Privately-held biotech QurAlis plans to begin clinical development for the first-in-class molecule QRL-201 for amyotrophic lateral sclerosis (ALS) in the second half of 2022.
QRL-201 is now the subject of IND-enabling studies.
The drug candidate targets the restoration of STATHMIN-2 (STMN2) expression in ALS. STMN2 is a protein encoded by the STMN2 gene that is involved in neural repair. ALS is often associated with decreased expression of STMN2.
In ALS patient-derived motor neuron disease models with TDP43 pathology, QRL-201 restored STMN2 loss of function.
A 2008 study in Current Opinion in Neurology found that TDP43 is involved in ALS and frontotemporal dementia (FTD) pathogenesis.
TDP43 pathology is also present in some Alzheimer’s and Parkinson’s disease patients.
“QRL-201 could potentially benefit ALS patients who have a loss of STMN2 due to TDP43 pathology, which could, in turn, slow disease progression,” explained Kasper Roet…