Trial of QRL-201, Aiming to Slow ALS Progression, Opening in Canada

Treatment designed to restore stathmin-2 protein levels in motor neurons

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by Andrea Lobo |

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Health Canada has given a green light to QurAlis‘ request to open a Phase 1 clinical trial of QRL-201, its candidate antisense oligonucleotide (ASO) molecule to protect and repair nerve cells, slowing disease progression, in people with amyotrophic lateral sclerosis (ALS).

The global trial, called ANQUR (NCT05633459), will investigate eight increasing doses of the medication against a placebo, given via injections into the spinal canal, in up to 64 ALS patients. Recruitment is to begin soon in Canada, and sites are expected to open in the U.S. and several European countries.

Its main goal is to test the safety and tolerability of QRL-201, with measures of the treatment’s pharmacokinetics — its movement into, through, and out of the body — as secondary objectives.

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“The authorization of our [clinical trial application] in Canada is a significant milestone for our company, representing our first program to receive clearance … to enter the clinic and the first ever clinical trial to evaluate a therapy that rescues [stathmin-2 protein] expression in ALS patients,” Kasper Roet, PhD, CEO and co-founder of QurAlis, said in a company press release.

“We are working diligently with our investigators in Canada to begin patient enrollment and dosing as quickly as possible,” Roet said.

ALS is a progressive neurological disorder that affects motor neurons, the nerve cells that control voluntary movements. With the loss of these neurons, muscles become smaller and weaker, and with time patients lose the ability to perform everyday tasks.

QRL-201 is an investigational ASO designed to increase the production of stathmin-2 (STMN2), a protein found at high levels in motor neurons and essential for neuronal growth and repair. This protein is at lower-than-normal levels in nearly all ALS patients.

Previous research using motor neurons derived from ALS patients showed that stathmin-2 deficiency in ALS patients is caused by the impaired function of TDP-43. This protein — involved in the regulation of gene activity, including that of making proteins like STMN2 — forms toxic clumps and contributes to nerve cell damage in about 97% of ALS cases.

Notably, raising stathmin-2 to normal levels was reported to help restore neuronal repair in cells with TDP-43 clumps, suggesting that QRL-201 may help slow disease progression in ALS patients with loss of stathmin-2 due to TDP-43 abnormalities.

“We will continue advancing the QRL-201 clinical program so that we can bring this potentially transformative treatment to patients rapidly and fulfill our mission to make a meaningful difference in patients’ lives,” Roet added.

Health Canada’s approval is part of QurAlis regulatory strategy for clinical development of QRL-201, with authorizations in other countries anticipated in the coming months.