Stealthy Biotech Revives Stalled Parkinson's Drug, Reveals Plans In Alzheimer's and ALS

Aug. 25, 2016

Denali CEO Ryan Watts

Denali Therapeutics, the stealthy South San Francisco biotech startup that raised $217 million last year to fight Alzheimer’s, Parkinson’s, and ALS, is finally lifting the curtain on its plans this morning. It is also announcing that it has raised another $130 million from investors in a private placement led by Baillie Gifford, a UK-based mutual fund.

“This is it,” says Ryan Watts, Denali’s chief executive and co-founder. He says the company is based around the discovery of genes that seem implicated in genetic disease. Twenty years ago, the identification of cancer genes set the stage for a dramatic change in how cancer is treated. “That’s where we are for neurodegeneration,” he says.

Watts is reviving a promising project he worked on at Genentech that seemed left for dead. It relates to LRRK2, a gene implicated in Parkinson’s disease. Sergey Brin famously carries a copy of it. 23andMe, the Google-backed genetics startup, has studied it. Pfizer, Merck, and Genentech were all working on drugs targeting the gene when it was discovered that those drugs seemed to cause lung problems in animals.

Denali has licensed the Genentech LRRK2 compounds. Watts says that he was convinced there was a dose at which the drug would block LRRK2 without causing the lung issues. But he also says that animal studies done by the Michael J. Fox Foundation, which created a unique collaboration between the three companies to study the lung side effect, seem to show that it does not cause any harm to the lungs anyway, at least in animals.

“This is a textbook example of what we exist to do: persevere to overcome issues that would otherwise set PD drug development back by years,” says Todd Sherer, chief executive of the Michael J. Fox Foundation. “We’re thrilled on behalf of everyone living with the disease that a highly promising target continues to move forward.”

That won’t be the first drug Denali tests, though. On August 22, Denali filed an application to start a clinical trial in Europe of healthy volunteers testing a new drug aimed at Alzheimer’s disease and Amyotrophic Lateral Sclerosis (ALS). The pill targets an enzyme called the Receptor-interacting protein 1 (RIP1) kinase. Work by Harvard researcher Junying Yuan has indicated that RIP1 has a key role in inflammation in glial cells, a type of brain cell.

http://www.forbes.com/sites/matthewherper/2016/08/25/stealthy-biotech-revives-stalled-parkinsons-drug-reveals-plans-in-alzheimers-and-als/#3aaa49c0d631