Vamorolone

Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and China New five-year data on vamorolone highlighted substantially improved safety pro reduced vertebral fractures, lower incidence of cataracts and that normal growth rate was maintained. With adverse effects being a key limitation of conventional corticosteroid therapy, vamorolone’s safety pro its comparable effectiveness, has the potential to displace existing steroid treatments Transaction advances Nxera’s mission to bring innovative medicines to patients in Japan/APAC in line with 2030 vision to build high-growth, highly profitable Japanese biopharma company Tokyo, Japan and Cambridge, UK, 8 January 2026 – Nxera Pharma Co. Ltd (“Nxera” or “the Company; TSE 4565) today announces that it has entered an exclusive licensing agreement for the development, manufacturing and commercialization of vamorolone for the treatment of Duchenne Muscular Dystrophy (DMD) in Japan, South Korea, Australia and New Zealand with Santhera Pharmaceuticals Holding (“Santhera”) (SIX: SANN). Vamorolone is approved and marketed as AGAMREE® for the treatment of DMD, a rare inherited neurodegenerative disease, in the US, European Union, UK and China. The addition of vamorolone brings into Nxera’s portfolio of innovative medicines for rare and specialty diseases, a late-stage development candidate with the potential to address significant unmet needs of patients in Japan and the Asia-Pacific (“APAC”) region living with DMD. This portfolio includes PIVLAZ™ (clazosentan) and QUVIVIQ™ (daridorexant), both of which are approved and marketed in Japan and under commercial development in other APAC countries. Under the terms of the agreement, Nxera will be responsible for obtaining regulatory approval of vamorolone in the licensed territories, including undertaking regulatory activities such as clinical trials where required, and will lead commercialization and manufacturing activities for those territories. This strategic partnership leverages Nxera's proven commercial capabilities and development and regulatory expertise across the APAC region, particularly in Japan. In addition, Nxera’s team brings significant prior development and manufacturing experience with vamorolone through its 2023 acquisition of Idorsia’s Japan and APAC business. Christopher Cargill, President and Chief Executive Officer of Nxera, commented: “We are excited to partner with Santhera to bring vamorolone to DMD patients in Japan, South Korea, Australia and New Zealand. Vamorolone’s differentiated safety and efficacy pro the potential to fundamentally change the standard of care by enabling early use, full dosing, and long-term treatment, addressing critical limitations of existing steroid therapies currently used in the region. “Furthermore, the transaction expands our portfolio of late- and commercial-stage products for Japan/APAC and advances our mission to bring innovative medicines to patients in these important regions in line with our 2030 vision to build high-growth, highly profitable Japanese biopharma company.” Dario Eklund, Chief Executive Officer of Santhera, added: “This strategic partnership represents a significant milestone in our mission to expand global access to AGAMREE® (vamorolone) and bring meaningful new treatment options to patients with DMD worldwide. Nxera's deep expertise and established infrastructure in Japan and across the wider APAC region, as well as prior experience of vamorolone, make them an ideal partner to unlock the full commercial and clinical potential of AGAMREE® (vamorolone) in these markets and accelerate access for patients with DMD.” Additional transaction terms In return for development, manufacturing and commercialization rights for vamorolone in the licensed territories, Nxera will make an upfront payment of USD 40 million to Santhera, consisting of USD 30 million in cash and USD 10 million as a strategic equity investment. Santhera is also eligible to receive sales and regulatory milestone payments of up to USD 165 million from Nxera as well as tiered royalties starting in the low teens on net sales of vamorolone in the licensed territories. About Duchenne Muscular Dystrophy 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. About vamorolone Vamorolone 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 vamorolone 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, vamorolone 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. Five-year data from the GUARDIAN study highlighted substantially improved safety pro reduced vertebral fractures, lower incidence of cataracts and that normal growth rate was maintained. Currently available data show that vamorolone, 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 . –END– About Nxera Pharma Nxera Pharma is a technology powered biopharma company in pursuit of new specialty medicines to improve the lives of patients with unmet needs in Japan and globally. The Company has built an agile, new-generation commercial business in Japan to develop and commercialize innovative medicines, including several launched products, to address this high-value, large and growing market and those in the broader APAC region. In addition, and powered by its unique NxWave™ GPCR structure-based drug discovery platform, the Company is advancing an extensive pipeline internally and in partnership with leading pharma and biotech companies. Nxera Pharma operates at key locations in Tokyo and Osaka (Japan), London and Cambridge (UK), Basel (Switzerland) and Seoul (South Korea) and is listed on the Tokyo Stock Exchange (ticker: 4565). For more information, please visit LinkedIn: @NxeraPharma | X: @NxeraPharma | YouTube: @NxeraPharma QUVIVIQ™ is trademark of Idorsia Ltd. About Santhera 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 Commission (EC), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA), in Hong Kong by the Department of Health (DoH) and in Canada by Health Canada. 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 . AGAMREE® is a trademark of Santhera Pharmaceuticals. Enquiries: For Nxera Pharma Media and Investor Relations Shinya Tsuzuki, VP, Head of Investor Relations Maya Bennison, Communications Manager +81 (0)3 5962 5718 | +44 (0)1223 949390 | IR@Nxera.life MEDiSTRAVA (for International Media) Mark Swallow, Frazer Hall, Erica Hollingsworth +44 (0)203 928 6900 | Nxera@medistrava.com For Santhera Catherine Isted, Chief Financial Officer IR@santhera.com ICR Healthcare Santhera@icrhealthcare.com Forward-looking statements This press release contains forward-looking statements, including statements about the discovery, development, and commercialization of products. Various risks may cause Nxera Pharma Group’s actual results to differ materially from those expressed or implied by the forward looking statements, including: adverse results in clinical development programs; failure to obtain patent protection for inventions; commercial limitations imposed by patents owned or controlled by third parties; dependence upon strategic alliance partners to develop and commercialize products and services; difficulties or delays in obtaining regulatory approvals to market products and services resulting from development efforts; the requirement for substantial funding to conduct research and development and to expand commercialization activities; and product initiatives by competitors. As a result of these factors, prospective investors are cautioned not to rely on any forward-looking statements. We disclaim any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

❒ FDA与NDA提交背景 2022年3月29日，Santhera制药与ReveraGen生物医药联合宣布，计划向美国食品药品管理局（FDA）逐步提交vamorolone针对杜氏肌营养不良（DMD）的新药上市申请（NDA）。此前，该药物已获得美国FDA的“快速通道认定”。同时，曙方医药于2022年1月获得了在大中华区独家开发和商业化vamorolone用于DMD及其他罕见病适应症的权益。 在2021年11月与美国食品药品管理局（FDA）成功举办pre-NDA会议后，Santhera和ReveraGen已启动NDA申请的滚动提交工作。FDA在会议中指出，申请方所提供的临床疗效与安全性数据足以支持vamorolone在杜氏肌营养不良（DMD）治疗领域的NDA申请。该申请的最终接受与否将取决于FDA对完整申报文件的详细审查。 ❒ vamorolone的国际申请及预期审评 预计在2022年第二季度，Santhera和ReveraGen将完成NDA申请材料的全部递交。根据FDA的审查时间表，预计在2022年8月将发出关于接受申请审查的通知，同时FDA还将决定是否授予vamorolone优先审评资格。一旦获得此资格，将有望缩短审查周期，使药物最早可能在2023年第一季度获得批准。 若vamorolone在美国获得FDA批准，Santhera计划将首先在美国市场上市该药物。同时，在欧洲方面，Santhera也计划于2022年第三季度向欧洲药品管理局（EMA）递交vamorolone用于DMD治疗的上市许可申请（MAA）。假设审核时间约为一年，且报销谈判顺利通过，则有望在2023年下半年获得欧洲市场的批准并上市。Santhera预计德国可能成为欧洲首个上市的国家。 ❒ 孤儿药与研发背景 值得注意的是，vamorolone已在美国和欧洲获得治疗DMD的孤儿药认定，并荣获美国FDA的快速通道与儿科罕见疾病认定，以及英国药监局（MHRA）的潜力创新药物（PIM）认定。尽管目前该药物尚处于临床试验阶段，尚未获得任何卫生监管部门的正式批准，但已显示出巨大的治疗潜力。 ❒ VISION-DMD临床研究 VISION-DMD是一项2b期临床研究，旨在评估vamorolone在杜氏肌营养不良（DMD）治疗中的有效性和安全性。该研究包含一个双盲、为期24周的关键临床环节，以及随后的24周研究以进一步确认疗效的持久性，并收集长期安全性和耐受性数据。共有121名4至7岁的DMD患儿参与了这项研究。在治疗的第24周（第一阶段），与安慰剂相比，vamorolone（6毫克/公斤/天）组在“由卧位至站立所需时间”这一主要终点上表现出显著优势（p=0.002）。同时，该组还达到了多个次要疗效终点，且与泼尼松相比无统计学差异。在接下来的48周研究中，所有参与者均接受了vamorolone的治疗。原安慰剂组和泼尼松组的参与者被随机分配至不同剂量的vamorolone，而vamorolone组则继续使用原有剂量。结果显示，vamorolone 6毫克/公斤/天剂量组在长达48周的时间内，多个临床终点均持续显示出24周的疗效。此外，该研究还确认了vamorolone的优良耐受性，为其作为目前标准治疗的潜在替代方案提供了有力支持。 ❒ 曙方医药的角色 曙方医药，这家成立于2019年的创新型企业，专注于中国罕见病药物研发与商业化。其治疗领域广泛，涵盖神经肌肉病、肺循环疾病及遗传代谢病等，致力于为医患提供临床可及的可靠产品与服务。通过构建符合中国罕见病市场特点的创新商业模式，曙方医药努力成为行业的佼佼者。企业得到了知名生物医药投资基金礼来亚洲基金和晨兴创投的支持，展现了其强大的实力与潜力。 ❒ Santhera制药的定位和研究方向 Santhera制药，这家瑞士专科制药企业，专注于罕见神经肌肉病和肺部疾病的创新药物研发与商业化。其产品管线丰富，包括正在进行DMD关键临床试验的Vamorolone、处于临床阶段治疗囊性纤维化及其他中性粒细胞肺病的Lonodelestat，以及一款探索性基因疗法用于治疗先天性肌营养不良。此外，Santhera已将其首款获批产品Raxone®（艾地苯醌）在北美和法国以外市场的权益授予凯西制药集团，用于治疗Leber遗传性视神经病变（LHON）。 ❒ 至爱进行性肌营养不良关爱中心的使命和成就 在至爱，我们秉持着一种信念：患者的命运并非不可改变，通过积极营造有利的社会环境，我们能为患者带来更多的医疗、教育及就业机会。与疾病共存，保持乐观，是提升患者及其家庭生活质量、延长患者寿命的关键。 至爱进行性肌营养不良关爱中心长期与多个社会福利、教育及儿童慈善机构，以及罕见病患者组织保持紧密合作，共同推动DMD相关的公益项目，充分利用社会资源，为全国各地DMD患者及其家庭提供支持。自2011年起，我们便与北京协和医院、解放军总医院第三医学中心（原武警总医院）、北京儿童医院、北京大学第一医院等三甲医院合作，开展丰富多彩的患者教育活动。2012年至今，我们与原武警总院等机构携手，汇聚各方力量，已成功举办了六届DMD全国医患交流大会。作为DMD联合门诊（MDT）的倡导者和推动者，我们与北京儿童医院、原武警总医院、北京大学第一医院等医疗机构紧密合作，为患者提供全方位的支持。我们致力于推动各地医院开展DMD联合门诊，为DMD的标准化治疗贡献力量，同时为全国范围内的DMD家长和患者提供专业的技术支持。