QurAlis Granted Clinical Trial Authorization (CTA) in the U.K. for QRL-201: A First-of-its-Kind Precision Therapy for ALS Targeting STATHMIN-2
Wednesday, July 12, 2023
QurAlis Corporation recently announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom has accepted their Clinical Trial Application (CTA) for QRL-201, a potential treatment for amyotrophic lateral sclerosis (ALS). QRL-201 is a precision therapeutic product candidate developed by QurAlis, aiming to restore the expression of STATHMIN-2 (STMN2) in ALS patients. STMN2 is an important protein for neural repair and axonal stability, and its expression is significantly reduced in ALS patients.
QRL-201 has shown promise in rescuing STMN2 loss of function in motor neuron disease models derived from ALS patients. QurAlis has already enrolled patients and dosed the first participant in the Cohort 1 portion of the Phase 1 ANQUR clinical trial in Canada. With the MHRA's clearance, the company plans to initiate the trial in the United Kingdom by the fourth quarter of 2023. QurAlis aims to bring breakthrough precision medicines to ALS and other neurodegenerative disease patients through their global strategy.
The ANQUR clinical trial (NCT05633459) is significant as it will be the first to evaluate a therapy that restores STMN2 expression in ALS patients. The trial is designed as a randomized, double-blind, placebo-controlled Phase 1 study to assess the safety, tolerability, and pharmacokinetics of QRL-201. It is expected to include 64 ALS patients across multiple sites in Canada, the European Union, the United States, and the United Kingdom.
QurAlis also plans to present a poster on the ANQUR study design and participate in the Therapeutic Section at the European Network to Cure ALS (ENCALS) meeting in Barcelona, Spain, in July 2023.
To support the clinical development of QRL-201, QurAlis has established a global regulatory strategy. Apart from the UK, they have already obtained cleared CTAs in Canada and the European Union, including Germany, Belgium, the Netherlands, and Ireland.
STMN2, also known as SCG-10, plays a crucial role in stabilizing microtubules, which are essential for the cytoskeleton and axonal structure. The expression of STMN2 is significantly reduced in almost all ALS patients. QurAlis co-founder Kevin Eggan, Ph.D., discovered that the expression of STMN2 is regulated by TDP-43, a protein associated with ALS and other neurodegenerative diseases. When TDP-43 function is impaired, it leads to a decrease in STMN2 expression and neuronal repair. Restoring STMN2 levels can rescue this impairment.
TDP-43 pathology is not only linked to ALS but also present in approximately 50% of frontotemporal degeneration (FTD) cases, the second most common form of dementia. It is also associated with a subset of Alzheimer's disease and Parkinson's disease patients.
Given the lack of effective therapies for ALS and FTD, the successful development of QRL-201 could offer hope for patients suffering from these devastating diseases.
