Pasithea Therapeutics Receives $1 Million ALS Association Grant to Advance PAS-004 MEK Inhibitor in Phase 1 ALS Trial
核心洞察
The ALS Association (搜索) awarded Pasithea Therapeutics approximately $1 million through its Hoffman Clinical Trial Awards Program to study PAS-004, a next-generation macrocyclic MEK (搜索) inhibitor, in ALS (搜索) patients.
The Phase 1 study will evaluate PAS-004 in twelve ALS (搜索) patients across three sequential dose cohorts over approximately 28 weeks, marking the first clinical trial of this MEK (搜索) inhibitor in ALS.
PAS-004 targets MAPK (搜索) and MEK (搜索) enzymes involved in TDP-43-related neurodegeneration and neuroinflammation, with promising preclinical results in the SOD mouse model of ALS (搜索).
Pasithea Therapeutics Corp. (NASDAQ: KTTA) has secured a significant milestone in its pursuit of novel ALS (搜索) treatments, receiving approximately $1 million from the ALS Association (搜索)'s Hoffman Clinical Trial Awards Program to advance PAS-004, a next-generation macrocyclic MEK (搜索) inhibitor, into its first clinical trial for amyotrophic lateral sclerosis (搜索).
The grant will fund a Phase 1 study evaluating the "efficacy, safety and tolerability of PAS-004 for the treatment of ALS (搜索)," representing a critical step forward for the clinical-stage biotechnology company's lead drug candidate.
Targeting Key Pathways in ALS Pathophysiology
Dr. Tiago Reis Marques, CEO of Pasithea, explained the scientific rationale behind the approach: "Inflammation and the aggregation of a protein called TDP-43 are well-recognized contributors to the development and progression of ALS (搜索). Two enzymes, mitogen-activated protein kinase (MAPK (搜索)) and extracellular signal-regulated kinase (MEK (搜索)), have been shown to play a role in TDP-43–related neurodegeneration and neuroinflammation, suggesting they represent promising therapeutic targets."
The company's preclinical work has shown encouraging results. According to Dr. Lawrence Steinman, Chairman of Pasithea, "PAS-004 targets a critical molecule in the pathophysiology of motor neuron disease and has delivered significant and promising results in the ALS (搜索) gold standard SOD mouse model."
Phase 1 Trial Design and Endpoints
The upcoming Phase 1 study will evaluate PAS-004 in twelve patients with ALS (搜索) enrolled across three sequential dose cohorts, with participants followed for approximately 28 weeks. Beyond assessing safety and tolerability as primary objectives, the study will measure changes in the ALS Functional Rating Scale–Revised (ALSFRS-R) scores and neurofilament light chain (NfL) levels to explore potential early signals of clinical activity.
The trial will be conducted at a leading ALS (搜索) institution in the United States, leveraging the expertise of specialized centers in neurodegenerative disease research.
Building on Existing Clinical Experience
PAS-004 is already demonstrating clinical promise in other indications. The drug candidate is currently being tested in a Phase 1 clinical trial in advanced cancer (搜索) patients (NCT06299839) and a Phase 1/1b clinical trial in adult patients with neurofibromatosis type 1 (搜索) (NF1)-associated plexiform neurofibromas (NCT06961565).
"PAS-004, already in the clinic for neurofibromatosis and advanced cancers, is showing a promising safety profile and initial monotherapy efficacy signal," noted Dr. Steinman.
Addressing Critical Unmet Medical Need
The grant comes at a time when ALS (搜索) patients face limited therapeutic options. Amyotrophic lateral sclerosis (搜索) is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord, leading to loss of movement, speech, and eventually breathing ability. The disease is invariably fatal, typically within five years of diagnosis, with few treatment options available.
Dr. Kuldip Dave, Senior Vice President of Research at the ALS Association (搜索), emphasized the importance of supporting early-stage clinical trials: "The ALS Association's Hoffman Clinical Trial Awards Program supports early-stage clinical trials of potential new treatments that hold promise for those living with ALS (搜索). We are pleased to support the first dosing of PAS-004 in people living with ALS. By funding programs at this critical stage, we are working to accelerate the development of therapeutic candidates that can help make ALS a livable disease until we can cure it."
Broader Therapeutic Potential
Beyond ALS (搜索), Pasithea is positioning PAS-004 as a potential best-in-class MEK (搜索) inhibitor for multiple indications. The company describes PAS-004 as intended for the treatment of RASopathies, MAPK (搜索) pathway-driven tumors, and other diseases, suggesting broad therapeutic applications for this next-generation macrocyclic MEK inhibitor.
The ALS Association (搜索), recognized as the world's leading funder of ALS (搜索) research and the largest ALS organization globally, created the Hoffman ALS Clinical Trial Awards Program specifically to fund early- to mid-stage biomarker-driven clinical trials of novel or repurposed therapeutics for ALS.