ACE-2223-1 / Acelot - LARVOL DELTA

Acelot takes TDP-43 ALS drug ACE-2223 into first-in-human trial (AllSci Corp) - "The trial will evaluate safety, tolerability, and pharmacokinetics in healthy adults. Enrollment size, dosing schedule, and trial completion date have not been disclosed."

First-in-human • Trial status • Amyotrophic Lateral Sclerosis

ACE-2223, a first-in-class oral small molecule for sporadic ALS, treats both gain- and loss-of-function TDP-43 pathology, extends survival and decreases NfL in ALS mouse models and has begun human safety studies (NEALS 2026) - No abstract available

Preclinical • TARDBP

Single and Multiple-Ascending Dose Study of ACE-2223-1 in Healthy Adult Participants (Part A and Part B) (ANZCTR) - P1 | N=96 | Recruiting | Sponsor: Acelot, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Apr 2026

Enrollment open • First-in-human • Trial initiation date • Amyotrophic Lateral Sclerosis • CNS Disorders