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October 1, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the positive outcome of the LIONHEART study, confirming vamorolone’s distinctive action also as a mineralocorticoid receptor antagonist, setting it apart from other corticosteroids. The LIONHEART study, an open-label randomized, placebo- and eplerenone-controlled study involving 30 healthy adult male subjects, met its primary endpoint. It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. This increased ratio in urine provides clinical evidence for vamorolone’s unique mode of action also as a mineralocorticoid receptor antagonist (MRA) in humans. Combined with its known properties as a dissociative glucocorticoid receptor agonist, these findings further differentiate vamorolone’s pharmacological profile, distinguishing it from other corticosteroids. Cardiac complications such as cardiomyopathy are a leading cause of morbidity and mortality in boys with Duchenne muscular dystrophy (DMD). While treatment with corticosteroids (and ACE inhibitors) has demonstrated a delay in the onset of cardiomyopathy, the addition of MRAs including eplerenone to standard of care has also shown an improvement in left ventricular systolic dysfunction, the benefit of which increases with earlier initiation [1-3]. “Mineralocorticoid receptor antagonists are strongly recommended but late when cardiac function is already reduced and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi, PhD, MD, Cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid” “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone,” stated Shabir Hasham, MD, Chief Medical Officer of Santhera. “We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterize long-term outcomes including any beneficial impact on cardiac complications in DMD.” 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 [4-7]. 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 [4-7]. 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 [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 been shown to be a dual mineralocorticoid antagonist and a dissociative glucocorticoid agonist based on animal experiments [7]. It has no restriction of growth [8] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [9]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom for the treatment of DMD. The LIONHEART study (SNT-I-VAM-026) is an open label randomized, 3-arm, parallel-group, placebo and eplerenone controlled study to evaluate the mineralocorticoid receptor antagonism (MRA) effect of vamorolone in 30 healthy adult male subjects after challenge with fludrocortisone, a known mineralocorticoid receptor agonist. The primary endpoint was defined as the ratio of sodium to potassium (Na/K) and the corresponding logarithm of the ratio (log10(10*Na/K) in urine at different time-points. Further analysis of the data is ongoing and will be presented at medical conferences. References: [1] Birnkrant et al. (2018) Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. [2] Raman et a. (2015) Lancet Neurol 14: 153–161 doi:10.1016/s1474-4422(14)70318-7 [3] Raman et al. (2017) Orphanet J Rare Dis 12: 39. doi:10.1186/s13023-017-0590-8 [4] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [5] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [6] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [7] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [8] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [9] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera 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), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.

BALLERUP, Denmark--( BUSINESS WIRE )-- NOT INTENDED FOR DISTRIBUTION IN THE UK LEO Pharma A/S, a global leader in medical dermatology, has today announced its submission of a New Drug Application (NDA) to the National Medical Products Administration (NMPA) for Enstilar ® (LEO 90100) to treat adult patients living with plaque psoriasis. Following a week of significant LEO Pharma treatment and data milestones in the EU and the U.S., today’s news of the NDA submission in China is the latest example of LEO Pharma’s global strategy in action across medical dermatology. If approved, China would join 50 other markets in the world to be approved to bring Enstilar to patients, and would be the largest country to date. Enstilar ® , a calcipotriol/betamethasone cutaneous foam, is an improved formulation of Daivobet ® ointment, which is one of the current standard treatment options in China for adults living with plaque psoriasis. 4 Both Daivobet ointment and Enstilar are LEO Pharma products. “ LEO Pharma strives to enhance patient care and elevate quality of life for those living with complex skin conditions like plaque psoriasis ,” said Byron Yin, General Manager for LEO Pharma, China. “ Today’s submission is a crucial step towards offering a much-needed new treatment option in our region, in addition to the existing portfolio offering from LEO Pharma China.” “ Today’s submission of our NDA for Enstilar is a testament to LEO Pharma’s truly global outlook and ambition in advancing dermatology, following on from a week of milestones across both the EU and U.S.” said Becki Morison, Executive Vice President, Global Product Strategy & International Operations for LEO Pharma. “ China is a key market for LEO Pharma, with dermatology growing rapidly in the country and our revenue steadily increasing through a strong performance from our team. We will continue to leverage our extensive dermatology expertise to enhance the treatments we can provide in the region, unwavering in our goal to provide support to those in China that need it most.” The NDA submission is supported by results from a phase 3 trial comparing the efficacy and safety of a once daily application with Enstilar and Daivobet ointment after 4 weeks of treatment in adult Chinese subjects with stable plaque psoriasis (N=604). 1,2 The trial achieved its primary and secondary endpoints, with Enstilar demonstrating superiority to Daivobet ointment. 2 “ The trial showed the potential that Enstilar has to improve the quality of life for millions of Chinese patients living with plaque psoriasis,” said Professor Zhang Jianzhong from Peking University People's Hospital, lead investigator of the phase 3 Enstilar trial. “ Through today’s milestone, we take a step towards new treatment options for the millions that live with plaque psoriasis in the near future.” Following the Enstilar submission, the Centre for Drug Evaluation (CDE) will validate the filing, before the full evaluation of the NDA is conducted by the CDE. The regulatory review process is expected to conclude in the first half of 2026. About Enstilar ® Enstilar ® is an aerosol spray foam containing calcipotriol monohydrate 50 mcg/g and betamethasone dipropionate 0.5 mg/g. In the EU, it is indicated for the treatment of psoriasis vulgaris in adults for up to 4 weeks. Patients who have responded at 4 weeks’ treatment using Enstilar once daily are suitable for long-term maintenance treatment. 4-7 About Psoriasis Psoriasis is a chronic, systemic inflammatory disease that primarily affects the skin in 125 million people worldwide. 8,9 Psoriasis is the result of skin barrier cell proliferation and the activation of cytokines (a family of proteins involved in immune responses) that cause inflammation. 10 About 80% to 90% of patients are affected by plaque psoriasis, the most common clinical form of psoriasis. 11 The symptoms of plaque psoriasis are itchy or painful raised scaly and inflamed plaques. Plaques may appear anywhere on the body, but often appear on the scalp, knees, elbows and torso. 11 About LEO Pharma LEO Pharma is a global company dedicated to advancing the standard of care for the benefit of people with skin conditions, their families and society. Founded in 1908 and majority owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, and today, the company offers a wide range of therapies for all disease severities. LEO Pharma is headquartered in Denmark with a global team of 4,200 people, serving millions of patients across the world. In 2023, the company generated net sales of DKK 11.4 billion. References ClinicalTrials.gov. National Library of Medicine (U.S.). A Study to Investigate Efficacy and Safety With LEO 90100 Compared With Daivobet® Ointment in Adult Chinese Subjects With Stable Plaque Psoriasis. Identifier: NCT05919082. https://clinicaltrials.gov/study/NCT05919082 . LEO Pharma Press Release. LEO Pharma Announces Topline Results of Phase 3 Trial in China for Enstilar® (LEO 90100) Demonstrating Superiority in Adult Chinese Subjects With Stable Plaque Psoriasis. Published: May 04, 2024. Last accessed: Sep 25, 2024. Available at: https://www.businesswire.com/news/home/20240507292614/en/LEO-Pharma-Announces-Topline-Results-of-Phase-3-Trial-in-China-for-Enstilar%C2%AE-LEO-90100-Demonstrating-Superiority-in-Adult-Chinese-Subjects-With-Stable-Plaque-Psoriasis . Ding X, Wang T, Shen Y, et al. Prevalence of psoriasis in China: a population-based study in six cities. Eur J Dermatol. 2012;22(5):663-667. Zhang, XJ et al., Chin J Dermatol, July 2023, Vol. 56, No. 7, Guideline for the diagnosis and treatment of psoriasis in China (2023 edition) Enstilar ® EU Summary of Product Characteristics July 2022. Koo J, Tyring S, Werschler WP, et al. Superior efficacy of calcipotriene and betamethasone dipropionate aerosol foam versus ointment in patients with psoriasis vulgaris--A randomized phase II study. J Dermatolog Treat. 2016;27(2):120-127. Leonardi C, et al. Efficacy and Safety of Calcipotriene Plus Betamethasone Dipropionate Aerosol Foam in Patients With Psoriasis Vulgaris—a Randomized Phase III Study (PSO-FAST). J Drugs Dermatol. 2015;14(12):1468-1477. Menter A, Korman NJ, Elmets CA, Feldman SR, Gelfand JM, Gordon KB, et al. Guidelines of care for the management of psoriasis and psoriatic arthritis. Section 3. Guidelines of care for the management and treatment of psoriasis with topical therapies. J Am Acad Dermatol 2009; 60: 643-659. National Psoriasis Foundation. Psoriasis Statistics. Last updated: Dec 21, 2022. Last accessed: Sep 25, 2024. Available at: https://www.psoriasis.org/content/statistics . Hawkes JE, Chan TC, Krueger JG. Psoriasis pathogenesis and the development of novel targeted immune therapies. J Allergy Clin Immunol. 2017;140(3):645-653. National Psoriasis Foundation. Plaque Psoriasis. Last updated: Feb 08, 2024. Last accessed: Sep 25, 2024. Available at: https://www.psoriasis.org/plaque/ . MAT-76612 September 2024

TAIPEI, Aug. 5, 2024 /PRNewswire/ -- Taiwan-based Formosa Pharmaceuticals ("Formosa", 6838.TWO) announced today that the company has entered into an exclusive licensing agreement with Apotex Inc. ("Apotex"), for exclusive rights in Canada to the commercialization of clobetasol propionate ophthalmic suspension, 0.05% (APP13007), a patented innovative medicine for the treatment of inflammation and pain following ocular surgery. Clobetasol propionate ophthalmic suspension, 0.05% (APP13007) was approved by the U.S. Food and Drug Administration (FDA) on March 4, 2024. The licensing deal will include certain payments, including an upfront payment and milestone payments upon achievement of certain regulatory and sales milestones. APP13007's active ingredient is the superpotent corticosteroid, clobetasol propionate, and is derived from Formosa Pharma's proprietary APNT® nanoparticle formulation platform. The novel formulation enables a convenient and straightforward dosing regimen (twice daily for 14 days) while providing rapid and sustained relief of inflammation and pain, which in Phase 3 trials proved statistically and clinically superior to its matching placebo (p<0.001). APP13007 will enter a ~$C50M market for topical ophthalmic steroids and steroid combinations, growing at around 5% annually, driven by an estimated ~500,000 ocular surgeries annually in Canada. "Formosa Pharma is honored to partner with Apotex, with its storied history and reputation for success," said Erick Co, President and CEO of Formosa Pharmaceuticals. "Their commitment to branded ophthalmology products gives us full confidence that APP13007 will flourish in the Canadian market. We look forward to working with the Apotex team in providing this differentiated therapy to Canadian physicians and patients." "We are pleased to provide Canadian patients with APP13007, a new treatment option for post-operative inflammation and pain following ocular surgery," said Allan Oberman, President & CEO, Apotex. "Expanding our portfolio of innovative medicines, including our ophthalmic portfolio, along with our commercial infrastructure, enables us to support more patients along their journey to health." About Formosa Pharmaceuticals, Inc. Formosa Pharmaceuticals, Inc. (6838.TWO) is a clinical stage biotechnology company with primary focus in the areas of ophthalmology and oncology. The company's proprietary nanoparticle formulation technology (APNT®), through which APP13007 was developed, improves the dissolution and bioavailability of APIs for topical, oral, and inhaler administration. Resulting formulations have high uniformity, purity, and stability, thereby allowing the utilization of poorly soluble or extremely potent drug agents which otherwise may face insurmountable challenges in delivery and penetration to target tissues. For more details about Formosa Pharma and APNT®, visit . About Apotex Inc. Apotex is a Canadian-based global health company. We improve everyday access to affordable, innovative medicines and health products for consumers worldwide, with a broad portfolio of generic, biosimilar, and innovative branded pharmaceutical products. Headquartered in Toronto, with regional offices globally, including in the United States, Mexico and India, we are the largest Canadian-based pharmaceutical company and a health partner of choice for the Americas for pharmaceutical licensing and product acquisitions. Learn more about us at . SOURCE Formosa Pharmaceuticals Inc.,