Latest news with #GianfrancoPittari
Business Wire
13-06-2025
- 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
Business Wire
12-05-2025
- Health
- Business Wire
MaaT Pharma Announces Promising Final Data Readout for Phase 1b Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS)
BUSINESS WIRE)--Regulatory News: These encouraging findings from the IASO Phase 1b trial confirm the favorable safety and tolerability profile of MaaT033 in ALS patients. They also highlight the therapeutic potential of microbiome modulation beyond oncology. 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, announced additional findings following full data readout for the exploratory single-arm, open-label Phase 1b clinical trial named IASO (NCT05889572) evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS). , the Company announced that the trial had met its primary endpoint assessing the safety and tolerability of MaaT033 with multiple doses, following the independent Data Safety and Monitoring Board (DSMB) conclusion. As a reminder, the exploratory Phase 1b trial enrolled a total of 15 participants across two centers in France Hôpital de la Pitié-Salpêtrière – AP-HP and the University Hospital of Lille. An external Scientific Advisory Committee took place at the end of March 2025 to review the full data. Below are the key takeaways from the Committee's review: MaaT033 demonstrated a favorable safety and tolerability profile, supported by biomarker and microbiome analyses. A rapid and sustained engraftment of bacterial species from MaaT033 was observed, mostly occurring within the first month and maintained during the 1-month follow-up period. A slower rate of disease progression (based on ALSFRS-R slopes) was noted to be interpreted with caution given the short follow up, limited sample size and single-arm Phase 1b design. The ALSFRS-R is a standard functional scale in ALS trials to track the progression of the disease. The slope reflects how many points are 'lost' (=disease progression) per month. In the final data readout for the IASO trial, the following was observed for the ALSFRS-R Total score slope: From first symptoms to baseline, the median slope was –0.7 points/month (range: –1.2 to –0.3) From baseline to Day 84 (D84), the median slope slowed to –0.3 points/month (range: –2.4 to +1.0) No variation at D84 in the levels of neurofilaments, a marker associated with neuronal injury in ALS. In addition, the Scientific Advisory Committee provided insights regarding the best population to target in a Phase 2 trial. 'These encouraging findings from the IASO Phase 1b trial confirm the favorable safety and tolerability profile of MaaT033 in ALS patients. They also highlight the therapeutic potential of microbiome modulation beyond oncology and open new avenues for development in neurodegenerative diseases, as evidence continues to grow around the gut-brain connection," said Gianfranco Pittari, MD, PhD, Chief Medical Officer of MaaT Pharma. The study was conducted in close collaboration with the French patient association Tous en Selles contre la SLA (TECS), highlighting the essential role of patients and their advocates in advancing science, and with the support of experts from the French academic networks FILSLAN and ACT4ALS-MND. 'Recognizing the significant unmet medical need in ALS and with the will to support those affected, we applied our discovery platform to this new disease area, demonstrating its potential beyond our primary focus in oncology with encouraging Phase 1 data. In light of the Company's strict financial discipline, and considering the recent successes achieved in oncology, we are actively seeking partners with a focus on ALS and the financial capacity to support new options to fight this disease," stated Hervé Affagard, CEO and co-founder of MaaT Pharma. –-- About MaaT033 MaaT033, a standardized, donor-derived, high-richness, high-diversity oral Microbiome Ecosystem Therapy TM containing anti-inflammatory Butycore TM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. MaaT033 is developed with the "pooling" technology, which allows pooling donations from multiple donors to create a standardized product with high microbial richness and diversity. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA). About Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease in the US and Charcot's disease in Europe, is a progressive neurodegenerative disorder affecting motor neurons in the brain and spinal cord. This leads to muscle weakness, loss of voluntary movement, and eventually, paralysis and on an average lead to death in 3 to 5 years. ALS could affect up to 60,000 patients in US and EU by 2040 and has currently no curative treatment and few symptomatic treatments. 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.
Yahoo
08-04-2025
- Business
- Yahoo
MaaT Pharma Announces Positive Safety Interim Analysis from DSMB for Phase 2b Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT
Following a milestone unblinded interim safety review, the independent Data Safety Monitoring Board (DSMB) has recommended that the study proceed without modification. MaaT033, a pooled donor-derived oral drug candidate developed for ambulatory use, continues to demonstrate a favorable safety profile and tolerability. LYON, France, April 08, 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 positive outcome of a key DSMB safety interim analysis for the Phase 2b trial PHOEBUS, the world's largest randomized controlled trial evaluating microbiome therapy in oncology to date. The study compares the efficacy and safety of MaaT033 (experimental arm) to placebo in patients undergoing an Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT). Early after allo-HSCT, patients are highly vulnerable and face a significant risk of non-relapse mortality. Therefore, the study protocol includes a specific safety analysis that would trigger a stopping rule in case a pre-defined mortality excess in the experimental arm would be identified after 30 patients have been randomized to receive MaaT033 (approximately 60 enrolled in the study) and monitored for 90 days after allo-HSCT. This analysis is distinct from the ongoing safety assessments conducted every six months, whose positive outcomes have last been communicated on January 21, 2025. As a result of their unblinded analysis, the DSMB recommended the trial to proceed as planned, showing no excessive mortality related to MaaT033 as of today. This additional positive outcome further reinforces MaaT033's safety profile and supports MaaT033's integration in the allo-HSCT setting without significant risks of severe adverse events. "We are pleased to report that MaaT033's safety profile continues to be positive. The confirmed absence of a pre-specified mortality excess in patients receiving MaaT033 is of critical relevance", said Gianfranco Pittari, MD, PhD, Chief Medical Officer of MaaT Pharma. "These patients would enormously benefit from innovative therapies enhancing hematopoietic stem cell transplantation outcomes while avoiding toxic effects." Patient enrollment is ongoing in France, Germany, Belgium, Spain, Netherlands and the United Kingdom. The Phoebus trial is an international, multi-center, randomized, double-blinded study comparing MaaT033 (a standardized, oral, freeze-dried, multi-donor microbiotherapy) to placebo in patients receiving an allo-HSCT. The trial is expected to enroll 387 patients and is set to be conducted in up to 60 clinical investigational sites (NCT05762211). Building on the demonstrated safety and efficacy profile of MaaT013, the capsule formulation MaaT033, a high-value asset and the second candidate from MaaT Pharma's native ecosystem platform, is designed to reach a larger patient population through its oral administration. By enabling outpatient use, MaaT033 also supports optimized patient care. Next steps: The next DSMB unblinded interim analysis with mortality monitoring is scheduled for Q3 2025 at the 120-patient mark. The routine DSMB review for ongoing safety, conducted every six months, is also expected for Q3 2025. About MaaT033 MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem TherapyTM containing anti-inflammatory ButycoreTM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by 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. View source version on Contacts MaaT Pharma – Investor Relations Guilhaume DEBROAS, of Investor Relations+33 6 16 48 92 50invest@ Rx Communications Group – U.S. Investor Relations Michael MillerManaging Director+1-917-633-6086mmiller@ MaaT Pharma – Media Relations Pauline RICHAUDSenior PR & Corporate Communications Manager+33 6 14 06 45 92media@ Catalytic Agency – U.S. Media Relations Heather SheaMedia relations for MaaT Pharma +1 617-286-2013 Sign in to access your portfolio
Yahoo
11-03-2025
- Business
- Yahoo
MaaT Pharma Receives Positive Opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for MaaT013
Positive EMA Pediatric Committee opinion has cleared the investigation clinical plan to evaluate the safety and efficacy of MaaT013 in patients from 6 years old to less than 18 years old with aGvHD Key regulatory milestone showing alignment with EMA expectations for pediatric investigation confirming MaaT013 is on track towards a marketing authorization submission to the EMA in June 2025 MaaT013 has the potential to be the first microbiome-driven therapy approved in Europe LYON, France, March 11, 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, 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). "We are very pleased with the productive dialogue with the EMA Pediatric Committee and the positive PIP opinion. This approval marks a major regulatory milestone towards the submission of our Marketing Authorization dossier with the EMA," said Gianfranco Pittari, MD, PhD, Chief Medical Officer at MaaT Pharma. "Through our Early Access Program, we have already successfully and safely treated two pediatric patients with aGvHD. We are committed to bringing MaaT013 to pediatric patients suffering from aGvHD, who currently have limited options." 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. "With this approval of our Pediatric Investigation Plan, we are now on track to submit our Marketing Authorization dossier in June this year. If approved, the Company could be positioned to generate revenues as soon as late 2026 with MaaT013 in third-line treatment in aGvHD," stated Hervé Affagard co-founder and CEO of MaaT Pharma, "Additionally, the Company will continue to provide the product through its Early Access Program for all patients in need." --- About the Pediatric Committee (PDCO)The Pediatric Committee (PDCO) is the European Medicines Agency's (EMA) scientific committee responsible for activities on medicines for children and to support the development of such medicines in the European Union by providing scientific expertise and defining pediatric needs. The PDCO issues an opinion on PIP as part of the regulatory process and the EMA adopts a final decision based on the PDCO's opinion. About the Pediatric Investigation Plan (PIP)A pediatric investigation plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorization of a medicine for children. As part of the regulatory process for the registration of new medicines in Europe, the EMA requires pharmaceutical companies to provide a PIP detailing their strategy for investigation of the new medicinal product in the pediatric population. An approved PIP is a prerequisite for filing a Marketing Authorization Application (MAA). About acute Graft-versus-Host DiseaseAcute 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 second-line treatment for steroid-refractory acute graft-versus-host disease (SR aGvHD) is ruxolitinib. Recently, remestemcel—L-rknd was approved in December 2024 in the US specifically for use in the paediatric population as a second-line treatment. About MaaT013MaaT Pharma's Microbiome Ecosystem TherapiesTM (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. MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, 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). 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. MaaT013 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). 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. View source version on Contacts MaaT Pharma – Investor Relations Guilhaume DEBROAS, of Investor Relations+33 6 16 48 92 50invest@ Rx Communications Group – U.S. Investor Relations Michael MillerManaging Director+1-917-633-6086mmiller@ MaaT Pharma – Media Relations Pauline RICHAUDSenior PR & Corporate Communications Manager+33 6 14 06 45 92media@ Catalytic Agency – U.S. Media Relations Heather SheaMedia relations for MaaT Pharma+1 Sign in to access your portfolio
