Pasithea Therapeutics Corp. (KTTA), a clinical-stage biotechnology company, announced it has received Orphan Drug Designation for PAS-004 in treatment of Amyotrophic Lateral Sclerosis by the U.S Food and Drug Administration or FDA.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease which affects the nerve cells of the brain and spinal cord.
PAS-004 is a macrocyclic MEK inhibitor in evaluation for the treatment of RASopathies, MAPK pathway-driven tumours and other diseases.
The company is currently testing PAS-004 in a Phase 1 clinical trial in patients with advanced cancer, and a Phase 1/1b clinical trial in patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas with symptomatic and inoperable, incompletely resected, or recurrent PN or Plexiform Neurofibroma. Under the FDA's Orphan Drug Designation, Pasithea receives several potential benefits, including eligibility for tax credits for qualified clinical trials, exemption from certain FDA fees, and, following approval, seven years of market exclusivity.
In November 2025, the company received a grant from the ALS Association to study the efficacy, safety, and tolerability of PAS-004 in ALS, with funding of up to $1 million depending on project milestones.
"This designation further supports our efforts to explore the potential of PAS-004 in ALS and other serious diseases where dysregulation of the MAPK pathway may play an important role" said Tiago Reis Marques, Chief Executive Officer of Pasithea.
KTTA is currently trading at $0.69, down 3.50%.
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