Latest news with #MarketingAuthorization
Cision Canada
4 days ago
- Health
- Cision Canada
Zemcelpro® (UM171 Cell Therapy) receives positive CHMP opinion for treatment of blood cancer patients without access to suitable donor cells Français
If approved, Zemcelpro ® is expected to: increase access to donor-derived stem cell transplantation, which offers a potentially curative option for haematologic malignancies, including leukemias and myelodysplastic syndromes be the first and only therapy in the European Union with marketing authorization for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available Approval decision from the European Commission expected within approximately two months MONTREAL, June 19, 2025 /CNW/ - ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, announced today the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending granting conditional marketing authorization for Zemcelpro ® for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. The European Commission (EC) is expected to make a final decision within approximately two months following CHMP recommendation, and the decision will apply to all 27 European Union (EU) Member States, Iceland, Norway and Liechtenstein. Zemcelpro ®, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. If approved, Zemcelpro ® is expected to be the first and only therapy in the EU with marketing authorization for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. Every year in Europe there are over 10,000 new cases of patients with haematological malignancies, including leukemias and myelodysplastic syndromes, requiring bone marrow transplant, and a number of these patients do not have access to suitable donor cells for different reasons, including the absence or unavailability of suitably matched donors. The positive CHMP opinion was based on the conditional Marketing Authorization Application (MAA) for Zemcelpro ®. Additional filings are planned for Zemcelpro ® with other health authorities, including in the US, Canada, the UK, and Switzerland. "Each year, thousands of people in Europe are diagnosed with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation, and it's an upsetting reality that a number of them don't have access to suitable donor-derived stem cells," said Dr. Guy Sauvageau, CSO and Founder of ExCellThera. "With today's positive opinion, we are closer to bringing the life-changing potential of Zemcelpro ® to patients with at-risk haematological malignancies in the EU," said David Millette, CEO of ExCellThera. "We are proud to bring our transformative cell therapy innovation to patients who continue to have unmet medical needs." The safety of Zemcelpro ® is consistent with the well-characterized safety profile of conventional allogeneic blood stem cell transplantation for haematological malignancies following myeloablative conditioning. ExCellThera extends its sincere gratitude to the patients and investigators who have contributed to the clinical development of Zemcelpro ®. About Zemcelpro ® Zemcelpro ®, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. Zemcelpro ®, developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients with haematologic malignancies in clinical trials in the United States, Europe and Canada. Zemcelpro ® has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA. Zemcelpro ® has been tested in Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. A pivotal Phase 3 trial in this patient population will be initiated as soon as possible. The use of Zemcelpro ® in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being explored. The product is not yet approved for marketing by the EMA and remains subject to European Commission decision. Its safety and efficacy have not been established by other regulatory agencies, such as the FDA and Health Canada. About ExCellThera and UM171 Technology ExCellThera is a world leader in enhanced blood stem cell therapies. ExCellThera's proprietary Enhance ™ platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding haematopoietic stem cells (HSCs) from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the Enhance ™ platform, including the proprietary molecule UM171 which has a first-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs. For additional information, visit and follow us on LinkedIn.
Yahoo
23-05-2025
- Business
- Yahoo
Outlook Therapeutics® Announces Pricing of $13.0 Million Public Offering
ISELIN, N.J., May 23, 2025 (GLOBE NEWSWIRE) -- Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company focused on enhancing the standard of care for bevacizumab for the treatment of retina diseases, today announced the pricing of an underwritten public offering of 9,285,714 shares of its common stock, together with accompanying warrants to purchase 18,571,428 shares of its common stock. The combined public offering price of common stock and accompanying warrant is $1.40. The common stock is being sold in combination with an accompanying warrant to purchase two shares of common stock issued for each share of common stock sold. The accompanying warrant has an exercise price of $1.40 per share, will become exercisable immediately and will expire five years from the date of issuance. The offering is expected to close on May 27, 2025, subject to the satisfaction of customary closing conditions. The gross proceeds from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by Outlook Therapeutics are expected to be approximately $13.0 million. Outlook Therapeutics intends to use the net proceeds from the offering for working capital and other general corporate purposes. BTIG, LLC is acting as sole book-running manager for the offering. The securities described above are being offered by Outlook Therapeutics pursuant to a shelf registration statement on Form S-3 (No. 333-278340) that was declared effective by the Securities and Exchange Commission (SEC) on April 5, 2024. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC's website located at Copies of the preliminary prospectus supplement and the final prospectus supplement relating to this offering may be obtained, when available, by contacting: BTIG, LLC, 65 East 55th Street, New York, New York 10022, by telephone at (212) 593-7555 or by email at ProspectusDelivery@ This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction. About Outlook Therapeutics, Therapeutics is a biopharmaceutical company focused on the development and commercialization of ONS-5010/LYTENAVA™ (bevacizumab-vikg; bevacizumab gamma) to enhance the standard of care for bevacizumab for the treatment of retina diseases. LYTENAVA™ (bevacizumab gamma) is the first ophthalmic formulation of bevacizumab to receive European Commission and Medicines and Healthcare products Regulatory Agency Marketing Authorization for the treatment of wet age-related macular degeneration (wet AMD). Outlook Therapeutics is working to initiate its commercial launch of LYTENAVA™ (bevacizumab gamma) in the European Union and the United Kingdom as a treatment for wet AMD, expected in the second quarter of calendar year 2025. In the United States, ONS-5010/LYTENAVA™ is investigational, and a Biologics License Application has been resubmitted to the U.S. Food and Drug Administration. If approved in the United States, ONS-5010/LYTENAVA™, would be the first approved ophthalmic formulation of bevacizumab for use in retinal indications, including wet AMD. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts are 'forward-looking statements,' including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as 'anticipate,' 'believe,' 'continue,' 'expect,' 'may,' 'plan,' 'potential,' 'will,' or 'would' the negative of terms like these or other comparable terminology, and other words or terms of similar meaning. These include, among others, statements regarding the gross proceeds from the offering, the use of proceeds from the offering, the completion of the offering, expectations concerning decisions of regulatory bodies and the timing thereof, plans for commercial launch of LYTENAVA™ in the EU and the UK and the timing thereof and other statements that are not historical fact. Although Outlook Therapeutics believes that it has a reasonable basis for the forward-looking statements contained herein, they are based on current expectations about future events affecting Outlook Therapeutics and are subject to risks, uncertainties and factors relating to its operations and business environment, all of which are difficult to predict and many of which are beyond its control. These risk factors include fluctuations in Outlook Therapeutics' stock price, changes in market conditions and satisfaction of customary closing conditions related to the offering, risks in obtaining necessary regulatory approvals, the content and timing of decisions by regulatory bodies, and the sufficiency of Outlook Therapeutics' resources, as well as those risks detailed in Outlook Therapeutics' filings with the SEC, including the Annual Report on Form 10-K for the fiscal year ended September 30, 2024, filed with the SEC on December 27, 2024, and subsequent filings with the SEC, which include uncertainty of market conditions and future impacts related to macroeconomic factors, including as a result of the ongoing overseas conflicts, tariffs and trade tensions, fluctuations in interest rates and inflation, and potential future bank failures on the global business environment. These risks may cause actual results to differ materially from those expressed or implied by forward-looking statements in this press release. All forward-looking statements included in this press release are expressly qualified in their entirety by the foregoing cautionary statements. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Outlook Therapeutics does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise, except as may be required under applicable securities law. Investor Inquiries:Jenene ThomasChief Executive OfficerJTC Team, LLCT: 908.824.0775OTLK@ Source: Outlook Therapeutics, 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
Yahoo
22-05-2025
- Business
- Yahoo
Soleno Therapeutics Announces Submission and EMA Validation of Marketing Authorization Application for Diazoxide Choline Prolonged-Release Tablets for the Treatment of Hyperphagia in Patients with Prader-Willi Syndrome
REDWOOD CITY, Calif., May 22, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that Soleno's Marketing Authorization Application (MAA) seeking regulatory approval of Diazoxide Choline Prolonged-Release Tablets (previously referred to as DCCR) for the treatment of adults and children four years and older with Prader-Willi syndrome (PWS) who have hyperphagia had been validated by the European Medicines Agency (EMA). 'The validation of our MAA represents the next significant milestone in our mission to deliver this important therapy to the broad PWS community, including those in the EU,' said Anish Bhatnagar, M.D., Chairman and Chief Executive Officer of Soleno Therapeutics. 'Based on the data generated, DCCR has the potential to help treat hyperphagia, which is the most life-limiting aspect of PWS. We look forward to working closely with European regulators during the review process and intend to make DCCR available to patients in the EU as expeditiously as possible, if approved.' Soleno estimates that there are approximately 9,500 patients with PWS in the United Kingdom, France, Germany, Italy and Spain combined1. The Company has been granted Orphan Drug Designation for diazoxide choline in the EU for the treatment of PWS, which could provide Soleno with up to 10 years of market exclusivity in the EU, if approved, in addition to certain other regulatory and financial incentives. DCCR was approved by the U.S. Food and Drug Administration (FDA) under the brand name VYKAT™ XR on March 26, 2025. About PWSPrader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in the gene expression on chromosome 15. The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The defining symptom of PWS is hyperphagia, a chronic and life-threatening condition characterized by an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety, which can severely diminish the quality of life for individuals with PWS and their families. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. U.S. INDICATIONVYKAT XR is indicated in the U.S. for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS). ________________________________ 1 Based on mid-point of Orphanet Birth Prevalence rate of 1 in 15,000 to 30,000 IMPORTANT SAFETY INFORMATION ContraindicationsUse of VYKAT XR is contraindicated in patients who have a known hypersensitivity to diazoxide, other components of VYKAT XR, or to thiazides. Warnings and Precautions HyperglycemiaHyperglycemia, including diabetic ketoacidosis, has been reported. Before initiating VYKAT XR, test fasting plasma glucose (FPG) and HbA1c; optimize blood glucose in patients who have hyperglycemia. During treatment, regularly monitor fasting glucose (FPG or fasting blood glucose) and HbA1c. Monitor fasting glucose more frequently during the first few weeks of treatment in patients with risk factors for hyperglycemia. Risk of Fluid OverloadEdema, including severe reactions associated with fluid overload, has been reported. Monitor for signs or symptoms of edema or fluid overload. VYKAT XR has not been studied in patients with compromised cardiac reserve and should be used with caution in these patients. Adverse ReactionsThe most common adverse reactions (incidence ≥10% and at least 2% greater than placebo) included hypertrichosis, edema, hyperglycemia, and rash. Please see the full Prescribing Information, including Medication Guide. About Soleno Therapeutics, is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company's first commercial product, VYKAT XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome and was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the regulatory approval process for Diazoxide Choline Prolonged-Release Tablets in the EU, the timing of commercialization of Diazoxide Choline Prolonged-Release Tablets in the EU, the potential market opportunity for Diazoxide Choline Prolonged-Release Tablets and the ability of Diazoxide Choline Prolonged-Release Tablets to address the unmet needs of the PWS community. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Corporate Contact:Brian RitchieLifeSci Advisors, LLC212-915-2578 Media Contact:media@ 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
Yahoo
27-03-2025
- Business
- Yahoo
MaaT Pharma Completes a Capital Increase of €13 Million with Historical Shareholders and Announces 2024 Annual Results
Positive results from Phase 3 trial for MaaT013 in acute Graft-versus-Host disease (aGvHD); topline results (January 2025) showed a 62% gastrointestinal overall response rate at Day 28 and 1-year expected Overall Survival of 54%, demonstrating high efficacy and significant improvement over currently available therapies; Positive final DSMB review confirming remarkable efficacy results and a positive benefit/risk profile. Marketing Authorization Application (MAA) in Europe for MaaT013 on track for EMA submission in June 2025. Initiation of Expanded Access for MaaT013 in aGvHD in the U.S. EAP Revenues of €3.2 million in 2024, a 44% increase over 2023, and the highest revenues generated from the EAP to date. As of December 31, 2024, cash and cash equivalents were €20.2 million. Capital Increase of €13 million supported by historical shareholders to advance towards its next significant operational and financing milestones, extending its cash runway into October 2025. LYON, France, March 27, 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 Therapies™ (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today reported the 2024 full-year annual results and announces the completion of a private placement of €13 million at market price through the issuance of 2,131,148 new ordinary shares of the Company at a price per New Share (as defined below) of €6.10, to the benefit of its existing shareholders, Biocodex, PSIM Fund represented by Bpifrance Investissement and a US/EU existing investor. The funds will support the Company's progress towards key value milestones, including market submission for EU approval of MaaT013 in aGvHD, its US expansion strategy, and the conclusion of a potential partnership for its hemato-oncology products in Europe. "I am proud of our team for the significant progresses MaaT Pharma made to date, marked by unprecedented results from our positive Phase 3 trial with our lead asset, MaaT013, supporting our transformational role in hemato-oncology. I'm deeply thankful to our historical shareholders for their renewed support, which will enable the Company to reach major value milestones in the coming months. Looking ahead, we look forward to bringing our innovation to patients in need and creating lasting value for all stakeholders," stated Hervé Affagard, CEO and co-founder of MaaT Pharma. Pipeline highlights In Hemato-Oncology In 2024, MaaT Pharma confirmed its leadership position with microbiome-based therapies for hemato-oncology applications, releasing breakthrough Phase 3 results from its ARES aGvHD trial. The Company is now preparing for the submission of its Market Authorization Approval dossier for its lead-asset MaaT013, while also actively discussing potential partnership options for commercialization in Europe. Acute Graft-versus-Host Disease (aGvHD) – MaaT013 In April 2024, the Company presented for the first time positive 18-month data for MaaT013 showing a clear Overall Survival advantage in aGvHD from the Early Access Program (EAP) at the 2024 EBMT Annual Meeting. Promising data included a gastrointestinal overall response rate (GI-ORR) of 52% at D28 and an Overall Survival (OS) of 47% at 12 months. In September 2024, MaaT Pharma announced that MaaT013 batches were ready for distribution for clinical supply in the US and Europe and that MaaT Pharma advanced the readiness phase for the initiation of clinical activities. In October 2024, MaaT Pharma announced the completion of patient recruitment for ARES, its European Phase 3 clinical trial evaluating the efficacy and safety of MaaT013 in the treatment of steroid refractory and ruxolitinib refractory or intolerant aGvHD (NCT04769895). In December 2024, the Company announced the initiation of the Single Patient Expanded Access in the U.S with the treatment of the first U.S. patient with aGvhD with MaaT013 at City of Hope Hospital, one of the largest and most advanced cancer research and treatment organizations in the United States. In December 2024, MaaT Pharma presented updated data, with a GI-ORR at Day 28 of 51% and an OS rate of 47% at 12 months, for 154 patients with acute Graft-versus-Host Disease (aGvHD) treated with MaaT013 in EAP in Europe during the 66th American Society of Hematology (ASH) Annual Meeting. In January 2025, the Company announces 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. More recently, the Company received positive feedback, further solidifying its plans to submit the MAA dossier with the EMA 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 a marketing authorization submission to the EMA, expected in June 2025. And also in March 2025, the Company received a positive outcome from the final DSMB meeting on ARES, confirming the remarkable efficacy results and positive risk/benefit profile of MaaT013 in third-line aGvHD. Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) - MaaT033 In July 2024, the Company announced that the DSMB completed its first safety assessment of the Phase 2b trial PHOEBUS (NCT05762211), aiming at assessing MaaT033 impact in improving overall survival in patients receiving hematopoietic stem cell transplantation, and recommended continuation of the trial without modification. The trial is a European, multi-center, randomized, double-blind study, testing MaaT033, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and expected to enroll 387 patients. 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 Immuno-Oncology In 2024, MaaT Pharma advanced its donor-derived MET-N drug candidates in immunotherapy combinations for solid tumors in proof-of-concept clinical phases, while developing its next-generation MET-C drugs using the Company's co-cultured platform, in view of addressing large immuno-oncology indications. MaaT013 and MaaT033 – Proof-of-Concept trials with donor derived drugs (MET-N platform) In March 2024, the Company completed patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 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 will contribute 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. Following an updated timeline from the academic sponsor, data readout is now expected in H2 2025, compared to Q1 2025 as previously announced. In May 2024, the Company announced its participation in the IMMUNOLIFE RHU 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 randomized multicenter Phase 2 clinical trial will include advanced non-small cell lung cancer (NSCLC) patients. In this investigator-sponsored trial, MaaT Pharma's financial commitment will be limited to clinical product supply. The trial is expected by the sponsor to start mid-2025. MaaT034 - Next-generation drug candidates with co-cultured technology (MET-C platform) In 2024, MaaT Pharma presented new in vitro data on MaaT034's metabolite production and immune modulation at the American Association for Cancer Research (AACR) Annual Meeting (April) and at the Society for Immunotherapy of Cancer (SITC) Annual Meeting (November). MaaT034, the first product from the MET-C platform, is a synthetic microbiota therapy aimed at enhancing immunotherapy responses in solid tumor patients, which represents a potentially large market. Given the current Company's prioritization of resources on its hemato-oncology programs, and particularly on MaaT013's registration activities in Europe, the pace of MaaT034 development activities will be contingent upon further financial resources. In neurodegenerative diseases Outside the Company's main focus in oncology, MaaT Pharma's donor-derived MaaT033 was successfully investigated in ALS, further demonstrating the versatility of the Company's drug platform in other therapeutic domains. The Study met its primary endpoint and positive Phase 1b results were announced in November 2024, confirming its safety and tolerability beyond oncology indications. Corporate update In 2024, MaaT Pharma significantly strengthened its leadership team to support its next development phase, enhancing expertise across key areas to drive its clinical, regulatory, and financial strategy forward. Jonathan Chriqui joined as Chief Business Officer in March, followed by Gianfranco Pittari as Chief Medical Officer and Carole Ifi as Head of Regulatory Affairs in the Summer 2024. In November, Eric Soyer was appointed Chief Financial Officer. These strategic appointments further strengthen execution capabilities of its clinical and development plans. Financial highlights The key financial audited results for the full year of 2024 are as follows. Condensed Income Statement In thousands of euros 31 December 2024 (12 months) 31 December 2023 (12 months) Revenue 3 216 2 228 Other Income 3 831 4 667 Sales, General and Administrative costs (7 781) (5 839) Research and Development costs (27 694) (20 999) Operating income (expense) (28 428) (19 943) Financial Income 401 639 Financial Expense (878) (413) Net financial income (expense) (477) 226 Net Income (loss) for the period (28 904) (19 717) In accordance with IFRS international standards. Detailed financial information available here (French only) The audit procedures for the 2024 financial statements were carried out by the Company's statutory auditor and the 2024 statutory accounts were closed by the Company's Board of Directors on March 24, 2025. The financial statements are available on the Company's website. The full financial reports will be included in the Company's Universal Registration Report (equivalent to the annual financial report), which will be filed with the Autorité des Marchés Financiers on 11 April 2025. Revenues totaled €3.2 million for the year ended December 31, 2024, the highest revenues generated thus far by the Company, mostly comprised of compensation invoiced from the Early Access Program in France and for which data was presented at the American Society of Hematology Annual Meeting in December 2024. Other income of €3.8 million included R&D tax credits of €3.5 million, stable from €3.6 million in the prior year, while grants decreased to €0.1 million in 2024 from €1.0 million in the prior year. Sales, General and Administrative expenses amounted to €7.8 million in 2024, compared with €6.0 million in 2023, reflecting mostly the expenses to support the early access program, as well as the increase in regulatory advisory costs and the strengthening of the team. Research and Development expenses were €27.7 million in 2024, an increase of €6.7 million from 2023, consistent with the advancement of clinical and operational activities as detailed in the pipeline highlights' section above. As a result, Operating expenses amounted to €28.4 million in 2024 compared with €19.9 million for 2023, an increase of €8.5 million. Net loss was to €28.9 million for the year ended December 31, 2024, compared with €19.7 million for the year ended December 31, 2023. Cash Position as of December 31, 2024 As of December 31, 2024, total cash and cash equivalents were €20.2 million, as compared to €27.0 million as of September 30, 2024, and €24.3 million as of December 31, 2023. The net decrease in cash position of €4.1 million between December 31, 2023, and December 31, 2024, was related to a net cash utilization in Operating and Investing activities of €22.0 million and €0.4 million, respectively, while cash generated in financial activities was €18.3 million, including the €17.2 million net proceeds from the May 2024 capital raise. Capital Increase with the support of historical shareholders In this exceptionally challenging financial and economical context, the Company has just carried out a Capital Increase with its historical shareholders, thus giving priorities to secure upcoming key milestones while limiting the dilution and preserving value for all shareholders. Based on its projected plans and its associated financing needs to date, and following the Capital Increase, the Company expects its cash and cash equivalents balance to be sufficient to fund its operations into October 2025. In order to finance its activities for the next twelve months, the Company will need to raise additional funds. The Company is actively discussing additional dilutive and non-dilutive financing options for 2025, which, together with a potential strategic partnership, if materialized, will further finance and accelerate its developments activities, allowing to extend the Company's cash runway. The Capital Increase will enable the Company to pursue its developments plan and confirm its potential for being a key player in hemato-oncology. Upcoming significant value-creation milestones include: the Company is targeting to submit its Market Authorization Application for MaaT013 in aGvHD with the European agency in June 2025, with a potential approval expected in H2 2026; the Company is also preparing for a commercial launch in Europe, based on compelling Phase 3 data released in January 2025; the Company is also actively assessing potential partnership options for the distribution of its hemato-oncology products in Europe, in view of strengthening the launch and market penetration strategy of MaaT013, if approved; in the US, the Company is preparing for the launch of a dedicated US Phase 3 trial in aGvHD, or, subject to FDA's approval, the submission of a US Biologics License Application (BLA) (subject to confirmatory trial) based on compelling European Early Access and Phase 3 data, in view of a late 2026 commercial launch, if approved; the Company will continue the ongoing allo-HSCT Phase 2b study "PHOEBUS" (1yr-OS read out expected in late 2027). Main characteristics of the Capital Increase MaaT Pharma's Board of Directors using the delegation of powers granted by the 25th resolution of the shareholders' general meeting held on May 28, 2024 (capital increase without preferential subscription rights reserved to specific categories of investors) (the "AGM") and in accordance with article L. 225-138 of the French Commercial Code (code de commerce), has authorized the principle of a Capital Increase on March 24, 2025 and the CEO has decided, pursuant to the sub-delegation of authority granted by the Board, to complete a capital increase of 13,000,002.80 euros, by way of issuance of 2,131,148 new shares with a nominal value of €0.10 each (the "New Shares") for a subscription price of €6.10 each (including premium) (the "Capital Increase"). The New Shares will be issued at a market price, corresponding to the closing price of the Company's shares on the Euronext Paris regulated market at the time of the last trading session preceding its setting (i.e. March 26, 2025). The participation of existing shareholders represents the aggregate gross amount of the Capital Increase, namely, Biocodex for 6 million euros, PSIM Fund for 5 million euros, and a US/EU existing investor for 2 million euros. PSIM Fund represented by Bpifrance Investissement and Biocodex are also represented on the Board of Directors of the Company, and as such, did not take part in the vote of the Capital Increase at the Board of Directors' meeting held on March 24, 2025. The New Shares will be of the same class and fully fungible with the existing shares of the Company and will be admitted to trading on the regulated market of Euronext in Paris under the ISIN FR0012634822 - MAAT. The settlement-delivery of the Capital Increase is expected to take place around March 31, 2025, subject to customary conditions. Impact of the Capital Increase on the share capital Following the completion of the Capital Increase, MaaT Pharma's share capital will amount to €1,611,525.10 divided into 16,115,251 shares and the issuance of the New Shares represents 13.2% of the share capital of the Company after the Capital Increase. On an illustrative basis, a shareholder holding 1% of the Company's share capital before the Capital Increase and who did not participate in the Capital Increase will hold 0.87% of the Company's share capital after the issuance of the New Shares. To the Company's knowledge, the shareholding structure, on a non-diluted base, before and after the Capital Increase, breaks down as follows: Shareholders Before Capital Increase (non-diluted basis) After Capital Increase (non-diluted basis) Number of Ordinary Shares held Percentage of Existing Share Capital Number of Ordinary Shares held Percentage of Existing Share Capital Karim Dabbagh 1,960 0.01% 1,960 0.01% Hervé Affagard 266,173 1.90% 266,173 1.65% Total of individual corporate officers 268,133 1.92% 268,133 1.66% Seventure Funds 2,586,379 18.50% 2,586,379 16.05% Crédit Mutuel Innovation SAS 1,412,364 10.10% 1,412,364 8.76% Biocodex SAS 1,859,185 13.29% 2,842,792 17.64% Symbiosis LLC 1,946,735 13.92% 1,946,735 12.08% FPCI Fonds PSIM 2,802,439 20.04% 3,622,111 22.48% US/EU existing investor 623,632 4.46% 951,501 5.90% Other Shareholders 196,128 1.40% 196,128 1.22% Total Historical shareholders 11,426,862 81.71% 13,558,010 84.13% Employees and consultants 183,573 1.31% 183,573 1.14% Public Float 2,105,535 15.06% 2,105,535 13.07% Total 13,984,103 100% 16 115 251 100% Absence of Prospectus In connection with the Capital Increase, no listing prospectus will be approved by the Autorité des Marchés Financiers (the "AMF"). This press release does not constitute a prospectus under Regulation (EU) 2017/1129 of the European Parliament and of the Council of June 14, 2017, as amended (the "Prospectus Regulation"), or a public offering. The Company will file with the AMF a document containing the information set out in Annex IX of the Prospectus Regulation (the "Information Document"), which will be made available on the Company's website ( Risk factors Investors' attention is drawn to the risk factors set out in the 2023 Universal Registration Document filed with the AMF on April 2nd, 2024, under number D.24-0225 as well as in the 2024 half-year financial report, which are available on the Company's website ( and the website of the AMF ( as updated by the risk factors presented in section 4 of the Information Document. The occurrence of any or all of these risks could have an adverse effect on the Company's business, financial situation, results, development or prospects. Financial calendar* May 13, 2025: Publication of revenues & cash for Q1 2025 June 20, 2025: Annual General Meeting September 16, 2025: Publication of H1 results November 4, 2025: Publication of revenues & cash for Q3 2025 *Indicative calendar that may be subject to change. Upcoming conferences participation April 2-3, 2025 – Kempen Life Sciences Conference, Amsterdam April 25 - 30, 2025 – American Association for Cancer Research (AACR) Annual Meeting 2025, Chicago, IL May 5-6, 2025 - Swiss Biotech Day, Basel May 13, 2025 – Forum Midcaps Gilbert Dupont, Paris June 12-15 - European Hematology Association (EHA) Congress, Milan, IT 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. Disclaimer This press release is an advertisement and not a prospectus within the meaning of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017 (as amended, the "Prospectus Regulation"). In France, the offering described above took place solely as a placement to a category of institutional investors, in accordance with Article L. 225-138 of the "Code de commerce" and applicable regulations. The Capital Increase does not constitute a public offering in France, as defined in Article L. 411-1 of the "Code monétaire et financier" and no prospectus reviewed or approved by the Autorité des marchés financiers will be published. With respect to Member States of the European Economic Area (including France), no action has been taken or will be taken to permit a public offering of the securities referred to in this press release which would require the publication of a prospectus in any Member State. This press release and the information it contains is not an offer to sell, nor the solicitation of an offer to subscribe for or buy, New Shares in the United States or any other jurisdiction where restrictions may apply. Securities may not be offered or sold in the United States absent registration under the Securities Act or an exemption from registration thereunder. MaaT Pharma does not intend to register the New Shares under the Securities Act or conduct a public offering of the New Shares in France, the United States, or in any other jurisdiction. This communication is being distributed only to, and is directed only at (a) persons outside the United Kingdom, (b) persons who have professional experience in matters relating to investments falling within Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (the "Order"), and (c) high net worth entities, and other persons to whom it may otherwise lawfully be communicated, falling within Article 49(2) of the Order (all such persons together being referred to as "relevant persons"). Any investment or investment activity to which this communication relates is available only to relevant persons and will be engaged in only with relevant persons. Any person who is not a relevant person should not act or rely on this communication or any of its contents. This distribution of this press release may be subject to legal or regulatory restrictions in certain jurisdictions. Any person who comes into possession of this press release must inform him or herself of and comply with any such restrictions. 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 Relations Michael 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 Relations Heather SheaMedia relations for MaaT Pharma +1 617-286-2013 Sign in to access your portfolio
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11-03-2025
- Business
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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
