Latest news with #PediatricInvestigationPlan
Yahoo
15-05-2025
- Health
- Yahoo
Positive EMA Opinion on Pediatric Investigation Plan for PolTREG's Treg Therapy in Type 1 Diabetes
Positive EMA Opinion on Pediatric Investigation Plan for PolTREG's Treg Therapy in Type 1 Diabetes The Paediatric Committee (PDCO) of the European Medicines Agency issues positive opinion on PolTREG's Pediatric Investigation Plan (PIP) for PTG-007 in pre-symptomatic type 1 diabetes (Stage 1). Positive opinion paves the way for potential marketing authorization in the EU and EEA. Recommendation to expand pediatric indication to ages 3–18 years (originally 6–16 years). In vivo data in murine models confirm preliminary safety and efficacy of CAR-TREG therapy, supporting upcoming Phase 1 clinical trials in multiple sclerosis and amyotrophic lateral sclerosis. Gdańsk, Poland – 15 May 2025 – PolTREG S.A. (Warsaw Stock Exchange: PTG) , a clinical-stage biotechnology company developing cellular therapies for a range of autoimmune diseases, announces that the Paediatric Committee (PDCO) of the European Medicines Agency has issued a positive opinion on the Pediatric Investigation Plan (PIP) for its investigational somatic cell therapy product, polyclonal Treg lymphocytes (PTG-007), aimed at preventing symptomatic type 1 diabetes in children. 'Securing a positive opinion from the PDCO brings PolTREG one step closer to the potential approval of PTG-007 for pediatric use across the European Union and the European Economic Area. Achieving the PIP-defined clinical endpoints may serve as the basis for obtaining marketing authorization. Pre-symptomatic type 1 diabetes (Stage 1) represents a significant unmet medical need, making this therapeutic area highly attractive,' said Prof. Piotr Trzonkowski, CEO of PolTREG. 'We are also delighted by the progress in developing next-generation Tregs. The in vivo murine results obtained a few days ago using CAR-TREG lymphocytes shows the preliminary safety and efficacy of our therapy. In the coming months, we will intensively prepare to initiate a Phase 1 clinical trial in multiple sclerosis and amyotrophic lateral sclerosis,' said Prof. Piotr Trzonkowski. The PDCO's positive opinion is based on PolTREG's original preTreg clinical trial protocol initiated in October 2024, which enrolled children aged 6–16 years in Stage 1 type 1 diabetes. In its assessment, the committee has recommended broadening the eligible population to include patients aged 3–18 years. PolTREG combines over 12 years of clinical data confirming the safety and efficacy of polyclonal Treg therapies with a broad portfolio of next-generation technologies, including allogeneic Tregs, CAR-TREGs, antigen-specific Tregs, and TCR-TREGs. PolTREG is the only company with 12 years' worth of patient-safety and efficacy data for autologous polyclonal Treg therapies—data derived from clinical trials and hospital exemptions in Poland. To read more about the clinical trials PolTREG has completed, please click on: In parallel, PolTREG has achieved a major milestone toward its CAR-TREG program, in collaboration with AZTherapies. In vivo studies in murine models demonstrate encouraging preliminary safety and efficacy for CAR-engineered Treg lymphocytes, supporting an application for a Phase 1 clinical trial in multiple sclerosis and Amyotrophic Lateral Sclerosis in the coming months. This cellular product will be further made allogeneic in the cooperation with Swiss-based company Antion manufactures its Treg therapeutics at its own GMP-certified manufacturing facility. It is the first company in the world to have administered Treg therapies to patients, and, under a hospital exemption valid in Poland, the first company to start receiving revenues from a Treg therapeutic for autoimmune disease. Its GMP manufacturing facility is one of Europe's largest and most advanced, boasting over 2,100 sqm of laboratory space, including 15 production lines. PolTREG has the option to substantially expand the facility to accommodate manufacturing of next-generation engineered therapies and cell therapies. It can ship its wide range of cellular therapy products across Europe within 24 hours. About PolTREG PolTREG is a global leader in developing autoimmune therapies based on T-regulatory cells (Tregs). Its lead product, PTG-007, autologous Treg treatment for early-onset Type-1 Diabetes (T1D) is ready for Phase 2/3 clinical testing, for which the company is seeking a partnership. PolTREG has established a robust platform encompassing a wide range of cell therapy approaches, including polyclonal TREG, CAR-TREG, allogeneic TREG, antigen-specific TREG, and TCR-TREG therapies. For more information please visit For further information please contact:PolTREG Piotr TrzonkowskiChief Executive Officerir@ 512 532 401 Important information The contents of this announcement include statements that are, or may be deemed to be, "forward-looking statements". These forward-looking statements can be identified by the use of forward-looking terminology, including the words "believes", "estimates," "anticipates", "expects", "intends", "may", "will", "plans", "continue", "ongoing", "potential", "predict", "project", "target", "seek" or "should", and include statements the Company makes concerning the intended results of its strategy. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. The company's actual results may differ materially from those predicted by the forward-looking statements. The company undertakes no obligation to publicly update or revise forward-looking statements, except as may be required by law.
Associated Press
14-03-2025
- Business
- Associated Press
Sensorion Reports Full-Year 2024 Results, Provides Corporate Update and Announces Availability of Full-Year Report
Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, today reported its full-year 2024 results, provided a corporate update, and announced the availability of the full-year report. 'This past year has been an exceptional period of progress on clinical and corporate development fronts,' commented Nawal Ouzren, Chief Executive Officer of Sensorion. 'Our portfolio of next generation treatments for hearing loss disorders achieved important development milestones highlighted by the first cohort being enrolled in Audiogene, our first gene therapy clinical trial, and with SENS-401's Phase 2a POC study meeting its primary and secondary endpoints in preserving residual hearing following cochlear implantation. On the operational side, we executed two successful financings totaling over €65 million which enabled us to maintain development pace for the entire pipeline of programs while welcoming some of the healthcare sector's leading institutional investors to the registry. We head into 2025 well positioned to execute on our clinical and corporate growth plan and on behalf of the entire team, I extend gratitude to our longstanding shareholders, to the patients and to the doctors for their continued support as we remain steadfast in bringing to market disruptive treatments for hearing loss disorders.' Pipeline Highlights and Upcoming Milestones Gene Therapies for Hereditary Monogenic Hearing Loss In 2024, Sensorion advanced its portfolio of gene therapies developed in collaboration with the Institut Pasteur. The Company achieved several notable milestones with lead candidate SENS-501, for the treatment of hearing loss caused by otoferlin deficiency. SENS-501: Gene therapy program to restore hearing in OTOF patients Sensorion's SENS-501 dual vector AAV (adeno-associated virus) gene therapy development product aims at restoring hearing in patients with mutations in OTOF gene who suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, with around 20,000 people affected in the US and Europe 1. On January 19, 2024, Sensorion announced the approval to initiate the Phase 1/2 gene therapy clinical trial of SENS-501, Audiogene. The study design consists of two cohorts of two doses followed by an expansion cohort at the selected dose. Safety will be the primary endpoint for the dose escalation cohort, and the auditory brainstem response (ABR) will be the primary efficacy endpoint of the dose expansion cohort. Audiogene will also assess the clinical safety, performance, and usability of the administration device system developed by Sensorion. Additionally, Sensorion received the European Medicines Agency's decision agreeing on a Pediatric Investigation Plan (PIP) for SENS-501, in September 2024. In September 2024, Sensorion reported the injection of the first patient recruited in the Audiogene trial, and, during the symposium it held during the World Congress of Audiology, reported initial safety data of the first patient. On December 27, 2024, Sensorion announced the patient recruitment completion of the first cohort of patients in the Audiogene study, with all first three toddlers and infants having received an injection of the gene therapy product, SENS-501. On February 21, 2025, Sensorion received a positive recommendation from the Data Monitoring Committee of Audiogene, after reviewing the first cohort tolerability and safety data. Sensorion plans on completing the recruitment of the second cohort of patients and on hosting a KOL event to present new data in H1 2025. GJB2-GT: Gene therapy program to restore hearing in GJB2 patients Sensorion's AAV-based GJB2 gene therapy program developed in collaboration with the Institut Pasteur, has the potential to address three pathologies related to GJB2 mutations: pediatric congenital deafness, progressive forms of hearing loss in children, and early onset of presbycusis in adults. The Company provided GJB2-GT Proof-of-Concept data at the European Society of Cell & Gene Therapy (ESGCT), which took place on October 22-25, 2024, Rome, Italy. Sensorion is advancing the candidate into CTA/IND-enabling activities for anticipated submission in Q1 2026. SENS-401, Sensorion's small molecule for the treatment and prevention of hearing loss SENS-401 (Arazasetron) is a small molecule that Sensorion develops in three indications: (i) to treat Sudden Sensorineural Hearing Loss SSNHL (Phase 2b completed), (ii) to preserve residual hearing following cochlear implantation (Phase 2a completed), and (iii) to prevent Cisplatin-Induced Ototoxicity (Phase 2a ongoing). SENS-401 is an orally available small molecule that aims at protecting and preserving inner ear tissue from damage, responsible for hearing impairment. SENS-401 has been granted Orphan Drug Designation in Europe for the treatment of SSNHL, and in the U.S. for the prevention of Cisplatin-Induced Ototoxicity in pediatric population. The Company is conducting strategic business discussions to partner it small molecule, SENS-401. SENS-401 to preserve residual hearing after cochlear implantation Sensorion's Phase 2a Proof of Concept clinical trial of SENS-401 in patients scheduled for a cochlear implantation was a multicentric, randomized, controlled open-label trial aimed at evaluating the presence of SENS-401 in the cochlea (perilymph) after 7 days of twice-daily oral administration in adult patients prior to cochlear implantation due to moderately severe to profound hearing impairment. Patients started treatment with SENS-401 7 days before implantation and continued to receive SENS-401 for a further 42 days. This study was developed in collaboration with Cochlear Limited, the global leader in implantable hearing solutions. On February 1, 2024, Sensorion announced the completion of patient inclusion in the Phase 2a POC clinical trial. On March 11, 2024, Sensorion announced that the primary endpoint of assessing the presence of SENS-401 in the inner ear perilymph at a level sufficient to elicit a therapeutic benefit was met in 100% of the patients sampled, 7 days after the start of the treatment. On September 20, 2024, study investigator Professor Stephen O'Leary, M.D., Ph.D., during a symposium organized by Sensorion at the World Congress of Audiology, and Professor Christophe Vincent in a dedicated session on auditory implants for adults, reported analysis of Sensorion's final data of SENS-401. After 7 weeks of treatment with SENS-401 (and 6 weeks after cochlear implantation), the reduction in residual hearing loss was systematically better at the 3 frequencies 250, 500 & 750Hz in the group treated with SENS-401. This protective effect was maintained 8 weeks after cessation of treatment (14 weeks after cochlear implantation). The results show that patients treated with SENS-401 have 'complete' hearing preservation (40% of patients) compared with the control group (0% of patients) according to the Skarzynski index. In addition, the favorable safety profile of SENS-401 has been validated, in line with previous studies on SENS-401. SENS-401 to prevent Cisplatin-Induced Ototoxicity (CIO) Cisplatin and other platinum-based compounds are essential chemotherapeutic agents in the treatment of many cancers. A serious side effect of these therapies is ototoxicity, permanent and irreversible hearing loss, which occurs in 40 to 60% 2 of adult and pediatric patients treated. This indication represents a significant unmet medical need for patients and constitutes a potential large global market. The Phase 2a Proof-of-Concept (POC) NOTOXIS trial is a multicenter, randomized, controlled, open-label study, designed to evaluate the efficacy of SENS-401 to prevent ototoxicity induced by cisplatin in adult patients with a neoplastic disease 4 weeks after the completion of cisplatin-based chemotherapy. The trial assesses several outcome measures, including the rate and severity of ototoxicity, the change from baseline in Pure Tone Audiometry (PTA) (dB) throughout the study and the tolerance. On July 23, 2024, Sensorion announced a positive recommendation from the Data Safety Monitoring Board (DSMB) regarding the continuation of NOTOXIS. On September 20, 2024, Professor Yann Nguyen reported preliminary safety and efficacy data in Sensorion's NOTOXIS trial, during the World Congress of Audiology. The preliminary data show that a cumulative dose of cisplatin is a key factor of ototoxicity severity. A good safety profile of SENS-401 is confirmed in the long term, with the drug being administered for the first time for an average duration of up to 23 weeks. The preliminary results suggest a trend toward an otoprotective effect of SENS-401 beyond a cisplatin dose of 300 mg/m2. Despite significant exposure to cisplatin in the treatment group, most participants showed only mild ototoxicity. On March 7, 2025, the Company announced the end of patient enrollment in NOTOXIS, its Phase 2a POC clinical trial of SENS-401 in Cisplatin-Induced Ototoxicity. Sensorion plans on reporting the topline data by the end of 2025. Strengthening the Board of Directors and senior leadership On January 25, 2024, Sensorion announced the nomination of Dr. Federico Mingozzi as board member. Dr. Federico Mingozzi has previously worked at Spark Therapeutics, where he served as Chief Science and Technology Officer. Federico brings over 25 years of experience in gene therapy, immunology, as well as biochemistry and molecular biology in academia and industry. He is well known for his significant contributions to the development of gene therapies for the treatment of various diseases. Furthermore, he has played a key role in advancing the understanding of the interactions between gene therapy vectors and the host immune system, as well as in the formulation of strategies to overcome immune responses to anti-AAV vectors. On June 27, 2024, Sensorion appointed Laurene Danon as Chief Financial Officer. Laurene brings to Sensorion more than 15 years of experience in investment banking and international equity capital markets. A graduate of HEC, she began her career in London with the investment bank J.P. Morgan in a corporate finance advisory capacity, before specializing in equity capital markets at J.P. Morgan and later at Jefferies International. Prior to joining Sensorion, she founded the strategic advisory firm Concorde Advisory, where she supported and managed the execution of strategic corporate finance projects for her clients. In total, Laurene has led executions for 70 transactions totalling over $35 billion raised. Expected future milestones and estimated timelines H1 2025 – SENS-501: Enrollment completion of the second cohort of patients in Audiogene trial and KOL event to present new data H2 2025 – SENS-401 in Cisplatin-Induced Ototoxicity: Topline results Q1 2026 – GJB2-GT: Clinical Trial Applications filing Full Year 2024 financial highlights Cash Position Cash & Cash Equivalents, and short-term deposits, amounted to c. €77m as of December 31, 2024, compared to €37.0m as of December 31, 2023. R&D expenses increased by 13 % from €22.8 million in 2023 to €25.7 million in 2024. General And Administrative (G&A) Expenses G&A expenses were €9.4 million for 2024, compared to €5.3 million for 2023. Net Loss Net loss was -€26.0million for 2024, compared to -€22.1 million for 2023. Financial guidance Based on cash and cash equivalents and short-term deposit classified in other current assets of €77.0 million at 31 December 204, the Company has sufficient net working capital to meet its cash requirements until the end of Q1 2026. Financial results The annual accounts as at December 31, 2024, were prepared according to IFRS standards and approved by the Board of Directors on March 13, 2025. The simplified income statement as of December 31, 2024, is as follows: In thousands of Euros – IFRS standards 31.12.2024 31.12.2023 Operating income 6,653 5,698 Research & Development expenses -25,664 -22,755 General & Administrative expenses -9,390 -5,253 Total operating expenses -35,054 -28,009 Operating loss -28,401 -22,310 Financial result 2,555 544 Corporate Income Tax -126 -297 Net loss -25,972 -22,063 The simplified balance sheet as of December 31, 2024, is as follows: In thousands of Euros – IFRS standards 31.12.2024 31.12.2023 Non-current Assets 3,574 3,236 Other Current Assets 18,934 6,292 Of which short term deposit 10 214 Cash & cash equivalent 66,769 36,974 Total Assets 89,277 46,502 Equity 72,138 33,275 Non-current Liabilities 3,486 3,646 Current Liabilities 13,653 9,581 Total Liabilities 89,277 46,502 2024 certified accounts On March 13, 2025, the Board of Directors approved the Company's full year results as of December 31, 2024. The Full Year Report can be found on Sensorion's website ( in the investor section under financial information. The full year accounts of 2024 have been subject to a limited review by the Company's statutory auditors and an unqualified report is being issued. About SENS-501 SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss. The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). This therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. Currently in the clinical research phase, this gene therapy program represents significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness. This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l'Audition. The project is partially financed by the French National Research Agency, through the 'investing for the future' program (ref: ANR-18-RHUS-0007). About the Audiogene Trial Audiogene aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is optimal, the chances of these young children with pre-linguistic hearing loss acquiring normal speech and language are maximized. The study comprises two cohorts of two doses followed by an expansion cohort at the selected dose. While safety will be the primary endpoint of the first part of the dose escalation study, auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the expansion. Audiogene will also evaluate the clinical safety, performance and ease-of-use of the delivery system developed by Sensorion. About SENS-401 SENS-401 (Arazasetron), Sensorion's clinical stage lead drug candidate, is an orally available small molecule that aims to protect and preserve inner ear tissue from damage responsible of progressive or sequelae hearing impairment. Sensorion developed SENS-401 in three Phase 2 clinical trials: (i) for the prevention of Cisplatin-Induced Ototoxicity, (ii) to prevent residual hearing loss in patients scheduled for cochlear implantation, and (iii) to treat sudden sensorineural hearing loss. The last two studies are completed. SENS-401 has been granted Orphan Drug Designation by the EMA in Europe for the treatment of sudden sensorineural hearing loss, and by the FDA in the U.S. for the prevention of platinum-induced ototoxicity in pediatric population. About Sensorion Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion's portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion's small molecule progressed in three Phase 2 proof of concept clinical study: firstly, in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing, for which the recruitment is completed and the follow-up is ongoing. Secondly, with partner Cochlear Limited, a study of SENS-401 for the residual hearing preservation in patients scheduled for cochlear implantation, completed in 2024. Thirdly, a Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in 2022. Label: SENSORION FR0012596468 Mnemonic: ALSEN Disclaimer This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2023 full year report published on March 14, 2024, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith. 1 Rodríguez-Ballesteros M, Reynoso R, Olarte M, Villamar M, Morera C, Santarelli R, Arslan E, Medá C, Curet C, Völter C, Sainz-Quevedo M, Castorina P, Ambrosetti U, Berrettini S, Frei K, Tedín S, Smith J, Cruz Tapia M, Cavallé L, Gelvez N, Primignani P, Gómez-Rosas E, Martín M, Moreno-Pelayo MA, Tamayo M, Moreno-Barral J, Moreno F, del Castillo I. A multicenter study on the prevalence and spectrum of mutations in the otoferlin gene (OTOF) in subjects with nonsyndromic hearing impairment and auditory neuropathy. Hum Mutat. 2008 Jun;29(6):823-31. doi: 10.1002/humu.20708. PMID: 18381613. 2 JCO Oncology practice, ASCO, volume 19, Issue 5/ CIO: a concise review of the burden, prevention and interception strategies, May 2024 Chattaraj. [email protected] Press Relations Bruno Arabian / 00 33(0)6 87 88 47 26 [email protected] Nicolas Entz / 00 33 (0)6 33 67 31 54 [email protected] KEYWORD: EUROPE UNITED STATES NORTH AMERICA FRANCE INDUSTRY KEYWORD: HEALTH GENETICS RESEARCH PHARMACEUTICAL SCIENCE BIOTECHNOLOGY SOURCE: Sensorion Copyright Business Wire 2025. PUB: 03/14/2025 02:30 AM/DISC: 03/14/2025 02:29 AM
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
