Pasithea reports early PAS-004 tumor reductions and FDA clearance for Phase 2
On November 13, 2026, Pasithea Therapeutics reported Phase 1/1b data for PAS-004 in adults with neurofibromatosis type 1. Each of the 15 evaluable cNF participants had a reduction in target tumor area, with a median best decrease of 41% across doses and 46% at doses planned for Phase 2. All 18 evaluable PN participants had lower tumor volume, two met the partial-response threshold, and the FDA cleared the investigational new drug application for a Phase 2 cNF study.
Why it matters
The FDA decision permits Pasithea to advance PAS-004 into a randomized, placebo-controlled Phase 2 study involving approximately 200 adults, according to the company. The current efficacy findings come from an ongoing early-stage study, so the Phase 2 trial will provide the next controlled test of the drug’s activity.
Key facts
- 1Among the 15 evaluable cNF participants, the median strongest reduction in target surface area was 41% across all tested doses. orthospinenews.com
- 2Participants treated at 4, 8 and 12mg had a median best cNF surface-area reduction of 46%. orthospinenews.com
- 3All 18 evaluable PN participants showed lower tumor volume, while two met the partial-response criteria; one recorded a 22.5% decrease at the first on-treatment MRI. tradingview.com
- 4The FDA’s Division of Dermatology and Dentistry cleared the IND for a Phase 2 PAS-004 trial in adults with NF1-associated cNF. orthospinenews.com
- 5Pasithea plans a randomized, placebo-controlled Phase 2 study of three once-daily doses in approximately 200 adults with NF1-associated cNFs. rttnews.com
- 6No treatment-related serious adverse events, treatment-related discontinuations, retinopathy or decreases in left ventricular ejection fraction were reported. orthospinenews.com
Open questions
- The Phase 1/1b efficacy results are from an ongoing open-label study without a comparator, and Phase 2 efficacy and safety results are not yet available.
Summary written by AlphAI AI Desk from 4 of 4 sources. Not investment advice. Figures are as stated by the linked sources.