QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, announced interim data from its Proof-of-Concept Phase 1/2 ANQUR clinical trial of QRL-201 in people living with amyotrophic lateral sclerosis (ALS). Results from this study demonstrated QRL-201 had an effect on disease progression in ALS patients; this effect was confirmed by markers of clinical efficacy and changes in a clinically relevant biomarker, along with target engagement.

QRL-201 is a first-in-class, potent antisense oligonucleotide (ASO) precision therapeutic in development to restore STATHMIN-2 (STMN2) expression in ALS patients with the aim to modify disease progression and improve outcomes. STMN2 is a protein important for muscle innervation that is regulated by TDP-43 and downregulated in the majority of ALS patients. ANQUR (QRL-201-01; NCT05633459) is the first-ever clinical trial to evaluate a potential therapy to rescue STMN2 expression in people with ALS. The ANQUR study is a double-blind, placebo-controlled study which dosed a total of 69 patients across the dose escalation (n=17) and dose-range finding (DRF) (n=52) phases of the study.

“I am encouraged by these data from the ANQUR clinical trial of QRL-201. 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,” said Merit E. Cudkowicz, M.D., M.Sc., director, Sean M. Healey & AMG Center for ALS and executive director of the Mass General Brigham Neuroscience Institute, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

“ALS is a devastating, fatal neurodegenerative disease with a significant unmet medical need. Our mission is to make a meaningful difference for people living with ALS. We believe QRL-201 has the potential to modify disease progression and improve outcomes for sporadic ALS patients,” said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. “These results from the ANQUR clinical trial are well aligned with our understanding of STMN2 biology and the potential of STMN2 restoration to have a positive impact on ALS disease progression. We are grateful to the ANQUR clinical trial team, participants and their families, and look forward to advancing the QRL-201 clinical program to deliver a much-needed precision medicine option for people living with ALS.”

Results from the interim analysis of the ANQUR clinical trial show:

• Target engagement, confirmed through tissue analysis, with broad distribution across the spinal cord and motor cortex, statistically significant increased levels of STMN2, above the estimated therapeutic target, and correction of STMN2 mis-splicing, as compared to a cohort of natural history samples.
• A statistically significant and clinically meaningful reduction in phosphorylated neurofilament heavy (pNfH) in the low dose group.
• An encouraging trend of slowing decline in ALSFRS-R in sporadic patients, including a strong trend on the gross motor sub-score, which is critical for patient independence.
• In a post-hoc analysis excluding patients with the highest levels of baseline neurofilament light, which is associated with faster disease progression, there was a statistically significant and clinically meaningful slowing of decline in ALSFRS-R at the six-month (24 week) timepoint in treated sporadic ALS patients compared to placebo.
• QRL-201 was generally safe and well tolerated with most adverse events (AEs) reported as mild or moderate. During all phases of the study, and most recently in December 2025, the unblinded Data Safety Monitoring Board has recommended the study continue without modification.

These data support an open-label extended dosing period to the ANQUR clinical trial protocol under which all eligible trial participants will be given the opportunity to receive QRL-201 at the low dose of the DRF portion of the study. The open-label extended dosing period to the ANQUR clinical trial has been approved in Canada and is currently under regulatory review in the European Union and the United Kingdom. In parallel, QurAlis is preparing to advance QRL-201 into a pivotal Phase 3 clinical trial in 2027.