Bimekizumab

2025年9月30日，MoonLake Immunotherapeutics因其唯一核心药物 sonelokimab 在两项关键 III 期临床试验中表现远逊预期。 sonelokimab工作机制 股价在周一暴跌逾 89%，市值蒸发逾 35 亿美元，市值从近 40 亿美元缩水至不足 4 亿美元。成为近期生物医药行业最惨烈的“黑天鹅”事件之一。 试验数据一成一败 MoonLake 于 9 月 28 日公布了 sonelokimab 用于治疗中重度化脓性汗腺炎（HS）的两项 III 期试验（VELA-1 与 VELA-2）的 16 周数据。尽管公司宣称试验“达到主要终点”，但市场普遍认为结果令人失望： - 在 VELA-1 试验中，sonelokimab 组的应答率为 35%，安慰剂组为 18%，差距仅为 17 个百分点； - VELA-2 试验中，差距进一步缩小至 9 个百分点，并未达到统计学显著性； - 合并分析显示，该药的安慰剂调整后疗效仅为 14%，远低于竞争对手 UCB 的 Bimzelx（18%）。 摩根士丹利、摩根大通、Stifel 等多家投行迅速下调评级，认为 sonelokimab 的疗效“劣于竞品”，并显著降低其获批与商业化概率。Jefferies 将目标价从 65 美元大幅下调至 8 美元，RBC 更是将目标价砍至 10 美元。 管理层强行“乐观表态” 尽管数据不佳，MoonLake 管理层在公告中仍试图传递积极信号。公司创始人兼首席科学官 Kristian Reich 表示：“我们对 VELA-1 的结果感到鼓舞，它符合 sonelokimab 在患者和医生关注的关键指标上的预期表现。” 然而，这一“乐观表态”并未获得投资者认同。市场普遍认为公司管理层在“强行正面解读”失败数据，甚至有分析师直言：“这是一场灾难性的数据发布，管理层的信心与市场现实严重脱节。” 结束语： sonelokimab 是 MoonLake 唯一进入临床后期的药物，此次试验失败意味着公司短期内几乎失去所有商业化前景。尽管公司表示将寻求与监管机构讨论审批路径，但分析师普遍认为其获批概率已大幅下降，甚至不排除项目被彻底搁置的可能。 值得注意的是，MoonLake 曾在今年早些时候拒绝默沙东（Merck & Co.）提出的超过 30 亿美元的非约束性收购要约。如今随着核心药物失败，公司并购价值大幅缩水，未来是否还能吸引大型药企青睐，成为市场关注焦点。 本稿件基于公开信息撰写，不构成投资建议。

MoonLake Immunotherapeutics’ one and only pipeline drug has generated both a hit and a miss in paired pivotal trials testing it in the painful skin disorder hidradenitis suppurativa. Expectations had been high for the drug, called sonelokimab, with investors hoping that an unequivocal win could lead to a lucrative partnership or an M&A deal. MoonLake’s stock $MLTX dropped around 85% premarket on Monday. Data released Sunday showed that sonelokimab, a nanobody that inhibits IL-17A/F, missed MoonLake’s stated expectations and also fell short of UCB’s established hidradenitis suppurativa therapy Bimzelx on a cross-trial basis. In the VELA-1 trial, 34.8% of patients taking sonelokimab hit a bar called HS clinical response (HiSCR) 75 after four months’ treatment. HiSCR75 represents a 75% reduction in the number of abscesses and inflammatory nodules that characterize the disease. In the trial’s placebo arm, 17.5% met this goal, and the difference was statistically significant. However, the HiSCR75 rate with sonelokimab in VELA-2 was 35.9%, versus 25.6% with placebo. Using a strict statistical analysis that controlled for multiple testing, the delta over placebo was just 9%. That trial did not hit statistical significance (p=0.053). MoonLake said the placebo response in VELA-2 was “higher than expected.” Leerink analysts wrote that the placebo-adjusted figures came in well below MoonLake’s expectations of at least 20%. The identical studies evaluated a 120 mg subcutaneous dose of MoonLake’s nanobody every two weeks for the first six weeks, and once a month starting at week eight. The trials enrolled a total of 838 patients with moderate-to-severe HS. When the data from the two trials were combined, sonelokimab was statistically significantly better than placebo, with a 13.8 percentage point difference over the control arm. But analysts from Stifel called the pooled data “less than competitive.” That’s short of what UCB’s drug achieved in its two trials. In those, Bimzelx achieved a margin over placebo of 15 and 20 percentage points on the four-month HiSCR75 measure, though they used a different statistical analysis. Bimzelx is also an IL-17A/F inhibitor and is a blockbuster thanks to its approval in several indications, including psoriasis as well as HS. Sonelokimab’s safety readout was clean, with 2.9% of patients on the drug discontinuing treatment across both studies because of side effects. That rate was around twice as high as with placebo. MoonLake said it would submit the data to regulators and discuss a path to approval. But the drug’s potential market looks slimmer. So do the chances of MoonLake scoring a deal with a partner or buyer. This summer, Merck was rumored to be interested in buying the biotech. Some had predicted the weak results: Stifel analysts wrote in September that the drug had “limited differentiation versus other IL-17s and an uncertain near-term regulatory path in HS.” They lowered their potential of success for the product from 75% to 33%.