Denali Therapeutics Inc (NASDAQ:DNLI)

Hope just got a little brighter for those who have Parkinson’s disease. Researchers indicate a signaling protein known as leucine-rich repeat kinase (LRRK2) is a key factor influencing the development of Parkinson’s disease.

High levels of LRRK2 activation are associated with malfunction of lysosomes, the “garbage collectors” in our brain cells. Some people have an overactive variant of the gene for this molecule.
And they also have an increased risk for developing this neurodegenerative disease.

DNLI) is making great progress in attacking this particular target as a way to treat Parkinson’s. The company has two different LRRK2-blocking drugs in clinical trials and recently updated investors on its progress.

One of the company’s therapies, DNL201, showed huge potential in patients by reducing biomarkers for LRRK2 by over 50% while showing no serious safety issues.

Denali’s second Parkinson’s drug, DNL151, showed early results in over 150 patients of a similar makeup — greater than 50% change — and dose-dependent reductions in lysosomal biomarkers as measured via urinalysis.

The company plans to move both drugs into Phase 2/3 clinical trials by the middle of 2020.

The market loved this news when it was released last week, driving DNLI shares up by some 30% in a single trading session.
But I suspect there’s much more to come beyond our current 41% gain in this brain health biotech. The company expects data to read out from its ALS trials by mid-2020, and its Alzheimer’s clinical trial is now fully enrolled.

Positive data from either of these programs would be a huge mover for DNLI shares, and the company has the potential to be worth thousands of percent more over time as it executes on its clinical pipeline.

What I like about Denali’s Alzheimer’s program is its unique approach to dealing with the disease.

We’ve seen huge high-profile drug trial failures in the Alzheimer’s space. Biogen Inc. recently tried to salvage a program that was discontinued in Phase 3 with an FDA submission for high-dose patients.

But high doses of Biogen’s drugs also meant high side effects.
Biogen’s drug, as well as many others, is based on what was the mainstream theory of this disease and repeated failures strongly suggested the theory was wrong.

Denali’s approach, however, is to attack inflammatory pathways in brain cells that lead to cell death. This concept may prove to have far more value, and if the company demonstrates this via clinical data, its own market value will rise.