ALS Association grant funds Phase 1 trial of Pasithea’s PAS-004
12 patients in Florida to participate in 28-week study
- ALS Association funds a Phase 1 trial for PAS-004, an experimental ALS therapy.
- PAS-004, a MEK inhibitor, targets neurodegeneration and neuroinflammation in ALS.
- The 28-week trial in 12 patients will assess PAS-004's safety, tolerability, and dosing.
An ALS Association grant totaling nearly $1 million will support a clinical trial testing Pasithea Therapeutics‘ PAS-004 in people with amyotrophic lateral sclerosis (ALS).
Funding for the Phase 1 trial is provided by the ALS Association’s Hoffman ALS Clinical Trial Awards Program, an initiative designed to support early clinical trials that gather safety, dosing, and biomarker data on a small number of patients to advance the development of experimental therapies.
These trials reduce the risk of negative results in the next stage of drug development — typically larger clinical trials — and help attract additional funding to complete those studies.
“While early phase studies cannot really determine whether a drug significantly slows or stops ALS, they are critical to understanding many important questions to determine a drug’s potential to become an effective therapy,” Shah said in a press release from the ALS Association. “We hope this trial will improve the field’s understanding of the role MEK inhibitors could play in the treatment of ALS.”
The company aims “to provide proof-of-concept that PAS-004 may be the best-in-class MEK inhibitor for the treatment of many indications,” Lawrence Steinman, MD, chairman and co-founder of Pasithea, said in a company press release. “We are honored that the ALS Association recognizes the promise of PAS-004.”
Making ALS ‘a livable disease’
The $951,976 grant was awarded to the Mayo Clinic in Jacksonville, Florida. It will support a project titled, “Efficacy, safety and tolerability of PAS-004 for the treatment of ALS,” which according to the project summary is “an initial step to see if PAS-004 is safe and well-tolerated in humans and to determine the best dose to use in a larger clinical trial in the future.” The trial will be conducted by Jaimin Shah, MD, a neurologist at the clinic, in collaboration with Pasithea.
ALS is a neurodegenerative disease characterized by the progressive loss of motor neurons, nerve cells that control voluntary movements. While its exact causes aren’t fully understood, about 97% of patients develop abnormal clumps of the TDP-43 protein inside nerve cells, which are toxic and disrupt normal cell processes.
“Inflammation and the aggregation of a protein called TDP-43 are well-recognized contributors to the development and progression of ALS,” said Tiago Reis Marques, MD, CEO and co-founder of Pasithea.
PAS-004 is designed to inhibit the MEK protein, which is believed to contribute to TDP-43-related neurodegeneration and neuroinflammation. This makes PAS-004 a potentially relevant therapy for ALS.
Marques said the upcoming trial will evaluate three dose levels of PAS-004 in 12 people with ALS, who will be followed for 28 weeks, or approximately 6.5 months.
The main goal is to assess for safety and tolerability. However, researchers will also investigate potential early signs of efficacy by examining changes in the ability to perform activities of daily living and in neurofilament light chain levels, a biomarker of nerve damage.
“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,” said Kuldip Dave, PhD, senior vice president of research at the association. “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.”
PAS-004 “delivered significant and promising results” in mouse experiments, said Steinman, noting that the treatment is also showing a promising safety profile in ongoing clinical trials for neurofibromatosis and advanced cancers. “We are excited for PAS-004 to enter the clinic for ALS, a disease in great need of advances in therapy.”
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