Pasithea Therapeutics Expands NF1 Study in South Korea, Leverages Significant Patient Population.

Pasithea Therapeutics (KTTA) has expanded its clinical trials to South Korea, targeting a significant NF1 patient population. The company operates in the biotechnology sector, focusing on CNS disorders and other diseases. Despite strong financial strength, the company faces challenges with negative earnings and high volatility. The expansion is expected to significantly contribute to the success of the trial and enhance treatment options for the NF1 community.

Pasithea Therapeutics (KTTA) has expanded its clinical trial for Pasithea’s Phase 1/1b Neurofibromatosis Type 1 (NF1) study to South Korea. The company, known for its focus on Central Nervous System (CNS) disorders and other diseases, is leveraging the trial to target a significant NF1 patient population in the region. This expansion comes amidst the company's strong financial position, despite facing challenges with negative earnings and high volatility.

The trial, which initially began in Australia and the U.S., has now moved to South Korea, adding to its cross-border site distribution. The study aims to test PAS-004, a macrocyclic MEK inhibitor, in adult NF1 patients with plexiform neurofibromas. The trial is designed in two parts: Part A will enroll up to 24 participants across four dose levels (4, 8, 12, 18 mg) to establish a recommended Part B dose, while Part B will randomize participants to the selected dose and a lower dose for up to six 28-day cycles to define the recommended Phase 2 dose (RP2D).

The expansion to South Korea is expected to significantly contribute to the success of the trial by enhancing recruitment efforts in a dispersed adult NF1 population. The trial is designed to assess the safety and efficacy of PAS-004, with a focus on volumetric PN shrinkage, pain, function, and quality of life. The study also includes exploratory tissue and molecular analyses in cutaneous neurofibromas (CNs).

The inclusion of cutaneous endpoints and symptom measures suggests that Pasithea is aiming for a broader clinical value proposition beyond volumetric PN shrinkage alone. The company is strategically positioning itself to compete in an increasingly segmented MEK landscape by targeting adult NF1, a population with fewer labeled options than pediatrics and a need for chronic, tolerable regimens.

The initial interim data from the first two cohorts is targeted for Q1 2026. This measured escalation pace indicates a focus on accruing sufficient exposure and imaging timepoints before calling a signal. The study's operational complexity, including volumetric MRI and dermatologic assessments, may pose logistical challenges for smaller centers, necessitating strong enablement from the sponsor and Contract Research Organization (CRO).

Key catalysts for the trial's success include the absence of dose-limiting toxicities (DLTs) at higher doses, the selection of the Part B dose, and the character of the first PN and CN signals in early 2026. Any signal indicating sustained dosing feasibility with manageable class-associated toxicities would justify the adult NF1 bet and inform the design of a registrational path. Conversely, tolerability setbacks or inconclusive PD-efficacy linkage could push PAS-004 back into a crowded MEK field.