Restoring Protein Levels With QRL-201 Has Potential to Stabilize Disease Progression in ALS: Angela Genge, MD, FRCPC, eMBA

WATCH TIME: 4 minutes

“The [antisense oligonucleotides] that we are bringing into the clinic right now is a splice switcher that will enable us to increase levels of normal STATHMIN-2. On cell models, it rescues the phenotype, it rescues the motor neurons from the dying back phenomenon that occurs with the loss of full length STATHMIN-2.”

QRL-201 (QuarAlis), an antisense oligonucleotide therapy, is currently in evaluation for the treatment of amyotrophic lateral sclerosis (ALS) in a first-in-human, phase 1 study called ANQUR (NCT05633459). The therapy is a first-in-class therapeutic product candidate aimed to restore STATHMIN-2 (STMN2) expression in patients with ALS, and the trial is the first-ever study to investigate a treatment that rescues STMN2 expression in patients with ALS.1

The primary objective of the ANQUR is to determine the safety and tolerability of multiple doses of QRL-201 in people with ALS, and it is expected to include 64 patients with ALS across sites in Canada, the U.S., the United Kingdom, Belgium, the Netherlands, Italy, Germany, and Ireland. Recently, QuarAlis announced that the first patient has been dosed in its trial at the University of Montréal Hospital Centre (CHUM).¹

Angela Genge, MD, FRCPC, eMBA, clinical director, executive director, clinical research unit, Montreal Neurological Institute, and chief medical officer, QurAlis, presented on therapy at the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas. In her talk, she spoke about gene directed therapies for patients with sporadic ALS and the potential of QRL-201 in ALS care.²

Genge sat down in an interview with CGTLive®, a sister publication to NeurologyLive®, to talk about the main highlights from her presentation. She expanded upon the advantages of QRL-201, how it may restore STMN2 and rescue the disease phenotype seen in ALS. Also, she discussed the different standards of care in ALS, comparing the current treatment landscape of symptom-relieving therapies. Additionally, Genge spoke upon the unmet needs in ALS care and research that remains to be addressed in regard to effectively treating the disease.

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REFERENCES
1. QurAlis Announces First Patient Dosed With QRL-201, a First-in-Class STATHMIN-2 Precision Therapy for ALS. News Release. QurAlis. Published April 6, 2023. Accessed April 13, 2023. https://quralis.com/quralis-announces-first-patient-dosed-with-qrl-201-a-first-in-class-stathmin-2-precision-therapy-for-als/
2. Genge, A. Gene Directed Therapy for Sporadic ALS: The Story of Stathmin. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Gene Therapy in ALS session.