Pasithea Therapeutics Presents Positive Initial Phase 1/1b Data for PAS-004, Showing Substantial Reductions in Cutaneous Neurofibroma Size and Early Activity in Plexiform Neurofibromas at the 2026 European Neurofibromatosis Conference
Globe Newswire•13/11/2026•02:00 ET
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Key Highlights
- ➤PAS-004 cutaneous neurofibroma reductions reached 41% median best across all doses
- ➤Phase 2 doses delivered 46% median best cNF surface-area reduction
- ➤4mg cohort median cNF reduction reached 71% by Cycle 15
- ➤FDA cleared Phase 2 IND for approximately 200 adults with NF1-associated cNF
- ➤All 18 evaluable participants showed plexiform neurofibroma volume reductions
Expert Statements
Tiago Reis Marques, Chief Executive Officer of Pasithea
“Cutaneous neurofibromas do not regress spontaneously and generally increase in number and size with age. The substantial reductions in cNF size reported today represent an encouraging clinical signal in a condition for which there are currently no FDA-approved systemic treatments”
Tiago Reis Marques, Chief Executive Officer of Pasithea
“Importantly, these reductions have continued to deepen with longer treatment, including at the lowest doses tested. With our IND now cleared by FDA’s Division of Dermatology and Dentistry, these findings reinforce our confidence in the 4mg to 12mg dose range planned for Phase 2 and support advancing PAS-004 into a randomized, placebo-controlled study in cNF.”
Kartik Krishnan, Chief Medical Officer of Pasithea
“The reductions in cNF size observed with oral PAS-004 highlight its potential to provide broad, visible improvements for patients through a systemic treatment approach, rather than relying on local procedures that can treat individual lesions.”
Kartik Krishnan, Chief Medical Officer of Pasithea
“We are also seeing early activity in plexiform neurofibromas, with reductions in tumor volume across all dose levels and two participants meeting the REiNS threshold for partial response, including one at the first on-treatment MRI.”
Kartik Krishnan, Chief Medical Officer of Pasithea
“Together with the favorable tolerability observed to date and a pharmacokinetic profile characterized by a 40-to-60-hour half-life and limited peak-to-trough fluctuations, these findings support the continued development of PAS-004 as a once-daily oral therapy for long-term treatment of NF1-associated neurofibromas.”
* 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
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