Trace Neuroscience Initiates Global Clinical Development Program for TRCN-1023, an Antisense Oligonucleotide Designed to Restore UNC13A Function for the Treatment of ALS

Phase 1/2 FUNCTION ALS trial initiated in Europe with additional global regions anticipated in 2026

First patients dosed in LAUNCH ALS, an investigator-initiated trial in China conducted in partnership with Tenacia Biopharmaceutical, to support accelerated global clinical development strategy

TRCN-1023 is designed to restore function of the UNC13A protein, a genetically validated target in 97% of people living with ALS

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Trace Neuroscience, Inc., a biopharmaceutical company expanding the promise of genomic medicine for people living with neurodegenerative diseases, today announced the initiation of its global clinical development program for TRCN-1023, an investigational antisense oligonucleotide (ASO) designed to restore UNC13A protein function for the treatment of amyotrophic lateral sclerosis (ALS).

The global TRCN-1023 clinical program includes the Phase 1/2 FUNCTION ALS trial, which has received clinical trial authorization in the United Kingdom and Netherlands, as well as LAUNCH ALS, an investigator-initiated trial (IIT) underway in China. The LAUNCH ALS trial is being conducted in partnership with Tenacia Biopharmaceutical, which provides deep expertise in neuroscience drug development and operational execution in China, and in collaboration with principal investigator Yilong Wang, M.D., Ph.D. at Beijing Tiantan Hospital, a leading neurological hospital in China. The first patients were dosed in LAUNCH ALS earlier this month.

“Our team helped establish UNC13A as one of the most compelling genetically validated targets in ALS, and we built Trace Neuroscience to translate that biology into a medicine,” said Eric Green, M.D., Ph.D., co-founder and CEO of Trace Neuroscience. “We are thrilled to now be advancing TRCN-1023 into the clinic with a global early development strategy that is poised to generate a robust clinical data package with the urgency that ALS demands.”

TRCN-1023 is a highly potent and durable ASO designed to re-establish healthy communication between nerves and muscle cells. Administered by intrathecal injection, TRCN-1023 is a targeted intervention that binds directly to UNC13A messenger RNA to regulate its processing and guide formation of functional UNC13A protein, potentially improving synaptic transmission and thereby nerve and muscle function.

“UNC13A is among the most promising targets in ALS research today with a strong grounding in human genetics and mechanistic biology,” said Dame Pamela Shaw, M.D., Professor of Neurology at the University of Sheffield and FUNCTION ALS Chief Investigator. “There is compelling rationale for restoring this protein's function, which has relevance to the vast majority of ALS patients. I look forward to contributing to a stronger understanding of TRCN-1023’s biological and clinical impact through the FUNCTION ALS trial.”

“People with ALS need meaningful therapeutic innovation beyond today’s limited treatment options. The potency, durability and biological rationale behind TRCN-1023 make it a particularly exciting drug candidate to bring into the clinic,” said Dr. Wang, LAUNCH ALS principal investigator who also serves as Executive Vice President at Beijing Tiantan Hospital and Professor of Neurology at Capital Medical University. “I am proud to partner with the Trace Neuroscience and Tenacia Biopharmaceutical teams to advance this program and accelerate a potential new treatment for people with ALS worldwide.”

About the FUNCTION ALS Phase 1/2 Clinical Trial & LAUNCH ALS IIT

FUNCTION ALS is a global Phase 1/2 randomized, double-blind, placebo-controlled clinical trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of TRCN-1023 in people living with ALS. The study is expected to enroll approximately 30 participants across sites in North America and Europe. Key eligibility criteria include age 18-75, symptom onset within the past two years or less, and slow vital capacity (SVC) of at least 60%. Individuals with SOD1 or FUS mutations are not eligible. Participants will receive TRCN-1023 or placebo, with 24 weeks of follow-up. Designed with input from people living with ALS and their caregivers, FUNCTION ALS incorporates biomarker analyses, digital movement and speech assessments, and operational measures intended to reduce participant burden.

LAUNCH ALS is an IIT conducted in collaboration with principal investigator Dr. Yilong Wang at Beijing Tiantan Hospital to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic activity of TRCN-1023 in people with ALS. The study is expected to enroll approximately 25 participants. Eligibility criteria for enrollment are consistent with the criteria for the FUNCTION ALS trial.

About ALS

Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease (MND) or Lou Gehrig’s disease), is a progressive and terminal neurodegenerative disease impacting nerve cells in the brain and spinal cord that reduces muscle function and control. As ALS advances, the ability to speak, swallow, move and breathe is increasingly impaired. In the U.S., approximately 30,000 people are living with ALS, and approximately 1 in 400 people will be diagnosed during their lifetime. Sporadic ALS that occurs without a clear family history or identified gene change is the most common form, accounting for 9 out of 10 cases, and has very limited treatment options.

About Trace Neuroscience

Trace Neuroscience is a biopharmaceutical company on a mission to expand the promise of genomic medicine for people living with neurodegenerative diseases. With an initial focus on ALS, the company is developing novel therapies to restore UNC13A protein function to re-establish healthy communication between nerves and muscle cells. Trace Neuroscience launched in 2024 with funding from leading life sciences investors and is headquartered in South San Francisco, California. For more information, please visit www.traceneuro.com and follow the company on LinkedIn and X.