Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025
Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13 – 17, 2025. Five patients, aged 6 to 10 years, were treated, 2 at the first dose level and 3 at the second dose level (3x10¹³ vg/kg). The initial part of the clinical trial aimed at selecting the optimal dose (dose escalation phase), evaluating the tolerance and preliminary efficacy of the treatment and determined the effective dose for the pivotal phase of the GNT-016-MDYF trial, which is expected to start in mid-2025 (3x10¹³ vg/kg).
Genethon CEO Frederic Revah observed, 'The results of our gene therapy GNT0004 are very positive in patients treated at the dose of 3x10¹³ vg/kg, both in terms of microdystrophin expression and clinical efficacy criteria. Besides these results, the advantage of our product lies in the selected dose for the pivotal phase, which is lower than those used in other gene therapy trials for Duchenne Muscular Dystrophy. We are currently preparing the pivotal phase that we will conduct in Europe and the US.'
Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase (CPK) levels, a key marker of muscle damage.
One-year post-treatment, the comparison of the three patients treated at the effective dose with a group of 34 untreated patients, matched by age and followed in the same centers and by the same practitioners, shows a difference of +4.7 points in the score obtained using the internationally recognized clinical evaluation scale NSAA between treated and untreated patients.
At 24 months post-treatment, key observations include:
About GNT0004 and the trial
The GNT0004 gene therapy is composed of an AAV8 (adeno-associated virus) vector and the optimized hMD1 transgene, a shortened but functional version of the gene encoding dystrophin, the protein deficient in people with DMD. This vector is designed to be expressed in muscle tissue and also in the heart, thanks to a tissue-specific Spc5-12 promoter sequence. GNT0004 is administered by a single intravenous injection. It was developed by Genethon, in partnership with the teams of Prof. Dickson (University of London, Royal Holloway) and the Institut de Myologie (Paris). The trial, sponsored by Genethon, combines Phases 1/2/3, a dose-escalation phase followed by a pivotal phase at the dose finally chosen. The trial is being carried out in France and the UK and includes boys aged 6 to 10 with DMD who have retained their ability to walk.
About Duchenne muscular dystrophy
DMD is a rare, progressive genetic disease affecting all the body's muscles, and mainly boys (1 in 5000). It is due to abnormalities in the gene responsible for producing dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of skeletal and cardiac muscles, loss of walking and respiratory capacity, cardiomyopathy and death between the ages of 20 and 40.
About Genethon
A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a nonprofit organization created by the AFM-Téléthon. The first gene therapy to treat spinal muscular atrophy, incorporating technologies developed at Genethon, is marketed worldwide. With over 240 scientists and professionals, Genethon pursues its goal of developing innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen products from Genethon's R&D or collaborations are in clinical trials for diseases of the liver, blood, immune system, muscles and eyes. A further seven products could enter clinical trials in the next five years. To find out more visit: http://www.genethon.com/.View source version on businesswire.com:https://www.businesswire.com/news/home/20250519497866/en/
CONTACT: Press contact:
Stephanie Bardon
[email protected]
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KEYWORD: EUROPE UNITED STATES NORTH AMERICA FRANCE WASHINGTON
INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS
SOURCE: Genethon
Copyright Business Wire 2025.
PUB: 05/19/2025 05:30 AM/DISC: 05/19/2025 05:31 AM
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These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans' and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. 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Ad hoc announcement pursuant to Art. 53 LR Allschwil, Switzerland, August 14, 2025 Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today that it has entered into an exclusive license agreement with Venatorx Pharmaceuticals, Inc., to acquire the global rights to ceftibuten-ledaborbactam etzadroxil, a clinical phase 3-ready oral beta-lactam/beta-lactamase inhibitor (BL/BLI) combination for the potential treatment of complicated urinary tract infections (cUTI), including pyelonephritis. Ceftibuten-ledaborbactam etzadroxil is the combination of ceftibuten, an orally bioavailable cephalosporin antibiotic, and ledaborbactam etzadroxil, the orally bioavailable prodrug of the novel beta-lactamase inhibitor ledaborbactam. 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We are committed to discovering, developing and commercializing innovative drugs to meet the needs of patients with severe bacterial and fungal infections. We have successfully launched two hospital brands, Cresemba for the treatment of invasive fungal infections and Zevtera for the treatment of bacterial infections. In addition, we have preclinical and clinical anti-infective assets in our portfolio. Basilea is listed on the SIX Swiss Exchange (SIX: BSLN). Please visit Disclaimer This communication expressly or implicitly contains certain forward-looking statements, such as "believe", "assume", "expect", "forecast", "project", "may", "could", "might", "will" or similar expressions concerning Basilea Pharmaceutica Ltd, Allschwil and its business, including with respect to the progress, timing and completion of research, development and clinical studies for product candidates. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Basilea Pharmaceutica Ltd, Allschwil to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd, Allschwil is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. For further information, please contact: Peer Nils Schröder, PhDHead of Corporate Communications & Investor RelationsBasilea Pharmaceutica International Ltd, Allschwil Hegenheimermattweg 167b4123 AllschwilSwitzerland Phone +41 61 606 1102 E-mail media_relations@ This ad hoc announcement can be downloaded from References T. P. Lodise, T. Chopra, B. H. Nathanson et al. Epidemiology of Complicated Urinary Tract Infections due to Enterobacterales Among Adult Patients Presenting in Emergency Departments Across the United States. Open Forum Infectious Diseases 2022, Jun 24;9(7):ofac315. M. D. Zilberberg, B. H. Nathanson, K. Sulham et al. Descriptive Epidemiology and Outcomes of Hospitalizations With Complicated Urinary Tract Infections in the United States, 2018. Open Forum Infectious Diseases 2022, Jan 10;9(1):ofab591 C. F. de Oliveira, M. B. Dorr, K. Lowe et al. Evaluation of Single and Multiple Dose Safety and Pharmacokinetics of Ledaborbactam Etzadroxil and Ceftibuten-Ledaborbactam Etzadroxil in Healthy Volunteers. Open Forum Infectious Diseases 2025, Jan 29;12(Suppl 1):ofae631.1425 J. A. Karlowsky, M. G. Wise, M. A. Hackel et al. Ceftibuten-Ledaborbactam Activity against Multidrug-Resistant and Extended-Spectrum-β-Lactamase-Positive Clinical Isolates of Enterobacterales from a 2018–2020 Global Surveillance Collection. Antimicrobial Agents and Chemotherapy 2022, Nov 15;66(11):e0093422 Attachment Press release (PDF)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
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Company announcement – No. 18 / 2025 Zealand Pharma Announces Financial Results for the First Half of 2025Petrelintide collaboration with Roche off to a strong start, while key leadership appointments and solid financial position enable exciting next chapter for Zealand Pharma. Collaboration and license agreement with Roche to co-develop and co-commercialize petrelintide as a future foundational therapy for weight management off to a strong start, with further insight and updates expected at Roche's Pharma Day in September and Zealand Pharma's Capital Markets Day in December. Petrelintide program advancing at full speed, with ZUPREME Phase 2 trials progressing towards key milestones, and Roche breaking ground this August on new high-volume, high-throughput manufacturing facility dedicated to next-generation obesity medicines. Strengthening of leadership team to optimally position Zealand Pharma for future growth, with Utpal Singh as Chief Scientific Officer to drive the next wave of innovation and Steven Johnson as Chief Development Officer to spearhead development and regulatory strategies. Copenhagen, Denmark, August 14, 2025 – Zealand Pharma A/S (Nasdaq: ZEAL) (CVR-no. 20045078), a biotechnology company focused on the discovery and development of innovative peptide-based medicines, today announced the interim report for the six months ended June 30, 2025, and provided a corporate update. Zealand Pharma ready for catalyst-rich and exciting chapter from position of strengthAdam Steensberg, President and Chief Executive Officer at Zealand Pharma said: 'I am very satisfied to see our historic and transformative collaboration with Roche on petrelintide off to a strong start and progressing at full speed. Our organizational strength and very solid financial position empower us to unlock the full value potential of petrelintide and to accelerate investments in the next wave of innovation. With petrelintide Phase 2 data and survodutide Phase 3 data in obesity rapidly approaching, we are entering a pivotal new chapter for the company.' Key financial results for H1 2025 DKK million H1-25 H1-24 Revenue 9,096 49 Operating expenses1 -9682 -559 Operating result 8,1282 -524 Net financial items -157 -1DKK million Jun-30,2025 Dec-31,2024 Cash position3 16,578 9,022 Notes:1. Operating expenses consist of R&D, S&M, and G&A. 2. Excluding transaction-related costs of DKK 196 million related to the Roche partnership agreement. Operating expenses including transaction fees in H1 2025 amount to DKK 1,164 million.3. Cash position includes cash, cash equivalents and marketable in the second quarter of 2025Obesity Petrelintide, amylin analog. 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In April 2025, Zealand Pharma initiated the Phase 2 ZUPREME-2 trial with petrelintide in people with overweight or obesity and type 2 diabetes, investigating the efficacy and safety of petrelintide over a treatment duration of 28 weeks. Petrelintide, amylin analog. In June 2025 at the American Diabetes Association's 85th Scientific Sessions in Chicago, Illinois, Zealand Pharma presented additional data from the 16-week Phase 1b trial showing greater weight loss efficacy in the few female participants. No differences were observed between females and males for any adverse events, including gastrointestinal. Dapiglutide, GLP-1/GLP-2 receptor dual agonist. Results from Part 1 of the Phase 1b trial investigating the efficacy and safety of dapiglutide over a treatment duration of 13 weeks were presented at the American Diabetes Association's 85th Scientific Sessions in Chicago, Illinois in June 2025. Dapiglutide, GLP-1/GLP-2 receptor dual agonist. In June 2025, Zealand Pharma announced positive topline results from Part 2 of the Phase 1b trial with higher doses of dapiglutide, demonstrating a placebo-adjusted weight loss of 11.4% from baseline after 28 weeks despite a relatively lean and almost entirely male trial population. Rare diseases Glepaglutide in SBS. In June 2025, Zealand Pharma submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for glepaglutide administered twice weekly for the treatment of adult patients with SBS. Corporate Appointed Utpal Singh as Chief Scientific Officer. Utpal joined the executive team in April 2025 to lead discovery research and translational sciences at Zealand Pharma. Utpal brings nearly 25 years of pharmaceutical industry experience spanning the full drug discovery and development lifecycle. Appointed Steven Johnson as Chief Development Officer. Steven joined the executive team in May 2025 to lead regulatory and development strategies at Zealand Pharma. Steven brings nearly 30 years of comprehensive global drug development experience, gained across leading pharmaceutical companies, contract research organizations, and the U.S. FDA. Upcoming events next 12 monthsObesity Petrelintide, amylin analog. In the first half of 2026, Zealand Pharma expects to report topline results from the Phase 2 ZUPREME-1 trial and complete the Phase 2 ZUPREME-2 trial with petrelintide. Petrelintide/CT-388, amylin+GLP-1/GIP fixed-dose combination. Zealand Pharma and Roche expect to initiate Phase 2 with petrelintide/CT-388 in the first half of 2026. Dapiglutide, GLP-1/GLP-2 receptor dual agonist. In the second half of 2025, Zealand Pharma expects to initiate a Phase 2 trial in a dedicated obesity-related comorbidity. Survodutide, glucagon/GLP-1 receptor dual agonist. Topline data from SYNCHRONIZETM-1 and SYNCHRONIZETM-2, the Phase 3 trials with survodutide in people with overweight or obesity without and with type 2 diabetes, respectively, are expected in the first half of 2026. Rare diseases Glepaglutide in SBS. In the second half of 2025, Zealand Pharma expects to initiate a Phase 3 clinical trial of glepaglutide (EASE-5) that is anticipated to provide further confirmatory evidence for a regulatory submission in the U.S. Following the submission of the MAA to the EMA in June 2025, the company expects potential regulatory approval in the EU in the first half of 2026. In parallel, the company is engaging in partnership discussions for future commercialization. Dasiglucagon in CHI. Zealand Pharma is ready to resubmit the New Drug Application for dasiglucagon for up to three weeks of dosing and to submit the requested analyses from existing continuous glucose monitoring datasets to support use beyond three weeks. The regulatory submissions are, however, contingent on an inspection classification upgrade of a third-party manufacturing facility. In parallel, Zealand Pharma is implementing a supply contingency plan, including the qualification of an alternative supplier to ensure that the product can be made available to patients in need as quickly as possible. Chronic inflammation ZP9830, Kv1.3 Ion Channel Blocker. Zealand Pharma expects to complete the first-in-human clinical trial with ZP9830 in the fourth quarter of 2025 and report topline data in the first half of 2026. Corporate Zealand Pharma Capital Markets Day. Zealand Pharma will host a Capital Markets Day in London on December 11, 2025. Speakers will include Management as well as external experts and thought leaders in obesity. Financial guidance for 2025 Guidance unchanged from February 20, 2025, excluding transaction-related costs associated with the Roche collaboration. DKK million 2025Guidance4,5 2024Actuals Revenue anticipated from existing and new license and partnership agreements No guidance 63 Net operating expenses 2,000-2,500 1,327 Notes:4. Excluding transaction-related costs related to the Roche collaboration.5. Financial guidance based on foreign exchange rates as of August 13, 2025. Conference call today at 2 PM CET / 8 AM ETZealand Pharma's management will host a conference call today at 2:00 PM CET / 8:00 AM ET to present results through the first half of 2025 followed by a Q&A session. Participating in the call will be Chief Executive Officer, Adam Steensberg; Chief Financial Officer, Henriette Wennicke; and Chief Medical Officer, David Kendall. The conference call will be conducted in receive telephone dial-in information and a unique personal access PIN, please register at The live listen-only audio webcast of the call and accompanying slides presentation will be accessible at Participants are advised to register for the call or webcast approximately 10 minutes before the start. A recording of the event will be available following the call on the Investor section of Zealand Pharma's website at Financial Calendar for 2025 Q3 2025 November 13, 2025 Q4/FY 2025 February 19, 2026About Zealand Pharma A/SZealand Pharma A/S (Nasdaq: ZEAL) is a biotechnology company focused on the discovery and development of peptide-based medicines. More than 10 drug candidates invented by Zealand Pharma have advanced into clinical development, of which two have reached the market and three candidates are in late-stage development. The company has development partnerships with a number of pharma companies as well as commercial partnerships for its marketed Pharma was founded in 1998 and is headquartered in Copenhagen, Denmark, with a presence in the U.S. For more information about Zealand Pharma's business and activities, please visit Forward-looking StatementsThis company announcement contains 'forward-looking statements', as that term is defined in the Private Securities Litigation Reform Act of 1995 in the United States, as amended, even though no longer listed in the United States this is used as a definition to provide Zealand Pharma's expectations or forecasts of future events regarding the research, development, and commercialization of pharmaceutical products, the timing of the company's clinical trials and the reporting of data therefrom and the company's significant events and potential catalysts in 2025 and financial guidance for 2025. These forward-looking statements may be identified by words such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'intend,' 'may,' 'plan,' 'possible,' 'potential,' 'will,' 'would', and other words and terms of similar meaning. You should not place undue reliance on these statements, or the scientific data presented. The reader is cautioned not to rely on these forward-looking statements. Such forward-looking statements are subject to risks, uncertainties and inaccurate assumptions, which may cause actual results to differ materially from expectations set forth herein and may cause any or all of such forward-looking statements to be incorrect, and which include, but are not limited to, unexpected costs or delays in clinical trials and other development activities due to adverse safety events or otherwise; unexpected concerns that may arise from additional data, analysis or results obtained during clinical trials; our ability to successfully market both new and existing products; changes in reimbursement rules and governmental laws and related interpretation thereof; government-mandated or market-driven price decreases for our products; introduction of competing products; production problems; unexpected growth in costs and expenses; our ability to effect the strategic reorganization of our businesses in the manner planned; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies, or may reject, fail to approve or may delay approval of our drug candidates or expansion of product labelling; failure to obtain regulatory approvals in other jurisdictions; exposure to product liability and other claims; interest rate and currency exchange rate fluctuations; unexpected contract breaches or terminations; inflationary pressures on the global economy; and political uncertainty. If any or all of such forward-looking statements prove to be incorrect, our actual results could differ materially and adversely from those anticipated or implied by such statements. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. All such forward-looking statements speak only as of the date of this press release/company announcement and are based on information available to Zealand Pharma as of the date of this release/announcement. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical Pharma® is a registered trademark of Zealand Pharma A/S. ContactsAdam LangeVice President, Investor RelationsZealand PharmaEmail: ALange@ Neshat AhmadiInvestor Relations ManagerZealand PharmaEmail: NeAhmadi@ Anna Krassowska, PhDVice President, Investor Relations & Corporate CommunicationsZealand PharmaEmail: AKrassowska@ Attachment Zealand Pharma H1 2025 Interim ReportError 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
