Pasithea’s PAS-004 Reduces Cutaneous Neurofibromas In All 15 Evaluable Participants, 41% Median Best Reduction, 46% At Planned Phase 2 Doses, With No Treatment-Related Serious Adverse Events

Benzinga · 2d ago
  • All participants showed cutaneous neurofibroma (cNF) reductions: All 15 evaluable participants had reductions in target cNF surface area, with a median best reduction of 41% across all doses and 46% at doses planned for Phase 2
  • cNF reductions deepened with continued treatment: The median reduction in target cNF surface area reached 49% at Cycle 12 among participants followed through 12 cycles and 71% at Cycle 15 in the 4mg cohort
  • Favorable safety and tolerability profile: No treatment related serious adverse events, treatment related discontinuations, retinopathy or adverse event of decreased left ventricular ejection fraction (LVEF)
  • IND cleared for Phase 2 in cNF: The FDA's Division of Dermatology and Dentistry has cleared the IND for a Phase 2 trial of PAS-004 in adults with NF1-associated cNF
  • Early activity in plexiform neurofibromas (PN): Reductions in target PN volume observed in all 18 evaluable participants, including two with reductions ≥20%, one achieving a 22.5% reduction at the first on-treatment MRI (Cycle 4)
  • PAS-004 Clinical Data Webcast: Pasithea will post a webcast on Friday, November 13, 2026, at 7:00 a.m. EST to discuss the data presented at the European Neurofibromatosis Conference