Trace Neuroscience launches first global clinical program for TRCN‑1023 ASO in ALS

Trace Neuroscience has initiated a global clinical development program for TRCN-1023, an investigational antisense oligonucleotide designed to restore UNC13A protein function in people with ALS. The UNC13A target is supported by genetics in about 97% of ALS patients, and TRCN-1023 aims to correct UNC13A mis‑splicing caused by TDP‑43 pathology, thereby improving synaptic transmission between nerves and muscles.

The program includes two key studies:
FUNCTION ALS, a Phase 1/2 randomized, double‑blind, placebo‑controlled trial in North America and Europe enrolling approximately 30 adults (18–75 years) with ALS symptom onset within the past two years and slow vital capacity ≥60%; and LAUNCH ALS, an investigator‑initiated trial (IIT) in China led by Dr. Yilong Wang at Beijing Tiantan Hospital, which will enroll about 25 participants. Both trials will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of intrathecally administered TRCN-1023 over a 24‑week follow‑up period.

TRCN‑1023 is a highly potent, durable ASO that binds UNC13A messenger RNA to guide formation of functional UNC13A protein. Nonclinical data show that TRCN‑1023 normalizes UNC13A splicing, increases correctly spliced UNC13A transcripts, and is well tolerated in preclinical models at doses higher than those expected to be used in humans. The data support the transition of TRCN‑1023 into these first‑in‑human clinical studies, which represent an early but important step in testing whether restoring UNC13A can meaningfully modify ALS disease course.