Pasithea Therapeutics Announces Positive PAS-004 Clinical Data from Phase 1 Advanced Cancer Study Presented at ESMO Congress 2026
Key Highlights
- ➤Four patients maintained stable disease on PAS-004 for over 300 days
- ➤Two patients continued PAS-004 treatment beyond 500 days with prolonged stable disease
- ➤37 patients enrolled across 10 dose cohorts in the Phase 1 study
- ➤No dose-limiting toxicities or treatment-related discontinuations were observed
- ➤PAS-004 showed approximately 58-hour half-life and supported once-daily dosing
Expert Statements
Kartik Krishnan, Chief Medical Officer of Pasithea Therapeutics
“The prolonged stable disease that we’re seeing in some patients across this heavily pre-treated population is highly encouraging, particularly in patients with BRAF V600E-mutant tumors who previously progressed on BRAFi monotherapy and/or BRAFi/MEKi combination therapy. The literature reports a median progression-free survival of approximately five months for combination BRAFi/MEKi rechallenge in this setting, and some patients in the BRAF-mutant subgroup are exceeding that benchmark with our MEKi monotherapy”
Kartik Krishnan, Chief Medical Officer of Pasithea Therapeutics
“What makes this data especially encouraging is that we’re seeing these signals of durable clinical benefit alongside a long-term tolerability profile that continues to hold up. No patient has discontinued treatment at any dose level due to treatment-related adverse events, and we continue to observe low rates of vomiting and other GI toxicity, low rates of rash, even in patients taking PAS-004 continuously for long periods of time. A tolerability profile like this is exactly what you want to see for the long-term treatment of chronic diseases including neurofibromatosis type 1 (NF1).”
-- Prolonged Stable Disease: PAS-004 achieved prolonged stable disease in a heavily pre-treated population, with four patients on PAS-004 for more than 300 days, including two who continue on once daily PAS-004 for over 500 days despite recurrent, metastatic melanoma carrying the BRAF V600E mutation and prior progression on targeted combination therapy with dabrafenib and trametinib.
-- Long-Term Tolerability: Overall, the safety profile of PAS-004 in the heavily pre-treated advanced cancer population appears favorable. No Grade 3 treatment-related adverse events (“AEs”), no discontinuations due to treatment-related AEs, and no related serious AEs have been reported. Treatment-related AEs remain low-grade and manageable, with low rates of vomiting, nausea, diarrhea, and rash, including none reported by the four patients on-study for >300 days. None of the MEKi class effects on the heart or the retina have been reported. To date, there have been no dose-limiting toxicities, and the maximum tolerated dose has not been reached.
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