Friday, 27 February 2026
Research
On 23 February 2026, US biotech company QurAlis gave an interim update on an early-stage clinical trial it is running for a gene therapy called QRL-201. The trial focuses on people with sporadic MND (i.e. with no family history of the disease) and those with MND caused by alterations in the C9orf72 gene. Although the company has not yet published data, it reported that the therapy was safe and well tolerated by trial participants. The update also suggested there could be a trend of slowing decline in the physical function of participants with sporadic MND. This is measured by an established questionnaire called ALSFRS-R. Although this update comes at an early point in the trial, it is encouraging and we look forward to further updates.
See below for a detailed look at the update.
US biotech QurAlis announced an interim update from its Proof-of-Concept Phase 1/2 ANQUR clinical trial of QRL-201. QRL-201 is a gene therapy that restores STATHMIN-2 (STMN2) expression. It was tested in people with both sporadic MND and C9orf72-MND. Key findings include:
STMN2 is a protein that plays an important role in motor neuron biology. It is controlled by a protein called TDP-43, which is dysregulated in ~97% of people living with MND. STMN2 expression itself is decreased in the majority of people living with MND. QRL-201 is the first treatment aiming to restore STMN2 expression that has reached clinical trial stage for MND. QRL-201 is a kind of gene therapy called an Antisense Oligonucleotide (ASO). Another example of an ASO is tofersen, which has now been approved by the MHRA for the treatment of SOD1-MND.
The ANQUR study included 69 patients who were given either QRL-201 or placebo in a double-blind manner (where neither the participants nor the clinical trial team know who is receiving the treatment or placebo, to avoid bias). In the first part of the study, participants were given increasing doses of QRL-201 to demonstrate safety and tolerability and that the treatment was getting into the right tissues at the right concentrations. Next, a different set of participants were given two different doses of QRL-201 and its effect on disease progression was assessed via biomarker measurements (including pNfH) and the ALSFRS-R questionnaire.
This update from the QurAlis sounds encouraging and the company is now applying for an open-label extension so that patients who participated in the trial and are still eligible for treatment can continue to receive QRL-201 at a low dose. This extension has already been approved in Canada and is currently under regulatory review in the European Union and the United Kingdom. QurAlis is also preparing to advance QRL-201 into a Phase 3 clinical trial in 2027. We do not yet know if this is planning to come to the UK, but will publish more about this as information emerges.
It’s great to see positive updates like this for potential new MND treatments. In particular, we’re excited to see the focus on ensuring that the treatment is effectively restoring the expression of STMN2 in the right tissues. This kind of data, that shows that a drug is working effectively, has been missing from many historic MND trials. This makes it difficult to know whether trials failed because the drug didn’t get to the right tissue in high enough quantities, or because it wasn’t effective. However, this is an early phase clinical trial and we still need to see the data. There are some unanswered questions from this update such as:
We will continue to provide updates and try to answer these questions as new information emerges.
You can find out more about this trial on QurAlis’ website: 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 - QurAlis.
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