Early-stage ALS research just received a powerful boost. The ALS Association has announced the 2025 Hoffman ALS Clinical Trial Award winners, providing up to $1 million per project to fuel phase 1 and 2a trials. These grants act as catalytic capital, designed to turn strong science into actionable clinical progress.
Why These Awards Matter?
ALS research moves slowly without early clinical testing. Most therapeutic candidates stall because phase 1 trials are expensive, risky, and hard to fund. The Hoffman Awards aim to change that.
“Funding for early testing is ignition fuel,” said Dr. Kuldip Dave. “These studies create critical data that make larger trials less risky and more attractive to investors.”
This year’s awardees were selected for strong biological rationale, rigorous preclinical evidence, and clear plans for clinical advancement.
2025 Hoffman ALS Clinical Trial Awardees
Mayo Clinic Jacksonville – PAS-004 (MEK Inhibitor)
Lead Investigator: Jaimin Shah, M.D.
Partner: Pasithea Therapeutics
Trial: Phase 1, 12 ALS participants
Why It Matters?
PAS-004 targets MEK, an enzyme linked to inflammation, nerve damage, and neuron loss in ALS.
The study will assess:
- Safety
- Tolerability
- Optimal dose
- Early biomarker shifts
“Phase 1 trials cannot confirm efficacy,” said Dr. Shah. “But they answer essential questions that shape the drug’s path forward.”
Molefy Pharma – AP-2 (TDP-43 Modulator)
Lead Investigator: Ana Martínez, Ph.D.
Focus: Restoring TDP-43 protein balance
Trial: Phase 1, first-in-human
Why It Matters?
TDP-43 imbalance is found in 97% of ALS cases. This protein misfolds, forms toxic aggregates, and drives neurodegeneration.
AP-2 aims to correct this imbalance.
The trial will define:
- Maximum safe dose
- Therapeutic window
- Early safety markers
“This step is crucial for developing a new TDP-43 modifying therapy,” said Dr. Martínez.
A Push Toward Faster, Better ALS Trials
ALS remains a devastating diagnosis. Most patients die within five years. Few treatment options exist. The need for new therapies is enormous.
The Hoffman Awards help compress timelines by backing trials that:
- Are biomarker-driven
- Have strong mechanistic rationale
- Show promise in preclinical work
- Can progress quickly to larger studies
“We want ALS to become livable while we pursue a cure,” Dr. Dave emphasized.
About the ALS Association
The ALS Association is the world’s largest ALS-focused organization. It funds global research, supports families through its care network, and advocates for better policies.
Its mission: make ALS livable and find a cure.
About the Hoffman ALS Clinical Trial Awards
These awards were made possible by a historic $58 million gift from philanthropist Hugh Hoffman. His contribution created the Hugh and Herbert Hoffman ALS Fund—now a major force accelerating:
- New treatments
- Clinical-care infrastructure
- Access to multidisciplinary care
