Drug Development, Results

As a new study spotlights a growing role for LRRK2 in Parkinson’s, Denali clears an early trial hurdle

Hard on the heels of new research that points to a much wider role for LRRK2 in Parkinson’s disease than had earlier been believed, investigators at Denali say they were able to nail down some hard — though still very early — clinical data to back up their lead effort in the field.

The biotech, run by a crew of ex-Genentech researchers out to blaze some new trails in the disaster-prone neurodegeneration field, has focused heavily on the genetic triggers believed to play a role in Parkinson’s and Alzheimer’s. In a Phase I study, they outlined evidence that DNL201, a small molecule inhibitor of leucine-rich repeat kinase 2, hit the biomarkers they were aiming at as they zero in on the ideal dose for more advanced human studies. And that builds on earlier work demonstrating their drug’s ability to tamp down on the protein in healthy volunteers.

Carole Ho

“We conclude from this clinical trial that DNL201 was able to achieve the targeted level of LRRK2 inhibition at doses that were safe and well tolerated,” said Carole Ho, the chief medical officer, in a statement.

That was good enough for a 7% pop on the stock price $DNLI in pre-market trading Thursday.

Denali didn’t ignore the recent work highlighting LRRK2’s expanding role as a likely culprit for Parkinson’s. CEO Ryan Watt highlighted “mounting evidence supporting a role of LRRK2 inhibition in the broader sporadic Parkinson’s disease population, in addition to Parkinson’s disease genetically associated with a LRRK2 mutation.”

J. Timothy Greenamyre

Dr. J. Timothy Greenamyre, chief of the Movement Disorders Division at the University of Pittsburgh Medical Center in Pennsylvania published a study just days ago asserting that a variety of triggers set LRRK2 on a destructive course in Parkinson’s patients.

“This discovery is extremely consequential for Parkinson’s disease because it suggests that therapies currently being developed for a small group of patients may benefit everybody with the disease,” he said.

You can be sure that news made the rounds at Denali, and everywhere else in the Parkinson’s community, which accounts for millions of patients worldwide.

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Sr. Manager, Regulatory Affairs, CMC
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