ryan watts is ready to catch (or, create) the next wave in drugs for neurodegenerative diseases. (Denali therapeutics)

A cadre of Genentech higher-ups left last year, with about $217 million in hand, to form a new company tackling neurodegenerative disease: Denali Therapeutics. The company just raised a $130 million Series B round, and has applied to launch its first set of clinical trials in Europe. Its pipeline of drugs hinges on what it has dubbed “degenogenes” — the genes that have been linked to causing diseases like Alzheimer’s, Parkinson’s, and amyotrophic lateral sclerosis.

STAT chatted with CEO Ryan Watts about Denali’s plans: 

Do we know enough about the basic biology of neurodegenerative disease to come up with legitimate therapeutics in this space? 

I want to draw on our previous lives: A lot of us have experience in oncology. I remember the dark days — about two decades ago — when a lot of the cancer drugs were failing. But then a fundamental understanding of oncogenes came about, and that laid the foundation for a first, second, and third new wave of cancer drugs. That’s where we are with neurodegenerative disease, because we’ve now discovered what we’re calling “degenogenes” that lead to diseases like Alzheimer’s and Parkinson’s.  

So how do you decide which “degenogenes” to pursue to develop drugs? 

I think you have to ask yourself, what’s a druggable gene? APOE has been known for 30 years, but no one has really figured out how to drug it — same with LRRC2. I think one of the reasons we’ve failed in Alzheimer’s is because we’ve treated it like it’s a generalized disease. The more we understand about genetic subpopulations, we can better test our drugs. Here’s another lesson from oncology: Herceptin failed at first in a phase 2 study, but went on to work incredibly effectively in HER2 positive breast cancer. The same could apply for Alzheimer’s.

So, tell me Denali’s origin story. You worked on one of Denali’s products when you were at Genentech. Did you start up Denali to continue working on a shelved compound?

Our origin story’s come and gone. After 15 months in existence, we’re past that. We have one partnership with Genentech now, but all of our other programs are essentially Denali-originated, and from various other partnerships. I was at Genentech 11 years. But the reality is: We were looking to have a singular focus in neurodegeneration. It’s a very high risk area, but with some rigor we think we can find success. 

Heather Bresch's defense of Mylan's price hikes included multiple sentences that began with "as a mother." (CNBC)

Mylan CEO Heather Bresch went on CNBC yesterday, looking to tamp down the increasingly loud outrage over her company’s repeated price increases for EpiPen. Her defense amounted to a lot of blame-shifting, some unexpected (and perhaps not strategically savvy) pricing transparency, and a few appeals to parenthood.

But the key questions — like, why is EpiPen so expensive — remain unanswered. Read more. 

And for a look at how controversies like this one go viral — and which drug makers may be next on the hot seat, read this.

Medivation, you probably heard, has 14 billion reasons to become part of Pfizer. And now comes the first high-profile defection, as Amy Peterson, Medivation’s vice president of clinical development, has joined Chinese-American biotech BeiGene as chief medical officer. The company, at work on treatments for cancer, raised more than $150 million in a US IPO earlier this year.

Pfizer’s acquisition of Medivation, announced Monday, has many insiders wondering what’s next for others in the upper echelons of Medivation. We're especially interested in the path ahead for founder and CEO Dr. David Hung, who is regarded as a canny dealmaker.

Back in 2012, Amgen paid $315 million for a company called KAI Pharmaceuticals and its treatment for dialysis patients who develop dangerously high levels of parathyroid hormone. In three late-stage studies, the drug did its job, and Amgen submitted it for FDA approval in August of last year.

Then the FDA rejected it, and now Amgen's not saying why. In a statement released this week, the company disclosed only that it's reviewing the agency's rejection letter and "anticipate(s) a post-action meeting with the FDA later this year."

Companies aren't required to be specific about these things, and the FDA, as a rule, doesn't comment on them. But drug makers as big as Amgen usually provide some detail to let investors know whether it's a manufacturing issue, a request for more data — whatever. But Amgen's staying silent in what Adam Feuerstein called "an egregious abdication of corporate transparency."

• Theranos is appealing the federal sanctions that have blocked it from conducting tests at a California lab. (Press release)
• Short seller Carson Block questioned the security of St. Jude Medical's cardiac implants, claiming the devices can be easily hacked and should be recalled. (Bloomberg)
• Novartis' new multiple sclerosis treatment succeeded in a late-stage trial. (Press release)