The ALS Association has announced the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to USD 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. 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," said Dr Kuldip Dave, PhD, Senior Vice President—Research, ALS Association.

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 pre-clinical data, an integrated biomarkers programme and a clear plan for future clinical development.

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

Ana Mart?nez, Co-Founder and Scientific Director, PhD, Molefy Pharma SL, will lead phase 1 clinical testing of an investigational therapy known as AP-2, that has shown promise in pre-clinical studies, helping to restore the balance of a protein called TDP-43. In approximately 97 percent 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. We will work for the success of this project with all our strength and energy," Dr Mart?nez said.

Clinical trials are the most reliable and 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. By funding the 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," said Dr Dave.