Today Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (‘BBB’) for neurodegenerative diseases, announced broad pipeline progress including positive results from its LRRK2 program for Parkinson’s disease.

Ryan Watts, Ph.D., the CEO of Denali Therapeutics said,  

“We are pleased with the continued progress across our pipeline, including a significant step forward for our LRRK2 program with encouraging clinical data in patients and healthy subjects for two molecules,” 

Denali reported that he LRRK inhibitor DNL201 Phase 1b demonstrated high levels of target and pathway engagement and improvement of lysosomal biomarkers in patients with Parkinson’s disease, as well as the inhibitor DNL151 Phase 1 in healthy volunteers, that continues in an expanded Phase 1b study in patients with Parkinson’s disease.

Watts added:

“We are also enthusiastic about advancing two new molecules toward the clinic, DNL343 for ALS and other neurodegenerative diseases and DNL310 for Hunter syndrome, which is also expected to generate proof-of-concept in humans for our Transport Vehicle blood-brain barrier delivery platform.”

The biopharmaceutical company further announced that it submitted an IND for DNL310 for Hunter syndrome, which is Denali’s first clinical submission for a large molecule therapeutic enabled by its Transport Vehicle platform technology.

Additionally, a CTA for a Phase 1 first-in-human healthy volunteer study of EIF2B activator DNL343 that is intended for the treatment of ALS and other neurodegenerative diseases, has been accepted.

Based in San Francisco, the biopharmaceutical company Denali Therapeutics Inc. is  pursuing new treatments by assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement.