New ALS treatment enters human testing in global program

Trace Neuroscience has launched its clinical development program for TRCN-1023, an experimental treatment for amyotrophic lateral sclerosis (ALS) that is designed to restore the function of UNC13A, a protein involved in nerve-muscle communication.

The global clinical program includes FUNCTION ALS, a company-sponsored Phase 1/2 clinical trial authorized in the U.K. and the Netherlands and expected to enroll participants at sites in the U.S., Canada, Germany, the Netherlands, and the U.K. as they open. Another trial in the program is LAUNCH ALS, an investigator-led clinical study underway in China in partnership with Tenacia Biopharmaceutical, where the first patients have already been dosed.

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Global program begins testing TRCN-1023

“Our team helped establish UNC13A as one of the most compelling genetically validated targets in ALS,” Eric Green, MD, PhD, Trace’s co-founder and CEO, said in a company press release. “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.”

ALS occurs when motor neurons — the nerve cells responsible for muscle movement — become damaged and die over time. As the disease progresses, muscle weakness causes patients to lose the ability to walk, speak, swallow, and eventually breathe. Current treatments are limited, creating a need for new therapeutic options.

TRCN-1023 is an antisense oligonucleotide, a short piece of genetic material, designed to bind to messenger RNA, the “instruction copy” cells use to produce proteins from DNA. By binding to UNC13A messenger RNA, TRCN-1023 is designed to help regulate how that RNA is processed, guiding the formation of functional UNC13A protein.

The target of TRCN-1023 is UNC13A, whose protein function is disrupted in most people with ALS. This occurs when TDP-43, a protein normally found in the nucleus, forms abnormal clumps outside that cellular compartment and disrupts the normal processing of UNC13A messenger RNA. As a result, faulty segments can remain in the RNA, making it unstable.

Therapy aims to restore nerve-muscle signaling

By binding to UNC13A messenger RNA, TRCN-1023 is designed to help restore proper RNA processing and guide the formation of functional UNC13A protein. This may help improve signaling between nerves and muscles.

“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, MD, a professor of neurology at the University of Sheffield in England 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.”

Designed with input from people living with ALS and their caregivers and with help from artificial intelligence tools, the FUNCTION ALS clinical trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of TRCN-1023 compared with a placebo in about 30 people with ALS whose symptoms started up to two years prior to enrollment.

Before being randomly assigned to receive TRCN-1023 or a placebo, patients will enter a two-week run-in period during which they will be asked to wear digital sensors on their dominant wrist and ankle to collect information about daily movement. They will also complete speech assessments at home.

TRCN-1023 and the placebo will be delivered into the spinal canal through intrathecal injection. Researchers will record side effects, how TRCN-1023 moves into, through, and out of the body, known as pharmacokinetics, and its biological effects in the body, known as pharmacodynamics. They will also track changes in movement, speech, functional abilities, and biomarkers.

Trials will track safety and early biological effects

After completing 24 weeks of follow-up, patients may have the option to enter a long-term extension.

In China, the LAUNCH ALS clinical trial is expected to enroll about 25 patients at Beijing Tiantan Hospital. Researchers led by principal investigator Yilong Wang, MD, PhD, will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of TRCN-1023.

“The potency, durability and biological rationale behind TRCN-1023 make it a particularly exciting drug candidate to bring into the clinic,” said Wang, LAUNCH ALS principal investigator, executive vice president at Beijing Tiantan Hospital, and professor of neurology at Capital Medical University in Beijing. “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.”