SAN DIEGO, CA, Dec 2025 — Repeat Therapeutics, a biotechnology company developing potential treatments for neurodegenerative diseases with a current focus on amyotrophic lateral sclerosis (ALS), today announced that the first participant has been dosed in its Phase 1 clinical study evaluating RTX-101, the company’s lead investigational candidate for ALS associated with the C9orf72 hexanucleotide repeat expansion.
C9orf72-associated ALS is the most common known genetic cause of familial ALS. RTX-101 and RTX-111 are orally administered investigational small-molecule combination therapies designed to restore cellular balance and reduce neurotoxic stress in motor neurons. It is hypothesized that the investigational therapies modulate key biochemical processes involved in oxidative injury and cellular stress in C9orf72-mediated ALS with potential broader application to other neurodegenerative diseases.
The Phase 1 study is designed to evaluate the safety, tolerability, and pharmacokinetics of RTX-101, RTX-111 in healthy volunteers, with planned expansion into individuals with C9orf72-associated ALS. The study is being conducted in Australia under the Therapeutic Goods Administration (TGA) Clinical Trial Notification (CTN) pathway, with Human Research Ethics Committee(HREC) approval.
“Dosing the first participant marks an important early milestone for Repeat,” said Susan Francia, co-founder of Repeat Therapeutics. “ALS remains a devastating disease with limited treatment options, and our team is committed to advancing this research thoughtfully and responsibly. These early studies are designed to help us better understand the safety of our investigational approaches as we work toward addressing an area of unmet need.”
Repeat Therapeutics is advancing a focused pipeline of investigational approaches for repeat expansion disorders, with an emphasis on understanding and addressing metabolic and cellular stress pathways.
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