KTTAW Overview

Pharmacokinetics of PAS-004: The tablet formulation of PAS-004 shows linear pharmacokinetics with a dose-proportional increase in exposure, achieving a Cmax/Cmin ratio of less than 2, indicating improved predictability and reduced variability compared to the capsule formulation.

Clinical Trial Results: In ongoing Phase 1/1b studies, the 4mg and 8mg tablet cohorts demonstrated significant pharmacokinetic parameters, with the 8mg tablet showing higher exposure levels than the 22mg capsule, suggesting enhanced efficacy for treating neurofibromatosis type 1-associated plexiform neurofibromas.

Long Half-Life: PAS-004 has a long half-life of approximately 57 hours, which supports once-daily dosing and may contribute to its therapeutic effectiveness in patients with symptomatic and inoperable plexiform neurofibromas.

Company Overview: Pasithea Therapeutics is focused on developing PAS-004 as a treatment for RASopathies and MAPK pathway-driven tumors, currently conducting clinical trials to evaluate its safety, tolerability, and efficacy in various patient populations.

New Clinical Trial Site Activation: Pasithea Therapeutics has activated a new clinical trial site at the University of Alabama at Birmingham for its Phase 1/1b study of PAS-004, targeting adult patients with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas.

Study Objectives and Design: The trial aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PAS-004, with a two-part design involving dose escalation and expansion phases across multiple international sites.

Community Engagement: Pasithea is also serving as the Platinum Sponsor for the 2025 NF Caregivers Symposium at UAB, emphasizing collaboration with caregivers, clinicians, and advocates to improve outcomes for NF1 patients.

Forward-Looking Statements: The press release includes forward-looking statements regarding the company's clinical trials and future business strategies, highlighting the inherent risks and uncertainties associated with such projections.

Trial Progression: Pasithea Therapeutics has received a recommendation from the external Safety Review Committee to escalate the dosage of PAS-004 to 8mg in their Phase 1/1b clinical trial for neurofibromatosis type 1 (NF1), following a review of safety data from initial participants.

Study Objectives: The trial aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PAS-004 in adult NF1 patients, with a focus on symptomatic and inoperable plexiform neurofibromas, and will be conducted across multiple sites in Australia, South Korea, and the U.S.

Clinical Trial Progress: Pasithea Therapeutics has completed enrollment and initial dosing of three subjects in Cohort 1 of its Phase 1/1b clinical trial for PAS-004, a MEK inhibitor aimed at treating neurofibromatosis type 1 (NF1) with symptomatic plexiform neurofibromas.

Future Expectations: The company anticipates presenting initial proof-of-concept data for PAS-004 in NF1 patients by Q1 2026, highlighting its potential as a once-daily treatment option that may improve patient compliance compared to existing therapies.

Appointment of Dr. James Lee: Pasithea Therapeutics has appointed Dr. James Lee to its scientific advisory board to assist in the development of PAS-004, a MEK inhibitor targeting ETS2 pathway inflammatory diseases such as IBD and Crohn's disease.

Current Clinical Trials: The company is conducting Phase 1 clinical trials for PAS-004 in advanced cancer patients and NF1-associated plexiform neurofibromas, with plans for further development based on non-dilutive financing options.

Potential of PAS-004: New preclinical data shows that PAS-004, a next-generation MEK inhibitor, significantly outperforms the FDA-approved selumetinib in inhibiting ETS2-driven inflammatory responses, suggesting its potential as a treatment for immune-mediated inflammatory diseases like IBD and ankylosing spondylitis.

Broader Implications: The study indicates that PAS-004 may provide a more effective approach to targeting multiple cytokines involved in chronic inflammation, positioning it as a promising oral treatment option for various inflammatory conditions beyond its initial focus on MAPK pathway-driven tumors.