Plozasiran

iStock, Afry Harvy Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. Arrowhead Pharmaceuticals’ shares have fallen 12% in the past month, but it had little to do with work going on at the RNA interference specialist. Instead, it was Arrowhead’s association with Sarepta Therapeutics—with which it has a partnership worth as much as $11 billion —that sowed the chaos at the markets. “We have been caught in that downdraft as well, unfortunately,” Arrowhead CEO Chris Anzalone told BioSpace , speaking to Sarepta’s gene therapy safety drama that has been playing out over the past month. “And it’s not unreasonable. I think that people were curious about Sarepta’s ability to perform on the partnership.” For the record, Anzalone isn’t worried about Sarepta dropping out of the alliance. He thinks the deal is too sweet for both sides. H.C. Wainwright Research agreed in a July 31 note, calling the partnership “symbiotic and mutually beneficial.” Annalee Armstrong for BioSpace “[Sarepta] have said quite clearly that—and I don’t want to put words in their mouth, but my interpretation is—the future of their company is based on the assets they got from us,” Anzalone said. Indeed, Sarepta last month announced a strategic restructuring and pipeline pivot away from its legacy gene therapies and toward a handful of siRNA platform assets that are partnered with Arrowhead. Even so, Anzalone understands why investors have been nervous. “Cash from Sarepta is a big part of our financial model over the next two years,” he said. HCW pointed out that the non-dilutive capital provided by Sarepta is key to Arrowhead’s plans to reach cash flow–positive operations by 2030. With some explaining of what happens if Sarepta doesn’t pay, Anzalone said that investors have been reaching the place he is at right now. “I’m almost at equivoce. The deal goes as planned. Great. The deal goes under, we get our assets back and we keep the cash. That’s also fine.” The Future of Sarepta Is Here Sarepta’s pipeline pivot should have been good news for Arrowhead—it was a vote of confidence for the biotech’s platform and a sign that Sarepta saw the massive partnership as its future. But a day later, the company’s months-long safety drama came to a head. On July 18, Sarepta revealed that a third patient had died after taking one of its gene therapies. This spurred a market tumble for both companies. While Arrowhead kept its head down, Sarepta became wrapped up in a very public dispute with the FDA. Analysts began to wonder if Sarepta could keep up its commitments to Arrowhead. The deal, signed in November 2024, is worth $825 million upfront, with as much as $10 billion possible later on through a series of consistent payments tied to enrollment and other clinical development milestones. The partnership is focused on rare genetic diseases of the muscle, central nervous system and lung, with two key Phase I muscular disease programs: ARO DM1 for type 1 myotonic dystrophy (DM1) and ARO-DUX4 for facioscapulohumeral muscular dystrophy type 1 (FSHD1). The deal also includes two other clinical-stage therapies and three preclinical programs. Arrowhead has been working swiftly behind the scenes on the Sarepta programs, already meeting one of the Phase I enrollment targets for ARO DM1 that would trigger a $100 million milestone. This payment is due by the end of the fourth quarter under the terms of the agreement. Anzalone doesn’t anticipate any issues with that payout. “I expect [Sarepta] to continue to perform, and we’ve seen no evidence that they will not,” Anzalone said. “I would read into that that they’re going to spend their last dollar to make sure that [the deal] stays intact, because it’s so important for them strategically.” But if they don’t, Anzalone said the contract has solid termination framework, which HCW also noted. If Sarepta stops paying, the assets fall back to Arrowhead and no cash has to be returned. “Look, I’m okay with either of those things—again, I don’t think the latter is going to happen. I think they’re going to perform,” Anzalone said. “The fail-safe here is that we get a bunch of really good assets back and move forward ourselves.” The CEO also expressed an openness to work with Sarepta in the future to tweak the deal if needed. While the situation is a little uncertain, Anzalone said he’s confident in one thing: “These are good assets.” Looking back at the deal process, Anzalone said he would still sign the same one today. Sarepta—which is known for its Duchenne muscular dystrophy therapies—was a natural fit for ARO-DUX4, an RNA interference (RNAi) conjugate in development for the progressive genetic muscle disease FSHD1. Anzalone remembers the scope of the partnership growing as discussions went on with Sarepta CEO Doug Ingram. Sarepta at that time was in the initial stages of commercializing Elevidys after its June 2023 approval—before it ran into the safety problems it faces today—but had a fairly empty pipeline. “They appear to be good at regulatory, commercial and late-stage development, but they didn’t have much of a pipeline. And we are nothing but pipeline,” Anzalone said. “So it just kind of grew. And next thing you knew, we were doing what was going to be a very large deal.” The final terms eventually work out to somewhere around $11 billion. Besides the $100 million ARO DM1 enrollment milestone, Anzalone expects to trigger another $200 million by the end of the year for the same program, plus a $50 million milestone in February 2026 for an R&D milestone. “We didn’t pick anybody’s pocket. This was, I think, as good a deal for Sarepta as it was for us,” Anzalone said. “This is one of those funny deals that I would be glad to take either side of the transaction.” Data from the first Sarepta-partnered programs ARO DM1 and ARO DUX4 are expected by the end of the year, and while there’s no firm date, Anzalone suspects Sarepta’s leadership will be eager to make the results public once they’re ready. He said those data are expected to be interpretable—which is to say, investors should get their first true assessment of the programs’ likelihood of success. “Too early data that doesn’t tell a story . . . can be confusing, but as soon as there is a nugget of a story, I’d like to put that out,” Anzalone said. Pizza and Beer Money The modality itself, RNAi, is derisked thanks to larger peer Alnylam Pharmaceuticals, and Arrowhead is largely seen as the next in line biopharma to capitalize on the technology. Besides Sarepta, Arrowhead is also partnered with Amgen, GSK and Takeda and is awaiting its first FDA approval this fall for the lead candidate in its wholly owned pipeline. Plozasiran is set for a Nov. 18 decision date for possible approval in familial chylomicronemia syndrome (FCS), a rare genetic metabolic disorder that prevents the body from breaking down fats. “[Arrowhead’s] RNAi pipeline is steadily advancing toward commercial-stage maturity, independent of partnership support,” HCW said. With such a focus on partnering, Anzalone said it was important to the team to take plozasiran all the way alone, even though Arrowhead could have continued as just an R&D company. With that kind of model, “you can stay capitalized and stay in what I call pizza and beer, but you can’t really make that leap to be an independent, longer-term valuable biotech company that say Vertex, Regeneron and the like have translated into,” the CEO said. To ensure success, Anzalone said the team has bulked up with regulatory experts and is focusing on the small FCS market first. “We, like any company who goes from an R&D-only organization to R&D plus commercial, are going to make a lot of dumb mistakes,” Anzalone admitted. “But it’s an ultra-rare indication and [has] allowed us to kind of get our feet wet and learn how to be a commercial organization.” Afterward, Arrowhead is targeting the much larger severe hypertriglyceridemia indication with plozasiran. The therapy is currently being tested in a trio of Phase III clinical trials with completion expected in mid-2026. These readouts could pave the way for Arrowhead’s second FDA nod. Besides the lead plozasiran program, Arrowhead has caught the obesity craze and is working on two early assets. Of the two, Anzalone is most excited about ARO-ALK7, which is meant to silence the ACVR1C gene, in turn reducing the risk of obesity-related metabolic complications. These two programs are not up for partnering, Anzalone says. Arrowhead is specifically focused on providing better weight loss and improving tolerability. “This is the health need of our time in western economies and so the GLP-1s are a very good start, but there’s a ton of white space there.”

跨国药企在中国重点资讯 文 | 苏丁 企业动态 碧迪医疗 碧迪医疗宣布，其首台本土化生产的流式细胞仪BD FACSLyric正式投入批量生产，在碧迪快速诊断产品（苏州）有限公司举办剪彩活动。流式细胞仪BD FACSLyric的本土化战略通过本地化生产全链路构建，使国内医疗及科研机构能够更高效地获取到先进的流式设备，直接提升了临床检验与科研工作的效率。更关键的是，该战略通过全价值链赋能体系的打造，有力地推动了中国流式细胞术创新生态链向高质量发展阶段迈进。 诺和诺德诺和诺德CEO齐亚尔·迈克·杜斯特达（Maziar Mike Doustdar）在电话会上回应了投资人关于司美格鲁肽在中国市场上面临的竞争挑战。杜斯特达表示，虽然中国GLP-1糖尿病药出现了下滑，但减重药销售是逐步增长的。他解释称，糖尿病药销量出现下滑是由于经销商库存发生的变化；而去年刚刚获批的司美格鲁肽减重药目前还在放量阶段，尚未进一步下沉到二、三、四线城市。 富士胶片8月7-8日，第八届生物药工艺发展大会（BPD）在上海张江科学会堂举办。富士胶片生命科学作为重要参展商亮相，围绕三大核心业务展示旗下最新成果：细胞培养解决方案，包括无血清培养基和细胞培养补充剂；iPSC衍生细胞工具，如人来源iPSC分化的心肌细胞、神经细胞等；实验室试剂与质量控制解决方案。 艾昆纬科兴制药与IQVIA艾昆纬达成全面战略合作关系，双方将链接彼此优势资源，共同推进科兴制药创新药和生物类似药在欧洲，乃至全球市场的临床设计、注册与商业化进程。 飞利浦 国内医疗信息化领域领先企业创业慧康公告，公司第二大股东飞利浦（中国）投资有限公司，计划减持创业慧康股份不超过4633.28万股（含），占剔除公司回购专用账户股份后总股本比例为3%。公开信息显示，飞利浦目前持有该公司股份1.55亿股，占其总股本比例为10.01%，占剔除公司回购专用账户股份后总股本比例为10.04%。 德达医疗7月25日，中国红十字基金会生命教育项目办主任陈珞一行赴上海德达医院，看望在此接受治疗的42名来自西藏、新疆的先心病患儿。此次接受治疗的患儿中，19人来自西藏林芝，23人来自新疆喀什地区和巴音郭楞蒙古自治州。他们在中国红十字基金会2025年“天使之旅”新疆、西藏先心病患儿筛查救助活动中，被上海德达心血管医院黄连军、刘建实率领的筛查医疗团队确诊为先心病。经专家确诊符合手术条件的患儿，分批前往德达医院接受治疗。 产业动态 赛诺菲停止波立达(阿利西尤单抗注射液)在中国市场的供应。对此，赛诺菲方面表示，停供是因为全球供应方面的问题，以及公司在中国心血管产品策略的升级：公司去年年底与箕星药业的Aficamten达成了大中华区BD合作协议，近期也宣布了获得维亚臻在研药物普乐司兰钠注射液大中华区权利，公司在华的心血管产品策略将进行整体升级。赛诺菲方面还强调，将持续提供不同领域的产品，满足国内患者的需求。 卫材（中国）药业有限公司宣布，首批创新药物达卫可（通用名：莱博雷生）已抵达中国并顺利完成药品检验流程，并已正式启程发往全国各大医院及指定药店，在北京大学第六医院成功开出全国首方。达卫可在京东健康全网首发。作为中国首个获批上市的双食欲素受体拮抗剂，该药通过精准调节睡眠－觉醒节律，为失眠患者提供全新治疗方案。 强生宣布，旗下创新治疗药物锐珂（埃万妥单抗注射液）正式获得国家药品监督管理局批准，联合利珂（甲磺酸兰泽替尼片）适用于携带表皮生长因子受体（EGFR）19号外显子缺失或21号外显子L858R置换突变的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者的一线治疗。此次获批标志着埃万妥单抗在中国迎来了今年第三个肺癌适应症。 阿斯利康宣布，其中国首个呼吸生物制剂凡舒卓（英文商品名：Fasenra，通用名：本瑞利珠单抗注射液）新适应症在中国正式批准，用于儿童（6至＜12岁）重度嗜酸粒细胞性哮喘（SEA）的维持治疗。本瑞利珠单抗是精准靶向嗜酸性粒细胞的抗IL-5R生物制剂，此前已在中国获批用于成人和12岁及以上青少年SEA的维持治疗。 CDE官网显示，诺华OAV101注射液申报上市，这是一种基于腺相关病毒（AAV）载体的基因治疗药物，适用于治疗6月龄及以上5q 型脊髓性肌萎缩症（SMA）患者。最初由AveXis公司开发，诺华在2018年以87亿美元收购AveXis公司后获得该产品。 华特达因公告，子公司北京达因康健医药有限责任公司与日本诺贝仁制药株式会社签署褪黑素颗粒总经销协议。根据协议，达因康健成为诺贝仁旗下褪黑素颗粒产品（曼乐静）在中国大陆地区（不含港澳台地区）的独家总经销商，负责进口、市场推广及销售。该产品已在中国获批上市，用于改善6-15岁神经发育障碍儿童的入睡困难。合作期限为10年，届满后可续签。 波士顿科学公司宣布旗下WATCHMAN FLX Pro带聚偏氟乙烯-六氟丙烯（PVDF-HFP）涂层的左心耳封堵器正式获得国家药品监督管理局批准。该产品适用于具有高卒中风险的非瓣膜性房颤患者，可替代口服抗凝药物治疗预防脑卒中风险。 泰利福宣布，其旗下Rüsch EZ-Blocker 一次性使用双侧支气管堵塞器已成功通过中国国家药品监督管理局（NMPA）的严格审评审批，并正式取得医疗器械注册证。此次获批，是泰利福深耕中国市场、满足中国患者医疗需求的又一重要举措。 晶泰科技与DoveTree完成总订单规模约470亿港元（59.9亿美元）的管线合作签约，并已收到协议约定的首付款约4亿港元（5100万美元）。根据协议条款，晶泰科技与DoveTree就多款处于临床前阶段的大分子及小分子创新药资产达成合作，并将针对一系列DoveTree指定的靶点进行新药研发，DoveTree独家享有相关药物全球范围的独家开发和商业化权利。除首付款外，晶泰科技还有权获得约3.85亿港元（4900万美元）的进一步付款，以及金额达约462亿港元（58.9亿美元）的潜在里程碑付款及销售分成。 乐普生物宣布与Excalipoint就两项临床前T细胞衔接器（TCE）资产的知识产权许可或转让事宜签订协议。Excalipoint将获得在全球范围内开发及商业化目标产品的独家权利。乐普生物将收取总计1,000万美元现金的首付款；通过全资子公司Innocube获得Excalipoint Cayman扩大后股本的10%普通股；总计最高8.475亿美元之开发及商业化里程碑现金付款。本次交易涉及的两款临床前资产是乐普生物基于其自有的TCE平台——TOPAbody平台开发的新一代TCE分子，其中CTM012已获IND批准，另一款为CTM013。 京新药业与Gedeon Richter Plc. 签署《专利许可协议》。Gedeon Richter将授予公司一项专利独占许可，公司可在许可区域（中国大陆）内使用该专利开发、制造和商业化盐酸卡利拉嗪制剂及原料药（API）。盐酸卡利拉嗪为非典型抗精神病药物，属于多巴胺D2、D3受体部分激动剂。 联系美通社 +86-10-5953 9500 info@prnasia.com