QurAlis Reports ANQUR Interim Data for STATHMIN-2-Targeting Agent QRL-201 in ALS

An investigational antisense oligonucleotide designed to correct a common splicing defect in amyotrophic lateral sclerosis (ALS) showed signs of biological activity in interim trial data. QurAlis's QRL-201 demonstrated target engagement, a neurofilament biomarker reduction, and a trend toward slower functional decline in the phase 1 ANQUR trial (NCT05633459) in people with sporadic and C9orf72-related ALS.¹

Merit E. Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, said she was “encouraged” by the findings. In the original February readout, she called the combination of target engagement, biomarker changes, and clinical measures “very promising and remarkable to see from an early stage clinical trial.”²

The full dataset was presented at the 2026 Northeast ALS Consortium (NEALS) Annual Meeting, held October 27-30, 2026, in Clearwater, Florida, by Manoj Malhotra, MD, chief medical officer of QurAlis, along with coauthors from the company.¹

STMN2 mis-splicing and the ANQUR trial

STMN2, a protein involved in neuronal repair, axonal stability, and neuromuscular junction innervation, is consistently mis-spliced in postmortem tissue from people with ALS. QRL-201 is designed to correct that mis-splicing and restore STMN2 protein function, administered intrathecally.

ANQUR is a first-in-human, phase 1 trial evaluating QRL-201's safety, tolerability, pharmacokinetics, and exploratory efficacy. It has dosed 69 participants, 17 in a completed dose-escalation phase and 52 in an ongoing dose-range-finding (DRF) phase, randomized 6:2 to QRL-201 or placebo within each cohort. Study drug is given up to 5 times over 12 weeks, with terminal pharmacokinetic assessment at 48 weeks and an option to enroll in open-label extended dosing.

QurAlis first advanced the trial into the DRF phase in November 2024, after pharmacokinetic analyses from the first 2 dose-escalation cohorts showed cerebrospinal fluid exposure levels meeting or exceeding the targeted therapeutic range.³

Results reported at NEALS 2026

In tissue analysis, QRL-201 showed broad distribution across the spinal cord and motor cortex, with statistically significant increases in STMN2 above the estimated therapeutic target and correction of STMN2 mis-splicing. The DRF phase showed a statistically significant reduction in neurofilaments in the low-dose group, alongside a trend toward slowing ALSFRS-R decline. In a post hoc analysis that excluded participants in the highest tertile of baseline neurofilament light, a marker associated with faster disease progression, sporadic ALS patients showed a statistically significant, clinically meaningful slowing of ALSFRS-R decline at 26 weeks.^1,2

QRL-201 was reported to be generally safe and well tolerated, with most adverse events mild to moderate. Specific adverse event counts or rates were not reported in the materials reviewed for this article. An unblinded data safety monitoring board most recently recommended, in December 2025, that the study continue without modification. Based on the interim results, QurAlis said it is preparing to advance QRL-201 into a pivotal phase 3 trial in 2027, with an open-label extended-dosing period already approved in Canada and under regulatory review in the UK and EU.^1,2

Pipeline context: QRL-101

QurAlis is also developing QRL-101, a Kv7.2/7.3 ion channel opener intended to reduce hyperexcitability-driven neurodegeneration in ALS, a distinct mechanism from QRL-201's STMN2-targeting approach. The company began dosing the first cohort of a phase 1 multiple-ascending-dose trial of QRL-101 in September 2024.⁴

REFERENCES
1. Gray T, Eon V, Tatterson L, et al. ANQUR: a phase 1 MAD and dose-range finding study to assess safety, tolerability, pharmacokinetics, and efficacy of QRL-201, a STMN2 targeting splice-switching ASO for the treatment of sporadic and C9orf72 ALS. Presented at: 2026 Northeast ALS Consortium (NEALS) Annual Meeting; October 27-30, 2026; Clearwater, FL.
2. QurAlis demonstrates effects on disease progression and target engagement in ANQUR clinical trial of QRL-201, a first-in-class precision medicine in development for sporadic ALS. News release. QurAlis. Published February 23, 2026. Accessed November 4, 2026. https://www.quralis.com/news/quralis-demonstrates-effects-on-disease-progression-and-target-engagement-in-anqur-clinical-trial-of-qrl-201-a-first-in-class-precision-medicine-in-development-for-sporadic-als/
3. QurAlis' ANQUR clinical trial of QRL-201 in ALS advances to dose range-finding (DRF) phase with first participant dosed. News release. QurAlis. Published November 19, 2024. Accessed November 4, 2026. https://www.prnewswire.com/news-releases/quralis-anqur-clinical-trial-of-qrl-201-in-als-advances-to-dose-range-finding-drf-phase-with-first-participant-dosed-302307803.html
4. QurAlis doses first participant cohort in phase 1 multiple-ascending dose (MAD) clinical trial evaluating QRL-101, a first-in-class Kv7 precision therapy for ALS. News release. QurAlis. Published September 10, 2024. Accessed November 4, 2026. https://www.prnewswire.com/news-releases/quralis-doses-first-participant-cohort-in-phase-1-multiple-ascending-dose-mad-clinical-trial-evaluating-qrl-101-a-first-in-class-kv7-precision-therapy-for-als-302239922.html