September 8, 2025 — Leads & Copy — Pasithea Therapeutics Corp. (NASDAQ: KTTA) announced its Phase 1/1b open-label study assessing PAS-004’s safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) in adult participants with neurofibromatosis type 1 (NF1) has been recommended to proceed to Cohort 2, 8mg tablet, without modification.
The Safety Review Committee’s decision was based on reviewing safety data from Cohort 1’s three patients, noting no dose-limiting toxicities (DLTs).
The primary objective of the study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives include identifying the recommended Part B dose (“RPBD”) or Maximum Tolerated Dose (MTD) of PAS-004, characterizing the PK and PD profile of PAS-004, evaluating the preliminary efficacy of PAS-004 on target PN volume, evaluating the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and evaluating the impact of PAS-004 on quality of life (“QOL”) and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology.
According to Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea, there is substantial enrollment demand, and the first three Cohort 2 patients have already been enrolled.
The trial is planned to be conducted at five clinical trial sites in Australia, South Korea, and the U.S.
Pasithea is focused on developing PAS-004, a next-generation macrocyclic MEK inhibitor, for treating RASopathies, MAPK pathway-driven tumors, and other diseases. They are currently testing PAS-004 in Phase 1 clinical trials in advanced cancer patients (NCT06299839) and in adult patients with NF1-associated plexiform neurofibromas (NCT06961565).
Patrick Gaynes
Corporate Communications
pgaynes@pasithea.com
Source: Pasithea Therapeutics Corp.
