According to a story from PR Newswire, the QurAlis Corporation, a clinical stage biotech company, recently announced the completion of the review of the Clinical Trial Regulation (CTR) in the European Union. The company has received notices of acceptance from the participating countries for QRL-201, an investigational therapy being developed as a treatment for amyotrophic lateral sclerosis (ALS), a rare disease. QurAlis is focused on developing precision breakthrough treatments for amyotrophic lateral sclerosis and other neurodegenerative diseases.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.
About QRL-201
QRL-201 is a precision medicine with a mechanism of action designed to restore the expression of STMN2 in people living with the disease. This is a protein that plays a vital role in axonal stability and neural repair. Nearly all people with ALS see their expression of this protein greatly diminish. In fact, it is the most consistently decreased gene across ALS patient data sets.
This is the second authorization that the company has received regarding the development of this treatment, having previously earned a similar authorization in Canada. QurAlis has completed enrollment and dosed its first patient in that country. The trial is anticipated to include 64 participants across Canada, the EU, the US and the UK. The company anticipates the start of its phase 1 trial in Europe to begin in late 2023.
