MorphoSys AG

加科思药业6月16号向国家药管局提交了BET抑制剂JAB-8263的新适应症——自身免疫性疾病的IIa临床试验申请，BET抑制剂有望像BTK或JAK抑制剂一样，从肿瘤适应症进入自免领域，打开更大的商业潜力。自身免疫性疾病是一组由免疫系统攻击自身组织导致的慢性疾病，包括类风湿关节炎、系统性红斑狼疮、银屑病等，影响全球5-9%的人口。目前大多数用于自免的药物为抗体注射剂，口服制剂仅有少数几种，而且因为安全性带有FDA黑框警告，所以临床急需新机制的口服制剂药物用于长期自免疾病的治疗。若加科思的JAB-8263能拿下自免适应症，临床及商业价值天花板将大幅提高。TONACEA01独特的作用机制：BET治疗自免的三重作用目前上市的自免药物中，或者是单一炎症因子的阻断抗体，或者是炎症因子下游信号通路的抑制剂，这些药物仅能抑制炎症，无法进一步重建正常免疫系统及组织修复。而BET抑制剂通过调控表观遗传学通路，可以从抑制炎症、重建免疫系统、组织修复三个角度全面起到治疗自免疾病的作用，具有广谱且多功能的治疗效果。首先，BET抑制剂能抑制炎症。人体的炎症是一个复杂网络，由多种炎症因子共同参与，彼此调控。不同于单一炎症因子的阻断抗体，BET抑制剂通过表观遗传学调控，能强效抑制绝大多数炎症因子表达，因此能更完全彻底地消除炎症，起效更快且避免耐药。其次，BET抑制剂可重建免疫系统。BET抑制剂对正常免疫细胞无杀伤，却有效清除异常激活增殖的T细胞和B细胞。因此BET抑制剂可将人体异常免疫系统恢复正常，而这种免疫重建的效果是现有抗体或者小分子自免药物所不具备的。第三，BET抑制剂可修复受损组织。现有药物只能减轻炎症但不能修复炎症导致的组织损伤，因此自免患者最终仍会发展到组织机能丧失。BET具有抗纤维化、骨保护和调节干细胞分化的特殊功能，对组织修复有重要作用，这是目前自免药物都不具备的独特优势TONACEA02有望长期给药：JAB-8263的临床数据展示了良好的安全性BET抑制剂JAB-8263和同为口服药的JAK抑制剂相比，具有无可比拟的安全性优势。对于需要长期用药的自免适应症来说，药物安全性尤为关键，而JAK抑制剂无论是一代还是二代，都有FDA的黑框警告（感染，心血管毒性，致癌性等）。可喜的是，BET抑制剂JAB-8263在所有JAK抑制剂的安全风险领域，都有明显的优势：在长期的JAB-8263人体给药中，无一例观察到感染问题；且JAB-8263作为BET抑制剂，本就是一种抗癌药物，也无致癌性风险；另外初步数据显示JAB-8263具有心脏保护功能。这些特性都决定了BET抑制剂JAB-8263相较于JAK抑制剂，可解决临床未满足的安全性需求，具有极大的市场前景。加科思的BET抑制剂已经在中美两国完成肿瘤适应症的一期临床试验，数据显示，JAB-8263的二期推荐剂量为0.3毫克，可每日给药，耐受性良好。而已完成临床III试验即将被美国FDA获批上市的同靶点产品pelabresib剂量为125毫克，给药14天停药7天。由此可见加科思的JAB-8263有效性和安全性都更胜一筹。在自免适应症用药，预测剂量会更低，因此安全性更好。TONACEA03更高的商业价值：从29亿美元收购说起诺华于2024年2月溢价六成收购德国公司MorphoSys，当时MorphoSys的BET抑制剂Pelabresib三期数据刚刚亮相，并计划提交首发适应症骨髓纤维化的NDA（与卢可替尼联用）。仅仅凭借单一适应症的联用，Pelabresib就获得诺华29亿美元的收购。JAB-8263的价值不仅仅要参考诺华29亿美元的收购，或许还应该参考年销售额过60亿美元的乌帕替尼。至少从早期机制研究来看，JAK只能抑制下游传导，不能抑制炎症因子表达。而BET比JAK更广谱、更具有安全性优势（BET在肿瘤适应症无黑框警告）。另一款自免大药IL-17（司库奇尤单抗2024年销售额超过60亿美元）则需要再临床中考虑Th细胞分型，和BET相比不具备广谱性。目前加科思已经公布了BET抑制剂JAB-8263骨髓纤维化临床数据——60%的患者（N=10）在第24周时总症状评分下降≥50%（2024年ASH公布），并且有望拿下自免适应症，这不仅意味着加科思将创造更多临床与商业价值，也标志着中国公司将引领全球BET抑制剂的开发。关于同写意同写意论坛是中国新药研发行业权威的多元化交流平台，二十一年来共举办会议论坛百余期。“同写意新药英才俱乐部”基于同写意论坛而成立，早已成为众多新药英才的精神家园和中国新药思想的重要发源地之一。同写意在北京、苏州、深圳、成都设立多个管理中心负责同写意活动的运营。尊享多重企业/机构会员特权● 分享庞大新药生态圈资源库；● 同写意活动优享折扣；● 会员专属坐席及专家交流机会；● 同写意活动优先赞助权；● 机构品牌活动策划与全方位推广；● 秘书处一对一贴心服务。入会请联系同写意秘书处 同写意创新链盟机构 （上下滑动查看更多）深势科技 | 新天地药业 | 快舒尔医疗 | 华赛伯曼 | 艾里奥斯 | 药明合联 | 皓元医药 | 希格生科 | 纽瑞特医疗 | 夸克医药 | 石药集团 | 源生生物 | 君赛生物 | 达尔文生物 | 浩博工程 | 怀雅特 | 赛立维 | 科伦博泰 | 赛隽生物 | 安升达/金唯智 | 卡替医疗 | 达科为生物 | 沙利文 | 天广实 | 拜耳 | 楚天科技 | 三生制药 | 三启生物 | 国通新药 | 通瑞生物 | 科济药业丨立迪生物 | 森西赛智 | 汇芯生物 | 申科生物 | 方拓生物 | 东抗生物 | 科盛达 | 依利特 | 翊曼生物丨锐拓生物丨复百澳生物丨圆因生物丨普洛斯丨华润三九丨皓阳生物丨人福医药丨广生堂药业丨澳宗生物丨妙顺生物 | 荣捷生物丨行诚生物 | 宜联生物 | 生命资本 | 恒诺康丨斯丹姆 | 益诺思 | 深圳细胞谷丨佰诺达生物 | 沃臻生物 | 金仪盛世 | 朗信生物 | 亦笙科技 | 中健云康 | 九州通 | 劲帆医药 | 沙砾生物 | 裕策生物 | 同立海源 | 药明生基 | 奥浦迈 | 原启生物 | 百力司康 | 宁丹新药 | 上海细胞治疗集团 | 滨会生物 | FTA | 派真生物 | 希济生物 | 优睿赛思 | 血霁生物 | 优睿生物 | 邦耀生物 | 华大基因 | 银诺生物 | 百林科医药 | 纳微科技 | 可瑞生物 | 夏尔巴生物 | 金斯瑞蓬勃生物 | 健元医药 | 星眸生物 | 格兰科医药 | 莱羡科学仪器 | 明度智云 | 玮驰仪器 | 康源久远 | 易慕峰 | 茂行生物 | 济民可信 | 欣协生物 | 泰楚生物 | 泰澧生物 | 谱新生物 | 思鹏生物 | 领诺医药 | 宜明生物 | 爱科瑞思 | 阿思科力 | 博格隆生物 | 百吉生物  | 迈邦生物 | 多宁生物 | 万邦医药 | ASCT | 为度生物 | 比邻星创投 | 赛桥生物 | 吉美瑞生 | 荣泽生物 | 科金生物 | 汉超医药 | 康日百奥 | 汉腾生物 | 力品药业 | 安必生 | 博瑞策生物 | 中盛溯源 | 深研生物 | 东方略 | 赛赋医药 | 克睿基因 | 安润医药 | 镁伽科技 | 科锐迈德 | 和元生物 | 申基生物 |楷拓生物|森松生命科技 | 凯理斯 | 尚德药缘 |  晟国医药 | 健新原力 | 纽福斯 | 华东医药 | 士泽生物 | 影研医疗科技 | 新格元生物 | 依生生物 | 腾迈医药 | 汉欣医药 | 恒驭生物 | 盛诺基 | 序祯达生物 | 乐纯生物 | 速石科技 | 耀海生物 | 新合生物 | 华龛生物 | 恺佧生物 | 成都凡微析 | 正帆科技 | 大橡科技 | 博雅辑因 | 因美纳 | 博雅控股集团 | 近岸蛋白 | 依科赛生物 | 利穗科技 | 东南科仪 | 倍谙基 | 辉诺医药 | 圣诺制药 | 埃格林医药 | 科镁信 | 爱思益普 | 复星医药 | 齐鲁制药 | 捷思英达丨荣昌生物丨泽璟制药丨奕安济世丨礼新医药丨维立志博丨派格生物丨赛生药业丨呈源生物丨启德医药丨双运生物丨宝船生物丨曙方医药丨澳斯康生物丨普莱医药丨维健医药丨海昶生物丨征祥医药丨智核生物丨望石智慧丨博生吉医药丨南京诺丹丨四星玻璃丨艾米能斯丨霁因生物丨普瑞康生物丨映恩生物丨康哲生物丨霍德生物丨海慈药业丨沃生生物丨睿健医药丨矩阵元丨斯微生物丨则正医药丨预立创投丨东立创新丨博安生物丨伟德杰生物丨星奕昂生物丨耀乘健康科技丨琅钰集团丨康德弘翼 | 原力生命科学丨上海科洲丨特瑞思丨药源丨健艾仕生物丨冠科美博丨微境生物丨天境生物丨合源生物丨泛生子丨创胜集团丨加科思药业丨丹诺医药丨凌科药业丨偶领生物丨凯斯艾生物丨成都圣诺丨松禾资本丨清普生物丨和其瑞丨开拓药业丨科兴制药丨玉森新药丨水木未来丨分享投资丨植德律所丨奥来恩丨乐明药业丨东曜药业丨君圣泰丨海创药业丨天汇资本丨再鼎医药丨济煜医药丨百英生物丨基石药业丨君实生物丨Sirnaomics,Inc.丨亦诺微丨博腾股份丨思路迪诊断丨艾博生物丨普瑞金生物丨未知君生物丨尚健生物丨阿诺医药丨有临医药丨赛业生物丨睿智医药丨博济医药丨晶泰科技丨药明康德丨创志科技丨奥星集团丨苏雅医药丨科贝源丨合全药业丨以岭药业丨科睿唯安丨DRG丨博瑞医药丨丽珠医药丨信立泰药业丨步长制药丨华素制药丨众生药业丨上海医药丨高博医疗集团丨药渡丨君联资本丨集萃药康丨诺思格丨精鼎医药丨百利药业丨Pfizer CentreOne丨默克中国创新中心丨奥来恩丨瑞博生物丨新通药物丨广东中润丨医普科诺丨诺唯赞丨康利华丨国信医药丨昆翎丨博纳西亚丨缔脉丨一品红丨和泽医药丨博志研新丨凯莱英医药丨汉佛莱丨英派药业丨京卫制药丨海思科药业丨宏韧医药丨开心生活科技丨哈三联丨Premier Research丨宣泰医药丨先声药业丨海金格丨普瑞盛医药丨Informa丨科特勒丨谋思医药丨HLT丨莱佛士丨辉瑞丨科林利康丨冠科生物丨科文斯丨卫信康丨龙沙（Lonza）丨美迪西丨阳光诺和丨润东医药丨勃林格殷格翰（中国）丨艾苏莱生物丨领晟医疗丨驯鹿医疗丨燃石医学丨中肽生化丨鸿运华宁丨泰格医药丨易迪希丨希麦迪丨百奥赛图丨迪纳利丨青云瑞晶丨鼎丰生科资本丨中源协和丨维亚生物丨青松医药丨中科谱研丨长风药业丨艾欣达伟丨鼎康生物丨中晟全肽丨海步医药丨勤浩医药丨奥萨医药丨太美医疗科技丨生特瑞丨东富龙丨Cytiva丨优辰实验室丨苏桥生物丨君达合创丨澎立生物丨南京澳健丨南京科默丨东阳光丨亚盛医药丨杰克森实验室丨上海科州丨三优生物丨三迭纪丨泰诺麦博丨Cell Signaling Technology丨PPC佳生丨澳斯康丨先为达丨智享生物丨锐得麦丨宜明昂科丨明济生物丨英百瑞丨六合宁远丨天津天诚丨百拓生物丨星药科技丨亓上生物丨真实生物丨引光医药丨方达医药丨高博医疗集团丨赞荣医药丨国投创新丨药明生物丨康哲药业丨高特佳投资丨普瑞基准丨臻格生物丨微谱医药丨和玉资本 | 倚锋资本

– Monjuvi® (tafasitamab-cxix) in combination with rituximab and lenalidomide is the first FDA-approved CD19- and CD20-targeted immunotherapy combination for adult patients with follicular lymphoma (FL) – Patients with relapsed or refractory FL achieved significantly improved progression-free survival with Monjuvi in combination with rituximab and lenalidomide in the Phase 3 registration trial – This milestone represents the second approved indication for Monjuvi in the United States WILMINGTON, DE, USA I June 18, 2025 I Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has approved Monjuvi® (tafasitamab-cxix), a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody, in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). “Patients living with relapsed or refractory FL have been waiting for new options that improve progression-free survival without substantial increase in side effects. Based on the data from the inMIND trial of Monjuvi, today’s approval brings to this patient population the first CD-19 and CD20-targeted immunotherapy combination and a potential new treatment standard,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “This second U.S. approval for Monjuvi reinforces our commitment to advancing innovation for the lymphoma community.” The Priority Review and FDA approval of the supplemental Biologics License Application (sBLA) for Monjuvi was based on data from the pivotal, randomized, double-blind, placebo-controlled Phase 3 inMIND trial evaluating the efficacy and safety of Monjuvi in combination with rituximab and lenalidomide in adult patients with relapsed or refractory FL. Data from the trial was featured in the Late-breaking Session (LBA-1) at the 2024 American Society of Hematology (ASH) Annual Meeting. 1 The study met its primary endpoint demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) by investigator assessment, which demonstrated 27.5% (N=273) of patients with an event in the Monjuvi group vs. 47.6% (N=275) of patients with an event in the control arm. Patients receiving Monjuvi in combination with rituximab and lenalidomide achieved a median PFS by investigator assessment of 22.4 months (95% CI, 19.2-not evaluable [NE]) compared to 13.9 months (95% CI, 11.5-16.4) in the control arm (hazard ratio [HR]: 0.43 [95% CI, 0.32-0.58]; P<0.0001). The PFS assessed by an Independent Review Committee (IRC) was consistent with investigator-based results. Median PFS by IRC was not reached (95% CI, 19.3-NE) in the Monjuvi group versus 16.0 months (95% CI, 13.9-21.1) in the control arm (HR: 0.41 [95% CI, 0.29-0.56]. The PFS benefit was consistent across prespecified patient subgroups, including number of previous lines of therapy. The safety of Monjuvi in patients with FL was evaluated in 546 patients in the inMIND trial. Serious adverse reactions occurred in 33% of patients who received Monjuvi in combination with rituximab and lenalidomide, including serious infections in 24% of patients (including pneumonia and COVID-19 infection). Other serious adverse reactions in ≥ 2% of patients included renal insufficiency (3.3%), second primary malignancies (2.9%), and febrile neutropenia (2.6%). Fatal adverse reactions occurred in 1.5% of patients, including from COVID-19, sepsis, and adenocarcinoma. The most common adverse reactions (≥ 20%) in recipients of Monjuvi, excluding laboratory abnormalities, were respiratory tract infections (including COVID-19 infection and pneumonia), diarrhea, rash, fatigue, constipation, musculoskeletal pain, and cough. The most common Grade 3 or 4 laboratory abnormalities (≥ 20%) were decreased neutrophils and decreased lymphocytes. “Follicular lymphoma is generally an indolent yet chronic cancer that frequently recurs after treatment, making long-term disease control a critical objective,” said Christina Poh, M.D., Assistant Professor of Medicine at the University of Washington and Fred Hutchinson Cancer Center. “The FDA approval of Monjuvi in combination with rituximab and lenalidomide marks a significant advancement, offering a chemotherapy-free option that has demonstrated a meaningful reduction in the risk of disease progression across a broad patient population, including those with high-risk disease.” FL is the second most common type of non-Hodgkin lymphoma (NHL) and represents up to 30% of NHL cases. 2 While considered an indolent, slow-growing disease with prolonged survival, FL is challenging to treat due to its tendency for frequent relapse, need for multiple lines of therapy and potential transformation into large B-cell lymphoma. 2,3 “While the initial responses to FL treatment are often positive, recurrence can become increasingly difficult for patients to manage as they navigate emotions and the next treatment steps related to relapse,” said Mitchell Smith, M.D., Ph.D., Chief Medical Officer, Follicular Lymphoma Foundation. “We are pleased that the FDA has approved tafasitamab, part of a treatment combination offering a new option for patients living with this chronic disease.” In July 2020, Monjuvi in combination with lenalidomide received FDA approval for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication was approved under accelerated approval by the U.S. FDA based on overall response rate (ORR). Continued approval of Monjuvi for this indication may be contingent on verification and description of clinical benefit in confirmatory trial(s). Tafasitamab is also being evaluated as a therapeutic option in an ongoing pivotal trial for first-line DLBCL. Incyte is committed to supporting patients and removing barriers to access medicines. Eligible patients in the U.S. who are prescribed Monjuvi have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering personalized patient support, including financial assistance and ongoing education and additional resources. More information about IncyteCARES is available by visiting www.incytecares.com or calling 1-855-452-5234, Monday through Friday, from 8 a.m. to 8 p.m. ET. About inMIND A global, double-blind, randomized, controlled Phase 3 study, inMIND (NCT04680052) evaluated the efficacy and safety of tafasitamab in combination with rituximab and lenalidomide compared with placebo in combination with rituximab and lenalidomide in patients with relapsed or refractory follicular lymphoma (FL) Grade 1 to 3a or relapsed or refractory nodal, splenic or extranodal marginal zone lymphoma (MZL). The study enrolled a total of 654 adults (age ≥18 years). The primary endpoint of the study is progression-free survival (PFS) by investigator assessment in the FL population, and the key secondary endpoints are PFS in the overall population as well as positron emission tomography complete response (PET-CR) and overall survival (OS) in the FL population. For more information about the study, please visit https://clinicaltrials.gov/study/NCT04680052 . About Monjuvi ® (tafasitamab-cxix) Monjuvi ® (tafasitamab-cxix) is a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb ® engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP). MorphoSys and Incyte entered into: (a) in January 2020, a collaboration and licensing agreement to develop and commercialize tafasitamab globally; and (b) in February 2024, an agreement whereby Incyte obtained exclusive rights to develop and commercialize tafasitamab globally. In the U.S., Monjuvi is approved by the U.S. Food and Drug Administration in combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). MONJUVI is not indicated and is not recommended for the treatment of patients with relapsed or refractory marginal zone lymphoma outside of controlled clinical trials. Additionally, Monjuvi received accelerated approval in the United States in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). In Europe, Minjuvi ® (tafasitamab) received conditional Marketing Authorization from the European Medicines Agency in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory DLBCL who are not eligible for ASCT. XmAb® is a registered trademark of Xencor, Inc. Monjuvi, Minjuvi, the Minjuvi and Monjuvi logos and the “triangle” design are registered trademarks of Incyte. Please see the full Prescribing Information including the Medication Guide for Monjuvi. About Incyte A global biopharmaceutical company on a mission to Solve On. , Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia. For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn , X , Instagram , Facebook , YouTube . SOURCE: Incyte

Global Phase 3 PROMINENT study will evaluate the efficacy and safety of felzartamab, as compared to tacrolimus, in adults with primary membranous nephropathy (PMN) There are currently no therapies specifically approved for PMN, a rare immune-mediated disease affecting the kidneys with an estimated prevalence of ~36k patients in the U.S.1 Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potentially differentiated therapeutic candidate with promise for a broad range of immune-mediated diseases CAMBRIDGE, Mass., June 30, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) – announced the initiation of dosing in the global clinical study, PROMINENT. The Phase 3 study will evaluate the efficacy and safety of the investigational drug felzartamab compared to tacrolimus in adults diagnosed with primary membranous nephropathy (PMN). PROMINENT is designed to enroll approximately 180 adults with PMN and expected to readout in 2029. PMN is a severe antibody-mediated disease of the kidney that is a leading cause of nephrotic syndrome and carries a significant risk of kidney failure. Felzartamab is an anti-CD38 antibody that has been shown in clinical studies to selectively deplete CD38+ cells, including plasma cells, the source of autoantibodies that mistakenly attack the body’s own tissues in a range of immune-mediated diseases. Importantly for felzartamab, it is estimated that up to 80% of PMN patients have autoantibodies against PLA2R (aPLA2R) generated by CD38-expressing plasma cells. Currently, there are no approved treatments for PMN and the standard of care includes treatments ranging from immunosuppressants to chemotherapy.2 “We are encouraged by the opportunity to advance a Phase 3 study for primary membranous nephropathy, a condition that carries a significant risk of kidney failure,” said Travis Murdoch, Head of the Biogen West Coast Hub. “This is the third Phase 3 trial of felzartamab launched by Biogen this year, underscoring our ongoing commitment to developing potential novel treatment options for patients living with kidney disease.” PROMINENT is a 104-week, randomized, open-label, global multicenter Phase 3 trial (NCT06962800) to evaluate the efficacy and safety of felzartamab compared with tacrolimus in moderate- to -high-risk participants with PMN, inclusive of newly diagnosed and relapsed patients, in achieving complete remission of proteinuria. Participants will be randomized to receive either felzartamab or tacrolimus with the primary endpoint being the percentage of participants who achieve complete remissions (CR) at week 104. The study will evaluate both anti-PLA2R (aPLA2R) autoantibody positive and negative patients, stratifying participants based on PLA2R levels. Key secondary endpoints include the impact of felzartamab on serum anti-phospholipase A2 receptor (PLA2R) antibodies and patient-reported outcomes.“Primary membranous nephropathy remains an unaddressed area of nephrology, for which there are no approved treatments. Felzartamab’s mechanism of action designed to deplete the cells that produce the pathogenic antibodies is exciting news for patients that are waiting for potential meaningful new treatment options,” said Mohamed El-Shahawy, M.D., MPH, MHA, Director of the Academic Research Institute in Los Angeles, Clinical Professor of Medicine at Keck-University of Southern California School of Medicine, and a principal investigator for the PROMINENT Phase 3 trial. “I’m grateful that Biogen is advancing research in this rare kidney disease and am eager to see the continued progress in the study’s enrollment.” Felzartamab was previously investigated in two Phase 2 studies, M-PLACE (n=31) and NewPLACE (n=24), which enrolled patients with aPLA2R-positive PMN. In the final analysis of M-PLACE, reductions in aPLA2R titers were observed in most patients as early as one week (median reduction of 45%), with responses (>50% reduction) in most patients at six months at end-of-treatment. In addition, improvements in proteinuria and serum albumin levels were observed with administration of felzartamab. Across both studies, the majority of treatment emergent adverse events (TEAEs) reported were mild to moderate and consistent with the known mechanism of action of felzartamab in the PMN population. The most common TEAE was infusion-related reactions on the first infusion that were mostly mild to moderate in intensity. In addition to beginning a Phase 3 study of felzartamab in PMN, Biogen also initiated two other Phase 3 studies of felzartamab this year, TRANSCEND (NCT06685757) for late antibody-mediated rejection in adult kidney transplant recipients and PREVAIL (NCT06935357) in IgA nephropathy.About FelzartamabFelzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Felzartamab is a potential first-in-class therapeutic candidate with promise as a pipeline-in-a-product across a range of immune-mediated diseases. Felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies. Felzartamab was originally developed by MorphoSys AG (now MorphoSys GmbH, a Novartis company). Human Immunology Biosciences (HI-Bio) exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding China (including Macau and Hong Kong and Taiwan). Biogen acquired HI-Bio in July 2024. Felzartamab is an investigational therapeutic candidate that has not yet been approved by any regulatory authority and its safety and effectiveness have not been established. About Primary Membranous Nephropathy (PMN)PMN is a severe antibody-mediated disease of the kidney that is a leading cause of nephrotic syndrome and carries a significant risk of kidney failure, with an estimated prevalence of ~36k patients in the United States.1 Patients with nephrotic syndrome often present with very severe swelling and fatigue related to high-grade proteinuria, and they are also at an increased risk of infection. There are no approved treatments for PMN and the current standard of care includes treatments ranging from immunosuppressants to chemotherapy.2 Even with these strategies, approximately one third of patients do not achieve remission.2 About BiogenFounded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube. Biogen Safe Harbor This press release contains forward-looking statements, relating to: our strategy and plans; potential of, and expectations for the design, timing and results of the PROMINENT study, the ability of felzartamab to treat AMR, PMN or IgAN, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, our future financial and operating results. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned “Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. References: