Adalyon Appoints Ulrik Zeuthen as CEO to Drive Next Phase of Growth in AI-driven Speech-based Biomarkers and Behavioural Analytics for Optimising Clinical Trials




Adalyon Appoints Ulrik Zeuthen as CEO to Drive Next Phase of Growth in AI-driven Speech-based Biomarkers and Behavioural Analytics for Optimising Clinical Trials

ESPOO, Finland, Nov. 25, 2025 (GLOBE NEWSWIRE) — Adalyon, a pioneering tech-bio company using its AI-powered speech biomarker platform to transform clinical drug development, today announced the appointment of Ulrik Zeuthen, as Chief Executive Officer (CEO). Ulrik is an experienced leader in driving digital transformation in the pharmaceutical industry through data integration and artificial intelligence (AI). Adalyon’s proprietary speech-based digital biomarkers provide actionable insights into cognitive and behavioural patient outcomes, helping optimise clinical research.

Ulrik brings extensive experience from the pharmaceutical and data science sectors, having previously held Director level positions at Novo Nordisk, working in Denmark and Japan, and as a data strategist at Deloitte. He has been responsible for the establishment and execution of partnerships between Novo Nordisk and primary technical partners, to drive the scaled and accelerated AI use case delivery across the company. He holds a Masters of Science (MSc) in Economics from the University of Copenhagen, Denmark.

“I’m excited to join Adalyon, to leverage the deep expertise of the team and the company’s strong heritage in behavioural science and technology R&D for real impact. Adalyon’s proprietary methodology utilising modern AI/ML capabilities in highly regulated environments stands out as market shaping. We’re pioneering quantified behavioural analytics to modernize clinical research,” said Ulrik Zeuthen, CEO of Adalyon. “I’m looking forward to showcasing the value of our AI-powered Behavioural Intelligence Platform in delivering real-time behavioural and emotional insights for clinical trials and healthcare applications. Specifically, our adaptable ‘patient-centric’ approach is designed as a plug-and-play technology, driving better patient stratification and retention in trials, and with the potential to identify benefits in additional indications, enabling product label extensions and early detection of treatment effects. Our technology has the potential to add tremendous value to pharmaceutical R&D programmes by improving clinical trial outcomes and ultimately delivering more effective drugs and patient outcomes.”

Founder of Adalyon, Katarina Cantell, will transition to the role of Chief Scientific Officer (CSO). Dr Cantell holds a PhD in information systems and has conducted doctoral research at Stanford University at the intersection of behavioural sciences and technology. She brings over 20 years of experience in digital health and strategic leadership, including executive roles at Aava Medical and TietoEvry with her earlier career stemming from Polar Electro, wearables and the evolution of HRV into a digital biomarker.

Dr Katarina Cantell, CSO and Founder of Adalyon commented, “Ulrik’s expertise in applying AI/ML & analytics in life sciences together with his entrepreneur background and growth mindset makes him ideal to lead Adalyon into its next chapter as we seek partnerships with pharmaceutical companies to support them in optimising clinical trials and enabling greater insights into a patient’s drug response profiles. Adalyon’s proprietary speech-based digital biomarker technology is designed to capture the true voice of the patient, not just what their blood tests may say. We could say that speech is the new blood, and we’re using it to strive for a digital revolution in clinical drug development.”

The current way of measuring behavioural phenomena and drug response signals in clinical trials relies heavily on questionnaires and invasive samples, which are often episodic, rigid, and burdensome for participants. This contributes to dropout rates reaching 40-50% and in many cases also incomplete and biased data. Adalyon’s technology aims to address this burden and data gap by providing a non-invasive, high-frequency signal that improves sensitivity, reduces noise, and accelerates go/no-go decisions. By seamlessly integrating into the current process, it enables sponsors to recruit and retain the right participants and engage and monitor them more effectively to support earlier detection of treatment responses and raise primary outcomes.

About Adalyon

Adalyon is a pioneering tech-bio company transforming natural human speech into next-generation digital biomarkers that optimise clinical trial design and execution. Founded on a deep behavioural science foundation, the company’s proprietary AI-based technology converts speech and text into quantitative biomarkers that capture subtle cognitive and emotional changes in trial participants, helping pharmaceutical sponsors and CROs improve patient selection, engagement, and retention in clinical trials.

Adalyon’s solution is designed as a ‘plug and play’ technology that seamlessly integrates into existing trial infrastructures to complement, rather than replace, traditional data collection. This approach helps reduce participant burden and dropout rates, deliver measurable quality-of-life insights, and enable more efficient, outcome-driven trials.

Using advanced natural language processing (NLP), large language models (LLMs), and its proprietary Ayda Insights behavioural analytics AI, Adalyon extracts real-time, unbiased intelligence from natural speech.

Adalyon is looking to collaborate with pharmaceutical partners and clinical research organisations to enable more effective patient stratification and retention, earlier detection of treatment effects, and deliver more data-driven, patient-centric outcomes.

With offices in Espoo, Finland, and Copenhagen, Denmark, Adalyon is led by a multidisciplinary, international team of experts in behavioural science, AI engineering, and computational linguistics, advancing the new era of speech biomarkers in life sciences.

Visit www.adalyon.com and follow Adalyon on LinkedIn.

Media contacts

Adalyon
Ulrik Zeuthen – CEO
ulrik.zeuthen@adalyon.com

Scius Communications 
Katja Stout
+44 778 943 5990
katja@sciuscommunications.com
Daniel Gooch
+44 7747 875479
daniel@sciuscommunications.com

Macomics Announces Updated Data of the ENIGMAC Drug Discovery Platform and The Role of Macrophages in Fibrotic Disease




Macomics Announces Updated Data of the ENIGMAC Drug Discovery Platform and The Role of Macrophages in Fibrotic Disease

EDINBURGH, United Kingdom and CAMBRIDGE, United Kingdom, Nov. 25, 2025 (GLOBE NEWSWIRE) — Macomics Ltd, a leader in macrophage drug discovery, announces that it has validated its ENIGMAC™ platform in macrophage mediated antifibrotic therapy.

Macomics’ ENIGMAC drug discovery platform is designed to discover therapeutic targets and unlock disease specific target biology. The platform enables identification and validation of novel targets and provides a translationally relevant path to the clinic through the development of more physiologically relevant human models combined with proprietary gene editing technology.

There is clear evidence of causal human genetics in fibrosis and the interplay between macrophages and fibroblasts in fibrotic diseases of the liver, lung and kidney. The identification and functional validation of potential first-in-class targets in this area has been limited by the lack of proper technology and macrophage expertise.

Having already proven its first-in-class target validation potential in oncology, ENIGMAC is a proprietary technology developed to produce gene editable macrophages. The platform can knock in or knock out genes of interest via CRISPRa or CRISPRi expression in iPSC derived cells, unlocking the opportunity for new target discovery informed by human data.

For fibrotic disease-related drug discovery, Macomics has focused on the interplay between macrophages and fibroblasts on the deposition of the extracellular matrix seen in fibrosis with a unique co-culture system. Our goal has been to promote fibrosis regression through macrophage targeting, exploring potential first in class approaches.

Luca Cassetta, PhD, co-founder and VP Immunology at Macomics, said, “We have completed and validated gene knock down screenings (pooled and array) in iPS-derived cells in disease relevant models and also demonstrated that gene editing is scalable from single to genome wide.”

In its in-house program, Macomics’ therapeutic strategy has utilized the validated in vitro co-culture assay enabling higher throughput arrayed screening for macrophage antibody-dependent cellular phagocytosis (ADCP) fibroblast targets, with validation in human ex vivo precision-cut lung slices (PCLS), as a strategy to identify additional first in class targets.

Simon Dew, CBO of Macomics, said, “We are now using our ENIGMAC platform for new target discovery in fibrotic diseases, as well as other disease areas, both in-house and in drug discovery partnerships.”

About Macomics – www.macomics.com

Macomics Ltd is a macrophage drug discovery company with a world-leading macrophage drug discovery platform, developing first-in-class medicines to deliver transformational impact for patients with macrophage-driven diseases: Macrophages are key to multiple diseases of high unmet medical need, including as key mediators of solid tumour immunosuppression and pathological inflammation in chronic inflammatory disorders.

The company is progressing a diversified portfolio of therapies targeting disease specific macrophages towards the clinic. Its ENIGMAC macrophage drug discovery platform enables identification and validation of novel macrophage therapeutic targets and provides a translationally relevant path to clinic through the development of more physiologically relevant human macrophage models combined with proprietary gene editing technology.

The company was co-founded in 2020 by Prof. Jeffrey Pollard and Dr. Luca Cassetta, University of Edinburgh, internationally recognised leaders in macrophage biology. It has R&D and office facilities in Edinburgh and Cambridge, UK. The company’s financing was led by Epidarex Capital, and the company is backed by Scottish Enterprise, LifeLink Ventures and Caribou Property Limited.

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For further information please contact:

Macomics

At the Company – Steve Myatt, CEO, Macomics E: myatt@macomics.com

Media enquiries (for Macomics) – Sue Charles, Charles Consultants T: +44 (0)7968 726585 E: sue@charles-consultants.com

Allegro and American Regent sign exclusive licensing deal for SynoglideTM, a novel osteoarthritis treatment in animal health, valued at up to $35 million




Allegro and American Regent sign exclusive licensing deal for SynoglideTM, a novel osteoarthritis treatment in animal health, valued at up to $35 million

• Global license provides American Regent, a wholly owned subsidiary of Daiichi Sankyo, with rights to commercialize Synoglide™ in the veterinary market in most countries of the world. Allegro retains rights to human applications for its technology, enabling the company to focus on human clinical development of its osteoarthritis candidate
• Under the terms of the agreement, Allegro has received an upfront payment and is eligible to receive commercialization and sales milestone payments up to $35 million, in addition to royalties on sales
• Product availability is planned for early 2026

Liege, Belgium and Shirley, NY – 25 November 2025 (08:30 CET) – Allegro NV, a biomedical company developing transformative nanotechnology-based treatments for degenerative joint disease, and American Regent, a leading pharmaceutical company in the U.S. specializing in injectables for human and animal health, announced today that the two companies have entered into an exclusive licensing deal for Allegro’s Synoglide™, a novel treatment for osteoarthritis in the equine market.

Under the agreement, Allegro will manufacture Synoglide™ in its state-of-the art cleanroom facility. “The partnership with leading animal health player American Regent is a clear validation of the potential of Allegro’s injectable hydrogel to treat osteoarthritis in the equine veterinary market. Horses serve as a strong translational model for humans in joint disease, an exciting prospect as we focus on human clinical development of the technology,” said Lucas Decuypere, Chief Executive Officer at Allegro.

American Regent plans to introduce Synoglide™ for horses at the American Association of Equine Practitioners conference in December 2025 and expects to achieve first sales in the U.S. in early 2026. “We are eager to add this novel treatment for osteoarthritis to our veterinary portfolio as part of our commitment to innovative solutions that improve animal health. Synoglide™ offers a new approach to help veterinarians manage this challenging chronic condition that affects the lives of so many animals,” said Paul Diolosa, Chief Executive Officer at American Regent.

Synoglide™ is an intra-articular injection for horses suffering from osteoarthritis and is based on Allegro’s proprietary nanotechnology platform, INTRICATE. This technology will be launched for use in horses initially, and is being researched for other animals, including canine patients.

American Regent Inc.® (www.americanregent.com), a Daiichi Sankyo Group company, maker of animal health products Adequan® and BetaVet® is an industry-leading injectable manufacturer. For over 50 years, American Regent has been developing, manufacturing, and supplying quality generic and branded injectables for healthcare providers.

Allegro NV (www.allegro.bio) is a private biomedical company developing transformative treatments for degenerative joint diseases based on its proprietary nanotechnology platform, INTRICATE. The company’s lead product candidate in humans, Hydrocelin, is a first-in-class, potentially disease-modifying medical device candidate for the treatment of osteoarthritis. Allegro is preparing to initiate human clinical studies of Hydrocelin in 2026. Synoglide™ is partnered with American Regent, a subsidiary of Daiichi Sankyo, for further development and global commercialization, excluding the Middle-East.

For more information, please contact:

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.

Attachment

• 20251125 Allegro PR licensing deal (approved final)

RAMSAY SANTE : Press release on November 25 2025




RAMSAY SANTE : Press release on November 25 2025

       
        PRESS RELEASE
                Paris, 25^th November 2025

Update on Ramsay Health Care Limited’s review of its strategic options associated with its majority shareholding in Ramsay Générale de Santé

On 27 February 2025, Ramsay Générale de Santé acknowledged the announcement of its Australian reference shareholder Ramsay Health Care Limited regarding the review of its strategic options associated with its majority shareholding in Ramsay Générale de Santé.

Today, Ramsay Générale de Santé takes note of the press release issued by Ramsay Health Care Limited announcing that it has completed this review with the support of Goldman Sachs.

According to this press release, Ramsay Health Care Limited indicates that its Board and Management are committed to executing one of the options considered as part of this strategic review, and that the market will be provided with an update no later than the publication of Ramsay Health Care Limited’s first half results in February 2026.

The press release issued by Ramsay Health Care Limited can be viewed on its website (www.ramsayhealth.com).

About Ramsay Santé

Ramsay Santé is the European leader in private hospitalisation and primary care. With 40,000 employees and 10,000 practitioners, the group welcomes 13 million patients each year in 492 facilities across five countries (France, Sweden, Norway, Denmark and Italy).

As a mission-driven company, Ramsay Santé covers the entire care pathways in medicine, surgery, obstetrics, medical and rehabilitation care, mental health and primary care centres, with constant innovation to improves everyone’s health and ensures equitable access to secure and qualitative care.

Facebook: https://www.facebook.com/RamsaySante 
Instagram: https://www.instagram.com/ramsaysante 
Twitter: https://twitter.com/RamsaySante 
LinkedIn: https://www.linkedin.com/company/ramsaysante 
YouTube: https://www.youtube.com/c/RamsaySante 

Code ISIN and Euronext Paris: FR0000044471 
Website:  www.ramsaysante.fr 

Investor / Analyst Relations        Press Relations

Clément Lafaix        Brigitte Cachon
Tél. +33 1 87 86 21 52        Tél. +33 1 87 86 22 11
clement.lafaix@ramsaysante.fr        brigitte.cachon@ramsaysante.fr

Attachment

• RAMSAY SANTE – Press Release on November 25 2025

Novartis data underscore pioneering scientific innovation in Hematology and Oncology at ASH and SABCS




Novartis data underscore pioneering scientific innovation in Hematology and Oncology at ASH and SABCS

• Positive results from ianalumab pivotal Phase III trial in ITP patients previously treated with corticosteroids to be presented as late-breaker
• Scemblix data across clinical and real-world settings offer new evidence informing CML care amid evolving patient needs
• 96-week pelabresib Phase III data represent longest follow-up of first-line myelofibrosis patients in randomized combination trial
• Kisqali NATALEE and MONALEESA data add to evidence of long-term benefits for early and metastatic breast cancer patients

Basel, November 25, 2025 – Novartis will present data from over 70 abstracts, including investigator-initiated trials at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition and 2025 San Antonio Breast Cancer Symposium^® (SABCS). Featured among these latest advances in hematology and oncology are 11 oral presentations, with the Phase III VAYHIT2 trial for ianalumab in immune thrombocytopenia (ITP) accepted as a late-breaker abstract.

“For decades, Novartis has redefined the future of hematology and oncology, and we’re building on that foundation with compelling new data presented at ASH and SABCS,” said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. “These data underscore how we seek to set new standards for transformative care, with the aim of turning cutting-edge innovation into meaningful impact for patients.”

Key highlights of data accepted by ASH include:

Key highlights of data accepted by SABCS include:

Product Information

For full prescribing information, including approved indications and important safety information about marketed products, please visit https://www.novartis.com/about/products.

Novartis in hematology
Our legacy in hematology runs deep, shaped by over 25 years of progress, partnerships and a commitment to keep asking questions, challenging norms and striving for better answers in a uniquely complex field. In the past two decades, we have delivered more than 10 medicines across more than 15 blood cancers and serious blood disorders including leading the era of targeted therapies in cancer and bringing the first CAR-T therapy to patients.

Innovation in hematology has brought significant progress, yet patients and clinicians continue to face persistent challenges. We’re forging the future of hematology, powered by our foundation in scientific discovery to deliver meaningful change for patients with unmet needs.

Novartis in breast cancer 
For over 30 years, Novartis has been at the forefront of driving scientific advancements for individuals affected by breast cancer and enhancing clinical practice in collaboration with the global community. With one of the most comprehensive breast cancer portfolios and pipeline, Novartis leads the industry in discovery of new therapies and combinations in HR+/HER2- breast cancer, the most common form of the disease. 

Disclaimer

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis 
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.

Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.

# # #

Pennsylvania Perio & Implants Announces Strengthened Commitment to Evidence-Based Periodontal and Dental Implant Evaluation in Pennsylvania




Pennsylvania Perio & Implants Announces Strengthened Commitment to Evidence-Based Periodontal and Dental Implant Evaluation in Pennsylvania

York, Pennsylvania, Nov. 24, 2025 (GLOBE NEWSWIRE) — Pennsylvania Perio & Implants today announced the reinforcement of its evidence-based framework for periodontal evaluation and dental implant assessment across its York and Hanover locations. Under the clinical direction of periodontist Dr. Dennis Sourvanos, the practice has placed continued emphasis on evidence-based methodologies to provide structured, measurable insights into periodontal conditions and implant suitability.

The organization states that the initiative reflects an ongoing commitment to clarity, diagnostic rigor, and transparent communication to its patients and referring partners. With periodontal and peri-implant disease affecting a substantial portion of adults in Pennsylvania and throughout the country, the practice highlights the importance of detailed clinical assessment that aligns with current literature and professionally recognized standards. According to the practice, this approach supports consistency in evaluation and fosters a stronger understanding among patients and referring providers to improve oral health in the context of Dental Medicine, without offering implied outcomes or promotional assurances.

In discussing the practice’s direction, Dr. Sourvanos noted the significance of using established scientific criteria when examining periodontal conditions.
“Our evaluation model relies on objective clinical indicators and recognized periodontal parameters,” said Dr. Sourvanos. “By integrating research-supported standards into each assessment, we aim to ensure that every diagnostic step reflects measurable findings and maintains alignment with contemporary expectations in the field of periodontics.”

The practice reports that its clinical methodology includes structured charting of periodontal measurements, such as probing depths and attachment levels; radiographic analysis to identify bone support and anatomical considerations; adjunctive diagnostics, and the use of standard indices that assist in determining the status and progression of gum and/or implant disease. Pennsylvania Perio & Implants notes that this emphasis on quantifiable data is foundational in periodontics and critical for ensuring consistency across patient evaluations.

As part of its focus on evidence-based assessment, the practice has detailed its approach to evaluating potential candidates for dental implants. The organization explains that diagnostic imaging, anatomical reviews, and bone density considerations support a systematic process that adheres to research-supported principles rather than subjective interpretation. Pennsylvania Perio & Implants emphasizes that all implant evaluations are driven by the latest standards of evidence-based research and occur exclusively during clinical consultations.

The practice indicates that the reaffirmation of its methodology also includes a continued effort to strengthen communication with general dentists, dental specialists, hospital systems, and referring partners throughout Pennsylvania and the surrounding region. Clear documentation, treatment rationale summaries, and structured diagnostic reports are provided to referring providers to support continuity of care. Pennsylvania Perio & Implants states that collaborative communication ensures that evaluations remain consistent and transparent from initial diagnosis through follow-up.

As periodontal health remains a central focus for many dental professionals across the region, the organization notes that its evidence-based structure allows for more standardized interpretation of gum disease severity. The practice highlights that conditions such as tissue inflammation, periodontal pocketing, and bone loss require detail-driven evaluation to understand progression, classify disease stages, and outline potential treatment considerations. The announcement emphasizes that Pennsylvania Perio & Implants’ goal is to maintain clarity while avoiding speculation or predictive statements that fall outside accepted clinical boundaries.

The company further notes that public awareness regarding periodontal health has grown, with increasing interest in the role of periodontists in evaluating complex or advanced gum concerns. Pennsylvania Perio & Implants states that this announcement is intended to inform the public about the practice’s continued dedication to structured evaluation methods and evidence-aligned review processes. The release does not present promotional claims, comparative statements, or advice related to treatment choices; it remains focused solely on the organizational update.

In addition, the practice reiterates that its website, pennsylvaniaperioandimplants.com, contains general information about periodontal conditions, dental implant considerations, and the role of a periodontist. These resources are intended for educational purposes and are not meant to replace clinical consultation. The site also includes announcements related to the organization’s operations and its locations serving York and Hanover.

Pennsylvania Perio & Implants emphasizes that this communication reflects its ongoing commitment to maintaining structured, research-informed practices within the field of periodontal care. The organization reports that continuous evaluation of academic literature, clinical guidelines, and diagnostic tools remains central to its long-term approach. The announcement is intended as a factual update for the community and referring dental professionals, consistent with newswire standards and without promotional or advisory intent.

For more information, please visit https://pennsylvaniaperioandimplants.com/.

About Pennsylvania Perio & Implants

Pennsylvania Perio & Implants is a periodontal practice located in York and Hanover, Pennsylvania. The organization provides evaluation and treatment related to periodontal conditions as well as assessment for dental implant considerations. The practice follows evidence-based methodologies designed to support consistent diagnosis and research-aligned decision-making within the field of periodontics.

Media Contact
Company Name: Pennsylvania Perio & Implants
Contact Person: Amin Nassiri
Email: amin@dentistnerds.com
Phone: 3105926293
Country: United States
Website: https://pennsylvaniaperioandimplants.com/

CONTACT: Media Contact
Company Name: Pennsylvania Perio & Implants
Contact Person: Amin Nassiri
Email: amin@dentistnerds.com
Phone: 3105926293
Country: United States
Website: https://pennsylvaniaperioandimplants.com/

Junshi Biosciences Announces Primary Endpoints Met in JS001sc’s Phase 3 Study for the 1ST-line Treatment of NSQ-NSCLC




Junshi Biosciences Announces Primary Endpoints Met in JS001sc’s Phase 3 Study for the 1ST-line Treatment of NSQ-NSCLC

SHANGHAI, Nov. 24, 2025 (GLOBE NEWSWIRE) — Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, announced that the JS001sc-002-III-NSCLC study has met its primary endpoints. JS001sc-002-III-NSCLC is a multi-center, open-label, randomized Phase 3 clinical study comparing toripalimab injection (subcutaneous injection) (code: JS001sc) or toripalimab injection (code: JS001) in combination with chemotherapy for the first-line treatment of recurrent or metastatic non-squamous non-small-cell lung cancer (“NSQ-NSCLC”) (NCT06505837). Junshi Biosciences plans to submit a new drug application (“NDA”) to the regulatory authorities in the near future.

According to data released by GLOBOCAN 2022, in 2022, China saw 1.06 million new lung cancer cases (22.0% of all new cancer cases in China) and 0.73 million lung cancer deaths (28.5% of all cancer-related deaths in China). NSCLC was the predominant subtype, accounting for approximately 85% of all lung cancer cases. Among NSCLC patients, non-squamous NSCLC constituted approximately 65% of cases.

Immunotherapy (I-O), represented by anti-PD-1 monoclonal antibodies, has become a cornerstone treatment for various malignant tumors including lung cancer, breast cancer, liver cancer, esophageal cancer, and nasopharyngeal carcinoma. Now, immunotherapy covers nearly all stages of treatment for cancer patients, encompassing adjuvant/neoadjuvant treatment for early-stage tumors, consolidation treatment after radical chemoradiation for locally advanced tumors, and first-line to last-line treatments for advanced tumors. Currently, most immunotherapy drugs in China are administered intravenously, and this not only requires lengthy infusion times, but also imposes significant inconveniences on patients. There is an urgent clinical need for more convenient administration methods for immunotherapy.

The JS001sc-002-III-NSCLC Study is a multi-center, open-label, randomized Phase 3 clinical study led by the principal investigator Professor Lin WU from Hunan Cancer Hospital. JS001sc-002-III-NSCLC is the first Phase 3 clinical study of a domestic anti-PD-1 monoclonal antibody subcutaneous formulation.

The study aims to compare the exposure, efficacy and safety of JS001sc plus chemotherapy or JS001 plus chemotherapy for the first-line treatment of recurrent or metastatic NSQ-NSCLC. The results have showed that the drug exposure of JS001sc was non-inferior to that of toripalimab injection with comparable efficacy and safety profiles. The study data will be presented at an upcoming international academic conference. Junshi Biosciences plans to communicate with the regulatory authorities and submit JS001sc’s NDA for all approved indications of JS001.

Dr. Jianjun ZOU, General Manager and CEO of Junshi Biosciences, said, “Since its launch as China’s first domestically developed PD-1 antibody drug, toripalimab has secured approvals for 12 indications, benefiting a significant number of patients. In clinical practice, we observed that patients undergoing immunotherapy, either as monotherapy or combination maintenance therapy, face challenges such as frequent intravenous catheterization and time-consuming infusions. The recent success of the Phase 3 study for JS001sc, achieved through the efforts of both patients and the research team, marks not only a pivotal breakthrough in transitioning I-O therapy from ‘efficacy’ to ‘convenience’, but also exemplifies Junshi Biosciences’ patient-centric ambition. By innovating drug delivery methods, we enhance treatment accessibility: simplifying procedures for patients, reducing their healthcare burden, and alleviating pressure on medical resources. We are committed to advancing the registration of JS001sc and providing more patients with a better treatment experience alongside clinical benefits.”

About JS001sc
JS001sc, developed by Junshi Biosciences, is a subcutaneous injection formulation based on the marketed product, toripalimab injection. JS001sc is the first domestic anti-PD-1 monoclonal antibody subcutaneous formulation to enter Phase 3 clinical study, and will potentially offer more convenient administration to patients. As of today, a multi-center, open-label, randomized Phase 3 clinical study comparing JS001sc plus chemotherapy or toripalimab injection plus chemotherapy for the first-line treatment of recurrent or metastatic NSQ-NSCLC (the JS001sc-002-III-NSCLC Study) has met its primary endpoints.

About Junshi Biosciences
Founded in December 2012, Junshi Biosciences (HKEX: 1877; SSE: 688180) is an innovation-driven biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapeutics. The company has established a diversified R&D pipeline comprising over 50 drug candidates, with five therapeutic focus areas covering cancer, autoimmune, metabolic, neurological, and infectious diseases. Five of the company’s products have received approvals in China and international markets, one of which is toripalimab, China’s first domestically produced and independently developed anti-PD-1 monoclonal antibody. Toripalimab has been approved in over 40 countries and regions including China, the US, and Europe. During the COVID-19 pandemic, Junshi Biosciences actively shouldered the social responsibilities of a Chinese pharmaceutical company through its involvement in developing etesevimab, MINDEWEI®, and other novel therapies for the prevention and treatment of COVID-19.

With a mission of “providing patients with world-class, trustworthy, affordable, and innovative drugs,” Junshi Biosciences is “In China, For Global.” At present, the company boasts approximately 2,500 employees in the United States (Maryland) and China (Shanghai, Suzhou, Beijing, Guangzhou, etc.). For more information, please visit: http://www.junshipharma.com.

Junshi Biosciences Contact Information

IR Team:
Junshi Biosciences
info@junshipharma.com
+ 86 021-6105 8800

PR Team:
Junshi Biosciences
Zhi Li
zhi_li@junshipharma.com
+ 86 021-6105 8800

Ascentage Pharma Announces Publication of Olverembatinib Phase Ib Safety, Efficacy and Novel Mechanism Data in Gastrointestinal Stromal Tumors in Nature’s Signal Transduction and Targeted Therapy




Ascentage Pharma Announces Publication of Olverembatinib Phase Ib Safety, Efficacy and Novel Mechanism Data in Gastrointestinal Stromal Tumors in Nature’s Signal Transduction and Targeted Therapy

Largest prospective clinical trial to-date in a rare subtype of GIST

ROCKVILLE, Md. and SUZHOU, China, Nov. 24, 2025 (GLOBE NEWSWIRE) — Ascentage Pharma Group International Inc. (NASDAQ: AAPG; HKEX: 6855), a global, commercial stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel, differentiated therapies to address unmet medical needs in cancer, announced the publication of clinical and translational data from a Phase Ib study (NCT03594422) on the Company’s novel drug, olverembatinib (HQP1351), in patients with gastrointestinal stromal tumors (GIST), by the renowned scientific journal Signal Transduction and Targeted Therapy (Impact Factor: 52.7). The published results demonstrated promising efficacy and safety of olverembatinib in patients with succinate dehydrogenase (SDH) deficient GIST, a type of rare tumor, and revealed a novel mechanism through which olverembatinib exerts antitumor effects by modulating lipid metabolism.

The world-renowned, peer-reviewed, open-access Nature journal, Signal Transduction and Targeted Therapy, publishes original research on cutting-edge experimental and clinical advances on signal-transduction-targeted therapeutics in cancer, autoimmune disorders, and other conditions, and has an impact factor of 52.7.

This Phase Ib study (NCT03594422), reported in the published paper titled Olverembatinib, a multikinase inhibitor that modulates lipid metabolism, in advanced succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase Ib study and translational research, was led by Prof. Ruihua Xu, Academician of the Chinese Academy of Engineering, and Prof. Haibo Qiu, at Sun Yat-sen University Cancer Center. The study evaluated the safety and antitumor activity of olverembatinib in 66 patients with unresectable/metastatic GIST/other solid tumors, including 26 patients with SDH-deficient GIST who had failed prior treatment with tyrosine kinase inhibitors (TKIs), making it the largest prospective clinical trial in this rare subtype of GIST to date. Among 26 patients with SDH-deficient GIST, the objective response rate (ORR) was 23.1%; the clinical benefit rate (CBR) was 84.6% (95% CI, 65.1-95.6), and the median progression-free survival (mPFS) was 25.7 months (95% CI, 12.9-not reached). Olverembatinib was well tolerated, and the median follow-up was 14.5 months.

Gastrointestinal stromal tumors are rare, occurring in only 10–15 cases per million people annually, yet they represent the most common mesenchymal tumor of the digestive tract. SDH-deficient GIST accounts for 5% to 7.5% of all GIST cases, predominantly affecting children, adolescents and young adults, and is prone to relapses and metastasis. Traditional targeted therapies such as tyrosine kinase inhibitors have shown limited efficacy in SDH-deficient GIST. Currently, there are no standard of care treatments for this patient population, and the rarity of this condition makes conducting clinical trials challenging.

In addition, translational study revealed a novel mechanism by which olverembatinib exerts antitumor effects through modulation of lipid metabolism. RNA sequencing and lipidomic analysis identified significant lipid metabolism dysregulation in SDH-deficient tumors, characterized by weakened endogenous lipid synthesis and markedly enhanced uptake of exogenous lipids. Further investigation revealed that SDH functional deficiency drives abnormal overexpression of CD36, a key cell membrane lipid transporter, thereby increasing cellular uptake of exogenous lipids. This discovery establishes, for the first time, a direct mechanistic link between SDH, a key mitochondrial enzyme regulating energy metabolism, and CD36, showing that SDH-deficient GISTs exhibit high dependence on exogenous lipids for survival and proliferation. The study confirmed that olverembatinib can effectively inhibit CD36 expression, thereby blocking tumor cells from acquiring exogenous lipids, an effect not observed with other TKIs clinically available to treat SDH-deficient GISTs.

Beyond targeting lipid metabolism, olverembatinib inhibits multiple tumorigenic signaling pathways, including HIF, FGFR, and VEGFR. Therefore, the antitumor efficacy of olverembatinib in SDH-deficient tumors likely results from synergistic effects of lipid metabolism modulation and the inhibition of tumorigenic signaling pathways. This study is the first to establish a direct link between SDH functional deficiency and dysregulated lipid metabolism in tumor cells, particularly CD36-mediated enhanced uptake of exogenous lipids. These findings provide a novel therapeutic approach and potential target for SDH-deficient GIST, a condition currently lacking effective treatments, and underscore olverembatinib’s promising potential for this patient population.

“SDH-deficient GIST is an extremely rare tumor type that lacks high-quality, prospective clinical data; and in Chinese and international clinical guidelines, there are currently no recommended treatments for unresectable SDH-deficient GIST,” said Prof. Ruihua Xu, Academician of the Chinese Academy of Engineering, President and Director of Sun Yat-sen University Cancer Center. “This Phase I study has yielded encouraging efficacy and safety results, suggesting that olverembatinib may offer a new treatment option for this indication.”

“SDH-deficient GIST represents a treatment gap that urgently needs new treatment options. It took us approximately 5 years to enroll 26 patients in this study, which is now the world’s largest prospective clinical trial in SDH-deficient GIST,” said Prof. Haibo Qiu, of Sun Yat-sen University Cancer Center. “In the study, olverembatinib demonstrated impressive clinical benefits, including a median PFS of 25.7 months, as well as favorable safety and tolerability profiles. We will continue additional studies to validate the drug’s efficacy and safety in patients with SDH-deficient GIST.”

“Our pioneering translational research identified a putative mechanism of action, patients with SDH-deficient GIST exhibit significant overexpression of lipid-uptake-related genes and proteins, and olverembatinib suppressed lipid uptake in GIST cells. Olverembatinib also conferred antitumor effects by suppressing tumorigenic signaling pathways related to angiogenesis, hypoxia, survival, and proliferation,” said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. “We are very encouraged by these results, as they signal a potential breakthrough in addressing an indication with urgent unmet clinical need. Remaining committed to our mission of addressing unmet clinical needs in China and around the world, we will press forward with this clinical development program to bring a safe and effective new treatment option to patients as soon as possible.”

Olverembatinib is an orally available third-generation BCR::ABL1 inhibitor TKI developed by Ascentage Pharma and represents the first third-generation BCR::ABL1 inhibitor approved in China. The drug is currently approved for two indications: adult patients with TKI-resistant chronic-phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation; and adult patients with CML-CP resistant to and/or intolerant of first-and second-generation TKIs. Olverembatinib is being jointly commercialized in China by Ascentage Pharma and Innovent Biologics.

Beyond hematologic malignancies, olverembatinib is being clinically evaluated in patients with GIST. Olverembatinib has been granted Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) for the treatment of patients with SDH-deficient GIST who have received first-line treatment. An international multicenter, open, single-arm, pivotal registrational Phase III registrational study (POLARIS-3; NCT06640361) is currently recruiting patients to evaluate the efficacy and safety of olverembatinib in patients with SDH-deficient GIST who have received prior therapy.

* Olverembatinib is currently under investigation and has not yet been approved by the FDA in the US.

About Ascentage Pharma

Ascentage Pharma Group International (NASDAQ: AAPG; HKEX: 6855) (“Ascentage Pharma” or the “Company”) is a global, commercial stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel, differentiated therapies to address unmet medical needs in cancer. The Company has built a rich pipeline of innovative drug products and candidates that includes inhibitors targeting key proteins in the apoptotic pathway, such as Bcl-2 and MDM2-p53, as well as next-generation kinase inhibitors.

The lead asset, olverembatinib, is the first novel third-generation BCR-ABL1 inhibitor approved in China for the treatment of patients with CML in chronic phase (CML-CP) with T315I mutations, CML in accelerated phase (CML-AP) with T315I mutations, and CML-CP that is resistant or intolerant to first and second-generation TKIs. All indications are covered by the China National Reimbursement Drug List (NRDL). The Company is currently conducting an FDA-cleared, global registrational Phase III trial, or POLARIS-2, of olverembatinib for CML, as well as global registrational Phase III trials for patients with newly diagnosed Ph+ ALL and SDH-deficient GIST patients.

The Company’s second approved product, lisaftoclax, is a novel Bcl-2 inhibitor for the treatment of various hematologic malignancies. Lisaftoclax is being commercialized in China following National Medical Products Administration (NMPA) approval for the treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have previously received at least one systemic therapy including Bruton’s tyrosine kinase (BTK) inhibitors. The Company is currently conducting four global registrational Phase III trials: the FDA-cleared GLORA study of lisaftoclax in combination with BTK inhibitors in patients with CLL/SLL previously treated with BTK inhibitors for more than 12 months with suboptimal response; the GLORA-2 study in patients with newly diagnosed CLL/SLL; the GLORA-3 study in newly diagnosed, elderly and unfit patients with acute myeloid leukemia ( AML); and the GLORA-4 study in patients with newly diagnosed higher-risk myelodysplastic syndrome (HR MDS), a study that was simultaneously cleared by the US FDA, the EMA of the EU, and China CDE.

Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with numerous leading biotechnology and pharmaceutical companies, such as Takeda, AstraZeneca, Merck, Pfizer, and Innovent, in addition to research and development relationships with leading research institutions, such as Dana-Farber Cancer Institute, Mayo Clinic, National Cancer Institute and the University of Michigan. For more information, visit https://ascentage.com/

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical facts, contained in this press release may be forward-looking statements, including statements that express Ascentage Pharma’s opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results of operations or financial condition.

These forward-looking statements are subject to a number of risks and uncertainties as discussed in Ascentage Pharma’s filings with the SEC, including those set forth in the sections titled “Risk factors” and “Special note regarding forward-looking statements and industry data” in its Registration Statement on Form F-1, as amended, filed with the SEC on January 21, 2025, and the Form 20-F filed with the SEC on April 16, 2025, the sections headed “Forward-looking Statements” and “Risk Factors” in the prospectus of the Company for its Hong Kong initial public offering dated October 16, 2019, and other filings with the SEC and/or The Stock Exchange of Hong Kong Limited we made or make from time to time that may cause actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. The forward-looking statements contained in this presentation do not constitute profit forecast by the Company’s management.

As a result of these factors, you should not rely on these forward-looking statements as predictions of future events. The forward-looking statements contained in this press release are based on Ascentage Pharma’s current expectations and beliefs concerning future developments and their potential effects and speak only as of the date of such statements. Ascentage Pharma does not undertake any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

Investor Relations:
Stella Yang
Ascentage Pharma
Stella.Yang@ascentage.com
+1 (301) 792-6286

Stephanie Carrington
ICR Healthcare
AscentageIR@icrhealthcare.com
+1 (646) 277-1282

Media Relations:
Sean Leous
ICR Healthcare
AscentagePR@icrhealthcare.com
+1 (646) 866-4012

Mesoblast Trading Update at Annual General Meeting Highlights Continued Strong Growth in Ryoncil® Revenue




Mesoblast Trading Update at Annual General Meeting Highlights Continued Strong Growth in Ryoncil® Revenue

NEW YORK, Nov. 24, 2025 (GLOBE NEWSWIRE) — Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided a trading update, together with a comprehensive operational overview, as part of the Chief Executive’s Annual General Meeting (AGM) address.

For the quarter ending December 31, 2025, management expects gross revenue of more than US$30.0 million from sales of Ryoncil^® (remestemcel-L-rknd). This represents more than 37% increase on the US$21.9 million in gross revenue from Ryoncil^® in the prior quarter ended September 30, 2025.

About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast’s Ryoncil^® (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.

Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil^® is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.

About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2044 in all major markets.

About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and X: @Mesoblast

Forward-Looking Statements
This press release includes forward-looking statements and forecasts that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise, except where required by law or the ASX Listing Rules. Past performance given in this announcement is given for illustrative purposes only and should not be relied on as (and is not) an indication of future performance.

Not financial product advice

This announcement does not constitute financial product advice or investment advice (nor tax, accounting or legal advice) and has been prepared without taking into account the objectives, financial situation or needs of individuals. Before making an investment decision, prospective investors should consider the appropriateness of the information having regard to their own objectives, financial situation and needs and seek appropriate professional advice.

Disclaimer

To the maximum extent permitted by law, Mesoblast and its directors, officers, employees, advisers and agents disclaim any obligation or undertaking to release any updates or revisions to the information to reflect any change in expectations or assumptions, and disclaim all responsibility and liability for these forward-looking statements (including, without limitation, any liability for negligence).

Release authorizedby the Chief Executive.

For more information, please contact:

Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)




Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

Ad hoc announcement pursuant to Art. 53 LR

• Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients regardless of SMA treatment history in Phase III studies
  
  
• One-time dose of Itvisma replaces SMN1 gene, potentially reducing the need for chronic SMA treatment
  
• Gene replacement therapy now available to eligible people of all ages living with SMA

Basel, November 24, 2025 – Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma^® (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene, making it the first and only gene replacement therapy available for this broad population. Itvisma is uniquely designed to address the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight.¹ By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.¹
  
“The FDA’s approval of intrathecal onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups,” said John W. Day, MD, PhD, Professor of Neurology and Pediatrics, Director, Division of Neuromuscular Medicine at Stanford University School of Medicine, and Co-Director of Stanford’s Neuro IGNITE Center. “This achievement is not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”
  
The approval of Itvisma is based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH study. Itvisma showed statistically significant improvements in motor function and stabilization of motor abilities typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up.^2,3 Additionally, Itvisma demonstrated a safety profile with adverse events that were consistent across both studies.^2,3 The most common adverse events in the STEER study were upper respiratory tract infection and pyrexia, and the most common adverse events in the STRENGTH study were common cold, pyrexia, and vomiting.^2,3 These data were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
   
“This new route of administration for a single dose of gene replacement therapy can mean so much more than what is measured by numbers on a functional motor scale – it could mean greater independence and freedom in activities of daily life,” said Kenneth Hobby, President, Cure SMA. “The SMA disease landscape has dramatically changed over the last six years, when the first gene therapy was approved. This is another welcome advancement, and it represents real progress in expanding access for many older patients and addressing the unmet needs that remain in our community.”
   
SMA is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene.^4,5 The SMN1 gene is responsible for producing most of the SMN protein a body needs for muscle function, including breathing, swallowing and basic movement.⁵ Without it, motor neurons are irreversibly lost, leading to progressive, debilitating muscle weakness.⁵ A second gene, the SMN2 gene, produces a small fraction (~10%) of functional SMN protein compared with the SMN1 gene.⁶ Individuals with more copies of the SMN2 gene generally have a less severe form of SMA than those with fewer copies.⁶ 
   
Approximately 9,000 people in the US live with SMA, and though there have been advancements in treating the disease, unmet needs remain for older children, teens, and adults in preserving motor neurons and maintaining physical strength.^7,8
   
Transforming care in SMA
“After redefining SMA care with the first gene replacement therapy for this challenging disease, we can now help address unmet needs across an even broader SMA population with the approval of Itvisma,” said Victor Bultó, President, Novartis, US. “We are proud to support the SMA community by empowering patients of all ages through our innovative, one-time therapies, offering the potential to reduce the burden that comes with chronic treatment.”
   
Itvisma will be available in the US in December. Novartis Patient Support is available to help eligible patients get started on treatment. Patients and providers can call 1-855-441-4363 for personalized assistance, including help understanding insurance coverage and identifying potential financial assistance options.
   
About Itvisma^® (onasemnogene abeparvovec-brve)
Itvisma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN1 gene to improve motor function through sustained SMN protein expression with a single, one-time intrathecal injection.
   
Novartis has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of adeno-associated virus 9 (AAV9) gene replacement therapy for the treatment of all types of SMA; an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene replacement therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA. 
   
Novartis in neuroscience
Neurological diseases are deeply personal, affecting people of any age, from newborns to seniors, often striking in the prime of life. At Novartis, we’re doubling down on our commitment to neurology, expanding our legacy of innovation in spinal muscular atrophy (SMA) and multiple sclerosis (MS) to work in neuroimmunology, neurodegeneration, and neuromuscular diseases. Our goal is to protect people’s health across their lifespan, developing more treatment options that lead to better outcomes.
   
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “believe,” “committed,” “commitment,” “pipeline,” “launch,” “potentially,” “step forward,” “goal,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Itvisma, or regarding potential future revenues from Itvisma. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Itvisma will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Itvisma will be commercially successful in the future. In particular, our expectations regarding Itvisma could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
   
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.
   
Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.
   
   
References

1. Itvisma^® (onasemnogene abeparvovec-brve). Prescribing Information. Novartis Gene Therapies, Inc.
2. Proud C, et al. Neuromuscul Disord. 2025;(53):0960-8966. 
3. Clinicaltrials.Gov. STRENGTH Study Results. https://clinicaltrials.gov/study/NCT05386680. Accessed November 2025. 
4. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895–908.  
5. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/. Accessed November 2025.
6. Lorson CL, et al. Hum Mol Genet. 2010;(15):111-8. 
7. Cure SMA. State of SMA 2024 Report. https://www.curesma.org/wp-content/uploads/2025/04/State-of-SMA-Report2024_vWeb-4.pdf. Accessed November 2025.
8. Cure SMA. Address the Unmet Needs of Children and Adults with Spinal Muscular Atrophy. https://www.curesma.org/wp-content/uploads/2024/08/NIH_SMA_Research_FactSheet.pdf. Accessed November 2025.

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