- Phase 1b study includes Parkinson’s disease patients with and without a genetic LRRK2 mutation
SOUTH SAN FRANCISCO, CA, USA I December 10, 2018 I Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, today announced initiation of dosing in a Phase 1b clinical study of DNL201 in patients with Parkinson’s disease.
“Based on the positive outcome of our Phase 1 study in 122 healthy volunteer subjects, we are excited to evaluate DNL201 in Parkinson’s disease patients,” said Carole Ho, M.D., Chief Medical Officer of Denali. “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). LRRK2 is a regulator of lysosomal function, which is 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.
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.
About the DNL201 Phase 1b study
This study (NCT03710707) is a 28-day, randomized, placebo controlled Phase 1b clinical trial in patients with mild to moderate Parkinson’s disease, with and without genetic LRRK2 mutations. Its purpose is to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and target and pathway engagement biomarkers in multiple oral doses of DNL201. Exploratory endpoints include certain clinical endpoints. The planned 30 patients in the study will be randomized to receive either a low dose of DNL201, a high dose of DNL201, or placebo.
Data readout from this study is expected during Q4 2019. Further details are available at ClinicalTrials.gov.
About Denali
Denali is a biopharmaceutical company developing a broad portfolio of therapeutic candidates for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the blood-brain barrier and guiding development with biomarker monitoring to demonstrate target engagement and select patients. Denali is based in South San Francisco. For additional information, please visit www.denalitherapeutics.com.
SOURCE: Denali Therapeutics
Post Views: 35
- Phase 1b study includes Parkinson’s disease patients with and without a genetic LRRK2 mutation
SOUTH SAN FRANCISCO, CA, USA I December 10, 2018 I Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, today announced initiation of dosing in a Phase 1b clinical study of DNL201 in patients with Parkinson’s disease.
“Based on the positive outcome of our Phase 1 study in 122 healthy volunteer subjects, we are excited to evaluate DNL201 in Parkinson’s disease patients,” said Carole Ho, M.D., Chief Medical Officer of Denali. “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). LRRK2 is a regulator of lysosomal function, which is 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.
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.
About the DNL201 Phase 1b study
This study (NCT03710707) is a 28-day, randomized, placebo controlled Phase 1b clinical trial in patients with mild to moderate Parkinson’s disease, with and without genetic LRRK2 mutations. Its purpose is to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and target and pathway engagement biomarkers in multiple oral doses of DNL201. Exploratory endpoints include certain clinical endpoints. The planned 30 patients in the study will be randomized to receive either a low dose of DNL201, a high dose of DNL201, or placebo.
Data readout from this study is expected during Q4 2019. Further details are available at ClinicalTrials.gov.
About Denali
Denali is a biopharmaceutical company developing a broad portfolio of therapeutic candidates for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the blood-brain barrier and guiding development with biomarker monitoring to demonstrate target engagement and select patients. Denali is based in South San Francisco. For additional information, please visit www.denalitherapeutics.com.
SOURCE: Denali Therapeutics
Post Views: 35
