食品药品监管局 FDA 解除 Aardvark 候选药 ARD-101 临床暂停：San Diego 公司将恢复普拉德-威利综合征试验
San Diego讯：总部位于 San Diego 的临床阶段生物制药公司 Aardvark Therapeutics（纳斯达克代码：AARD）10月22日宣布，美国食品药品监管局（Food and Drug Administration，FDA）已解除对其候选药 ARD-101 新药临床试验申请的临床暂停（clinical hold）。ARD-101 用于治疗普拉德-威利综合征（Prader-Willi Syndrome，PWS）患者的过度进食（hyperphagia），临床开发工作可以恢复。
这项暂停源于今年早些时候的一项安全监测发现。2026年2月27日，Aardvark 主动暂停了三期 HERO 试验及其开放标签扩展试验的给药，原因是另一项健康志愿者研究在例行安全监测中观察到可逆的心电图变化；同年5月，FDA 对这两项试验正式实施临床暂停。此后，公司按与 FDA 协商一致的方案，在更多健康志愿者中完成了一项全面的心电图研究，并完成了一项体外心脏钠通道（NaV1.5）研究；公司称，在新完成的心电图研究中，每日两次200毫克与400毫克剂量下均未观察到心脏事件。
Aardvark 创始人兼首席执行官 Tien Lee 表示，公司对 FDA 解除暂停、让 ARD-101 继续推进临床开发感到欣慰，并称普拉德-威利综合征患者及其家庭仍面临深切的未满足医疗需求。公司表示，将在与 FDA 就三期试验设计进一步沟通并达成一致后，恢复 ARD-101 在 PWS 患者中的临床开发；此前公司已对 HERO 与开放标签扩展两项已终止试验累积的数据进行揭盲评估，并定于10月23日上午8时（美东时间）举行网络会议介绍相关数据。
普拉德-威利综合征是一种罕见遗传疾病，特征之一是无法满足的饥饿感。ARD-101 是一种口服小分子药物，通过激活苦味受体刺激肠道肽类激素释放，以抑制饥饿感。据道琼斯通讯社报道，消息公布后，Aardvark 股价在盘后交易中上涨14%，至每股4.52美元。
FDA Lifts Clinical Hold on Aardvark's ARD-101, Clearing San Diego Biotech to Resume Prader-Willi Trial Work
San Diego: Aardvark Therapeutics, Inc. (Nasdaq: AARD), a clinical-stage biopharmaceutical company based in San Diego, announced on October 22 that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Investigational New Drug application for its candidate ARD-101, being developed to treat hyperphagia in people with Prader-Willi Syndrome (PWS). Clinical development of ARD-101 may now resume.
The hold traced back to a safety monitoring finding earlier this year. On February 27, 2026, Aardvark voluntarily paused dosing in its Phase 3 HERO trial and its open-label extension trial after reversible electrocardiogram (ECG) changes were observed during routine safety monitoring in a separate study in healthy volunteers. In May 2026, the FDA placed a formal clinical hold on both trials. Working in alignment with the FDA, the company then completed a thorough ECG study in additional healthy volunteers and an in vitro cardiac sodium channel (NaV1.5) study; it said no cardiac events were observed in the recently completed ECG study at doses of 200 mg and 400 mg twice daily.
Tien Lee, M.D., Aardvark's founder and chief executive officer, said the company was pleased the FDA lifted the hold, allowing it to continue advancing the program, and noted that people living with PWS and their families continue to face profound unmet need. The company said it plans to resume clinical development of ARD-101 in patients with PWS following further engagement and alignment with the FDA on the Phase 3 trial design. Aardvark has unblinded data accumulated across the terminated HERO and open-label extension trials and will host a webcast on October 23 at 8:00 a.m. Eastern Time to review the available dataset.
Prader-Willi Syndrome is a rare genetic disorder characterized in part by insatiable hunger. ARD-101 is an oral, small-molecule therapeutic designed to stimulate the release of gut-peptide hormones through activation of bitter taste receptors. According to Dow Jones Newswires, Aardvark shares rose 14% to $4.52 in after-hours trading following the announcement.
来源：GSM·美国（原文链接）
图片来源：图：生物技术实验室内的研究人员（资料图）。Idaho National Laboratory 摄，CC BY 2.0，经维基共享资源。Photo: Researchers in a biotechnology laboratory (file photo). Photo by Idaho National Laboratory, CC BY 2.0, via Wikimedia Commons.
