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, with 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) plexiform and cutaneous neurofibromas, addressing large unmet needs in both indications.

• NF1-PN and NF1-CN represent significant market opportunities, with estimated total addressable markets of over $2 billion and $1 billion, respectively.

• MEK inhibitors have broad potential in multiple diseases, including various cancers and rare disorders.

Clinical development and pipeline

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

• The drug has orphan drug designation, fast track, and rare pediatric disease status for NF1.

• Ongoing trials assess safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in both solid tumors and NF1 patients.

• Assessment schedules include volumetric MRI for PN and digital caliper/photography for CN, with response criteria based on established regulatory precedents.

Differentiation and preclinical/clinical data

• PAS-004’s macrocyclic structure provides improved metabolic stability, sustained pathway inhibition, and once-daily dosing, differentiating it from existing MEK inhibitors.

• Preclinical models show robust efficacy, including significant tumor volume reduction and better tolerability compared to selumetinib.

• In vitro and in vivo studies demonstrate superior potency and sustained activity in NRAS-mutant and NF1-mutant models.

• Human pharmacokinetic data confirm a long half-life (>60 hours) and favorable steady-state exposure, supporting chronic dosing.