Pasithea Therapeutics Announces Positive Phase 1 Data Including Partial Response, Demonstrating Monotherapy Clinical Activity and Favorable Safety Profile for PAS-004 in Advanced Cancer Study

Core Insights - Pasithea Therapeutics Corp. announced positive interim Phase 1 data for PAS-004, a macrocyclic oral MEK inhibitor, targeting neurofibromatosis type 1-associated plexiform neurofibromas and advanced solid tumors with specific mutations [1][2]. Interim Phase 1 Results - Initial signals of clinical activity were observed, with a partial response in a melanoma patient previously treated with a MEK + BRAF inhibitor, remaining on trial for over 11 months [3][4]. - Among 21 efficacy evaluable patients, the disease control rate was 71.4%, with 5 out of 7 patients with BRAF-mutated tumors achieving stable disease [3][7]. - PAS-004 demonstrated a favorable safety profile, with all treatment-related adverse events classified as Grade 1 or 2, and no ocular or cardiovascular toxicities reported [3][7]. Pharmacokinetics and Drug Exposure - At the highest reported dose of 30mg, PAS-004 showed significant drug exposure with an Area Under the Curve (AUC) greater than 5,400 ng·h/mL, indicating sustained pathway inhibition [2][3]. - The pharmacokinetic profile was characterized by a Cmax/Cmin ratio of less than 2, with a long half-life of approximately 60 hours [7]. Safety and Tolerability - The drug was well-tolerated across all dose levels, with no dose-limiting toxicities or treatment discontinuations reported [7]. - Limited adverse effects included rash (7.4%), nausea (18.5%), vomiting (14.8%), and diarrhea (7.4%) [7]. Company Overview - Pasithea Therapeutics is focused on developing PAS-004 for RASopathies, MAPK pathway-driven tumors, and other diseases, currently conducting Phase 1 clinical trials in advanced cancer patients and NF1-associated plexiform neurofibromas [5].