Pasithea Therapeutics (KTTA) Corporate presentation summary

Pipeline and clinical development

• PAS-004 is in development for chronic diseases, focusing on neurofibromatosis type 1 (NF1) and MAPK pathway-driven cancers.

• Ongoing Phase 1 trials target both NF1 (PAS-004-103) and advanced cancers (PAS-004-102), with interim data expected in Q4 2026 and at ESMO Congress 2026.

• PAS-004 aims to improve on current MEK inhibitors with once-daily dosing, better tolerability, and robust efficacy.

• Preclinical and early clinical data show PAS-004 reduces tumor volume and number in NF1 models, with activity in BRAF-mutated tumors.

• Phase 1 studies assess safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy in both NF1 and cancer populations.

Market opportunity and unmet need

• NF1 affects about 1 in 3,000 people, with ~115,000 U.S. patients; >95% develop cutaneous neurofibromas (CN), and 30-50% develop plexiform neurofibromas (PN).

• NF1-PN represents a concentrated, high-need group (~16,000 MEK inhibitor-eligible U.S. patients), while NF1-CN is a large, universal opportunity (~86,000 moderate/severe U.S. patients).

• Current treatments for PN have significant toxicity and dosing burdens; no systemic therapy exists for CN, representing a major unmet need.

• Willingness to try new treatments is high, especially among those with severe CN burden.

• Worldwide MEK inhibitor sales for NF1-PN are growing, with a multi-billion dollar total addressable market.

Clinical data and competitive positioning

• PAS-004 demonstrates favorable pharmacokinetics with once-daily dosing and a lower Cmax:Cmin ratio compared to competitors.

• Early clinical data show a 71.4% disease control rate in BRAF-mutated tumors, with some patients on treatment for over a year.

• Safety profile is favorable, with mostly grade 1/2 adverse events such as rash, diarrhea, and nausea.

• Compared to other MEK inhibitors, PAS-004 aims for improved tolerability and adherence, addressing key physician and patient concerns.

• Intellectual property protection extends to at least 2045, with multiple patents and FDA designations for orphan and rare pediatric diseases.