Denali Therapeutics Announces Broad Pipeline Progress Including Positive Results From Its LRRK2 Program for Parkinson’s Disease

• LRRK2 inhibitor DNL201 Phase 1b demonstrated high levels of target and pathway engagement and improvement of lysosomal biomarkers in patients with Parkinson’s disease 
• LRRK2 inhibitor DNL151 Phase 1 demonstrated high levels of target and pathway engagement and modulation of lysosomal biomarkers in healthy volunteers and continues in an expanded Phase 1b study in patients with Parkinson’s disease
• IND submitted for DNL310 (ETV:IDS) for Hunter syndrome, Denali’s first clinical submission for a large molecule therapeutic enabled by its Transport Vehicle platform technology
• CTA approved for DNL343, a small molecule activator of EIF2B for ALS and other neurodegenerative diseases
• RIPK1 inhibitor DNL747 Phase 1b trials in Alzheimer’s and ALS fully enrolled and open label extension in ALS ongoing with data readouts on track for mid-2020

SOUTH SAN FRANCISCO, Calif., Jan. 14, 2020 (GLOBE NEWSWIRE) -- 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, today announced the results of its Phase 1b clinical trial of LRRK2 inhibitor DNL201 in patients with Parkinson’s disease and its Phase 1 clinical trial of LRRK2 inhibitor DNL151 in healthy volunteers.

Denali also announced that it submitted an IND for DNL310 for Hunter syndrome, which is the company’s first submission with a biotherapeutic product candidate engineered to cross the BBB enabled by the Transport Vehicle technology. Furthermore, a CTA for a Phase 1 first-in-human healthy volunteer study of EIF2B activator DNL343, intended for the treatment of ALS and other neurodegenerative diseases, has been approved. This is Denali’s third small molecule program engineered to cross the BBB to advance to clinical testing.

“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,” said Ryan Watts, Ph.D., CEO. “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.”