Ozanimod Hydrochloride

iStock, Jorm Sangsorn Well-known for its oncology efforts and making recent headlines for a rumored mega merger with AstraZeneca, Bristol Myers Squibb is also dedicating considerable resources to neurological and psychiatric diseases, with a “continuum of care” mindset. When it comes to neuroscience, Bristol Myers Squibb watchers are laser-focused on the upcoming readouts for Cobenfy—the schizophrenia drug the pharma acquired in the $14 billion purchase of Karuna Therapeutics—in Alzheimer’s disease psychosis. But BMS has a deeper neuro pipeline, built upon a much larger transaction—the $74 billion mega-merger with Celgene in 2019. Readouts for Cobenfy —approved in 2024 as the first new schizophrenia treatment in decades—in Alzheimer’s disease psychosis have been repeatedly delayed by clinical trial challenges and are now expected in 2027 , with a possible interim analysis later this year. But BMS touts a growing pipeline of neuroscience assets in Alzheimer’s, multiple sclerosis and other neuropsychiatric diseases. BMS stepped away from neuro in 2013, instead throwing its primary capital and resources behind immuno-oncology and other priority focuses. Ken Rhodes, vice president of neuroscience, told BioSpace he joined BMS in July 2023 “out of excitement about what the company was doing as it re-entered [the space].” The company’s neuroscience renaissance began with its $74 billion mega-merger with Celgene in 2019, Rhodes continued. “Through the acquisition and looking at the portfolio that the Celgene crew had put together, I think BMS realized the value in that portfolio and chose to sort of re-enter the therapeutic area in earnest.” In March 2020, BMS won approval for Celgene’s ozanimod—now Zeposia—in multiple sclerosis in March 2020. A S1P receptor modulator , Zeposia blocks the capacity of immune cells to enter the central nervous system, reducing inflammation. Based on that foundation, BMS continued to build out neuroimmunology and neuroinflammation drug development programs and capabilities to study new approaches to treating diseases like multiple sclerosis (MS) and Alzheimer’s, Rhodes said. One of these programs, a dual FAAH/MAGL inhibitor called irafamdastat, is currently in Phase 2 trials for MS spasticity and agitation associated with Alzheimer’s. Another is the Prothena Biosciences–partnered anti-MTBR-tau antibody moponetug. The asset is being tested in Phase 2 development as a disease-modifying therapy intended to slow or halt the progression of Alzheimer’s. Like Rhodes, Laura Gault said she came to BMS to help oversee this collection of assets. “Really, what brought me here is the promise of our pipeline and the opportunity to really build a world-class team to deliver on this very promising pipeline,” the senior vice president and head of neuroscience development told BioSpace . As for BMS’ business development efforts, deals “may be done to expand core TAs [therapeutic areas] (e.g., neuroscience) but doesn’t appear to be [a] primary focus,” Truist wrote in April after speaking with Chief Medical Officer Cristian Massacesi and Commercialization Head of CV & Immunology Al Reba. Schizophrenia As BMS’ Cobenfy struggles to gain traction, MapLight knocks on the door Despite the recent emergence of a new drug class, schizophrenia remains one of the most challenging neuropsychiatric diseases to treat—and Cobenfy’s first steps into the market indicate there is still room to grow. August 10, 2026 · 6 min read · Michael Gibney Read more A neuroscience renaissance Neuroscience overall has experienced a comeback recently, with several companies—including BMS, AbbVie and Sanofi—returning after the complexity of the brain and high clinical trial failure rates drove many away. “What really has changed the probability of success, what’s demonstrated success now in disease-modifying therapies in Alzheimer’s disease, is all the investments that academia and industry have made . . . in biomarkers and in genetics,” Rhodes said. “The genetics have taught us what the most valuable targets are likely to be. The biomarkers have taught us how to make sure that we have the right patients in our clinical trials and how to monitor the response to therapy.” As for psychiatric diseases—where the clinical failure rate is especially high —Rhodes acknowledged that the science is further behind. “In psychiatric disease, our understanding of the causal biology is not quite as mature as it is in neurologic diseases,” he said. However, he added, “I think there’s been some important advances there as well, and hopefully, the lessons we learned about how to succeed in Alzheimer’s disease will be replicated in other indications, including psychiatric disease, although that may be a little further off.” BMS is currently evaluating Cobenfy in bipolar-1 mania, Alzheimer’s disease agitation, Alzheimer’s cognition and pediatric autism irritability. The company also has evifacotrep, a TRPC4/5 inhibitor, in Phase 1 testing for mood and anxiety disorders, among other programs. “Cobenfy is viewed as an anchor asset in a broader neuroscience portfolio,” Truist wrote in the April note, adding that two of the ADEPT trials in Alzheimer’s psychosis “need to hit for label expansion and mgmt has indicated that any stat sig result would be positive.” While agreeing that the underlying pathobiology is less understood in psychiatric disease than in other neurologic indications, Gault said the field has “really invested a lot in terms of identifying potential biomarkers that could be used to define subtypes within a disease category or predict treatment response to certain agents.” She specifically noted EEG and functional MRI as diagnostic tools researchers are gaining experience with. A continuum of care What sets BMS apart from some of its peer companies in Alzheimer’s disease, Rhodes said, is a focus on a continuum of care strategy. “We’re interested in developing both disease-modifying therapies and treatments like Cobenfy to treat some of the debilitating symptoms that affect patients with Alzheimer’s disease,” he explained, adding that this is an approach BMS looks to replicate in in other disease areas. Gault agreed, saying that beyond Alzheimer’s, BMS is thinking about how to develop treatments “in other areas that are symptomatic” that have a different pro risk, cost and time than is involved in a trial for a disease-modifying asset. Elsewhere in the pipeline, Rhodes is also “really excited” about discovery and early development work around brain shuttle technologies and the possibility of delivering different therapeutic modalities across the blood-brain barrier [BBB] to interact with targets inside the central nervous system. In December 2024, BMS inked a global license agreement with Swedish biotech BioArctic to leverage the company’s BrainTransporter technology against Alzheimer’s. One preclinical program, BAN2803, is an antibody that uses the transferrin receptor protein to facilitate transport across the BBB. Alzheimer’s disease BMS Taps Eisai’s Alzheimer’s Partner in Massive $1.35B Biobucks Deal BioArctic received $100 million upfront with another $1.25 billion in potential milestone payments on the line for two pyroglutamate-amyloid-beta antibodies. December 19, 2024 · 1 min read · Kate Goodwin Read more “It actually binds to the pyroglutamate form of amyloid, which is the same target, or same form of amyloid, that’s targeted by Eli Lilly’s drug Kisunla,” Rhodes said. “So it’s a clinically validated approach to modifying amyloid pathology and improving or offering disease modification in Alzheimer’s disease.” In addition to this BioArctic-targeted program, BMS has earlier-stage programs that “incorporate various types of shuttle technologies” that the company has not yet disclosed, Rhodes said. “We and perhaps others see [this technology] as an important area for future innovation in the neuroscience space.” Certainly, BMS is far from alone in advancing BBB-crossing platforms. Roche’s Alzheimer’s next-gen candidate trontinemab is an intravenously delivered Brainshuttle Aβ antibody engineered to provide enhanced access to the brain to rapidly reduce amyloid. The asset succeeded in a mid-phase trial in December 2025. A month earlier, Roche put $2 billion-plus on the line to work with Manifold Bio on BBB shuttles for multiple targeted neurological and neurodegenerative disease therapies. Rhodes discussed the evolution of the anti-amyloid space, and where brain shuttle tech could fit within it. “I think data are suggesting that the earlier you begin to treat, the greater the benefit with anti-amyloid therapies,” he said. The results so far “beg the question as to whether [BBB-crossing technologies] will offer a really compelling opportunity with subcutaneous or a more convenient route of administration for patients.” Such technology also enables the delivery of therapeutic modalities besides antibodies, Rhodes added, opening the door to targets that have historically “been very difficult to drug.” Antisense oligonucleotides (ASOs) or small interfering RNAs (siRNAs), for example, are also being investigated for neuro indications. “There’s good clinical support now in a couple of programs suggesting that shuttle technology can deliver those types of medicines into muscle, into heart, into other tissues, including the nervous system,” Rhodes said.

Pharma R&D Intelligence新药研发观察 2026-09-02 星期三第 13 期全球新药获批 · 临床里程碑 · 前沿创新信号 今日头条先声再明 × 罗氏 SIM0660 全球独家授权 15.3 亿美元CD79a/CD19/CD3 三特异性 TCE 出海，2026 中国 BD 最大单 先声药业集团（2096.HK）旗下先声再明 9 月 1 日公告，与罗氏（Roche）就三特异性抗体 SIM0660 达成开发与商业化全球独家授权：首付款 7500 万美元+ 里程碑最高 14.55 亿美元，总额 15.30 亿美元，加双位数分级特许权使用费。SIM0660 同时靶向 CD79a + CD19 + CD3，由先声再明自有 TCE 多特异性抗体平台开发，定位 B 细胞血液瘤 + B 细胞介导自免疾病双线适应症，先声再明截至目前已完成 6 项对外授权、潜在总金额 超 61 亿美元。当日先声港股盘中明显异动。SIM0660 尚处临床前阶段。 来源：先声药业官网 / en.simcere.com / 财联社 / 南京日报 | 事件性质：跨境 BD 授权 壹主简报 T-1 HEADLINES · 2026-09-011.恒瑞 SHR-A1811（瑞康曲妥珠单抗）HER2+ 乳腺癌一线 III 期预设期中分析双终点均达：头对头击败 THP 标准方案 恒瑞医药 9 月 1 日公告，子公司盛迪亚生物自主研发 HER2 ADC 瑞康曲妥珠单抗用于 HER2 阳性复发或转移性乳腺癌一线治疗的关键 III 期研究预设期中分析显示：两个试验组（联合或不联合帕妥珠单抗）对比多西他赛+曲妥珠单抗+帕妥珠单抗（THP 标准方案）均达到 PFS 主要终点，且具统计学显著与临床意义改善。即将向 NMPA 递交上市前沟通交流申请。SHR-A1811 已凭 HORIZON-CRC01 数据于 2025-2026 年陆续拿下 HER2 突变 NSCLC、HER2+ 乳腺癌、HER2+ 结直肠癌 3 项适应症，本次一线乳腺癌 III 期阳性将打开最大单瘤种空间。 关注原因：这是国产 HER2 ADC 首次在乳腺癌一线头对头击败国际标准 THP 方案，里程碑意义大于 DS-8201 在二线确立地位；反向风险在于对照组选择 THP 而非 DS-8201+帕妥珠新一代标准、且 PFS 双终点 vs OS 仍未读出，长期生存与海外注册路径尚待 ASCENT-style 验证。 来源：恒瑞医药官方公告 / 每日经济新闻2.诺华 Remibrutinib REMODEL-1/-2 III 期双阳性：高选择性口服 BTK 抑制剂头对头击败 Aubagio，复发型多发性硬化症（RMS）首个全阳性读出 诺华 9 月 1 日公告，remibrutinib（口服、高选择性 BTK 抑制剂）治疗 RMS 的两项平行 III 期 REMODEL-1 与 REMODEL-2 均达到主要终点：在降低年化复发率（ARR）和炎症性脑 MRI 病灶数上对比特立氟胺（Aubagio）显示统计学显著优效；6 个月确认残疾进展（6mCDP）预设汇总分析也达到名义显著。安全性干净：4500+ 受试者项目中未观测到肝安全信号、无符合 Hy's Law 病例。详细数据将在 10 月 21-23 日多伦多 MSToronto2026（ECTRIMS-ACTRIMS 联合大会）公布。 关注原因：目前 RMS 一线被注射型抗 CD20（Ocrevus/Kesimpta/Briumvi）+ S1P 调节剂主导，remibrutinib 是首个高效口服 BTKi 全 III 期阳性、且对肝安全信号"清白"，对赛诺菲 Aubagio 形成直接替代；反向风险为 MS 口服赛道近年扎堆（Zeposia/Ponvory/Mavenclad），remibrutinib 商业差异化仍需等头对头 Ocrevus 的数据与真实世界依从性结果。 来源：Novartis 官方新闻稿 / BioPharm International / Fierce Pharma3.Plozasiran（Redemplo）SHASTA-3/SHASTA-4 III 期双阳性：APOC3 siRNA 严重高甘油三酯血症（sHTG）首个广泛人群获益证据，胰腺炎风险降低 78% Arrowhead/Ionis 合作的 APOC3 siRNA 疗法 plozasiran 9 月 1 日在 ESC 2026 慕尼黑 Hot Line 公布 SHASTA-3 与 SHASTA-4 两项 III 期数据：对比安慰剂首次注射后 3 个月起甘油三酯降幅分别 79% 与 81%（P<0.0001），12 个月时 91-93% 治疗组 TG 降至 <500 mg/dL（vs 安慰剂 50-51%），超半数治疗组 <150 mg/dL（vs <10%）。预设合并分析：急性胰腺炎事件 减少 78%（P=0.008），有胰腺炎史亚组减少 91%，TG>880 mg/dL + 胰腺炎史亚组减少 100%（需考虑亚组样本量）。此前 plozasiran 已凭 PALISADE 研究获批用于 FCS（家族性乳糜微粒血症综合征），本次为更广泛 sHTG 人群补充数据。 关注原因：sHTG 是胰腺炎反复发作的核心代谢诱因，目前标准为贝特类/Omega-3/烟酸，无针对 APOC3 的获批疗法；与同类 olezarsen 数据互补形成 siRNA 心血管风险因子调控新证据；反向风险为最高剂量 TG>880 + 既往胰腺炎亚组 NNT=24 看似惊艳，但样本量偏小、动脉粥样硬化性心血管硬终点仍待 ESC 后续报道确认。 来源：MedPage Today 报道 / 西澳大学 Watts 教授 ESC 2026 报告4.BeOne × Jazz Ziihera（zanidatamab）HERIZON-GEA-01 IA2 OS 显著阳性：HER2 双抗一线 GEA 26 个月 OS 新标准坐实 BeOne Medicines 与 Jazz Pharmaceuticals 9 月 1 日联合公告，HER2 双抗 Ziihera 一线治疗 HER2 阳性胃食管腺癌（GEA）的关键 III 期 HERIZON-GEA-01 第二次期中分析（IA2）达到 OS 统计显著，HR 较 IA1 进一步改善；Ziihera+化疗对比曲妥珠+化疗 OS 阳性、Ziihera+曲妥珠+化疗三联方案 HR 也改善（数值未披露）。回看 IA1：n=914，OS 26.4 vs 19.2 月（HR 0.72，P=0.0043），PFS 12.4 vs 8.1 月，30+ 国家 300 中心。FDA 已于 8 月 25 日凭 IA1 批准双方案一线 GEA。 关注原因：Ziihera 是首个把 HER2 一线 GEA OS 拉到 26 个月的双抗方案，触发 Zymeworks $2.5 亿里程碑；百济神州 + Jazz + BeOne 三角联盟使 HER2 双抗一线 GEA 商业化路径在亚洲与北美同步落地；反向风险为 OS HR 改善幅度未披露，且三联方案（Z+曲妥+化疗）成本/毒性是否被医保/指南接受仍存分歧。 来源：BeOne PR / Jazz IR / BioPharm International5.Stoke × Biogen Zorevunersen Dravet 综合征 4 年随访阳性：全球首个 ASO 疾病修饰疗法候选，EMPEROR III 期读出 Q3 2027 Stoke Therapeutics 与 Biogen 9 月 1 日公告，将在 9 月 5-9 日雅典 EEC 大会公布 zorevunersen（靶向 SCN1A 的反义寡核苷酸 ASO）治疗 Dravet 综合征的 4 年开放标签延伸（OLE）数据：认知、行为改善持续 4 年，癫痫发作持续减少，安全性可控；新增针对最严重发作类型（SUDPE 首要风险因素）与生活质量亚组分析。截至 2026/7/31 已给药 930+ 剂，部分患者治疗超过 5 年；CSF 蛋白升高见于 94% 患者（59% 归为 TEAE），无相关严重临床表现，无脑积水报告。EMPEROR 全球 III 期数据预计 Q3 2027 读出。 关注原因：Dravet 综合征 90% 由 SCN1A 单倍体功能不全导致，目前无获批的疾病修饰疗法（DMT），抗癫痫药仅对 ≤43% 患者实现 ≥50% 发作减少；zorevunersen 是首个通过上调野生型 SCN1A 拷贝恢复 NaV1.1 蛋白的 ASO，具备 DMT 长期获益潜质；反向风险为 4 年 OLE 为开放标签非随机对照、且 CSF 蛋白升高机制尚不明晰，EMPEROR III 期才是真正的注册门槛。 来源：Stoke Therapeutics & Biogen 联合 PR / NEJM 2026 年 3 月已发表 1/2a+早期 OLE 贰三新雷达 NEW TARGET · TECH · DIRECTION 新靶点CDE BTD石药巨石生物 SYS6010 EGFR ADC 第 3 项 CDE 突破性治疗认定：复发/转移性头颈鳞癌 石药创新（300765.SZ）9 月 1 日公告，控股子公司巨石生物 EGFR ADC SYS6010 被 CDE 纳入突破性治疗品种名单，拟定适应症为经免疫和含铂化疗治疗失败的复发或转移性头颈鳞癌患者；该适应症关键 III 期临床正在开展。SYS6010 此前已获 2 项 BTD（EGFR 突变阳性晚期 NSCLC、晚期食管鳞癌）。NMPA 受理智飞生物冻干人用狂犬病疫苗（ZFB-3 细胞）临床试验申请获 NMPA 受理 智飞生物（300122）9 月 1 日公告，全资子公司智飞龙科马研发的冻干人用狂犬病疫苗（ZFB-3 细胞）获得 NMPA 药物临床试验申请受理通知书；自受理之日起 60 日内未收到否定或质疑意见，即可按方案开展临床试验。人用狂犬病疫苗为生物制品大单品，ZFB-3 细胞路线与人二倍体细胞工艺形成差异化。 新技术Ph2→Ph3GSK mRNA 流感疫苗 HA+NA 双抗原设计推进 III 期：OPTIONS XIII 数据阳性，2026 年 9 月启动 Ph3 GSK 9 月 1 日公告，HA+NA 双抗原 mRNA 季节性流感疫苗 Ph2 OPTIONS XIII 数据阳性，对所有测试株免疫应答高于许可标准剂量与高剂量灭活流感疫苗（含年轻与年长人群），耐受良好；将于 2026 年 9 月推进 III 期。GSK 候选是首个 Ph3 阶段同时靶向 HA+NA 的 mRNA 流感疫苗（已上市流感疫苗几乎只靶 HA）。FDA 已于 7 月授予快速通道认定；与 Moderna mNEXSPIKE（2025/5 获批）形成直接竞争。TCE 平台先声再明 CD79a/CD19/CD3 三特异性 TCE 平台验证：SIM0660 临床前阶段被 15.3 亿美元重金下注 SIM0660（详见今日头条）由先声再明自有 TCE 多特异性抗体平台开发，将 CD3 T 细胞衔接臂与 CD79a/CD19 双 B 细胞抗原结合域融合，可诱导强效 T 细胞介导细胞毒同时限制细胞因子释放；平台具备潜在 First-in-Class 价值。2026 年迄今国内 TCE 出海 BD 累计已超 60 亿美元，SIM0660 是单笔最大单一资产授权。 新方向TYK2 亚组Alumis Envudeucitinib LUMUS IIb 失败但 IFNGS-high 亚组阳性：biomarker-enrichment 自免设计新范式 Alumis 9 月 1 日公告，envudeucitinib（口服选择性变构 TYK2 抑制剂）治疗中重度 SLE 的 IIb 期 LUMUS 试验 n=408，48 周 BICLA 主终点 + 关键次要终点（CLASI-50、SRI-4、LLDAS）全部未达；预设 IFNGS-high 亚组显示强阳性，最高剂量 BICLA 52.6% vs IFNGS-low 24.4%。公司将启动 IFNGS 富集 Ph3 设计，与 FDA/EMA 沟通；银屑病 NDA 维持 Q4 2026 申报。化学药品 5.1华润双鹤洛莫司汀胶囊获 NMPA 注册证书：原发性恶性脑肿瘤，6 年数据保护期 华润双鹤（600062.SH）9 月 1 日公告，作为境内代理人申报的洛莫司汀胶囊获 NMPA《药品注册证书》（规格 40mg，化药 5.1 类），用于经手术和/或放疗后的原发性恶性脑肿瘤儿童及成人患者，同时获得 6 年数据保护期；公司与 Latina Pharma S.P.A 于 2024 年初签订合作协议，累计研发投入约 223 万元。 ◈ 猎头视角 · 本周人才信号TCE 多特异性抗体平台负责人 / 工艺与早期临床运营： 先声再明 SIM0660 被罗氏 15.3 亿美元下注印证"中国 TCE 平台 = 全球大药企首选"逻辑，2026 年内中国 TCE 累计出海已超 60 亿美元；和铂、康诺亚、德琪、剂泰、和铂、和铂、岸迈、亿腾嘉和等具备 TCE 平台的中国 Biotech 在研管线溢出加速，对肿瘤/血液瘤 BD 与全球早期临床转化医学岗构成结构性需求。HER2 ADC 头对头试验设计与乳腺癌一线转化医学： 恒瑞 SHR-A1811 一线乳腺癌 III 期双终点阳性，国产 ADC 头对头击败国际标准方案（THP）；中国头部 ADC 平台（恒瑞、科伦博石、荣昌、百利天恒、礼新、映恩、宜联、康联瑞臻、复旦天昊等）正集中构建"全球多瘤种头对头 vs 国际 SOC"的转化医学团队，对乳腺癌、肺癌、消化道瘤种的临床策略 + 生物标志物分层人才需求明显。神经炎症 ASO 平台与基因型分层的 DMT 临床团队： Stoke × Biogen zorevunersen 4 年 OLE 阳性 + EMPEROR III 期 2027 Q3 读出，SCN1A 单倍体功能不全 Dravet 综合征是 ASO 平台最被看好的 DMT 候选；中国 ASO（小核酸）平台（瑞博/圣诺/舶望/艾博生物/泰克/中美华东/时安/靖因/维亚臻/知源/尧唐/海昶/纳诺/科镁信/赫吉亚/锐正/中合基因/百力司康/中美瑞康/科赛津/博恩特/因诺纬/纽尔/元思/华道/和元艾研/逐典/迈睿/苏博/西湖欧米/明聚/华大吉诺/茵诺贝森/通灵生物/泰泽恒慧/辉大/杰毅/泰励/新芽/厚存/嘉晨西海）将密集招聘神经领域 DMT 临床策略 + 罕见病注册医学人才。心血管风险因子 siRNA 临床开发与代谢领域 BD： plozasiran SHASTA-3/-4 III 期阳性首次把 APOC3 siRNA 推到广泛 sHTG 人群，胰腺炎减少 78% 是硬终点级别证据；中国 APOC3 / Lp(a) / ANGPTL3 / ANGPTL4 / PCSK9 siRNA（恒瑞、瑞博、信达 IBI3042、亚盛、君实、绿叶、丽珠、康缘、康希诺、海昶、圣兆、圣诺、舶望、泰克、因诺纬、纽尔、维康、纽欧津、瑞博、博恩特、和元艾研、嘉晨西海、尧唐、海昶、华大吉诺、纳诺、科镁信、辉大）将密集招聘心血管代谢转化医学与海外注册人才。自免赛道 biomarker enrichment 临床设计与转化医学： Alumis envudeucitinib LUMUS 失败 + IFNGS-high 亚组阳性提示自免 III 期必须前置 biomarker 假设与富集设计；中国 IFN、TNF、IL-17、IL-23、TLSP、JAK/TYK2 平台（恒瑞、信达、康方、君实、康宁杰瑞、亚盛、三生、丽珠、华东医药、海正、辉瑞、罗氏、AZ、辉瑞、艾伯维、勃林格、UCB、协和发酵、赛诺菲、强生）将密集招聘生物标志物富集临床设计与转化医学人才。 新药研发观察 · PHARMA R&D INTELLIGENCE 出品：新药研发领域高级猎头顾问工作室  |  信息采集：FDA / NMPA / EMA 官网、药企公告、SEC 备案及主流医药媒体 本刊仅供行业研究与人才 mapping 参考，不构成任何投资建议；原文未披露的信息均已标注，未作推测性补充 © 2026 新药研发观察编辑部 · 第 13 期 · 每日 09:00 出版