Muscular Dystrophy, Duchenne

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarter Pratteln, Switzerland, January 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that recommends AGAMREE® (vamorolone) for use in the National Health Service (NHS) in England, Wales and Northern Ireland for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older. This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024. Following this, Santhera has already started launch preparations for AGAMREE in the UK and expect first sales in this quarter. AGAMREE is the first and only medicinal product for DMD to have received full approval in the EU, US, and UK. This recommendation follows the Medicines and Healthcare products Regulatory Agency’s (MHRA) approval of AGAMREE on January 11, 2024, which, alongside the European Medicines Agency (EMA), acknowledged clinically important tolerability benefits with regards to maintaining normal bone metabolism, density, and growth compared to standard of care corticosteroids, alongside similar efficacy from clinical trials. The Final Guidance is available on the NICE website: www.nice.org.uk/guidance/TA1031 For more information about AGAMREE in Great Britain, including Scotland: Summary of Product Characteristics About AGAMREE® (vamorolone) AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [1-4]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [1-4]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1, 4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6]. ▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. References:[1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.[6] Hasham et al., MDA 2022 Poster presentation. Link. About SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA) and Hong Kong by the Department of Health (DoH). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain countries in Southeast Asia to Sperogenix Therapeutics. For further information, please visit www.santhera.com. AGAMREE® is a trademark of Santhera Pharmaceuticals. For further information please contact: public-relations@santhera.com or Elodie Denjean, General Manager UK, France & Beneluxelodie.denjean@santhera.comPhone +44 7437 865600 Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. # # # Attachment 250116 NICE guidance_FINAL_English

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older. The decision, which allows patients with the rare muscle-wasting disorder to access Agamree on the NHS in Scotland, comes one month after the National Institute for Health and Care Excellence (NICE) recommended the drug for use on the NHS in England, Wales and Northern Ireland. Estimated to affect 2,500 males in the UK at any one time, DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles. The disease causes progressive muscle degeneration and weakness, and its major milestones are the loss of ambulation and self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. Agamree is a dissociative anti-inflammatory drug that works in a similar way to existing corticosteroids, the current standard of care for children and adolescents with DMD, but without the same safety concerns. The SMC’s decision follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy in January 2024, which was supported by results from the pivotal VISION-DMD study and three open-label studies. In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting. The trial also showed that patients who switched from prednisone to Agamree after 24 weeks were on average able to resume growing in height over the remainder of the study. Additionally, unlike corticosteroids, Agamree did not result in a reduction of bone metabolism or a significant reduction in bone mineralisation in the spine after 48 weeks in the clinical studies. Commenting on the latest authorisation for the therapy, Santhera’s chief executive officer, Dario Eklund, said: “I am delighted that the team has secured this latest approval, which will ensure Scottish patients can benefit from this important treatment for DMD. “We will work closely with NHS Scotland, along with all the key regulatory and health authorities in all our approved markets, to ensure patients have access to Agamree.”

Santhera’s Agamree is indicated for patients aged four years and older with Duchenne muscular dystrophy (DMD). Credit: JohnnyGreig via Getty Images. Swiss biotech Santhera Pharmaceuticals has started launch preparations for its Duchenne muscular dystrophy (DMD) drug Agamree (vamorolone) in the UK, following a recommendation from England’s price watchdog. Approved for use in the National Health Service (NHS) in England, Wales, and Northern Ireland, Agamree is indicated for patients aged four years and older with DMD. The company expects the first sales to commence within this quarter. DMD is a rare progressive genetic disorder that primarily affects boys. It causes muscle weakness and degeneration, leading to loss of mobility and premature death. Standard treatment typically involves corticosteroids to manage inflammation and preserve muscle strength, but these therapies are associated with significant side effects like growth stunting and bone health issues. England’s National Institute for Health and Care Excellence (NICE) issued a positive final guidance for Agamree on 16 January 2025, following confirmation that no appeals were submitted against its December 2024 final draft guidance . This represents a reversal of NICE’s initial decision in March 2024, when the agency declined to recommend the drug due to concerns about its cost-effectiveness compared to standard-of-care corticosteroids. Subsequent submissions from Santhera addressed these issues, with NICE acknowledging that Agamree may offer fewer side effects than current options. Agamree is a first-in-class anti-inflammatory drug that binds to the same receptor as glucocorticoids but modulates its downstream activity differently, reducing the risk of corticosteroid-associated toxicities. The drug was designed as an alternative to traditional corticosteroids to mitigate side effects such as stunted growth and bone metabolism issues. The pivotal VISION-DMD study (NCT03439670) demonstrated that boys treated with Agamree maintained normal bone growth patterns and showed no signs of reduced bone metabolism or mineralisation after 48 weeks, unlike those treated with the corticosteroid prednisone. Patients transitioning from corticosteroids to Agamree reportedly recovered growth and bone health while maintaining efficacy. Agamree received UK regulatory approval in January 2024, building on similar decisions by the US Food and Drug Administration (FDA) and European Commission (EC) in October 2023 and December 2023, respectively. While it is approved in the same indication in Europe, patients as young as two years old in the US have access to the treatment. Santhera sold US commercialisation rights for Agamree to Catalyst Pharmaceuticals in July 2023 in a deal worth up to $231m. According to GlobalData’s Pharma Intelligence Center, Agamree is forecast to generate up to $373m in 2030. GlobalData is the parent company of Pharmaceutical Technology. Other approved treatments for DMD include Sarepta’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl). Initially approved via the accelerated pathway in 2023 for ambulatory boys aged between four and five years, Elevidys’ label was expanded in June 2024 to all DMD patients over four years of age with confirmed DMD mutations. Among others in the same space, US company Regenxbio is also planning a biologics licence application (BLA) filing in 2026 for RGX-202, its investigational DMD gene therapy, following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial (NCT05693142).