MIAMI, November 4, 2025: Pasithea Therapeutics Corp. has announced the activation of a new clinical trial site at the University of Alabama at Birmingham (UAB) for its ongoing Phase 1/1b trial of PAS-004, a next-generation macrocyclic oral MEK inhibitor developed for adult patients with neurofibromatosis type 1 (NF1). The UAB activation marks a key milestone in Pasithea’s global multi-site effort to assess the safety, pharmacokinetics, and efficacy of PAS-004 in patients with symptomatic, inoperable, or recurrent plexiform neurofibromas.
Science Significance
PAS-004 represents an innovative macrocyclic MEK inhibitor engineered for selective and durable modulation of the RAS/MAPK pathway, implicated in NF1 and various cancers. The study’s design includes both dose escalation (Part A) and expansion phases (Part B) to determine the recommended Phase 2 dose (RP2D) while exploring tumor volume reduction and quality-of-life improvements. As a potential next-generation therapy, PAS-004 aims to overcome limitations of first-generation MEK inhibitors in safety, selectivity, and sustained pathway control.
Regulatory Significance
The trial (NCT06961565) operates under U.S. FDA regulatory oversight and adheres to Good Clinical Practice (GCP) standards. Activation of the UAB site enhances the study’s geographic diversity, joining sites in Australia and South Korea, which strengthens the regulatory data package for potential future filings. Successful completion of this Phase 1/1b trial could enable accelerated pathways toward Breakthrough Therapy designation or Orphan Drug status, given NF1’s rare disease classification.
Business Significance
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This milestone reinforces Pasithea’s transition into a clinically mature biotech, positioning PAS-004 as its lead development asset. The collaboration with UAB underscores the company’s strategic focus on academic partnerships and its dedication to rare disease innovation. By combining scientific advancement with community engagement—highlighted by its platinum sponsorship of the NF Caregivers Symposium—Pasithea strengthens its brand visibility and investor appeal within the rare disease and oncology sectors.
Patients’ Significance
NF1 patients often face limited therapeutic options and debilitating tumor growths that compromise quality of life. PAS-004 offers a potential oral, non-invasive treatment alternative with the goal of shrinking plexiform neurofibromas and reducing associated pain and disfigurement. The company’s collaboration with UAB ensures access to specialized NF1 care and strengthens the bridge between research innovation and patient support systems, empowering a historically underserved community.
Policy Significance
The study aligns with broader national rare disease policies encouraging academic-industry collaboration and expedited therapeutic development for orphan indications. By advancing NF1 research, Pasithea contributes to the FDA’s Rare Diseases Program objectives and supports public health initiatives promoting early access to life-improving treatments. Policy frameworks favoring precision oncology and genetic pathway modulation could further accelerate PAS-004’s pathway to clinical availability.
The activation of UAB as a clinical site for PAS-004’s Phase 1/1b study represents a crucial advancement in NF1 drug development. Combining robust scientific inquiry with a strong community engagement strategy, Pasithea Therapeutics continues to exemplify how biotech innovation can translate into tangible progress for rare disease patients. With ongoing global trials and regulatory momentum, PAS-004 could soon redefine treatment paradigms in NF1 and MAPK-driven pathologies.
Source: Pasithea Therapeutics Corp. press release
