Phase 1 trial of QRL-201 to open for patients at sites across Europe

Experimental ALS treatment aims to restore key protein's production

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by Andrea Lobo |

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QurAlis soon expects to open its ongoing Phase 1 clinical trial testing QRL-201 in people with amyotrophic lateral sclerosis (ALS) at sites in various European Union countries, including Germany, Belgium, the Netherlands, and Ireland.

The company’s announcement comes after these countries reviewed and accepted an application for the study. The Phase 1 ANQUR trial (NCT05633459), which is currently recruiting in Canada, could start enrolling patients at European sites by year’s end.

“This EU Clinical Trial Authorisation marks the second regulatory clearance for QRL-201 as we execute 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.

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ALS is a progressive neurodegenerative disorder that damages motor neurons, the nerve cells that control voluntary movements, leading to muscle weakness and muscle atrophy (shrinkage).

In most ALS cases, toxic aggregates of the TDP-43 protein accumulate in nerve cells, and they are believed to directly contribute to neurodegeneration. This protein normally works to process messenger RNA, an intermediated molecule made when genes are “read” to produce proteins.

Stathmin-2 (STMN2) is the protein most significantly regulated by TDP-43 in people. This protein is important for neural repair and stability, and it is abundant in motor neurons. But its levels drop significantly when TDP-43 is faulty, and stathmin-2 is known to be at lower-than-normal levels in nearly all ALS patients.

QRL-201 is a first-in-class experimental therapy designed to raise STMN2 levels in patients. This has been shown to lessen the damage caused by TDP-43 aggregates, and to restore nerve cells’ ability to repair damage. As such, QRL-201 could work to slow or prevent ALS progression.

ANQUR is the first trial evaluating a therapy designed to rescue STMN2 production. It aims to test the safety, tolerability, and pharmacological properties of QRL-201 in up to 64 people with ALS.

Participants will be randomly assigned to one of eight increasing doses of the therapy or to a placebo, both given by an injection into the spinal canal (intrathecal injection). Each dosing group will include eight patients, six being treated with QRL-201 and two being given a placebo. A first group in Canada is fully enrolled and dosing is underway.

The company plans to open additional sites in the U.S. and U.K.

“We believe QRL-201 has the potential to halt disease progression, which could transform the care and treatment of ALS. We are dedicated to ensuring efficient advancement of our ANQUR clinical trial so that we can fulfill our mission to make a meaningful difference in patients’ lives,” Roet said.