Pasithea11月13日公布PAS-004一期数据：15名可评估者皮肤神经纤维瘤均缩小，二期许可已获批
佛罗里达州迈阿密市消息，Pasithea Therapeutics 11月13日公布其在研口服药 PAS-004 的一期与一期 b 数据，数据在维也纳举行的 2026 年欧洲神经纤维瘤会议上发布，公司当日经 GlobeNewswire 发布。
该研究登记号为 NCT06961565，对象为患 1 型神经纤维瘤病相关丛状神经纤维瘤的成人及部分皮肤神经纤维瘤患者。截至 2026 年 9 月 15 日，共有 19 名参加者进入 6 个剂量组，剂量为 4 至 24 毫克，其中 15 人可评估皮肤神经纤维瘤、18 人可评估丛状神经纤维瘤。
结果显示，15 名可评估者的目标皮肤神经纤维瘤表面积均缩小，最佳缩小幅度的中位数为 41%；在计划用于二期的 4、8、12 毫克剂量组，中位最佳缩小为 46%；随访至第 12 周期者中位缩小达 49%，4 毫克组第 15 周期达 71%。18 名可评估者的目标丛状神经纤维瘤体积均缩小，其中 2 人达到 20% 或以上的缩小，1 人在首次治疗中核磁检查时缩小 22.5%。
安全性方面，公司称未见与治疗相关的严重不良事件、停药、视网膜病变或左心室射血分数下降不良事件。美国食品药品管理局皮肤科与牙科部门已批准其二期研究申请，该二期为随机、双盲、安慰剂对照试验，计划纳入约 200 名患相关皮肤神经纤维瘤的成人。公司称，目前尚无获该局批准的针对该类皮肤神经纤维瘤的全身治疗药物。本条只报道数据公布与研究安排信息，不构成对任何个体的诊疗建议。
Pasithea Reports Phase 1 Data for PAS-004 on November 13: All 15 Evaluable Participants Show Cutaneous Neurofibroma Reductions, Phase 2 IND Cleared
Miami, Florida — Pasithea Therapeutics announced on November 13, 2026, positive clinical data from its ongoing Phase 1 and 1b study of PAS-004, an oral MEK inhibitor, presented at the 2026 European Neurofibromatosis Conference in Vienna, Austria, in a GlobeNewswire release.
The study, listed as NCT06961565, covers adults with neurofibromatosis type 1 associated plexiform neurofibromas and a subset of patients with cutaneous neurofibromas. As of September 15, 2026, 19 participants had been enrolled across six cohorts at doses from 4 to 24 milligrams, of whom 15 were evaluable for cutaneous neurofibroma and 18 for plexiform neurofibroma response.
All 15 evaluable participants showed reductions in target cutaneous neurofibroma surface area, with a median best reduction of 41 percent across all doses and 46 percent at the doses planned for Phase 2. The median reduction reached 49 percent at Cycle 12 among participants followed through 12 cycles and 71 percent at Cycle 15 in the 4 milligram cohort. All 18 evaluable participants showed reductions in target plexiform neurofibroma volume, including two with reductions of 20 percent or more, one achieving a 22.5 percent reduction at the first on-treatment MRI.
The company said there were no treatment-related serious adverse events, treatment-related discontinuations, retinopathy, or adverse events of decreased left ventricular ejection fraction. The FDA's Division of Dermatology and Dentistry has cleared the investigational new drug application for a randomized, double-blind, placebo-controlled Phase 2 trial in approximately 200 adults with NF1-associated cutaneous neurofibromas, and the company said there are currently no FDA-approved systemic treatments for that condition. This report covers the published data and study plans only and offers no individual treatment advice.
来源：Pasithea Therapeutics
发布：GSM·美国（发布链接）
图片来源：图：1 型神经纤维瘤病皮肤表现（临床资料图）。作者与授权：Haymanj，Public domain，维基共享资源。 / Photo: Cutaneous signs of neurofibromatosis type 1 (clinical file image). Wikimedia Commons (Haymanj, Public domain).
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