诺华公司首席执行官瓦斯·纳尔辛汉（Vas Narasimhan）周二对CNBC表示：“这款药物可能成为我们有史以来最大规模的药物之一。”此前，这家瑞士制药商对其用于治疗多发性硬化症（MS）的实验性药物remibrutinib寄予厚望，预计其年销售额峰值将超过40亿美元。
"This medicine could be one of our largest medicines in history," Novartis CEO Vas Narasimhan told CNBC on Tuesday, as the Swiss drugmaker backed remibrutinib, its experimental MS medicine, to generate more than $4 billion in peak annual sales.
几天前，巴克莱银行下调了该药物峰值销售额的预测，此前两项后期试验的详细结果显示，该药物减少多发性硬化症患者复发的效果低于分析师预期。
Days before, Barclays cut its forecast for the drug's peak sales after detailed results from two late-stage trials showed the drug had reduced relapses in MS patients by less than analysts expected.
这突显出诺华在准备于11月中旬概述其长期增长战略时面临的一项挑战。
It highlights a challenge for Novartis as it prepares to outline its long-term growth strategy in mid-November.
尽管重磅心脏病药物Entresto失去了关键的专利保护，这家制药商本周仍报告了销售额的增长。但巴克莱分析师表示，市场已经在关注下一个专利悬崖，畅销药基斯卡利（Kisqali）和凯辛帕（Kesimpta）的专利分别定于2032年前后到期。
The drugmaker reported sales growth this week despite losing key patent protection for blockbuster heart drug Entresto. But Barclays analysts said that the market is already looking to the next patent cliff, with bestsellers Kisqali and Kesimpta facing patent expirations around 2032.
巴克莱分析师周三表示，这是“市场更大的问题”，并指出仅基斯卡利和凯辛帕的专利到期就占到180亿美元的峰值销售额。
That's "the market's bigger question," Barclays analysts said on Wednesday, noting Kisqali and Kesimpta expiries alone represent $18 billion in peak sales.
在此背景下，诺华预计remibrutinib的总销售额将超过80亿美元，其中包括复发型多发性硬化症以外的适应症。
Against that backdrop, Novartis has guided for $8 billion-plus in total sales of remibrutinib, including indications other than relapsing MS.
宾夕法尼亚大学的多发性硬化症专家阿米特·巴尔-奥尔（Amit Bar-Or）对CNBC表示，remibrutinib似乎具有良好的安全性，但不太可能像诺华自身的凯辛帕等高效抗体那样有效地控制复发型多发性硬化症。
Amit Bar-Or, a multiple sclerosis specialist at the University of Pennsylvania, told CNBC that remibrutinib appeared to offer a promising safety profile but was unlikely to control relapsing MS as effectively as high-efficacy antibodies like Novartis' own Kesimpta.
他补充说，如果正在进行的试验能够证明该药物对治疗选择仍然有限的进展型多发性硬化症有效，那么该药物扩大市场的最大机遇可能会显现。
He added that the drug's biggest opportunity to expand the market could emerge if ongoing trials demonstrate a benefit in progressive multiple sclerosis, where treatment options remain limited.
Remibrutinib的工作原理 Remibrutinib属于一类被称为BTK抑制剂的药物，旨在减少损害多发性硬化症患者神经的免疫系统活动。
How remibrutinib works Remibrutinib belongs to a class of medicines known as BTK inhibitors, which aim to reduce the immune system activity that damages nerves in MS patients.
凯辛帕的工作原理则不同。它是一种单克隆抗体，能与CD20蛋白结合，清除多发性硬化症中参与自身免疫反应的B细胞。
Kesimpta works differently. It is a monoclonal antibody that binds to the CD20 protein, depleting B cells involved in the autoimmune response in MS.
与凯辛帕不同，remibrutinib旨在调节细胞活动而不消除它们。
Unlike Kesimpta, remibrutinib aims to regulate the cells' activity without eliminating them.
巴尔-奥尔将患者经历病情爆发的复发型多发性硬化症的生物学特性，与随着时间逐渐恶化的进展型多发性硬化症区分开来。
Bar-Or distinguished the biology of relapsing MS — in which patients experience flare-ups — from progressive MS, which worsens gradually over time.
巴尔-奥尔表示，remibrutinib在治疗复发型多发性硬化症方面的临床结果似乎与罗氏（Roche）的竞争对手BTK抑制剂fenebrutinib相似，但在预防复发方面不如凯辛帕等抗CD20疗法有效。
Clinical results for remibrutinib in relapsing MS appear to be similar to Roche's rival BTK inhibitor fenebrutinib, but less effective at preventing relapses than anti-CD20 treatments such as Kesimpta, according to Bar-Or.
目前尚未开展将remibrutinib与fenebrutinib或抗CD20疗法进行对比的头对头试验，这使得直接比较变得困难。
No head-to-head trials have yet compared remibrutinib with fenebrutinib or anti-CD20 treatments, making direct comparisons difficult.
他补充说，对于在使用抗CD20药物期间病情出现进展的患者来说，它仍然可能是一个具有吸引力的转换选择。一项旨在测试remibrutinib能否有效治疗进展型多发性硬化症的正在进行的临床试验预计将于2031年公布结果。
It could still be an attractive switch for patients who are experiencing progressive MS while on an anti-CD20, he added. An ongoing clinical trial testing whether remibrutinib can effectively treat progressive MS is expected to read out in 2031.
巴尔-奥尔表示，BTK抑制剂的一个潜在优势是它们调节免疫细胞而不是消除它们，这可能使其长期安全性比某些现有的多发性硬化症治疗方法更好。不过，他补充说，目前尚没有足够的长期安全性数据来证实这一点。
One potential advantage of BTK inhibitors is that they regulate immune cells rather than eliminate them, which could make them safer over the long term than some existing MS treatments, Bar-Or said. However, there is not yet enough long-term safety data to confirm this, he added.
诺华发言人对CNBC表示，该公司主要将remibrutinib视为扩大其多发性硬化症产品组合的机遇，而不是替代凯辛帕的销售。
A Novartis spokesperson told CNBC that it views remibrutinib primarily as an opportunity to expand its MS portfolio, rather than replace sales of Kesimpta.
该公司的目标群体包括新确诊患者、目前正在接受口服治疗的患者，以及可能符合转换条件的接受抗CD20疗法的患者。
The company is targeting newly diagnosed patients, those currently taking oral treatments, and patients receiving anti-CD20 therapies who may be eligible to switch.
诺华认为，凯辛帕的年销售额峰值机遇超过60亿美元，而remibrutinib在复发型多发性硬化症领域的年销售额峰值机遇则超过40亿美元。
Novartis sees a $6 billion-plus peak annual sales opportunity for Kesimpta alongside $4 billion-plus for remibrutinib in relapsing MS.
Remibrutinib销售前景 晨星公司（Morningstar）分析师杰伊·李（Jay Lee）对CNBC表示，诺华对remibrutinib在多发性硬化症领域销售额峰值超过40亿美元的预测“在可能范围内”，但并非“没有风险”。李补充说，这取决于remibrutinib与罗氏竞争对手fenebrutinib的对比情况、药品定价谈判、其潜在的耐受性优势是否能转化为患者延长用药时间，以及临床试验是否支持将患者从现有的一线多发性硬化症治疗药物转换过来。
Remibrutinib sales outlook Morningstar analyst Jay Lee told CNBC that Novartis' $4 billion-plus peak sales forecast for remibrutinib in MS was "within the realm of possibility" but wasn't "de-risked." Lee added that this would depend on how remibrutinib compares with Roche's rival fenebrutinib, drug pricing negotiations, whether its potential tolerability advantages translate into patients staying on treatment longer, and whether clinical trials support switching patients from existing MS treatments.
“在短期内，我预计remibrutinib最大的市场将是口服转换市场，即需要转换药物的服用仿制口服治疗药物的患者，特别是出于耐受性原因的患者。”目前，投资者正将注意力集中在RESHAPE试验上，该试验招募了年龄在40至70岁之间、正在使用罗氏抗CD20抗体Ocrevus的患者，预计将于2028年公布结果。
"In the near term, I expect the largest market for remibrutinib will be the oral switch market, i.e. patients taking a generic oral treatment who need to switch, particularly due to tolerability reasons." For now, investors are focusing on the RESHAPE trial, which enrolled patients aged 40 to 70 taking Roche's anti-CD20 antibody Ocrevus, and is expected to read out in 2028.
李表示，虽然这可能会让remibrutinib从Ocrevus和凯辛帕等CD20药物手中夺取一些市场份额，“但如果这意味着他们从目前引领市场的罗氏手中获得了整体RMS（复发型多发性硬化症）患者份额，这可能仍然对诺华有利”，他并补充说，诺华估计在符合转换条件的CD20药物患者中，大约有75%没有使用凯辛帕。
While that could allow remibrutinib to take some market share from CD20 drugs like Ocrevus and Kesimpta, it "may still work in Novartis' favor if it means they gain overall RMS patient share from Roche, which currently leads the market," Lee said, adding that Novartis estimates about 75% of switch-eligible patients on CD20 drugs are not on Kesimpta.
此外，remibrutinib已被批准用于治疗某些形式的慢性荨麻疹，一项测试其用于治疗慢性炎症性皮肤病化脓性汗腺炎（HS）的后期临床试验预计将在11月19日之前公布结果。
Additionally, remibrutinib is already approved to treat certain forms of chronic hives, and a late-stage clinical trial testing it for the chronic inflammatory skin disease hidradenitis suppurativa (HS) is expected to read out before Nov. 19.
巴克莱估计，其在化脓性汗腺炎领域的年销售额峰值机遇为20亿美元，但鉴于中期试验中出现的不寻常的剂量反应模式以及以往在化脓性汗腺炎后期试验中失败的历史，该行给予该药物的成功概率仅为50%。
Barclays estimates a $2 billion peak annual sales opportunity in HS but assigns the drug only a 50% probability of success, citing an unusual dose-response pattern in mid-stage trials and a history of late-stage trial failures in HS.