Trace Neuroscience, a member of BioSpace’s NextGen Class of 2026, has learned from the success of Biogen’s Qalsody and aims to bring more treatment options to the ALS community.
Not much has changed for people diagnosed with amyotrophic lateral sclerosis since the days of Lou Gehrig. The clinic is littered with failed candidates from pharma and biotech alike. But Trace Neuroscience, which launched in November 2024, has found a target to shoot for in an attempt to offer some hope to patients with the neurodegenerative disease.
The biotech’s technology comes from University College London, where neurologist Pietro Fratta and his team discovered that people with ALS have insufficient levels of the UNC13A protein, which helps coordinate neuronal communication. At the same time, a group of scientists at Stanford made a similar discovery, and the two teams published papers simultaneously in 2022, according to Trace CEO Eric Green, who spoke to BioSpace as a member of the NextGen Class of 2026.
The rivals teamed up to form the biotech, recognizing the importance of the work and the possibility of helping people with ALS.
Trace is developing medicines to restore the function of UNC13A in hopes of re-establishing healthy communication between nerves and muscle cells and thus improving muscle function. The biotech’s lead clinical candidate is TRCN-1023, an antisense oligonucleotide delivered intrathecally to the cerebral spinal fluid, Green said.
He pointed to the success of Biogen’s tofersen, now known as Qalsody, which was approved in 2023 for patients with ALS who have a mutation of the SOD1 gene. This occurs in about 10-20% of all familial ALS cases, according to the ALS Association. The development of Qalsody provided important lessons for a company like Trace, including the utility of using biomarkers to offer a targeted approach. Specifically, Biogen tracked neurofilament light chain, which is released into the bloodstream when neurons degenerate.
Trace will use the same biomarker for studies of UNC13A-targeting therapeutics. However, the biotech hopes to be able to provide medicines for a broader swath of the ALS population.
“We very much see the work that was done in developing tofersen as setting the stage for what we plan to do,” Green said.
Trace has arrived amid biopharma’s neurodegenerative renaissance. Many Big Pharmas are striking deals, with Bristol Myers Squibb doubling down on a contract with insitro to develop ALS therapeutics in October 2025. Eli Lilly, meanwhile, has signed two recent deals in the space, including investing $415 million to work with Alchemab and up to $622 million to partner on a preclinical asset from QurAlis.
Green credits the renaissance to the improved understanding of biomarkers for the disease and unmet need in the space. Additionally, ALS moves so quickly that clinical benefit can be revealed faster than in your average clinical trial—an awful trait of the devastating disease but helpful for researchers in determining when to move on or double down.
“Six months in an untreated person with ALS, you expect to see considerable changes in their clinical function and loss of function, and so that gives us an opportunity to see relatively quickly if we’re seeing benefit with our treatment,” Green said.
Over the next year, Trace will be working to get TRCN-1023 into the clinic, running trials in patients with ALS rather than starting with healthy volunteers.
Green said that since the 2022 ALS findings, the understanding of UNC13A has grown, and evidence suggests that the target could also important in other neurodegenerative diseases, such as frontotemporal dementia or Alzheimer’s disease.
“ALS is where we’re starting, but [we] see a number of opportunities to expand quickly into these other disease areas,” Green said.
