Pasithea Therapeutics (KTTA) Corporate presentation summary

Investment highlights and market opportunity

• PAS-004 is a next-generation macrocyclic MEK inhibitor designed to overcome limitations of earlier MEK inhibitors, showing superior target binding, oral bioavailability, and pharmacokinetics.

• Interim clinical data show encouraging safety, pharmacokinetics, and early efficacy, with no dose-limiting toxicities or severe adverse events; all treatment-related adverse events are Grade 1/2.

• PAS-004 targets neurofibromatosis type 1 (NF1) with initial focus on plexiform neurofibromas (PN), followed by cutaneous neurofibromas (CN), addressing large unmet needs in both indications.

• NF1-PN represents a $2+ billion total addressable market, while NF1-CN is estimated at $1+ billion, with MEK inhibitors' worldwide sales projected at $701 million in 2025 for NF1-PN.

Clinical development and differentiation

• PAS-004 is in Phase 1/1b trials for advanced cancer and adult NF1-PN, with protocol amendments expanding dose cohorts and extending treatment duration.

• The drug demonstrates robust preclinical efficacy, outperforming existing MEK inhibitors in several models, and offers once-daily dosing with a long half-life (>60 hours), supporting sustained MAPK pathway inhibition.

• PAS-004 shows a differentiated safety profile, with all adverse events to date being mild (Grade 1/2), and no observed dose-limiting toxicities, ocular, or cardiac toxicities.

• PK data indicate dose-proportional exposure and steady-state concentrations above the cellular IC50, supporting constant pathway suppression.

Unmet need and competitive landscape

• NF1 affects ~1 in 3,000 newborns, with ~115,000 U.S. patients; 30-50% develop PN and >95% develop CN, both causing significant morbidity.

• Only two MEK inhibitors are approved for NF1-PN (Koselugo and Gomekli), both with notable toxicity and no approved systemic therapy for NF1-CN.

• PAS-004 aims to be the first approved systemic therapy for NF1-CN and improve tolerability and dosing convenience for NF1-PN.

• A ≥25% reduction in CN tumor volume is targeted, exceeding the regulatory threshold for meaningful benefit.