Core Viewpoint - The stock of Valiant Pharmaceuticals-B (09887) has increased by over 5%, currently trading at 55.2 HKD with a transaction volume of 18.0035 million HKD, following the announcement of orphan drug designation for its dual-specific antibody, Viligene (Oparatuzumab, LBL-024) for the treatment of pulmonary neuroendocrine carcinoma by the European Commission [1] Group 1 - Valiant Pharmaceuticals-B's stock rose by 4.94% to 55.2 HKD as of the report [1] - The company reported a transaction volume of 18.0035 million HKD [1] - The European Commission granted orphan drug designation for Viligene on January 22, 2026, marking a significant milestone in its global development [1] Group 2 - This designation follows the previous orphan drug recognition by the U.S. Food and Drug Administration (FDA) [1] - The dual-specific antibody Viligene is aimed at treating pulmonary neuroendocrine carcinoma [1] - The orphan drug designation is a critical step in the drug's development process, enhancing its potential marketability and support [1]

Core Viewpoint - The European Commission has granted orphan drug designation to Viligene (Opalizumab, LBL-024) for the treatment of extra-pulmonary neuroendocrine carcinoma, marking a significant milestone in its global development process after receiving similar recognition from the FDA [1][2]. Group 1 - The orphan drug designation is intended for medicines used to diagnose, prevent, or treat life-threatening or chronically debilitating diseases with a prevalence of less than 5 in 10,000 in the EU [2]. - The designation provides several key incentives, including scientific advice from the European Medicines Agency (EMA) on research design related to drug quality, benefits, and risks [2]. - The orphan drug status grants a 10-year market exclusivity period in the EU, during which no similar drugs for the same indication can be approved, ensuring protection even without patent coverage [2]. Group 2 - Additional benefits of orphan drug designation include fee reductions for regulatory guidance, marketing authorization applications, and post-marketing changes [2]. - Companies may also access funding from the European Commission, Horizon Europe, and other sources upon receiving orphan drug designation [2]. - Extra incentives are available for micro, small, and medium-sized enterprises, including regulatory, administrative, and procedural assistance, as well as fee reductions [2].

根据EC的定义，孤儿药是拟用于诊断、预防或治疗危及生命或慢性衰弱性疾病且患病率在欧盟低于万 分之五的药品。授予孤儿药认定提供多项关键激励措施，包括但不限于：(1)方案指导，据此，欧洲药 品管理局("EMA")提供涵盖药品质量、获益及风险等不同方面相关研究设计意见的科学建议；(2)欧盟市 场十年独占权，据此，自上市许可批准日期起，授予10年的市场独占期，期间，欧盟及其成员国的监管 部门不得批准其他公司针对相同适应症的类似药品，且该市场独占权与专利保护分别施行，即使在无任 何专利的情况下亦维持有效；(3)监管费用减免，包括方案指导、上市许可申请及核查、上市后变更及 年度维护相关费用减免；(4)获取资金，获授予孤儿药认定后，可自EC、Horizon Europe及其他来源获得 资金；及(5)针对微型、小型及中型企业的额外激励措施，包括监管、行政及程序性援助以及费用减 免。 格隆汇1月22日丨维立志博-B(09887.HK)公告，公司欣然宣布于2026年1月22日，欧盟委员会("EC")发布 关于PD-L1/4-1BB双特异性抗体维利信™(奥帕替苏米单抗，LBL-024)用于治疗肺外神经内分泌癌获授 孤儿药认定的20 ...

Core Viewpoint - The company Valiant Biosciences-B (09887) has received orphan drug designation from the European Commission (EC) for its PD-L1/4-1BB bispecific antibody, Velesin™ (Oparizumab, LBL-024), for the treatment of pulmonary neuroendocrine carcinoma, marking a significant milestone in its global development process [1]. Group 1 - The orphan drug designation was granted on January 9, 2026, following a similar recognition from the U.S. Food and Drug Administration (FDA) [1]. - Orphan drugs are defined by the EC as those intended for the diagnosis, prevention, or treatment of life-threatening or chronically debilitating conditions with a prevalence of less than 5 in 10,000 in the EU [2]. Group 2 - The orphan drug designation provides several key incentives, including scientific advice from the European Medicines Agency (EMA) on research design related to drug quality, benefits, and risks [2]. - It grants a 10-year market exclusivity period in the EU from the date of marketing authorization, during which no similar drugs for the same indication can be approved [2]. - Additional benefits include fee reductions for regulatory guidance, marketing authorization applications, and post-marketing changes, as well as potential funding from the EC and Horizon Europe [2].

Core Viewpoint - The company announced that its PD-L1/4-1BB bispecific antibody, Velesin™ (Oparizumab, LBL-024), received orphan drug designation from the European Commission for the treatment of pulmonary neuroendocrine carcinoma, marking a significant milestone in its global development process after previously receiving similar recognition from the FDA [1]. Group 1 - The European Commission's orphan drug designation is granted to drugs intended for the diagnosis, prevention, or treatment of life-threatening or chronically debilitating conditions with a prevalence of less than 5 in 10,000 in the EU [2]. - The orphan drug designation provides several key incentives, including scientific advice from the European Medicines Agency (EMA) on research design related to drug quality, benefits, and risks [2]. - The designation grants a 10-year market exclusivity period in the EU from the date of marketing authorization, during which no similar drugs for the same indication can be approved [2]. Group 2 - Additional incentives include fee reductions for regulatory guidance, marketing authorization applications, and post-marketing changes, as well as access to funding from the EC, Horizon Europe, and other sources [2]. - There are also extra incentives for micro, small, and medium-sized enterprises, including regulatory, administrative, and procedural assistance, along with fee reductions [2].

香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不 負 責，對 其 準 確 性 或 完 整 性 亦 不 發 表 任 何 聲 明，並 明 確 表 示，概 不 對 因 本 公 告 全部或任何部份內容而產生或因倚賴該等內容而引致的任何損失承擔任何責 任。 Nanjing Leads Biolabs Co., Ltd. 南 京 維 立志博生物科技股份有限公司 （於 中 華 人 民 共 和 國 成 立 的 股 份 有 限 公 司） （股 份 代 號：9887） 自願公告 維利信™（PD-L1/4-1BB雙特異性抗體 奧 帕 替 蘇 米 單 抗，LBL-024） 用於治療肺外神經內分泌癌 獲歐盟委員會授予孤兒藥認定（ODD） 本公告由南京 維 立志博生物科技股份有限公司（「本公司」）自 願 作 出，以 告 知 本 公 司 股 東 及 潜 在 投 資 者 有 關 本 公 司 的 最 新 業 務 發 展 情 況。 本公司欣然宣布於2026年1月22日，歐 盟 委 員 會（「EC」）發布關於PD-L1/4-1BB雙 特 異性抗體 維利信™（奧 帕 替 蘇 米 單 抗，LBL-024）用於治療肺外神經內分泌癌獲 ...

Group 1 - Sanofi's investigational BTK inhibitor, rilzabrutinib, received orphan drug designation from the European Medicines Agency (EMA) for treating IgG4-related disease (IgG4-RD) [1][7] - IgG4-RD is a chronic, immune-mediated rare condition that can affect multiple organs, leading to inflammation, swelling, and fibrosis [1] - Rilzabrutinib is currently undergoing a phase II study for IgG4-RD, showing a reduction in disease flare and glucocorticoid sparing over 52 weeks [2][7] Group 2 - Rilzabrutinib is also being developed for immune thrombocytopenia (ITP) and has orphan drug designation for this indication in the United States, EU, and Japan [3] - A regulatory application for rilzabrutinib's approval for ITP is under review in the U.S., with a decision expected by August 29, 2025 [4] - The FDA has granted orphan drug designation to rilzabrutinib for warm autoimmune hemolytic anemia (wAIHA) and sickle cell disease (SCD) [5] Group 3 - Upon potential approval, rilzabrutinib may face competition from existing therapies, such as Rigel Pharmaceuticals' Tavalisse, which recorded $68.5 million in sales in the first half of 2025, a 44% increase year-over-year [6] - Amgen's Uplizna became the first FDA-approved drug for IgG4-RD in April 2025, following its acquisition of Horizon Therapeutics [8]

Core Insights - The FDA has granted Orphan Drug Designation (ODD) to NeuroNOS's lead investigational therapy, BA-102, for treating Phelan-McDermid Syndrome (PMS), a condition associated with Autism Spectrum Disorder (ASD) [1][3] - NeuroNOS plans to initiate first-in-human clinical trials for ASD in the U.S. in 2026 [1] Company Overview - NeuroNOS is a biopharmaceutical company focused on developing treatments for autism and Alzheimer disorders, and is a subsidiary of Beyond Air [1][5] - The company specializes in therapies based on small molecules that can cross the blood-brain barrier to regulate Nitric Oxide (NO) levels in the brain [5] Disease Background - Phelan-McDermid Syndrome (PMS) is a rare genetic disorder primarily caused by deletions or mutations affecting the SHANK3 gene, leading to symptoms such as global developmental delay, intellectual disability, and severe speech impairments [2][4] - There are currently no FDA-approved treatments specifically indicated for PMS [2] Development Incentives - The ODD provides several key development incentives, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, waiver of FDA application fees, and access to FDA protocol assistance [3] Commitment to Development - NeuroNOS is committed to collaborating with the FDA, patient advocacy groups, scientific foundations, and clinical investigators to accelerate the development of therapies for PMS [4]