Sonelokimab

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。

Analysts at Leerink Partners described the readout as “arguably falling into the worst case outcome” for the MoonLake program.\n MoonLake Immunotherapeutics’ stock has taken a pummeling after the biotech blamed an unexpectedly high placebo response rate for its much-hyped inflammatory disease prospect missing the key goal of one of a pair of phase 3 studies.The company has been evaluating a single 120-mg dose of the IL-17 inhibitor, called sonelokimab, across two identical studies in 838 patients with moderate to severe hidradenitis suppurativa (HS). The primary endpoint was measured by the proportion of patients who saw at least a 75% reduction in abscess and inflammatory nodule count when compared to placebo.The proportion of patients who achieved this primary endpoint was similar for both the VELA-1 and VELA-2 studies—at 34.8% and 35.9%, respectively. However, the placebo response rates were more varied—at 17.5% and 25.6%. The “higher-than-expected” placebo response rate in VELA-2 meant that the study by itself missed the endpoint.Still, MoonLake said it had previously specified that it was entitled to analyze the data “irrespective of intercurrent events,” which still allowed the company to claim a win overall. Both studies also hit key secondary endpoints related to lesion count and patient-reported outcomes, the biotech noted.“We are encouraged by the results of VELA-1, which follow the expected performance of sonelokimab in all the important metrics for patients and treating physicians,” MoonLake Chief Scientific Officer Kristian Reich, M.D., Ph.D., said in the release.“The higher-than-expected placebo response rate in VELA-2 is disappointing but we are encouraged by the consistent performance of sonelokimab arms across all endpoints in both studies,” Reich said. “We are pleased to see a favorable safety profile consistent with previous studies, with no new safety signals.”The warm words weren’t enough to reassure investors, which sent the company’s stock plummeting 87% to $7.81 in premarket trading Monday morning from a Friday closing price of $61.99. MoonLake will be discussing the unexpected placebo data with regulators while the company continues to chart a path toward a hoped-for approval for the drug. As well as the VELA-1 and VELA-2 studies, which are due to read out 52-week data in the second quarter of 2026, there is also a phase 2 study in palmoplantar pustulosis due to read out this year, and next year brings the results of a phase 2 study in axial spondyloarthritis and phase 3 studies in adolescent HS and psoriatic arthritis.Analysts at Leerink Partners described yesterday’s readout as “arguably falling into the worst case outcome” for the VELA program.“We believe these results have introduced uncertainty around sonelokimab’s path to approval in HS on basis of the two VELA trials, though we await more details on a potential path forward and FDA’s receptivity to pooled results, treatment policy analyses, and MoonLake’s arguments around the totality of data,” they wrote in a Sept. 28 note.Reich said the company is still convinced by sonelokimab’s “convenient dosing, the efficacy data in the lesion-based metrics and the patient-reported outcomes.” But yesterday\'s mixed data may take the shine off an asset that was reportedly attracting Big Pharma attention.The Financial Times reported in June that Merck & Co. had submitted a nonbinding offer that valued MoonLake at more than $3 billion earlier this year. The offer was rejected, but talks could be revived, the FT reported at the time.MoonLake licensed sonelokimab from Germany’s Merck KGaA in 2021. The molecule originated at the nanobody specialist Ablynx, which is now part of Sanofi.