8-K: Pasithea Expands NF1 Trial with New UAB Site Activation

Pasithea Therapeutics announced the activation of a new U.S. clinical trial site at the University of Alabama at Birmingham for its Phase 1/1b study of PAS-004 in adult NF1 patients.

Summary

• Pasithea Therapeutics Corp. (Nasdaq: KTTA) activated a new U.S. clinical trial site at the University of Alabama at Birmingham (UAB) for its ongoing Phase 1/1b open-label study of PAS-004.
• The trial evaluates PAS-004, a next-generation macrocyclic oral MEK inhibitor, in adult patients with neurofibromatosis type 1 (NF1) who have symptomatic and inoperable, incompletely resected, or recurrent plexiform neurofibromas.
• Enrollment at the UAB site is expected to begin immediately, joining other global clinical centers in Australia, South Korea, and the U.S.
• The Phase 1/1b study (NCT06961565) aims to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of PAS-004.
• Secondary objectives include identifying the recommended Part B dose (RPBD) or Maximum Tolerated Dose (MTD), characterizing PK/PD, and evaluating preliminary efficacy on target plexiform neurofibroma (PN) volume, cutaneous neurofibroma (CN) size/appearance/symptoms, and quality of life (QOL).
• The trial consists of two parts: Part A (dose escalation) with up to 24 participants across four planned dose levels (4mg, 8mg, 12mg, 18mg) to identify RPBD, and Part B (expansion) with approximately 24 participants at two dose levels (RPBD and below RPBD) to identify the recommended phase 2 dose (RP2D).
• Pasithea will also serve as Platinum Sponsor for the 2025 NF Caregivers Symposium hosted at UAB on November 8, 2025.

Sentiment

Score: 7

Explanation: The announcement is positive, indicating progress in a key clinical trial and strong community engagement. It reflects expected operational advancements in drug development, without any negative surprises or financial disclosures.

Positives

• Activation of a new U.S. clinical trial site at UAB expands the reach and capacity for the Phase 1/1b study of PAS-004, potentially accelerating patient enrollment.
• UAB brings deep clinical expertise in NF1 and a strong commitment to advancing patient care, enhancing the credibility and potential success of the trial.
• Platinum sponsorship of the NF Caregivers Symposium demonstrates Pasithea's commitment to the NF1 community and fosters collaboration with academia, industry, caregivers, and patient advocates.
• The ongoing Phase 1/1b trial for PAS-004 addresses a significant unmet medical need for adult NF1 patients with symptomatic and inoperable plexiform neurofibromas.

Risks

• Future clinical trial results may not match results observed to date, may be negative or ambiguous, or may not reach the level of statistical significance required for regulatory approval.
• The company's business is subject to numerous conditions, many of which are beyond its control.
• Actual results could be materially different from forward-looking statements due to various risks and uncertainties.

Future Outlook

Pasithea Therapeutics remains focused on advancing PAS-004 through clinical development with urgency and purpose. The company expects enrollment at the new UAB site to begin immediately for its Phase 1/1b trial in adult NF1 patients. The trial aims to identify recommended doses and evaluate preliminary efficacy, safety, tolerability, PK, and PD of PAS-004.

Management Comments

• "We are pleased to activate UAB as a clinical site for our ongoing Phase 1/1b PAS-004 trial. UAB brings deep clinical expertise in NF1 and a strong commitment to advancing care for patients with NF1 neurofibromas. Alongside our continued engagement with the NF community, including our recent platinum sponsorship of the NF Caregivers Symposium, we remain focused on advancing PAS-004 through clinical development with urgency and purpose." Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea.
• "Collaborations between academia, industry, caregivers, and patient advocates improve outcomes and support for NF1 patients. We look forward to participating in this clinical trial and supporting other activities that promote resources for the NF community." Dr. Rebecca Brown, Director of NF Clinical Programs at UAB.

Industry Context

The activation of a new clinical trial site for an MEK inhibitor targeting NF1 is a positive development in the biotechnology sector, particularly for rare diseases. NF1 is a genetic disorder with significant unmet medical needs, and MEK inhibitors represent a promising class of drugs for treating associated tumors. Expanding trial sites and engaging with patient communities are standard and crucial steps in advancing drug candidates through clinical development, signaling progress in a competitive therapeutic area.

Stakeholder Impact

• **Shareholders**: Positive news regarding clinical trial progress and expansion could instill confidence, potentially leading to increased investor interest.
• **Patients (NF1)**: The activation of a new trial site offers more access points for eligible patients to participate in the study, potentially leading to new treatment options for a debilitating condition.
• **Caregivers**: The sponsorship of the NF Caregivers Symposium directly supports caregivers by providing resources and fostering community, which can indirectly benefit patients.
• **Medical Community/Researchers**: Collaboration with UAB, a reputable academic institution, enhances the scientific rigor and visibility of the trial, contributing to broader medical knowledge in NF1.

Next Steps

• Immediate commencement of patient enrollment at the University of Alabama at Birmingham site for the Phase 1/1b study of PAS-004.
• Participation as Platinum Sponsor in the 2025 NF Caregivers Symposium at UAB on November 8, 2025.
• Continuation of the Phase 1/1b study (NCT06961565) across five planned global sites to evaluate PAS-004's safety, tolerability, PK, PD, and preliminary efficacy.
• Advancement of PAS-004 through Part A (dose escalation) to identify the Recommended Part B Dose (RPBD) and subsequently Part B (expansion) to identify the Recommended Phase 2 Dose (RP2D).

Key Dates

Recommendation

The announcement is a positive operational update, indicating progress in a key clinical trial. While expanding trial sites and community engagement are good signs, this filing does not contain new clinical data or financial results that would significantly alter the fundamental valuation or risk profile of the company. It reinforces the ongoing development strategy but doesn't provide a strong catalyst for a 'buy' or 'sell' recommendation at this stage. Investors should 'hold' and await further clinical data or significant corporate developments.

Keywords

Pasithea Therapeutics, PAS-004, NF1, Neurofibromatosis Type 1, Clinical Trial, Phase 1/1b, MEK Inhibitor, Biotechnology, Plexiform Neurofibromas, UAB, Drug Development