Pasithea Therapeutics is a clinical-stage biotech developing treatments for diseases driven by abnormal activation of the MAPK signaling pathway. The company's pipeline centers on PAS-004, a next-generation MEK 1/2 inhibitor, which Pasithea is developing primarily for neurofibromatosis type 1 (NF1), a rare genetic disorder affecting roughly 114,000 patients in the U.S. that causes painful tumor growths along nerve sheaths and skin. PAS-004 has received FDA orphan drug designation for NF1, which would confer 7 years of market exclusivity upon approval. Pasithea is running two active Phase 1 trials: one in advanced solid tumors with MAPK pathway mutations, where 34 patients have been dosed across 8 cohorts with no dose-limiting toxicities observed, and one in adult NF1 patients, initiated in mid-2025. Pasithea's commercial thesis rests on PAS-004's pharmacokinetic profile — a roughly 60-hour half-life and once-daily dosing versus the twice-daily dosing of approved MEK inhibitors — which Pasithea believes could translate to better tolerability and patient adherence than current standard of care. Pasithea also has a discovery-stage program, PAS-001, targeting schizophrenia via the C4A complement protein, for which Pasithea is seeking a development partner. The company is pre-revenue, funds operations through equity and debt raises, outsources manufacturing to a CMO, and runs with only 5 full-time employees.
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