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Pasithea Therapeutics Announces FDA Clearance of IND for PAS-004 in Neurofibromatosis Type 1 (NF1)-Associated Cutaneous Neurofibromas (cNF)

KTTAKTTAW
  • The FDA cleared Pasithea Therapeutics' Investigational New Drug (IND) application for a Phase 2 trial of PAS-004 in patients with NF1-associated cutaneous neurofibromas (cNF).
  • PAS-004 is a next-generation oral macrocyclic MEK inhibitor designed as a once-daily treatment for chronic conditions including NF1.
  • The Phase 2 study will be a randomized, placebo-controlled trial overseen by the FDA Division of Dermatology and Dentistry.
  • There are currently no FDA-approved systemic therapies for NF1-associated cNF, which affects over 95% of individuals with NF1.
  • Pasithea aims to make PAS-004 the first FDA-approved systemic treatment for cNF, targeting an estimated 80,000 patients in the United States with moderate to severe disease.
  • The IND submission was supported by nonclinical safety, clinical safety, tolerability, pharmacokinetic data, and early treatment response signals from ongoing Phase 1/1b trials in plexiform neurofibromas and advanced cancer.
  • Cutaneous neurofibromas are benign skin tumors that cause significant disfigurement, morbidity, and reduced quality of life, with current management relying on procedural approaches like surgery or laser therapy.
  • Pasithea Therapeutics is developing PAS-004 for RASopathies, MAPK pathway-driven tumors, and other diseases, with current trials in advanced cancer (NCT06299839) and adult NF1 patients with plexiform neurofibromas (NCT06961565).