Latest news with #Xervyteg®
Yahoo
2 days ago
- Health
- Yahoo
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
Oral presentation highlights updated data in Early Access Program (EAP) for 173 patients with acute Graft-vs-Host Disease (aGvHD) treated with Xervyteg® Independent dataset from EAP reinforces the findings from the pivotal ARES trial and has also been included in the EMA Marketing Authorization Application submitted on June 2nd, 2025 EAP for Xervyteg® in aGvHD is currently running in 11 countries* providing expanded access to patients with high unmet medical needs LYON, France, June 13, 2025--(BUSINESS WIRE)--Regulatory News: MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced that Professor Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University, will present updated data for Xervyteg® (MaaT013) in treating acute Graft-versus-Host Disease (aGvHD) under the Early Access Program (EAP) at the European Hematology Association (EHA) Annual Congress 2025. This independent EAP dataset further supports the efficacy and safety profile of Xervyteg® previously shown in the pivotal ARES trial. It also confirms the breakthrough potential of Xervyteg® for aGvHD patients with limited treatment options and it also serves as supportive data within the Marketing Authorization Application (MAA) recently submitted to the European Medicines Agency (EMA). Key highlights: aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. The patients (N=173) treated in EAP previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (49%) or IV (38%) aGvHD. The real-world data presented underscores the favorable safety profile of Xervyteg® the strong and durable responses, translating into increased overall survival: 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). Median survival in all patients was 312 days. In responder patients, median survival was 834 days vs 69 days in non-responders. A subset of patients (n=70) failing both steroid resistant (SR) and ruxolitinib resistant (RR) and thus resembling the cohort enrolled in the pivotal Phase 3 ARES trial (NCT04769895), exhibited a significant and consistent efficacy profile: 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. The complete data may be found here. "The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg®'s clinical benefit for patients with severe, treatment-resistant aGvHD," said Dr. Gianfranco Pittari, PhD, Chief Medical Officer at MaaT Pharma. "This is particularly meaningful for clinicians and patients, as it confirms the potential of microbiome therapies to deliver long-term survival benefits in a population with historically poor outcomes." In comparison, historical data from Abedin et al. 2021 demonstrated that in a similar population of patients, i.e. third-line aGvHD patients receiving additional treatment after ruxolitinib failure, the median survival was only 28 days. "Among patients who responded by Day 28, the majority achieved a complete resolution of aGvHD symptoms — a strong predictor of sustained disease control over time. The overall safety profile is favorable in this high-risk patient population," outlines Professor Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University. Details of the Oral Presentation: Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe Abstract number: S260 Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University Session title: s424 Stem cell transplantation - Session 2 Date & Time: 13/06/2025 (17:00 - 17:15 CEST) - Brown Hall 3 MaaT Pharma also presented a poster on the design of its ongoing Phase 2b trial (PHOEBUS) evaluating MaaT033 to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites. --- About MaaT PharmaMaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking StatementsAll statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as "target," "believe," "expect," "aim", "intend," "may," "anticipate," "estimate," "plan," "project," "will," "can have," "likely," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. * France, Austria, Belgium, Canada, Germany, Italy, Lebanon, Spain, Sweden, Switzerland & USA View source version on Contacts MaaT Pharma – Investor RelationsGuilhaume DEBROAS, of Investor Relations+33 6 16 48 92 50invest@ Rx Communications Group – U.S. Investor RelationsMichael MillerManaging Director+1-917-633-6086mmiller@ MaaT Pharma – Media RelationsPauline RICHAUDSenior PR & Corporate Communications Manager+33 6 14 06 45 92media@ Catalytic Agency – U.S. Media RelationsHeather SheaMedia relations for MaaT Pharma+1 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
2 days ago
- Health
- Business Wire
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
LYON, France--(BUSINESS WIRE)--Regulatory News: 'The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg®'s clinical benefit for patients with severe, treatment-resistant aGvHD,' said Dr. Gianfranco Pittari, Chief Medical Officer at MaaT Pharma. MaaT Pharma (EURONEXT: MAAT – the 'Company'), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem Therapies TM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced that Professor Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University, will present updated data for Xervyteg ® (MaaT013) in treating acute Graft-versus-Host Disease (aGvHD) under the Early Access Program (EAP) at the European Hematology Association (EHA) Annual Congress 2025. This independent EAP dataset further supports the efficacy and safety profile of Xervyteg ® previously shown in the pivotal ARES trial. It also confirms the breakthrough potential of Xervyteg ® for aGvHD patients with limited treatment options and it also serves as supportive data within the Marketing Authorization Application (MAA) recently submitted to the European Medicines Agency (EMA). Key highlights: aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. The patients (N=173) treated in EAP previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (49%) or IV (38%) aGvHD. The real-world data presented underscores the favorable safety profile of Xervyteg ® the strong and durable responses, translating into increased overall survival: 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). Median survival in all patients was 312 days. In responder patients, median survival was 834 days vs 69 days in non-responders. A subset of patients (n=70) failing both steroid resistant (SR) and ruxolitinib resistant (RR) and thus resembling the cohort enrolled in the pivotal Phase 3 ARES trial (NCT04769895), exhibited a significant and consistent efficacy profile: 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. The complete data may be found here. 'The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg ® 's clinical benefit for patients with severe, treatment-resistant aGvHD,' said Dr. Gianfranco Pittari, PhD, Chief Medical Officer at MaaT Pharma. 'This is particularly meaningful for clinicians and patients, as it confirms the potential of microbiome therapies to deliver long-term survival benefits in a population with historically poor outcomes.' In comparison, historical data from Abedin et al. 2021 demonstrated that in a similar population of patients, i.e. third-line aGvHD patients receiving additional treatment after ruxolitinib failure, the median survival was only 28 days. 'Among patients who responded by Day 28, the majority achieved a complete resolution of aGvHD symptoms — a strong predictor of sustained disease control over time. The overall safety profile is favorable in this high-risk patient population,' outlines Professor Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University. Details of the Oral Presentation: Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe Abstract number: S260 Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University Session title: s424 Stem cell transplantation - Session 2 Date & Time: 13/06/2025 (17:00 - 17:15 CEST) - Brown Hall 3 MaaT Pharma also presented a poster on the design of its ongoing Phase 2b trial (PHOEBUS) evaluating MaaT033 to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites. --- About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking Statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as 'target,' 'believe,' 'expect,' 'aim', 'intend,' 'may,' 'anticipate,' 'estimate,' 'plan,' 'project,' 'will,' 'can have,' 'likely,' 'should,' 'would,' 'could' and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. * France, Austria, Belgium, Canada, Germany, Italy, Lebanon, Spain, Sweden, Switzerland & USA
Yahoo
02-06-2025
- Business
- Yahoo
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
MaaT Pharma submitted today a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its product candidate MaaT013, under the registered brand name of Xervyteg®. Xervyteg® has the potential if approved, to become the first microbiota therapeutic approved by the EMA and the first one in hemato-oncology globally. The MAA submitted to the EMA is based on data from the Pivotal ARES study, evaluating the safety and efficacy of Xervyteg® in adult patients with acute Graft-versus-Host Disease including gastro-intestinal involvement who received two prior lines of therapy and supported by data from the ongoing Early Access Program. MaaT Pharma prepares for a potential 2026 commercial launch through a strategic partnership to address this key unmet need in hemato-oncology. LYON, France, June 02, 2025--(BUSINESS WIRE)--Regulatory News: MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today 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. "Submitting our MAA to the EMA marks a major regulatory milestone for MaaT Pharma and a meaningful advancement for patients with refractory aGvHD—a life-threatening complication of stem cell transplantation with no approved therapies," said Hervé Affagard, Co-founder and CEO of MaaT Pharma. "We are now closer to providing a much-needed treatment option and remain deeply committed to advancing immunomodulating microbiota technologies in hemato-oncology, where new solutions are urgently needed." While advancing toward the commercialization of Xervyteg® (if approved) in Europe, MaaT Pharma is also actively exploring strategic partnerships to ensure broad access in timely fashion. The Company has active discussions with experienced partners who share its mission of delivering meaningful advancements to patients. In parallel to the MAA submission, MaaT Pharma continues to provide access to Xervyteg® in Europe and the U.S. through its Early Access Program (EAP)1 for patients with aGvHD and other indications. In 2024, physician demand for Xervyteg® under the EAP (n=107) increased by 75% compared to 2023, driven by growing adoption across Europe and in the U.S. In France where the EAP first started, MaaT Pharma has captured 25% of the addressable market on a yearly basis in 2024. Overall, this position reflects the increasing recognition of Xervyteg® as a valuable treatment option for GI-aGvHD patients. aGvHD is the most severe complication of allogeneic stem cell transplantation, a standard-of-care treatment with curative intent offered to patients with blood cancers and some non-malignant hematological conditions. aGvHD refractoriness to current treatments is frequently encountered and severely impacts prognosis. In particular, patients with aGvHD failing both steroid and ruxolitinib typically exhibit a dismal prognosis, with a median survival of 28 days and 85% mortality at one year (Abedin et al 2021). Currently, no therapy is approved for third-line aGvHD, underscoring the urgent need for innovative therapies capable of improving survival and quality of life. 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. Notably, the study met its primary endpoint, achieving a gastrointestinal overall response rate (GI-ORR) of 62% at Day 28, significantly exceeding the expected 38% response rate, and an overall response rate across all organs of 64% at Day 28. Among responding patients at Day 28, the majority exhibited full resolution of GvHD clinical manifestations (i.e., complete response), an important finding predictive of durable control of aGvHD clinical manifestations over time. The 12-month probability of survival was 54% (vs 15% Abedin et al, 2021). Importantly, patients who exhibited gastrointestinal response at Day 28 had a significantly better probability of survival than non-responders (67% vs 28% respectively, p <0.0001), indicating that Xervyteg®-mediated aGvHD control is associated with a remarkable survival benefit. Additional secondary endpoints, including overall survival, will become available in late H2 2025. The Company also integrated supporting safety and efficacy data from 186 aGvHD patients2 treated under its ongoing EAP, which aligns with the positive topline results of the ARES trial and further supports Xervyteg®'s strong efficacy and favorable safety profile in aGvHD. The safety and tolerability of Xervyteg® has been monitored by an independent Data Safety Monitoring Board (DSMB). In March 2025, the DSMB reviewed the overall safety of the trial (after all patients completed Day 28 visit or were discontinued earlier) and confirmed that "given the remarkable efficacy results, the study results show an acceptable safety profile and a favourable benefit /risk ratio". The DSMB members will continue to review safety on an ongoing basis until the 1-year follow-up. The EMA will review the application under the centralized marketing authorization procedure and potentially a marketing authorization could be granted in H2 2026. This centralized procedure means that a single marketing authorization application can be submitted to the EU, and if granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway. About acute Graft-versus-Host Disease Acute Graft-versus-Host Disease occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant, where the transplanted cells initiate an immune response and attack the transplant recipient's organs, causing inflammation of the skin, liver and/or gastro-intestinal tract and leading to significant morbidity and mortality. GI involvement is associated with severe complications such as profound diarrhea, abdominal pain, intestinal bleeding, and death. These complications are often life-threatening, with increased mortality risk, due to the challenges of managing severe GI inflammation and the associated risks of infection, malnutrition, and organ failure. The standard first line therapy for treating aGvHD is the use of systemic steroids. If patients do not respond to steroids, they are considered Steroid Resistant (SR) and other agents can be administered. Currently the only agent approved for treating SR aGvHD after failure of steroid treatment is ruxolitinib, which is currently approved for this indication in USA and has received approval from the European Medical Agency's Committee for Human Medicinal Products (CHMP) on March 25, 2022. About Xervyteg® (MaaT013) MaaT Pharma's Microbiome Ecosystem Therapies (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases. Xervyteg® (MaaT013) is a full-ecosystem, off-the-shelf, standardized, pooled-donors, enema Microbiome Ecosystem TherapyTM for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of ButycoreTM (a group of bacterial species known to produce anti-inflammatory metabolites). Xervyteg® (MaaT013) aims to restore the symbiotic relationship between the patient's functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)-aGvHD. Xervyteg® (MaaT013) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking Statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as "target," "believe," "expect," "aim", "intend," "may," "anticipate," "estimate," "plan," "project," "will," "can have," "likely," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. 1 Updated data from the Early Access Program will be presented at the EHA Annual Congress in Milan, June 12–16, 20252 The cutoff date was October 3, 2024 View source version on Contacts MaaT Pharma – Investor RelationsGuilhaume DEBROAS, of Investor Relations+33 6 16 48 92 50invest@ Rx Communications Group –U.S. Investor RelationsMichael MillerManaging Director+1-917-633-6086mmiller@ MaaT Pharma – Media RelationsPauline RICHAUDSenior PR & Corporate Communications Manager+33 6 14 06 45 92media@ Catalytic Agency –U.S. Media RelationsHeather SheaMedia relations for MaaT Pharma+1 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
02-06-2025
- Business
- Business Wire
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
LYON, France--(BUSINESS WIRE)--Regulatory News: 'Submitting our MAA to the EMA marks a major regulatory milestone for MaaT Pharma and a meaningful advancement for patients with refractory aGvHD—a life-threatening complication of stem cell transplantation with no approved therapies" Share MaaT Pharma (EURONEXT: MAAT – the 'Company'), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem Therapies TM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today 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. 'Submitting our MAA to the EMA marks a major regulatory milestone for MaaT Pharma and a meaningful advancement for patients with refractory aGvHD—a life-threatening complication of stem cell transplantation with no approved therapies,' said Hervé Affagard, Co-founder and CEO of MaaT Pharma. 'We are now closer to providing a much-needed treatment option and remain deeply committed to advancing immunomodulating microbiota technologies in hemato-oncology, where new solutions are urgently needed.' While advancing toward the commercialization of Xervyteg ® (if approved) in Europe, MaaT Pharma is also actively exploring strategic partnerships to ensure broad access in timely fashion. The Company has active discussions with experienced partners who share its mission of delivering meaningful advancements to patients. In parallel to the MAA submission, MaaT Pharma continues to provide access to Xervyteg ® in Europe and the U.S. through its Early Access Program (EAP) 1 for patients with aGvHD and other indications. In 2024, physician demand for Xervyteg ® under the EAP (n=107) increased by 75% compared to 2023, driven by growing adoption across Europe and in the U.S. In France where the EAP first started, MaaT Pharma has captured 25% of the addressable market on a yearly basis in 2024. Overall, this position reflects the increasing recognition of Xervyteg ® as a valuable treatment option for GI-aGvHD patients. aGvHD is the most severe complication of allogeneic stem cell transplantation, a standard-of-care treatment with curative intent offered to patients with blood cancers and some non-malignant hematological conditions. aGvHD refractoriness to current treatments is frequently encountered and severely impacts prognosis. In particular, patients with aGvHD failing both steroid and ruxolitinib typically exhibit a dismal prognosis, with a median survival of 28 days and 85% mortality at one year (Abedin et al 2021). Currently, no therapy is approved for third-line aGvHD, underscoring the urgent need for innovative therapies capable of improving survival and quality of life. 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. Notably, the study met its primary endpoint, achieving a gastrointestinal overall response rate (GI-ORR) of 62% at Day 28, significantly exceeding the expected 38% response rate, and an overall response rate across all organs of 64% at Day 28. Among responding patients at Day 28, the majority exhibited full resolution of GvHD clinical manifestations (i.e., complete response), an important finding predictive of durable control of aGvHD clinical manifestations over time. The 12-month probability of survival was 54% (vs 15% Abedin et al, 2021). Importantly, patients who exhibited gastrointestinal response at Day 28 had a significantly better probability of survival than non-responders (67% vs 28% respectively, p <0.0001), indicating that Xervyteg ® -mediated aGvHD control is associated with a remarkable survival benefit. Additional secondary endpoints, including overall survival, will become available in late H2 2025. The Company also integrated supporting safety and efficacy data from 186 aGvHD patients 2 treated under its ongoing EAP, which aligns with the positive topline results of the ARES trial and further supports Xervyteg ® 's strong efficacy and favorable safety profile in aGvHD. The safety and tolerability of Xervyteg ® has been monitored by an independent Data Safety Monitoring Board (DSMB). In March 2025, the DSMB reviewed the overall safety of the trial (after all patients completed Day 28 visit or were discontinued earlier) and confirmed that 'given the remarkable efficacy results, the study results show an acceptable safety profile and a favourable benefit /risk ratio'. The DSMB members will continue to review safety on an ongoing basis until the 1-year follow-up. The EMA will review the application under the centralized marketing authorization procedure and potentially a marketing authorization could be granted in H2 2026. This centralized procedure means that a single marketing authorization application can be submitted to the EU, and if granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway. About acute Graft-versus-Host Disease Acute Graft-versus-Host Disease occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant, where the transplanted cells initiate an immune response and attack the transplant recipient's organs, causing inflammation of the skin, liver and/or gastro-intestinal tract and leading to significant morbidity and mortality. GI involvement is associated with severe complications such as profound diarrhea, abdominal pain, intestinal bleeding, and death. These complications are often life-threatening, with increased mortality risk, due to the challenges of managing severe GI inflammation and the associated risks of infection, malnutrition, and organ failure. The standard first line therapy for treating aGvHD is the use of systemic steroids. If patients do not respond to steroids, they are considered Steroid Resistant (SR) and other agents can be administered. Currently the only agent approved for treating SR aGvHD after failure of steroid treatment is ruxolitinib, which is currently approved for this indication in USA and has received approval from the European Medical Agency's Committee for Human Medicinal Products (CHMP) on March 25, 2022. About Xervyteg ® (MaaT013) MaaT Pharma's Microbiome Ecosystem Therapies (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases. Xervyteg ® (MaaT013) is a full-ecosystem, off-the-shelf, standardized, pooled-donors, enema Microbiome Ecosystem Therapy TM for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of Butycore TM (a group of bacterial species known to produce anti-inflammatory metabolites). Xervyteg ® (MaaT013) aims to restore the symbiotic relationship between the patient's functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)-aGvHD. Xervyteg ® (MaaT013) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking Statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as 'target,' 'believe,' 'expect,' 'aim', 'intend,' 'may,' 'anticipate,' 'estimate,' 'plan,' 'project,' 'will,' 'can have,' 'likely,' 'should,' 'would,' 'could' and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. presented at the EHA Annual Congress 2 The cutoff date was October 3, 2024
