outcomes and improve patient survival.'"

Trial status • Acute Graft versus Host Disease

MaaT Pharma to Present Updates from Early Access Program at the 2024 ASH Annual Meeting Demonstrating Prolonged Long-Term Survival in Patients Receiving MaaT013 in aGvHD (Businesswire) - P3 | N=75 | ARES (NCT04769895) | Sponsor: MaaT Pharma | "MaaT Pharma...announced that updated results from its Early Access Program of MaaT013 in 154 patients with steroid-refractory (SR) or dependent (SD) gastrointestinal acute Graft-versus-Host Disease (GI-aGvHD)...The gastrointestinal overall response rate (GI-ORR) at day 28 was 51% with complete response (CR) occurring in 46 patients (30%). ORR considering all organs was 49% with CR occurring in 41 patients....Subset receiving 2nd line ruxolitinib (n=58) resembling the population enrolled in the Phase 3 ARES trial: Compared to the full patient cohort, improved responses were observed. The GI-ORR was 59% at day 28 (CR 48%). ORR considering all organ was 55% (CR 43%)....'As we eagerly anticipate the forthcoming results of our Phase 3 ARES trial, we are encouraged by these positive long-term results which underscore MaaT013’s potential to address a significant unmet need for patients with refractory GI-aGvHD.'"

P3 data • Acute Graft versus Host Disease

MaaT Pharma Provides Third Quarter 2024 Business and Financial Results and Announces Management Changes (Businesswire) - "MaaT Pharma reported revenues from its Early Access Program of EUR 0.6 million for the quarter ended September 30, 2024, an increase of EUR 0.2 million over the third quarter of 2023. Total revenues for the first nine months of 2024 amounted to EUR 2.3 million compared with EUR 1.8 million for the same period of 20231. This trend is a direct reflection of the continued demand from the medical community for MaaT Pharma’s drug candidate MaaT013."

Commercial • Acute Graft versus Host Disease

ARES: MaaT013 As Salvage Therapy in Ruxolitinib Refractory GI-aGVHD Patients (clinicaltrials.gov) - P3 | N=66 | Active, not recruiting | Sponsor: MaaT Pharma | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2024 ➔ Nov 2025 | Trial primary completion date: Sep 2023 ➔ Nov 2024

Enrollment closed • Trial completion date • Trial primary completion date • Acute Graft versus Host Disease • Gastrointestinal Disorder • Graft versus Host Disease • Immunology

MaaT Pharma Completes Recruitment of its ARES Phase 3 Trial for MaaT013 to Treat Acute Graft-versus-Host Disease (Businesswire) - "Registration process to commence with submission expected in 2025 in Europe....MaaT Pharma...today announced full recruitment of its Phase 3 clinical trial designed to evaluate efficacy and safety of its lead asset MaaT013 in the treatment of steroid refractory and ruxolitinib refractory or intolerant acute Graft-versus-Host Disease (aGvHD) (NCT04769895). Next steps and milestones for MaaT013 in GvHD: Early December 2024: Company to host Key Opinion Leaders discussion on updated data from Early Access Program in Europe for MaaT013 in treating aGvHD. January 2025: Topline results with primary endpoint evaluation: GI Overall Response Rate (GI-ORR) at day 28....By end of 2025: Secondary endpoint evaluation including One-year Overall Survival."

EMA filing • Enrollment closed • P3 data: top line • Acute Graft versus Host Disease

MaaT Pharma Publishes its Half Year 2024 Results and Provides a Business Update (Businesswire) - "MaaT013:...In March 2024, the Company informed on the completion of patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO)....A total of 70 patients have been enrolled since April 2022.....Data readout is expected in Q4 2024/Q1 2025....MaaT034:...Clinical activities for MaaT034 are now expected to begin in 2026 and not 2025, as previously announced."

Clinical • P2a data • Trial status • Melanoma