QurAlis became the latest biotech to join a steadily teeming amyotrophic lateral sclerosis (ALS) space with the a Phase I trial for a therapy designed to restore motor neuron function.
On April 6, Cambridge, Massachusetts-based QurAlis announced it had dosed the first patient in the Phase I ANQUR trial (NCT05633459) of its lead candidate QRL-201, antisense oligonucleotide (ASO).
QRL-201 is designed to restore the expression of the protein STATHMIN-2, which is implicated in neuronal repair and axonal stability. STATHMIN-2 is highly expressed in motor neurons, but patients with ALS often experience significant decreases in the protein.
QurAlis enters an active ALS drug development landscape amid a tumultuous period. Earlier this week, Cytokinetics discontinued its Phase III ALS program after a Data Monitoring Committee determined reldesemtiv failed an interim analysis.
Meanwhile, major ALS players Biogen and Brainstorm Cell Therapeutics closed March with promising news. On March 22, a US Food and Drug Administration (FDA) Advisory Committee (AdCom) overwhelmingly recommended an accelerated approval for Biogen’s ALS drug tofersen based on its impact on a surrogate biomarker. Less than a week later, the FDA reversed an earlier decision and elected to hold an AdCom for BrainStorm’s ALS stem cell therapy NurOwn.
The rise of ASOs
As the pharma world increasingly shifts focus toward precision medicine, interest in ASOs like QRL-201 has quickly gained steam. ASOs like QRL-201 work by binding to RNA in order to block or modify specific protein production.
Many researchers see ASOs as a strong treatment option for rare and ultra-rare diseases given their ability to target specific gene mutations. However, the increased investment in ASOs has also garnered concerns over their high potential costs, which could have far-reaching impacts on healthcare access and equity. Some researchers are advocating for more regulatory flexibility, which would allow sponsors to develop ASOs that can target a variety of similar diseases.
While the first patient in the Phase I QRL-201 study was dosed in the University of Montréal Hospital Centre (CHUM), Canada, QurAlis stated the safety study will also be conducted at other sites in the US, the UK, Belgium, the Netherlands, Italy, Germany, and Ireland. ANQUR has an estimated completion date of May 2025, according to the trial listing.