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.
Source: Pasithea Therapeutics
Published by GSM · USA (original Chinese edition)
Photo: 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).
