CAMBRIDGE, Mass. — QurAlis said interim data from its Phase 1/2 ANQUR clinical trial suggest its investigational therapy QRL-201 may slow disease progression in people living with sporadic amyotrophic lateral sclerosis, supported by evidence of target engagement and favorable changes in key biomarkers.
QRL-201 is a first-in-class antisense oligonucleotide precision medicine designed to restore expression of STATHMIN-2, a protein critical for muscle innervation that is reduced in most ALS patients due to TDP-43 dysfunction. The ANQUR trial is the first clinical study to evaluate a therapy aimed at rescuing STATHMIN-2 expression in ALS.
According to the company, interim results showed evidence of target engagement, statistically significant effects on a neurofilament biomarker associated with neuronal injury, and an overall trend toward slower functional decline as measured by the ALS Functional Rating Scale–Revised. A subgroup analysis demonstrated a statistically significant and clinically meaningful slowing of ALSFRS-R decline in treated patients compared with placebo.
“I am encouraged by these data from the ANQUR clinical trial of QRL-201,” said Merit E. Cudkowicz, M.D., M.Sc., director of the Sean M. Healey & AMG Center for ALS and executive director of the Mass General Brigham Neuroscience Institute. “The combination of effects on target engagement biomarkers, disease biomarkers such as neurofilament, and clinical measures is very promising and remarkable to see from an early-stage clinical trial.”
ANQUR is a randomized, double-blind, placebo-controlled study that enrolled 69 patients across dose-escalation and dose-range-finding phases. Interim findings showed statistically significant increases in STATHMIN-2 levels and correction of mis-splicing compared with natural history samples, as well as broad distribution of the therapy across the spinal cord and motor cortex. The study also demonstrated a statistically significant reduction in phosphorylated neurofilament heavy in the low-dose group.
In addition, QurAlis reported an encouraging trend toward slower functional decline in sporadic ALS patients, including effects on gross motor function, which is closely linked to patient independence. In a post-hoc analysis excluding patients with very high baseline neurofilament light levels, treated patients showed a statistically significant slowing of ALSFRS-R decline at six months compared with placebo.
QRL-201 was generally safe and well tolerated, with most adverse events reported as mild or moderate. The independent Data Safety Monitoring Board has repeatedly recommended continuation of the study without modification, most recently in December 2025.
“These results are well aligned with our understanding of STATHMIN-2 biology and its potential role in modifying ALS disease progression,” said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. “Our mission is to make a meaningful difference for people living with ALS, and we are grateful to the trial participants, their families, and the ANQUR study team as we work to advance this program.”
Based on the interim data, the company said the ANQUR protocol will be expanded to include an open-label extended dosing period, allowing eligible participants to receive QRL-201 at the low dose identified in the study. This extension has been approved in Canada and is under regulatory review in the European Union and the United Kingdom.
QurAlis said it is also preparing to advance QRL-201 into a pivotal Phase 3 clinical trial in 2027.
