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3 days ago
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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
14-05-2025
- Business
- Business Wire
MaaT Pharma To Present Updated Data for MaaT013 in Early Access Program at the European Hematology Association (EHA) Annual Congress
LYON, France--(BUSINESS WIRE)--Regulatory News: 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 updated data from its Early Access Program for 173 patients with aGvHD treated with MaaT013 have been selected for oral presentation at the European Hematology Association (EHA) Annual congress, taking place in Milan from June 12–15, 2025. The oral presentation of MaaT013 data at EHA—Europe's leading hematology conference—underlines the growing recognition of the drug's clinical potential and the Company's leadership in hemato-oncology using microbiome-based approach. These updated EAP results for 173 patients, to be presented at EHA Congress, are consistent with the positive topline results of the Phase 3 trial announced in January 2025 and further confirm MaaT013's high efficacy and favorable safety profile in treating patients with gastrointestinal aGvHD. There are currently no approved options for patients with GI-aGvHD who are refractory to steroids and either refractory or intolerant to ruxolitinib, despite the poor prognosis with one-year survival rates of 15% (Abedin et al., 2021). MaaT Pharma has observed a 75% increase in physician demand with MaaT013 under the ongoing EAP in 2024 compared to 2023, across Europe and, more recently, in the United States. This steady demand for access to MaaT013 reflects its growing adoption by the medical community as a treatment option for patients with GI-aGvHD. To date, the Company has safely treated more than 300 patients with aGvHD across clinical trials and the Company's EAP ongoing in both Europe and the U.S. With upcoming regulatory milestones in Europe including a Marketing Authorization submission expected in June 2025, growing global physician interest, and continued clinical validation, MaaT013 has the potential to become the first approved third-line treatment for GI-aGvHD, significantly improving survival outcomes for approximately 3,000 third-line patients annually across the U.S., Canada, and Europe. 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 - 18:15 CEST) - Brown Hall 3 MaaT Pharma will also present its ongoing Phase 2b trial (PHOEBUS) design for MaaT033 developed as an adjunctive therapy 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. To date, the independent Data Safety Monitoring Board (DSMB) has conducted two safety reviews and one unblinded interim analysis, all of which concluded positively with the recommendation that the PHOEBUS trial proceed as planned. 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.
Business Wire
13-05-2025
- Business
- Business Wire
GenSight Biologics Announces Approval of All Resolutions Supported by the Board of Directors at the Combined General Meeting of May 13, 2025
PARIS--(BUSINESS WIRE)--Regulatory News: The Combined General Meeting of shareholders of GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, took place on May 13, 2025, at 2:00 pm CEST at the Company's headquarters, 74 rue du Faubourg Saint-Antoine, 75012 Paris, France. The General Meeting was chaired by Laurence Rodriguez, Chief Executive Officer of the Company. All resolutions recommended by the Board of Directors and submitted to the Combined General Meeting were adopted, with the exception of Resolution A, which was rejected, in accordance with the recommendations of the Board. The quorum on first convening amounted to 56.51%. During the General Meeting, the Chief Executive Officer reviewed the key achievements of 2024, a year marked by careful cash management, strengthened financial structure with the participation of new investors and renewed support from historical shareholders, as well as the renegotiation of the Company's financial obligations. She also highlighted progress in manufacturing, including the optimization of GenSight's technology, preparation for technology transfer to a new manufacturing partner, and the successful mixing and pharmaceutical release of two active substance batches. On the regulatory and clinical fronts, the year saw the preparation of the Phase III RECOVER protocol, the planned submission of the Early Access Program (AAC) application in November 2024, and the continuation of strong scientific communication through international congresses and high-impact journal publications. Looking ahead to 2025, the Company will maintain strict spending discipline while actively pursuing refinancing opportunities. Strategic priorities include the finalization of the Phase III RECOVER protocol in alignment with health authorities, the submission of the LUMEVOQ ® registration dossier to the UK's Medicines and Healthcare products Regulatory Agency (MHRA), and the resumption of the Early Access Program in France through continued engagement with the French agency ANSM. On the manufacturing side, efforts will focus on successfully completing the technology transfer initiated at the end of 2024, optimizing LUMEVOQ ® production yields, and finalizing the quality control plan. In parallel, the Company will continue to explore strategic options through discussions already underway. The results of the vote by resolution and the videocast of the Annual General Meeting will be available on the Company's website in the Investors section ( About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
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
