QRL-201

QurAlis is a private biotech in Cambridge, Massachusetts, focused on ALS and related diseases. QRL-201 is its lead drug. The company told the trade press in February 2026 that it hoped these results would help it raise a new funding round (Series C) to pay for the pivotal trial. It is also developing QRL-101, a pill for nerve overexcitability in ALS, and a backup stathmin-2 drug, QRL-203.

In nearly all people with ALS, a protein called TDP-43 leaves the cell nucleus. Without it, the instructions for stathmin-2, a protein motor nerves need to repair themselves and stay connected to muscle, are read wrongly and too little is made. QRL-201 is a short strand of genetic material that sticks to those instructions and corrects the error. In spinal cord and brain tissue from trial participants who died, stathmin-2 levels were at least twice those in stored tissue from other people with ALS; that is a comparison with outside samples, not with placebo.

ClinicalTrials.gov lists ANQUR as Phase 1 with safety as its main goal, and QurAlis calls it a Phase 1/2 proof-of-concept study. We list it as Phase 1/2 because it compared QRL-201 with placebo in people with ALS and measured function and nerve-damage markers, not only safety. All 69 people have been dosed; participants can now get the drug in an open-label extension. QurAlis says a pivotal trial should start in 2027: in May 2026 it described a Phase 3 of about 270 people starting in early 2027, and its September 2026 slides call it a pivotal "Phase 2b".

Function was an exploratory measure, not the trial's main goal. In February 2026 QurAlis reported only a "trend" of slower decline in sporadic ALS, plus a significant result in an after-the-fact subgroup; in May, with more follow-up, it reported that the 10 mg group lost 5.1 fewer ALSFRS-R points than placebo after about 8.5 months, while the 3 mg group's 3.7-point difference was not reported as significant. Only 6 of 10 placebo patients were still in the study by then (40% had left through death, progression or other reasons, versus 7% on 10 mg), and the company says the data were not yet quality-checked. Spinal-fluid nerve-damage markers fell from baseline on 3 mg but rose on 20 mg, and none of these results has been published in a journal.

This is the most favourable result reported. See all key studies for the full picture.

QRL-201 is aimed at the large majority of people with ALS whose nerve cells lose TDP-43, which includes sporadic ALS and C9orf72 ALS. People with SOD1 or FUS gene changes were excluded. ANQUR's later groups enrolled adults with symptoms for less than 24 months, breathing capacity above 50% and signs of lower motor nerve involvement. The C9orf72 group was small and unbalanced, so the company says its results are hard to interpret.

QurAlis has said since February 2026 that it is preparing a pivotal trial to start in 2027. Its chief medical officer told BioSpace in May 2026 it should start in the first quarter of 2027 with about 270 people. No such trial is registered yet. First results from the open-label extension are also listed as upcoming.

ANQUR has finished enrolling and is not recruiting. Its open-label extension is only for people who took part in the blinded study. The pivotal trial has not been registered yet, and we found no expanded access programme.

• ANQUR (QRL-201-01) and open-label extension (NCT05633459) active not recruiting · Canada, UK, Ireland, Belgium, Germany, Netherlands