ALS Association Commits $2 Million to Support Early-Stage Clinical Trials of Two Emerging ALS Therapies

This funding was awarded through the ALS Association's Hoffman ALS Clinical Trial Awards Program, which aims to accelerate the development of new ALS treatments

ARLINGTON, Va., Dec. 4, 2025 /PRNewswire/ -- The ALS Association is pleased to announce the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate important data about things like safety, dosing, and biomarkers to help speed up the availability of effective new ALS treatments.

"Funding for early-stage clinical testing acts like 'ignition fuel' for the R&D engine," said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. "The knowledge gained from these initial trials helps propel drug development forward by providing critical data that can be used to inform larger efficacy studies. It also helps make these next steps less risky and more attractive to potential investors and partners."

This year's awardees were selected from a competitive group of applicants from around the world. Their applications demonstrated a compelling biological rationale for the investigational therapy, robust preclinical data, an integrated biomarkers program, and a clear plan for future clinical development.

2025 Awardees 
Mayo Clinic Jacksonville – Consultant Jaimin Shah, M.D., will conduct a phase 1 trial of an investigational therapy called PAS-004 in partnership with Pasithea Therapeutics Corp. PAS-004 targets an enzyme called MEK, which is involved in the inflammation, nerve damage, and nerve loss seen in ALS. The trial will recruit 12 people living with ALS to evaluate the safety and tolerability of PAS-004 and determine the best dosage to test in future studies. Biomarkers will also be measured to identify any early signs of potential disease-modifying effects.

"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," Dr. Shah said. "We hope this trial will improve the field's understanding of the role MEK inhibitors could play in the treatment of ALS."

Molefy Pharma SL – Co-founder and Scientific Director Ana Martίnez, Ph.D., will lead phase 1 clinical testing of an investigational therapy known as AP-2. AP-2 has shown promise in preclinical studies, helping to restore the balance of an important protein called TDP-43. In approximately 97% of people with ALS, TDP-43 imbalances lead to the buildup of abnormal, disease-causing clumps or aggregates. Results from this trial will help determine the highest safe dose of AP-2, its therapeutic window, and any possible side effects.

"This first-in-human trial represents a crucial initial step toward testing in ALS patients and paves the way for the clinical development of a new TDP-43–modifying drug," Dr. Martίnez said. "We will work for the success of this project with all our strength and energy."

Clinical trials are the most reliable—and ultimately the fastest—way to translate promising laboratory science into better ways to treat ALS. Through its Hoffman ALS Clinical Trial Awards, the ALS Association is paving the way for more, better, faster ALS clinical trials.

"People living with ALS urgently need new treatments," said Dr. Dave. "By the funding early-stage clinical trials like these, we are working to accelerate the development of promising therapeutic candidates so we can make ALS livable until we can cure it."

About the ALS Association 
The ALS Association is the largest ALS organization in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at www.als.org.

About ALS
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression.

About the Hoffman ALS Clinical Trial Awards Program 
Hoffman ALS Clinical Trial Awards support early- to mid-stage (phase 1 or 2a) biomarker-driven clinical trials of novel or repurposed therapeutics for ALS (either disease-modifying or symptomatic). These annual awards are made possible through the historic $58 million gift from the late Hugh Hoffman, a Cincinnati native and philanthropist whose life was shaped by the loss of his father, Herbert, to ALS. Mr. Hoffman's contribution, the largest single philanthropic gift ever made to an ALS organization, established the Hugh and Herbert Hoffman ALS Fund, which is dedicated to advancing new treatments, strengthening clinic infrastructure, and reduce barriers to accessing a multidisciplinary care model.

SOURCE The ALS Association