Aflibercept

EYLEA HD demonstrated non-inferior vision gains with an every 8-week dosing regimen compared to EYLEA® (aflibercept) Injection 2 mg dosed every 4 weeks Safety data remains consistent with the known EYLEA HD and EYLEA safety profiles Supplementary biologics license application planned for submission to the U.S. Food and Drug Administration in the first quarter of 2025 Dec. 17, 2024 -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the primary endpoint was met in the Phase 3 QUASAR trial investigating EYLEA HD® (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO), including those with central, branch and hemiretinal vein occlusions. In the trial, patients treated with EYLEA HD every 8 weeks (after initial monthly doses) experienced non-inferior vision gains compared to those treated with the approved monthly dosing regimen of EYLEA® (aflibercept) Injection 2 mg, the current standard of care. These data will be submitted to regulatory authorities around the world, with a submission to the U.S. Food and Drug Administration (FDA) planned for the first quarter of 2025, and are planned for presentation at an upcoming medical meeting. “All currently FDA-approved anti-VEGF therapies for retinal vein occlusion require monthly dosing, which can be burdensome for a patient. These impressive data from QUASAR demonstrated that EYLEA HD patients with retinal vein occlusion experienced improved vision with fewer injections than EYLEA – which could offer a significant advancement in this treatment setting,” said Seenu M. Hariprasad, M.D., Chair of the Department of Ophthalmology and Visual Science, The University of Chicago. “Furthermore, about 90% of EYLEA HD patients were able to maintain 8-week dosing intervals through 36 weeks.” QUASAR is a global, double-masked, active-controlled Phase 3 trial evaluating the efficacy and safety of EYLEA HD, compared to EYLEA, in patients with RVO. EYLEA HD patients were treated with an 8-week dosing regimen (after 3 or 5 initial monthly doses), and EYLEA patients were treated every 4 weeks. The primary endpoint was met at 36 weeks, with both groups of EYLEA HD patients achieving non-inferior visual acuity gains compared to those receiving EYLEA. EYLEA HD results were consistent across patients with branch retinal vein occlusions, and those with central retinal or hemiretinal vein occlusions. Outcomes at 36 weeks were as follows: BCVA: best corrected visual acuity *Non-inferiority (1-sided) p-values are for the difference in least squares mean compared to EYLEA with margin of 4 letters. EYLEA HD groups met non-inferiority. The safety profile of EYLEA HD (n=591) was similar to EYLEA (n=301) in QUASAR and remained generally consistent with the known safety profile of EYLEA HD in its pivotal trials. Ocular treatment emergent adverse events (TEAEs) occurring in ≥5% of all EYLEA HD patients included increased ocular pressure (5%), and there was one case each of endophthalmitis and retinal vasculitis. The rate of intraocular inflammation was 0.5% for EYLEA HD and 1.3% for EYLEA. Hypertension at baseline was present in 66% of EYLEA HD patients and 62% of EYLEA patients. Hypertension during the trial was reported in 8.1% of EYLEA HD patients and 4.7% of EYLEA patients. Thromboembolic events (APTC) occurred in 0.5% of EYLEA HD patients and 1.7% of EYLEA patients. “With these pivotal results in retinal vein occlusion, EYLEA HD with extended dosing has again met the high bar of vision gains and safety seen with standard-of-care EYLEA,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron, and a principal inventor of EYLEA. “EYLEA HD has already made a significant impact on the treatment of its three approved indications – wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy – and now has the potential to substantially reduce the treatment burden for patients with retinal vein occlusion. We look forward to sharing these results with regulatory authorities around the world as soon as possible.” EYLEA HD (known as Eylea™ 8 mg in the European Union and Japan) is being jointly developed by Regeneron and Bayer AG. In the U.S., Regeneron maintains exclusive rights to EYLEA and EYLEA HD. Bayer has licensed the exclusive marketing rights outside of the U.S., where the companies share equally the profits from sales of EYLEA and EYLEA HD. The safety and efficacy of EYLEA HD for the treatment of RVO has not been evaluated by any regulatory authority. QUASAR is a global double-masked, active-controlled Phase 3 trial evaluating the efficacy and safety of EYLEA HD in patients with macular edema secondary to RVO, including those with central retinal vein occlusion, branch retinal vein occlusion, or hemiretinal vein occlusion. In the trial, patients were randomized into three groups to receive either: EYLEA HD every 8 weeks following 3 initial monthly doses; EYLEA HD every 8 weeks following 5 initial monthly doses; or EYLEA every 4 weeks. The primary endpoint was mean change in BCVA from randomization through week 36, as measured by the Early Treatment Diabetic Retinopathy Study letter score. Patients in the EYLEA HD groups can have their dosing intervals shortened to a minimum of every 4 weeks throughout the trial if protocol-defined criteria for disease progression are met. Dosing intervals may be extended based on protocol-defined criteria starting at week 32 for patients who receive EYLEA or EYLEA HD after 3 initial monthly doses or at week 40 for patients who receive EYLEA HD after 5 initial monthly doses, with follow-up planned through week 64. QUASAR is being operationalized by Bayer under a collaboration agreement with Regeneron. RVO is a common cause of vision loss in adults and the second most common retinal vascular disease. RVO occurs when there is a blockage in a vein in the retina, which leads to a buildup of blood, restricted blood flow, increased pressure and sometimes pain in the eye. RVO may cause sudden blurry vision or vision loss and can ultimately result in serious complications like swelling in the eye called macular edema. A protein called vascular endothelial growth factor (VEGF) is instrumental in causing the vascular leakage that leads to macular edema. When a vein in the retina is blocked, the levels of VEGF increase, which spurs new blood vessel growth. Too much VEGF can lead to the formation of abnormal blood vessels and may cause vision to become blurry. Anti-VEGF injections are commonly used to treat macular edema due to RVO. There are two main types of RVO: central retinal vein occlusion (CRVO) and branch retinal vein occlusion (BRVO). In CRVO, the buildup occurs in the eye’s central retinal vein and in BRVO, the buildup occurs in one of the smaller branch veins. Globally, RVO affects over 28 million people. At Regeneron, we relentlessly pursue groundbreaking innovations in eye care science to help maintain the eye health of the millions of Americans impacted by vision-threatening conditions. Over a decade ago, our breakthrough scientific research resulted in the development of EYLEA, a vascular endothelial growth factor (VEGF) inhibitor designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels in the eye. EYLEA has since brought fundamental change to the retinal disease treatment landscape and is supported by a robust body of research. Regeneron continues to advance our anti-angiogenesis expertise with new solutions with the aim of offering optimal flexibility for a broad group of patients and eye care professionals. This includes EYLEA HD, which has been developed with the aim of extending the time between injections, while maintaining the vision gains, anatomic benefits and safety previously observed with EYLEA. Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. 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点击蓝字 关注我们 本周，热点不少。首先看审评审批方面，非常值得一说的就有两个，一个是礼来阿尔茨海默病新药多奈单抗在中国获批上市，另一个是信达生物/葆元医药ROS1抑制剂获批上市；其次看研发方面，多个药取得重要进展，其中，诺和诺德CagriSema Ⅲ期减重数据公布，68周减重22.7%；最后是交易及投融资方面，翰森制药以20亿美元授予默沙东口服GLP-1受体激动剂全球独家许可权。 本期盘点包括审评审批、研发和交易及投融资三大板块，统计时间2024.12.16-12.20，包含27条信息。  审评审批 NMPA 上市 批准 1、12月18日，NMPA官网显示，礼来1类新药Donanemab注射液获批上市，用于治疗早期阿尔茨海默病。Donanemab是一种用于静脉输注的药物，每四周注射一次。该产品已经于2024年7月获美国FDA率先批准，用于治疗出现早期症状的阿尔茨海默病成年人，商品名为Kisunla。 2、12月20日，NMPA官网显示，由葆元医药申报的己二酸他雷替尼胶囊（Taletrectinib）获批上市，用于治疗经ROS1-TKI治疗失败的ROS1阳性局部晚期或转移性非小细胞肺癌（NSCLC）成人患者。他雷替尼为新一代ROS1酪氨酸激酶抑制剂（TKI），葆元医药与信达生物共同负责该产品在大中华区的开发和商业化。 申请 3、12月17日，CDE官网显示，云顶新耀引进的伊曲莫德（etrasimod）申报上市，用于治疗中重度活动性溃疡性结肠炎（UC）。伊曲莫德（APD334/PF-07915503）是Arena Pharmaceuticals开发的一款高选择性鞘氨醇-1-磷酸（S1P）受体调节剂。2017年12月，云顶新耀以2.24亿美元的总交易额引进伊曲莫德在大中华区和韩国的独家开发、生产和商业化权利。 临床 批准        4、12月16日，CDE官网显示，东阳光药1类新药HEC169584胶囊获批临床，拟开发治疗非酒精性脂肪性肝炎（NASH）。HEC169584是一款在研THR-β激动剂药物，是该公司AI驱动的药物设计（AIDD）实验室开发的首款在研小分子新药。临床前研究结果表明，HEC169584对THR-β细胞的体外活性高，亦具有改善肝纤维化的潜力。 5、12月16日，CDE官网显示，金赛药业1类新药GenSci122片获批临床，拟开发治疗晚期实体瘤。GenSci122片是一种有效的选择性KIF18A抑制剂，具有显著区别于其他细胞周期和抗有丝分裂药物靶点的抗肿瘤特征，已经于2024年10月在美国获批临床。 6、12月16日，CDE官网显示，武田1类新药Mezagitamab注射剂获批临床，拟开发治疗原发性免疫性血小板减少症（ITP）。Mezagitamab是一种全人源免疫球蛋白IgG1单克隆抗体（mAb），对表达CD38的细胞（包括浆母细胞、浆细胞和自然杀伤细胞）具有高亲和力。武田已经于2024年12月启动了mezagitamab治疗成人慢性ITP患者的国际多中心Ⅲ期临床研究。 7、12月16日，CDE官网显示，和誉医药的依帕戈替尼（Irpagratinib、ABSK011）获批临床，用于治疗FGF19过表达的晚期或不可切除肝细胞癌（HCC）。依帕戈替尼是和誉医药自主研发的一款强效的高选择性小分子FGFR4抑制剂，被开发用于治疗FGF19过表达的晚期或不可切除肝细胞癌（HCC）。 8、12月19日，CDE官网显示，百济神州1类新药注射用BG-C137获批临床，拟开发用于晚期实体瘤患者。BG-C137是一款靶向FGFR2b的抗体偶联药物（ADC），百济神州拟开发该产品用于上消化道瘤种和乳腺癌。该公司研发的靶向FGFR2b的ADC具有差异化抗体骨架，有效载荷为拓扑异构酶I抑制剂，DAR为8。 9、12月19日，CDE官网显示，阿斯利康1类新药AZD6234注射液获批临床，拟开发用于在至少有一种肥胖相关合并症的超重或者肥胖成年人中长期体重管理。AZD6234是一款多肽类药物，为每周一次皮下注射的长效胰淀素受体激动剂。该产品正在国际范围内进行Ⅱb期临床研究，阿斯利康拟开发该产品作为肠促胰岛素不耐受肥胖患者的替代治疗方案。 申请 10、12月19日，CDE官网显示，第一三共1类新药DS-3939a申报临床。DS-3939a是一种靶向TA-MUC1的ADC，其结构由人源化抗TA-MUC1抗体、酶切可切割的四肽连接体和具有高药抗比（DAR）的DNA拓扑异构酶I抑制剂（DXd）组成，正在全球范围内处于Ⅰ/Ⅱ期临床研究阶段。 突破性疗法 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16、12月16日，住友制药宣布，一款Menin-KMT2A相互作用抑制剂Enzomenib（DSP-5336）用于治疗复发/难治性急性白血病的Ⅰ/Ⅱ期临床试验数据积极。结果显示，在23例KMT2A重排患者中，客观缓解率为65.2%，达到CR加部分血液学恢复的完全缓解（CRh）的患者比例为30.4%。 17、12月17日，拜耳宣布，Ⅲ期QUASAR研究取得了积极结果，该研究旨在评估阿柏西普8mg相较于标准治疗方案阿柏西普2mg（Eylea）在治疗继发于视网膜静脉阻塞（RVO）的黄斑水肿患者的疗效和安全性。该研究在第36周时达到其主要终点，结果显示，在初始月度给药后，阿柏西普8mg每8周给药1次与当前标准疗法Eylea每4周给药1次相比，患者获得了非劣效性的视力提升。 18、12月17日，赛诺菲与梯瓦宣布，其共同开发的在研单抗duvakitug在临床Ⅱb期研究RELIEVE UCCD中达到了主要终点，接受duvakitug治疗的溃疡性结肠炎（UC）和克罗恩病（CD）患者在第14周时达成缓解，并展现出“best-in-class”疗法的潜力。这是首个评估肿瘤坏死因子样配体1A（TL1A）靶向单抗用于CD治疗的随机、安慰剂对照研究。 19、12月18日，智康弘义宣布，高选择性内皮素受体A（ETA）小分子拮抗剂SC0062在一项针对伴有蛋白尿的慢性肾脏病（CKD）开展的2-SUCCEED临床Ⅱ期试验中，其糖尿病肾病（DKD）队列研究结果达到12周的主要临床终点。试验结果表明：有效性方面，SC0062与安慰剂组相比，高剂量组（20mg）能够显著降低白蛋白尿，结果具有临床意义和统计学意义。 20、12月18日，Immutep宣布，其LAG-3激动剂抗体IMP761在含安慰剂对照、双盲的首个人体Ⅰ期临床试验中获得初步积极安全性数据。在健康受试者中，前三个单次递增剂量队列中均未观察到与治疗相关的不良事件。在临床前研究中，IMP761显著降低炎症细胞因子水平，并显示出抑制抗原特异性T细胞介导免疫反应的效果。 21、12月20日，诺和诺德宣布了Ⅲ期试验REDEFINE 1的结果。在第68周时，CagriSema的减肥效果明显优于安慰剂，达到了主要终点。在评估所有患者都坚持治疗情况下的治疗效果时，68周后，接受CagriSema治疗的患者体重减轻了22.7%，而接受cagrilintide治疗的患者体重减轻了11.8%，接受司美格鲁肽治疗的患者体重减轻了16.1%，单独接受安慰剂治疗的患者体重减轻了2.3%。 交易及投融资 22、12月16日，信达生物宣布，和礼来就非共价（可逆）BTK抑制剂匹妥布替尼片（50mg/100mg、商品名：捷帕力）在中国大陆权益达成合作，信达将负责该药的进口、销售、推广和分销工作；礼来将负责研发和上市后医学事务相关工作。该药于2023年1月获FDA批准，并于2024年10月获NMPA批准，单药适用于既往接受过至少两种系统性治疗（含BTK抑制剂）的复发或难治性套细胞淋巴瘤（MCL）成人患者。 23、12月16日，岸迈生物宣布，和Candid Therapeutics达成战略研究合作，以发现和开发自身免疫适应症的新型T细胞接合分子的候选药物。根据协议，假如多个候选药物能够成功进入商业化阶段，岸迈生物有权获得首付款和总计超过10亿美元的开发和销售里程碑付款，以及基于净销售额的特许权使用费。Candid拥有开发和商业化合作项目的全球独家权利。 24、12月18日，翰森制药与默沙东宣布，双方已签署一种在研的口服小分子GLP-1受体激动剂HS-10535的全球独家许可协议。根据协议，翰森制药授予默沙东开发、生产和商业化HS-10535的全球独家许可权，翰森制药将获得1.12亿美元的首付款，并有资格根据候选药物的开发、注册审批和商业化进展获得最高19亿美元的里程碑付款，同时还将获得基于产品销售的特许权使用费。 25、12月19日，复宏汉霖宣布，与Palleon签署合作协议，以开发和商业化人唾液酸酶融合蛋白E-602与利妥昔单抗（汉利康）的联合疗法，用于治疗包括狼疮肾炎（LN）在内的自身免疫疾病。根据协议，复宏汉霖获得E-602在中国独家许可。Palleon有资格获得高达9530万美元的开发和商业化里程碑付款，以及E-602实现中国商业化后的销售特许权使用费。复宏汉霖将负责E-602联合汉利康用于治疗LN在中国的临床开发并承担相应资金。 26、12月19日，Sangamo Therapeutics宣布，与安斯泰来签署一项许可协议。安斯泰来将使用Sangamo开发的专有神经营养型腺相关病毒（AAV）衣壳STAC-BBB。根据协议，Sangamo将负责完成与STAC-BBB衣壳相关的技术转移，而安斯泰来将承担所有研究、临床前及临床开发、监管事务、生产以及最终基因治疗产品的全球商业化工作。Sangamo将从安斯泰来获得2000万美元的预付款，并有资格在五个潜在神经疾病靶点中，通过许可费用和里程碑付款获得高达13亿美元的额外付款。 27、12月19日，BioArctic宣布，已与百时美施贵宝就焦谷氨酸修饰型β淀粉样蛋白（PyroGlu-Aβ）抗体项目达成全球独家许可协议，包括抗体BAN1503和 BAN2803，其中BAN2803抗体还包括BioArctic的BrainTransporterTM技术。BioArctic将获得1亿美元的预付款和高达12.5亿美元的里程碑付款。BioArctic还有权从全球产品销售中获得分级特许权使用费。 END 【智药研习社近期直播】 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~