Pasithea Therapeutics Corp（KTTA）資訊與事件KTTAPasithea Therapeutics Corp

Pasithea Receives FDA Approval for PAS-004 Clinical Trial

Pasithea Therapeutics announced that the FDA has cleared its Investigational New Drug application for a randomized, placebo-controlled Phase 2 clinical trial for PAS-004 in patients with neurofibromatosis type 1-associated cutaneous neurofibromas. PAS-004 is a next-generation oral macrocyclic MEK inhibitor. Cutaneous neurofibromas affect over 95% of people with neurofibromatosis type 1, and there are currently no FDA-approved systemic therapies for them. "FDA clearance of our IND for PAS-004 in cutaneous neurofibromas is an important milestone for Pasithea and a significant step forward for people living with NF1 who have cNFs. Cutaneous neurofibromas are among the most visible and burdensome manifestations of NF1, and there are currently no approved systemic therapies for them. PAS-004 is designed to address this unmet need as a once-daily oral MEK inhibitor whose long half-life and flat pharmacokinetic profile may support chronic dosing," said Kartik Krishnan, chief medical officer of Pasithea.

Pasithea Releases PAS-004 Clinical Data Showing Good Tolerability

Pasithea Therapeutics announced updated safety, tolerability, and preliminary efficacy data from its ongoing Phase 1 dose-escalation study of PAS-004 monotherapy in a heavily pre-treated population of patients with MAPK pathway-driven advanced solid tumors harboring RAS, NF1 or RAF mutations, including patients who have failed prior BRAF/MEK inhibitor treatment. The data, reflecting durable clinical benefit, including prolonged stable disease in four patients, as well as long-term tolerability and flat pharmacokinetics, are being presented at the ESMO Congress 2026, taking place October 23-27, in Madrid, Spain. PAS-004 achieved prolonged stable disease in a heavily pre-treated population, with four patients on PAS-004 for more than 300 days, including two who continue on once daily PAS-004 for over 500 days despite recurrent, metastatic melanoma carrying the BRAF V600E mutation and prior progression on targeted combination therapy with dabrafenib and trametinib. Overall, the safety profile of PAS-004 in the heavily pre-treated advanced cancer population appears favorable. No Grade 3 treatment-related adverse events, no discontinuations due to treatment-related AEs, and no related serious AEs have been reported. Treatment-related AEs remain low-grade and manageable, with low rates of vomiting, nausea, diarrhea, and rash, including none reported by the four patients on-study for greater than300 days. None of the MEKi class effects on the heart or the retina have been reported. To date, there have been no dose-limiting toxicities, and the maximum tolerated dose has not been reached. PAS-004 demonstrated a long half-life, approximately 58 hours on average, with a low Cmax/Cmin ratio of less than 1.5. AUC increased proportionally in this dose escalation study. The flat pharmacokinetic curve with a long half-life supports long-term, chronic dosing.

Pasithea Updates PAS-004 Clinical Trial Data

Pasithea Therapeutics announced updated interim safety and clinical activity data from its ongoing first-in-human trial evaluating PAS-004 in patients with MAPK pathway-driven advanced solid tumors with a documented RAS, NF1 or RAF mutation, including patients who have failed prior BRAF/MEK inhibitor treatment. The company also announced details of its protocol amendment to extend dose escalation and initiate a pilot food effect assessment. In total, 34 patients have been enrolled and dosed. No dose-limiting toxicities and no discontinuations due to TRAEs with 10 patients on study for greater than 90 days, of which six patients greater than 150 days, for patients greater than 300 days, and two patients greater than 365 days. All TRAEs were Grade 1 or Grade 2; low cumulative rates of rash and diarrhea across all patients at all doses throughout the study, including no rash or diarrhea in the four patients on study greater than 300 days and two patients greater than 365 days and no events of retinal or cardiac toxicity. Multiple patients demonstrating durable clinical benefit and tumor shrinkage, including several MEK/BRAF-pretreated patients who have remained on study for greater than six months and two patients on study for over one year - exceeding the approximately five-month median PFS reported in the literature for BRAF/MEK rechallenge in this setting. The final PFS data will not be known until the end of the trial. The company plans to submit and present a more robust data set at a future scientific conference.