QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial

Cambridge, Massachusetts-based QurAlis has dosed the first patient with  the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment.

Stathmin-2 (STMN2), also known as superior cervical ganglia neural specific 10 (SCG10), belongs to the stathmin protein family involved in the regulation and dynamics of microtubules. Predominantly expressed in neurons, STMN2 serves key functions in neuronal development, axonal growth and regeneration.

Recent research has revealed the significance of STMN2 in neurodegenerative diseases, especially ALS and frontotemporal dementia (FTD). Researchers have linked the pathogenesis of those conditions to the loss of STMN2 function.

“In mice, STMN2 deletion causes axonal degeneration and loss of muscle innervation, w…

Read more
  • 0

QurAlis selects development candidate for ALS

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…

Read more
  • 0