Denali Therapeutics has started dosing Parkinson’s disease patients with its therapeutic candidate for slowing the progression of disease, in a Phase 1b clinical study.
Denali’s Chief Medical Officer, Carole Ho, in the press release announcement, was referred to as saying that the study will evaluate the therapeutic candidate DNL201 on Parkinson’s patients, based on the positive outcome of the company’s Phase 1 study in 122 healthy volunteers.
She said: “This study will provide additional important safety and biomarker data in patients to support rational dose selection. The results from this study will inform decisions on further clinical testing of DNL201, including potential registrational trials.”
DNL201, Denali’s lead LRRK2 therapeutic candidate, is a small molecule inhibitor of leucine-rich repeat kinase 2 (LRRK2) – a regulator of lysosomal function impaired in Parkinson’s disease and may be restored by LRRK2 inhibition. Inhibition of LRRK2 activity may potentially slow the progression of disease in patients with a genetic LRRK2 mutation as well as in patients with sporadic Parkinson’s disease, as explained by Denali’s press release.
The company also noted that mutations in the LRRK2 gene are among the most frequent genetic causes of Parkinson’s disease and a major driver of lysosomal dysfunction, which contributes to the formation of Lewy body protein aggregates and neurodegeneration.