Latest news with #HervéAffagard
Business Wire
02-07-2025
- Business
- Business Wire
MaaT Pharma Announces Exclusive Commercialization Partnership With Clinigen for Xervyteg ® in acute Graft-versus Host Disease in Europe
LYON, France--(BUSINESS WIRE)--Regulatory News: Clinigen's hemato-oncology expertise and leading European position in hospital distribution and market access make this team the ideal fit to bring this therapy to patients. 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 announces the signature of a license and commercial agreement with Clinigen, a global specialty pharmaceutical services group and a leading European player in hospital distribution and market access, to streamline the pathway for ensuring access to this medicine across Europe 1. With this partnership, MaaT Pharma demonstrates its capability to supply products to pharmaceutical companies, including those specializing in rare diseases while ensuring scale-ups for commercial and extending its cash runway into January 2026. Hervé Affagard, CEO and co-founder of MaaT Pharma says: 'This deal is a pivotal step in bridging MaaT Pharma's innovation with healthcare professionals who care for patients with aGvHD. Clinigen's hemato-oncology expertise and leading European position in hospital distribution and market access make this team the ideal fit to bring this therapy to patients and we're confident that this new relationship will maximize the full revenue generation potential of full revenue generation potential of Xervyteg ® (MaaT013). I look forward to working closely with the Clinigen team as we prepare for a successful launch.' Jerome Charton, CEO of Clinigen, says: ' Following the EMA's acceptance of MaaT Pharma's submission of an application for assessment for MaaT013 in June, we are very excited about this new relationship. This collaboration brings a novel technology to the forefront of rare disease and oncology care. We're proud to play a leading role in ensuring access across Europe to this innovative therapy, and we look forward to working closely with MaaT Pharma as we prepare for potential launch.' Transaction Terms Under the terms of the agreement, MaaT Pharma will grant Clinigen exclusive European rights to distribute this medicine for the treatment of patients with aGvHD, if approved by the EMA. MaaT Pharma will receive an upfront payment of €10.5 million and additional eligible payments of up to €18 million depending on the achievement of pre-specified regulatory and sales milestones. MaaT Pharma will also be eligible to receive royalty payments on net sales of a percentage in the mid-thirties and regular cash flow as per the supply agreement. The hematology community has expressed interest in this medicine and this class of medicines to treat patients with aGvHD, as evidenced by the growing requests of Early Access Program between 2023 and 2024 (+75%). This program has been active in Europe since 2019. Under the terms of the agreement, Clinigen will take over this activity to meet the growing expectations of physicians while allowing MaaT Pharma to optimize its internal resources. This transition enhances MaaT Pharma's capacity to focus on clinical development, regulatory milestones, and industrial scale-up. MaaT Pharma management will host a conference call and webcast tomorrow Thursday, July 3 nd, 2025, at 3:00pm CET/ 9:00am EDT/ 6:00am PT/ 9:00pm CST. To register, please click here. Participants can also attend the conference by phone by dialing the following number: +33 1 78 42 94 76 + and using the PIN code 43 92 58 1 Includes the following countries: European Union, Iceland, Norway, Liechtenstein and the United Kingdom About Clinigen Clinigen is a global, specialist pharmaceutical services company focused on providing ethical access to medicines. Its mission is to accelerate access to medicines for patients in every corner of the globe. The Group supports pharmaceutical and biotech companies across the medical product lifecycle, from clinical through to commercial and operates from sites in North America, Europe, Africa, and the Asia Pacific. Clinigen has more than 1,100 employees across five continents in 15 countries and provides access in more than 130 countries every year. For more information on Clinigen, please visit 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. 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 Medicines Agency's Committee for Human Medicinal Products (CHMP) on March 25, 2022. About MaaT013 (Xervteg®) 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. MaaT013 (Xervyteg ®) 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). MaaT013 (Xervyteg ®) 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 (Xervyteg ®) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Business Wire
19-06-2025
- Business
- Business Wire
MaaT Pharma Provides a Business Update and Highlights Key Milestones Expected in 2025
LYON, France--(BUSINESS WIRE)--Regulatory News: Following the submission of the Marketing Authorization Application to the EMA for our lead asset, we are excited to advance Xervyteg® toward commercialization — a potential world first for a microbiota therapeutic in oncology. 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 provides a business update and highlights its key milestones expected for the second half of 2025. 'Following the submission of the Marketing Authorization Application to the EMA for our lead asset, Xervyteg ®, earlier this month, we are excited to advance Xervyteg ® toward commercialization — a potential world first for a microbiota therapeutic in oncology — and are now fully focused on progressing registration activities across Europe', said Hervé Affagard, CEO and co-founder of MaaT Pharma. 'This marks a major step in confirming our commitment to address high unmet medical needs and, importantly, it serves as a stepping stone toward international expansion, as we aim to bring our therapies to patients worldwide" Pipeline highlights In Hemato-Oncology Acute Graft-versus-Host Disease (aGvHD) – Xervyteg ® (MaaT013) In January 2025, the Company announced positive topline results from the pivotal Phase 3 ARES Study evaluating Xervyteg ® (MaaT013) in aGvHD. The study met its primary endpoint with a significant gastrointestinal overall response rate at Day 28 of 62% and demonstrates the unprecedented efficacy of Xervyteg ® as third-line treatment of aGvHD with gastrointestinal involvement (GI-aGvHD) consistent with communicated Early Access Program results. On June 02, 2025, the Company announced the submission of a 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, the first one in hemato-oncology worldwide and the first approved therapy in third-line GI-aGvHD. On June 13, 2025, the Company presented positive updated data in Early Access Program for 173 patients at the 2025 annual EHA Congress supporting the high efficacy and good safety profile of Xervyteg ®. This dataset confirms the breakthrough potential of Xervyteg ® for aGvHD patients with limited treatment options. Final results from the pivotal ARES study, including 12-month overall survival data, are expected before the end of 2025 and will be incorporated into the filing dossier. The potential marketing authorization could be delivered around mid-2026, enabling the start of the commercialization of Xervyteg ® in Europe. MaaT Pharma is advancing discussions with potential partners to accelerate the commercialization plan across Europe. MaaT Pharma primarily focuses on the commercialization of its most advanced asset with the completion of regulatory steps in Europe, and dedicated preparation activities for the European launch of Xervyteg ®. In parallel, the Company continues discussions with the FDA to optimize a dedicated pivotal study in the U.S., with the objective of enabling the earliest possible access to Xervyteg ® for U.S. patients. Such a study could be initiated in 2026 (instead of Q4 2025), subject to regulatory confirmation as MaaT Pharma continues watching the evolving regulatory policies and process in the United States. The Company continues the ongoing Early Access Program in the United States, initiated in December 2024. Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) - MaaT033 Over the past 12 months, three DSMB safety assessments were conducted for MaaT033 in the Phase 2b PHOEBUS randomized trial designed to be pivotal: two routine evaluations and one interim analysis focused on excess mortality. All confirmed a favorable safety profile and recommended continuation of the trial without modifications. The last patient enrollment in the trial is anticipated for mid-2026 while the 1-year OS results are expected in H2 2027. In Immuno-Oncology Xervyteg ® and MaaT033 – Proof-of-Concept trials using the MET-N platform (donor derived conducted as Investigator-Sponsored Trials (ISTs). In March 2024, the Company completed patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP in Paris and in collaboration with INRAE and Institut Gustave Roussy, evaluating Xervyteg ® in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy ®) and nivolumab (Opdivo ®), in metastatic melanoma patients. The Company provided its Xervyteg ® 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 is overseeing data collection and analysis. Data readout is expected in H2 2025 as previously announced. In May 2024, the Company announced its participation in the IMMUNOLIFE 'RHU' (university hospital trial) program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. MaaT033 will be tested as a concomitant treatment to cemiplimab (Regeneron), an anti-PD1 therapy, to assess the potential increase in response rate in patients having received antibiotics. This investigator-sponsored, randomized, multicenter Phase 2 trial will evaluate MaaT033 in patients with advanced non-small cell lung cancer (NSCLC), with MaaT Pharma supplying the investigational product. The trial is expected by the sponsor to start mid-2025. MaaT034 – Next-generation drug candidates with co-cultured technology (MET-C platform) In April 2025, the Company presented new preclinical data for MaaT034, its next generation product, showing compelling anti-tumor efficacy results in germ-free mice at the American Association for Cancer Research (AACR) Annual Meeting 2025. Key results included: Metagenomic analysis shows that MaaT034 reproduces the microbial functions of Xervyteg ® MaaT034 improves DC-mediated T cell activation and potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade in vitro. 70% of MaaT034 microbial species engraft in mice, ensuring an enduring presence of beneficial bacteria in the gut environment. MaaT034 increases the production of key microbial-derived metabolites such as short-chain fatty acids in germ-free mice. This translates into an improved gastrointestinal physiology as evidenced by gut mucosal restoration. MaaT034 optimizes anti-PD1 mediated activity in tumor-bearing, germ-free mice. While anti-PD1 alone reduced tumor growth by 10%, the combination of anti-PD1 and MaaT034 resulted in a 83.7% tumor growth reduction (compared to a 24.2% reduction when using a single strain of Akkermansia muciniphila bacteria). In Neurodegenerative Diseases In May 2025, MaaT Pharma announced positive final Phase 1b results for MaaT033 in Amyotrophic Lateral Sclerosis (ALS), showing a favorable safety and tolerability profile supported by biomarker and microbiome analyses. Moving forward, the Company is seeking a partner to further advance clinical evaluation in ALS. As a reminder, the Company's Annual General Meeting will take place on Friday, June 20, 2025, at 9:30am CET at the Company's headquarter in France located at 70 avenue Tony Garnier, 69007 Lyon and will also be broadcasted live. A presentation by the management team on recent developments and perspectives will take place from 9:00 to 9:30 a.m. CET, prior to the General Meeting. For more information, please visit the investors section on the Company's website. --- 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
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
Business Wire
13-05-2025
- Business
- Business Wire
MaaT Pharma Provides Business Update and Reports Financial Results for the First Quarter 2025
LYON, France--(BUSINESS WIRE)--Regulatory News: The first months of 2025 have marked a pivotal moment for MaaT Pharma with the positive results from our Phase 3 trial of MaaT013, our lead asset. 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 provided a business update and reported its cash position as of March 31, 2025. 'The first months of 2025 have marked a pivotal moment for MaaT Pharma with the positive results from our Phase 3 trial of MaaT013, our lead asset. This milestone enables us to advance toward a Market Authorization submission in Europe and reflects over a decade of dedication and close collaboration with physicians. We are deeply grateful for the trust and participation of patients, whose support has been essential in achieving this progress,' said Hervé Affagard, CEO and co-founder of MaaT Pharma. 'We are also making strong progress across our pipeline, with encouraging outcomes for MaaT033 and MaaT034. Additionally, by extending our cash runway to October 2025, we are well-positioned to deliver on our upcoming value-creating milestones.' Pipeline highlights In Hemato-Oncology Acute Graft-versus-Host Disease (aGvHD) – MaaT013 In January 2025, the Company announced positive topline results from the pivotal Phase 3 ARES Study evaluating MaaT013 in aGvHD. The study met its primary endpoint with a significant gastrointestinal overall response rate at Day 28 of 62% and demonstrates the unprecedented efficacy of MaaT013 as third-line treatment of aGvHD with gastrointestinal involvement (GI-aGvHD) consistent with previously communicated EAP results. The Company anticipates MAA submission in Europe in June 2025. In March 2025, the Company received positive opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for MaaT013, a key milestone achieved towards the MAA submission to the EMA. In March 2025, the Company received a positive outcome from the final DSMB meeting on ARES Phase 3 trial, confirming the remarkable efficacy results and positive risk/benefit profile of MaaT013 in third-line aGvHD. Earlier this year, the Company announced its intent to partner the distribution of MaaT013 in Europe, while retaining MaaT013 rights in the U.S. Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) - MaaT033 In January 2025, the Company announced that the DSMB completed its second safety assessment of the Phase 2b trial PHOEBUS and recommended continuation of the trial without modification. In April 2025, the Company announced the positive outcome of a key DSMB safety interim analysis for the Phase 2b trial PHOEBUS. 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. In Immuno-Oncology MaaT034 - Next-generation drug candidates with co-cultured technology (MET-C platform) In April 2025, MaaT Pharma presented promising preclinical data for MaaT034 at the American Association for Cancer Research (AACR) Annual Meeting 2025, demonstrating strong anti-tumor efficacy and immune activation in germ-free mice. MaaT013– Proof-of-Concept trials with donor derived drugs (MET-N platform) 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. In Neurodegenerative Diseases Amyotrophic Lateral Sclerosis (ALS) - MaaT033 In May 2025, MaaT Pharma announced positive final Phase 1b results for MaaT033 in ALS, showing a favorable safety and tolerability profile supported by biomarker and microbiome analyses. Rapid and sustained microbial engraftment was observed, along with a slower rate of disease progression (ALSFRS-R slope to be interpreted with caution. The Company is seeking a partner to further advance clinical evaluation in ALS. Corporate update In April 2025, the Company announced the initiation of coverage of its stock by H.C. Wainwright & Co. With a research report named ' In With the Gut and Out With the Bad in GvHD; Initiating at Buy With a €21 PT', H.C. Wainwright & Co initiated a Buy recommendation and a Target Price of €21. Cash position 1 As of March 31, 2025, total cash and cash equivalents were EUR 24.4 million, as compared to EUR 20.2 million as of December 31, 2024. The net increase in cash position of EUR 4.2 million during the first quarter of 2025 includes the capital increase of €13 million supported by historical shareholders, while investment in R&D activities continued across the pipeline. The Company believes it has sufficient cash to cover its current needs and planned development programs into October 2025 and is exploring several options to further extend its cash horizon. Revenues in Q1 2025 1 MaaT Pharma reported revenues of EUR 1.1 million for the first quarter of 2025 compared with EUR 0.8 million for the same period of 2024 representing a constant growth, quarter to quarter, year to year. Upcoming financial communications* June 20, 2025: Annual General Meeting September 16, 2025: Publication of H1 2025 results November 4, 2025: Publication of revenues & cash for Q3 2025 *Indicative calendar that may be subject to change. Upcoming investor and business conferences participation June 12-15 – European Hematology Association (EHA) Congress, Milan June 16-19, 2025 – Bio International Convention, Boston, MA June 18-19, 2025 – Portzamparc Conference Mid & Small Caps 2025, Paris September 25, 2025 – KBC Healthcare Conference, Brussels --- 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 unaudited financial results
