Fenebrutinib

PRESS RELEASE AB SCIENCE RECEIVES JAPANESE PATENT PROTECTION FOR THE USE OF MASITINIB IN PROGRESSIVE FORMS OF MULTIPLE SCLEROSIS (MS) UNTIL 2041 THIS IS THE FIRST COUNTRY DELIVERING THE MS PATENT MASITINIB HAS UNIQUE AND COMPETITIVE POSITIONING IN PROGRESSIVE FORMS OF MULTIPLE SCLEROSIS DUE TO ITS DISTINCTIVE SAFETY AND EFFICACY PROFILE AND MECANISM OF ACTIONS TARGETING BOTH MICROGLIA AND MAST CELLS Paris, January 21, 2026, 6pm CET AB Science SA (Euronext - FR0010557264 - AB) today announced that the Japanese Patent Office has formally granted a patent for methods of treating progressive multiple sclerosis (MS) with its lead compound masitinib. This new patent (JP 7788154) ensures intellectual property protection for masitinib until February 2041. This is the first country to deliver the patent to protect the use of masitinib in progressive forms of MS. AB Science has followed for the protection of masitinib in progressive forms of MS the same methodology as for the use of masitinib in ALS. This last patent was granted everywhere in the world. AB Science is optimistic in its chance to receive the protection of the use of masitinib in progressive MS worldwide. The same secondary medical use patent strategy is pursued for several indications, including amyotrophic lateral sclerosis until 2037 (with 5 years possible extension), progressive forms of MS and Alzheimer’s Disease until 2041, sickle cell disease until 2040, and prostate cancer until 2042, severe mastocytosis until 2036. Masitinib unique and competitive positioning in progressive forms of MS, both primary progressive multiple sclerosis (PPMS) and non-active secondary progressive multiple sclerosis (nSPMS) Masitinib is positioned as a treatment of progressive MS patients, including the subvariants of PPMS and nSPMS. The development of masitinib in progressive forms of multiple sclerosis is based on the positive phase 2b/3 study (AB07002) and confirmatory phase 3 MAXIMS study (AB20009) Study AB07002 (656 enrolled patients) achieved its primary analysis endpoint, showing a statistically significant reduction in the cumulative change in EDSS with masitinib 4.5 mg/kg/day (p=0.0256) [1]. This treatment effect was consistent for both PPMS and nSPMS. Masitinib significantly reduced the risk of first disability progression by 42% and improved manual dexterity, as measured by the 9-hole peg test (p=0.0388). Furthermore, the risk of reaching an Expanded Disability Status Scale (EDSS) score of 7.0, indicating a level of disability severe enough to confine the patient to a wheelchair, was significantly reduced (p=0.0093). Masitinib’s safety was consistent with the known risk pro masitinib, with no increased risk of infection. Study AB20009 (MAXIMS) is a randomized, double-blind, phase 3 study of masitinib 4.5 mg/kg/day in patients with PPMS and nSPMS. The objective of this study is to confirm the positive results of study AB07002. Study AB20009 will enroll patients with an EDSS score of 3.0-6.0, disease progression within 2 years of randomization, and the absence of T1 Gadolinium-enhancing brain lesions. The primary endpoint of this study is the effect of masitinib on the time to confirmed disability progression over 96 weeks, with progression defined as a 1-point worsening when the EDSS baseline score is ≤5.5 or a 0.5-point worsening if the EDSS baseline score is >5.5. Masitinib safety pro based on a large database The safety pro masitinib is well characterized based on a safety population of more than 4,300 patients, including close to 2,000 patients receiving masitinib for more than 6 months and 1,200 patients receiving masitinib for more than a year. Interestingly, masitinib does not target B-cells, unlike BTK inhibitors, another class of tyrosine kinase inhibitors developed for multiple sclerosis. Targeting B cells, which are not a therapeutic target in progressive forms of multiple sclerosis, is associated with an increased risk of infection and immunological changes. Safety population Patients exposed to Masitinib All ≤ 3 months More than 3 months More than 6 months More than 1 year More than 2 years All 4,318 1,674 2,644 1,924 1,255 560 Healthy volunteers 96 96 0 0 0 0 Non-oncology subjects 2,184 565 1,619 1,307 958 453 Oncology subjects 2,038 1,013 1,025 617 297 107 ICH Topic E1 requirements for non-orphan drugs 1,500 300-600 100 Masitinib is the first in class and only drug in phase 3 designed to specifically target both mast cells and microglia, with strong evidence that this dual targeting the innate immune system is an effective strategy for the treatment of progressive forms of multiple sclerosis Masitinib is a first-in-class oral tyrosine kinase inhibitor, capable of slowing functional decline in progressive MS by selectively targeting the innate immune system through both mast cells and microglia, and without any apparent negative impact on B-cell or T-cell activity. Masitinib has demonstrated the ability to decrease serum neurofilament light chain (NfL) concentration in an animal model of MS, and by extension, possibly neuronal damage [2]. Masitinib targets microglia and mast cells, both of which play crucial roles in progressive MS and the experimental autoimmune encephalomyelitis (EAE) model of MS, as evidenced by numerous publications [3-13]. There is a strong medical need for the progressive forms of multiple sclerosis Multiple Sclerosis is an autoimmune disease of the central nervous system that affects more than 100,000 people in France and for which no definitive treatment exists to date. It is characterized by progressive degradation of the nerve cells of the central nervous system by the patient's immune system and comes in two main forms. The relapsing-remitting form (RRMS) is characterized by relapses of the disease. During these relapses, patients experience the onset of new symptoms or worsening of existing symptoms. These flare-ups are usually followed by recovery periods of varying lengths, after which some symptoms may persist. Relapsing-remitting forms of multiple sclerosis are mostly associated with dysfunction of adaptive immunity 1 (B and T cells). The progressive form (PPMS and nSPMS) is characterized by a constant and regular worsening of the symptoms of the disease without a distinct relapse or period of recovery. The rate of onset of severe, disabling, and irreversible disability is much higher in the progressive forms of the disease than in the relapsing-remitting forms. In progressive multiple sclerosis, innate 2 immune cells, such as macrophages, microglia, and mast cells, play a major role. To date, the vast majority of treatments for the management of multiple sclerosis target the patient's adaptive immune system and, therefore, mainly apply to relapsing-remitting forms of the disease. However, patients with progressive forms of the disease currently account for approximately 50% of MS cases. The medical need in PPMS remains very high, following mixed results from BTK inhibitors in multiple sclerosis. Tolebrutinib (Sanofi) failed in phase 3 studies in RRMS and PPMS, while showing a positive result in nSPMS that nonetheless resulted in an FDA Complete Response Letter due to significant safety concerns, including a high risk of fatal drug-induced liver injury. Evobrutinib (Merck/EMD Serono) and fenebrutinib (Roche/Genentech) failed to demonstrate efficacy in RRMS Phase 3 trials, with fenebrutinib showing only non-inferiority to ocrelizumab in PPMS. Consequently, evobrutinib development was discontinued. Remibrutinib (Novartis) is the only BTK inhibitor with ongoing phase 3 trials in RRMS. References [1] Vermersch P, et al. Efficacy and Safety of Masitinib in Progressive Forms of Multiple Sclerosis: A Randomized, Phase 3, Clinical Trial. Neurol Neuroimmunol Neuroinflamm. 2022 Feb 21;9(3):e1148. [2] Hermine O, et al. Tyrosine kinase inhibitor, masitinib, limits neuronal damage, as measured by serum neurofilament light chain concentration in a model of neuroimmune-driven neurodegenerative disease. PLoS One. 2025;20(5):e0322199. [3] Sandhu JK, Kulka M. Decoding Mast Cell-Microglia Communication in Neurodegenerative Diseases. Int J Mol Sci. 2021 Jan 22;22(3):1093. [4] Pinke KH, et al. Should mast cells be considered therapeutic targets in multiple sclerosis? Neural Regen Res. 2020 Nov;15(11):1995-2007. [5] Pinke KH, et al. Calming Down Mast Cells with Ketotifen: A Potential Strategy for Multiple Sclerosis Therapy? Neurotherapeutics. 2020 Jan;17(1):218-234. [6] Brown MA, Weinberg RB. Mast Cells and Innate Lymphoid Cells: Underappreciated Players in CNS Autoimmune Demyelinating Disease. Front Immunol. 2018;9:514. [7] Skaper SD, Facci L, Zusso M, Giusti P. An Inflammation-Centric View of Neurological Disease: Beyond the Neuron Front Cell Neurosci. 2018;12:72. [8] Hendriksen E, et al. Mast cells in neuroinflammation and brain disorders Neurosci Biobehav Rev. 2017;79:119-133. [9] Elieh-Ali-Komi D, Cao Y. Role of Mast Cells in the Pathogenesis of Multiple Sclerosis and Experimental Autoimmune Encephalomyelitis. Clin Rev Allergy Immunol. 2017;52(3):436-445. [10] Conti P, Kempuraj D. Important role of mast cells in multiple sclerosis. Mult Scler Relat Disord. 2016;5:77-80. [11] Skaper SD, Facci L, Giusti P. Mast cells, glia and neuroinflammation: partners in crime?. Immunology. 2014;141(3):314-327. [12] Skaper SD, et al. Microglia and mast cells: two tracks on the road to neuroinflammation. FASEB J. 2012;26(8):3103-3117. [13] Zappulla JP, Arock M, Mars LT, Liblau RS. Mast cells: new targets for multiple sclerosis therapy?. J Neuroimmunol. 2002;131(1-2):5-20. About AB Science Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science’s website: . Forward-looking Statements - AB Science This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB Science Financial Communication & Media Relations investors@ab-science.com 1 Adaptive immunity corresponds to the immune protection that an individual builds over the course of his or her life according to the pathogens to which his or her organism is exposed. 2 An individual's innate immunity represents his immune protection from birth. Attachment MS Patent JP VENG VF

原文作者：BioPlus编辑部；老狼略有删改。 前 言 摩根大通医疗健康大会（J.P. Morgan Healthcare Conference）是全球规模最大、最具影响力的医疗健康投资与产业合作峰会，被誉为行业的“春晚”和风向标。2026年的第44届大会于1月12日至15日在美国旧金山举行，汇聚了全球顶尖药企、数千家生物科技公司及超过8000名行业领袖与投资者。在 JPM Healthcare Conference 2026 上，多家跨国药企明确了2026年的战略重点与执行重心。本文选取 Pfizer、Merck、Sanofi、Roche、Novartis 与 Bristol Myers Squibb 等代表性 MNC，总结各家公司在2026年的优先事项、资源配置方向等信息。 01 Pfizer 在JPM 2026 上，Pfizer将其2026年战略重点明确聚焦于四个方面：最大化已完成关键交易的价值、按期交付核心研发里程碑、为 2028 年后的增长持续投资，以及在全业务范围内规模化应用AI。在执行层面，公司强调通过系统性的生产力提升推动经营利润率扩张，包括持续推进制造优化与成本重整计划。根据披露，Pfizer已超额完成 2025 年既定目标，并预计到2026年实现大部分约72亿美元的净成本节省，其中约5亿美元将被再投资于研发体系，以提升研发效率与产出质量，体现出“降本—再投资—支撑中长期增长”的明确资本配置逻辑。02 Novartis Novartis重申其长期一致且聚焦的战略路径：围绕心血管-肾-代谢、免疫、神经科学和肿瘤四大核心治疗领域，依托化学与生物药两大基础平台，并重点推进 xRNA、放射性配体疗法（RLT）以及基因与细胞治疗等新技术方向，同时将美国、中国、德国和日本作为优先市场。在战略重点上，公司强调一方面加速增长并兑现回报，通过高价值药物与上市执行力推动业绩；另一方面夯实长期基础，包括释放组织潜能、规模化数据科学与技术能力，并强化社会信任。在执行层面，Novartis 将重点放在运营卓越、资源配置灵活性以及研发效率提升，以支撑其通过技术领导力持续输出高价值创新药物的长期目标。03 Bristol Myers Squibb BMS 将其战略重点明确为以变革性药物构建长期增长能力：聚焦治疗危及生命疾病的高价值创新，推动 Growth Portfolio 的执行与动能释放，并在严格的财务纪律与以股东回报为导向的资本配置框架下，支持研发与业务拓展。在研发层面，公司以 “Science + Execution = Value” 为核心方法论，从资产导向转向疾病导向的组合管理，强化每项研究的成功概率，通过 AI 与技术赋能提升速度与效率，并将资源集中投向最具确定性的机会，以持续交付可转化为长期增长的创新疗法。04 Roche Roche Pharma 明确其 2026 年的核心重点在于在保持商业动能的同时推进组合焕新。公司计划通过三条主线实现增长延续：其一，最大化现有在售产品组合的价值，依托 17 款重磅产品支撑增长至 2028 年；其二，确保关键新品顺利上市，以 Gazvya、giredestrant、fenebrutinib、vamikibart 和 satralizumab 等为代表，推动 2026 年起的新一轮增长动能；其三，在 BAR 后时代加速新分子实体（NME）推进，重点布局阿尔茨海默病、肥胖等大规模疾病领域。公司同时强调“商业端的纪律性与科学端的严谨性”并行推进，并在展望中指出，2026 年将迎来显著的临床与注册性数据读出节奏，以支持持续输出具备 FIC/BIC 潜力的转化型药物。05 Merck & Co. Merck 将战略重点清晰聚焦于通过下一波创新实现组合转型：一是持续推进以科学为驱动的BD，主动获取具备长期价值创造潜力的外部创新；二是加速新增长引擎的落地，预计未来几年将带来 20 余项新产品/新适应症上市，其中绝大多数具备重磅潜力；三是依托广泛且多元的后期管线持续推进研发执行，当前约 80 项 III 期研究在研。公司同时量化了这一战略的中长期目标，预计至 2030 年代中期，新增长驱动带来的潜在商业机会超过 700 亿美元，以支撑 KEYTRUDA 专利到期后的长期增长连续性。在执行层面，Merck 强调其已进入一个以结果交付为导向的管线推进阶段：一方面，通过持续加速与扩充研发管线，已形成 20 余个潜在新增长驱动；另一方面，公司正步入数据密集期（data-rich period），预计 至 2027 年将迎来多项具有实质影响力的 III 期临床读出。上述执行与节奏安排服务于公司长期使命，即通过推进前沿科学，在提升患者获益的同时创造长期股东价值，并为组合转型提供持续的数据与产品支撑。06 SanofiSanofi 将其近一年的战略进展概括为以研发驱动、AI 赋能的持续增长路径。公司强调通过主动的组合管理与资本配置来支撑长期发展。在研发与管线方面，公司于 2025 年推出 3 款新药/疫苗，多项关键项目取得积极临床数据，并启动新的 III 期研究。整体来看，Sanofi 将 2026 年定位为在保持盈利增长的同时，持续输出可验证管线进展的重要一年。