Phase 1 clinical trial of QRL-201 for ALS approved in the UK
ANQUR study is recruiting patients at three sites in Canada
The announcement follows the trial’s clearance by the country’s Medicines and Healthcare Products Regulatory Agency.
“We look forward to advancing ANQUR in the U.K. as part of our global strategy focused on bringing breakthrough precision medicines to patients with ALS and other neurodegenerative diseases,” Kasper Roet, PhD, CEO and co-founder of QurAlis, said in a company press release.
The placebo-controlled Phase 1 ANQUR study (NCT05633459) is recruiting patients at three sites in Canada, but is expected to start enrollment in Germany, Belgium, the Netherlands, and Ireland by the end of the year following its regulatory clearance in those countries. The company also plans to open sites in the U.S.
According to QurAlis, the trial, which will test several QRL-201 doses, has completed enrollment of the first group in Canada and has already dosed the first patient.
ANQUR’s design was recently presented at the European Network to Cure ALS (ENCALS) meeting, July 12-14 in Barcelona, Spain.
QRL-201 works to restore STMN2 levels
ALS is marked by the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movements. With neuronal loss, the brain can no longer control movement, leading to muscle weakness and atrophy.
In most patients, toxic aggregates of the TDP-43 protein build up inside nerve cells, including motor neurons, contributing to their degeneration. This protein is found at high levels in the brain and spinal cord and normally regulates the first step of protein production from a gene.
Research by Kevin Eggan, PhD, QurAlis’ co-founder and former Harvard professor, showed TDP-43 regulates Stathmin-2 (STMN2), a protein produced in motor neurons and involved in neuronal repair. Toxic TDP-43 clumps affect the protein’s function, resulting in low STMN2 levels being produced.
QRL-201 is a first-in-class investigational therapy designed to increase STMN2 levels. It was shown to effectively increase STMN2 levels in lab-grown motor neurons and mouse models, reversing several neurodegenerative processes. Because of this, the therapy is expected to slow ALS progression.
The first in-human ANQUR trial will test the safety, tolerability, and pharmacokinetics of multiple ascending doses of QRL-201 in up to 64 adults, ages 18-80, who developed ALS symptoms within the past two years. Pharmacokinetics refers to a therapy’s movement into, through, and out of the body.
Participants will be randomly assigned to receive either QRL-201 at one of eight doses or a placebo via an injection into the spinal canal. The study is expected to be completed in May 2025.