QurAlis presents interim results from phase 1/2 ANQUR trial

Biotech company QurAlis has presented interim results from the ANQUR study, a phase 1/2 clinical trial investigating the effects of QRL-201 in people living with ALS. The data show that the compound acts on the intended protein in nerve cells and affects biomarkers. The results were recently presented at the Target ALS Annual Meeting.

What do the interim results show?

The interim analysis of the trial yields promising findings. Researchers observed that the compound restores levels of the STMN2 protein in nerve cells. In addition, the group receiving the low dose showed a statistically significant reduction in blood pNfH, a biomarker for nerve cell damage. The compound was also found to be well tolerated, with no new safety concerns identified.

It is important to note that this is a phase 1/2 trial, which is primarily designed to demonstrate in a smaller group of participants whether a compound has an effect on biomarkers. To draw meaningful conclusions about the drug’s effect on disease progression, a larger phase 3 trial will be required.

About the investigational compound

QRL-201 is an antisense oligonucleotide (ASO), a small piece of synthetic DNA or RNA that can alter protein activity, potentially slowing a disrupted disease process. This approach is comparable to the existing Tofersen treatment, which has been used successfully in people with ALS caused by a SOD1 mutation. QRL-201 is specifically designed to restore the production of the protein STATHMIN-2 (STMN2). In the vast majority of people with ALS, STMN2 production is severely reduced. Without sufficient STMN2, nerve cells are less able to repair themselves and their connections to muscles become impaired, contributing to the muscle weakness and muscle loss that are hallmarks of ALS.

About the ANQUR study

The ANQUR study is a clinical trial investigating a treatment aimed at restoring STMN2 levels in people with ALS. The trial is double-blind and placebo-controlled, meaning neither participants nor researchers know who is receiving the investigational compound or the placebo. A total of 69 people with ALS took part. The study is running in Canada, the United Kingdom, and the European Union.

What are the next steps?

As a phase 1/2 trial, the ANQUR study assessed the effect and safety of the compound in a relatively small group of participants. QurAlis has announced that it is now preparing for a phase 3 trial of QRL-201, which is planned for 2027.