Core Insights - Pasithea Therapeutics Corp. announced promising interim results for PAS-004, a next-generation macrocyclic MEK inhibitor, in a Phase 1 clinical trial targeting advanced cancer patients with MAPK pathway-driven tumors [1][6] Group 1: Clinical Trial Overview - The ongoing Phase 1 study employs a multi-center, open-label, dose escalation design to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of PAS-004 [1] - As of April 2, 2025, 21 patients have been enrolled across six cohorts, with pancreatic cancer (28.6%), colorectal cancer (28.6%), and melanoma (23.8%) being the most common diagnoses [2] Group 2: Efficacy and Safety - PAS-004 demonstrated a dose-dependent PK profile and preliminary clinical activity, with 10 out of 16 efficacy evaluable patients achieving stable disease at some point during the trial [5] - One patient with stage 4 BRAF-mutated melanoma showed a tumor volume reduction of -14.9% and has maintained stable disease for over 5 months [1][5] - All treatment-related adverse events were grade 1 or 2, with no known MEK inhibitor class-related adverse events observed [3] Group 3: Pharmacokinetics - Preliminary PK analysis indicates a linear PK profile with an estimated half-life exceeding 60 hours, and sufficient exposures for target engagement were achieved [4] - The peak to trough ratio was below 2 at steady state across all dose levels, supporting the potential for effective treatment [4] Group 4: Future Outlook - The CEO of Pasithea highlighted the potential of PAS-004 as a best-in-class MEK inhibitor, emphasizing its high selectivity and sustained pathway suppression, which may benefit both monotherapy and combination therapy [6] - The company is also conducting a Phase 1/1b clinical trial for PAS-004 in adult patients with neurofibromatosis type 1-associated plexiform neurofibromas [7]
Pasithea Therapeutics Presents Updated Interim Data from Ongoing Phase 1 Study of PAS-004 at the ASCO Annual Meeting 2025