How Sean Parker’s biotech education is shaking up science

Sean Parker, who made his billions on the likes of Napster and Facebook, has spent the last few years focused on cancer immunotherapy, bringing with him loads of money, some self-taught expertise, and an infusion of glamor to the often staid world of academy.

Speaking to STAT in his most extensive interview on the topic, Parker explained how he became fascinated with immuno-oncology and why he put $250 million of his own fortune into the Parker Institute for Cancer Immunotherapy. It’s a story that spans from Sting’s villa in Tuscany to a dream home next door to the Playboy Mansion, with a cameo from an 8-foot ice sculpture of a Nobel Prize sweating it out in the Texas heat.

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As for PICI, the ambitious goal is to take on some of the most vexing hurdles in cancer research, and scientists say Parker’s work has already moved the needle. Now, with that initial $250 million mostly spoken for, the institute is increasingly looking to other sources of funding, including some outside-the-box approaches.

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So what if Trump is serious about 'most favored nation'?

He made it sound so easy. Last week, President Trump told reporters that his White House is “working on a favored nations law where we pay whatever the lowest nation’s price is,” something he promised to take care of through an executive order.

But as STAT’s Nicholas Florko reports, Trump’s seemingly offhand remark put a simplistic spin on an infinitely complex issue, and it’s leaving experts with quite a few more questions than answers.

Among them is whether that’s even possible via executive order. Another is whether Trump knows what he’s talking about. And, assuming the answers to those questions, there’s still the possibility that such a policy would “blow up everything that is in drug pricing today,” as Gerard Anderson, a professor at Johns Hopkins’ Bloomberg School of Public Health, put it.

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Gene therapy has already changed medicine. Now it might get some competition

In the U.S., there are two approved gene therapies. Each set a record for the cost of a single medicine, and each manufacturer had a compelling defense for that cost: In one case, the alternative is blindness; in the other, it’s certain death.

But what about when the alternative is simply a different gene therapy? That could soon be the reality for patients with hemophilia A, as three one-time treatments are moving toward the market.

At a medical conference over the weekend, BioMarin said it’s on track to file for FDA approval by the end of this year. Spark Therapeutics is about a year behind with a gene therapy of its own. And Sangamo Therapeutics, partnered with Pfizer, presented promising early data suggesting it could compete with the other two in about two years.

If each company succeeds, hemophilia A will become the first competitive market for gene therapy, which — if standard economic principles have any application in the drug industry — should have an effect on pricing.

Just how understanding can the FDA be?

Intra-Cellular Therapies got good news and bad news yesterday. In one study, its drug significantly outperformed placebo in improving the symptoms of bipolar depression. In a second study, the treatment was basically indistinguishable from placebo.

The company, which blamed the failure of the second study on an uncommonly high placebo effect, still plans to make its case to the FDA. And there’s precedent for regulatory wiggle room: Johnson & Johnson won FDA approval for the depression treatment esketamine despite a single failed trial among a handful of positive ones.

But analysts say the most likely scenario is that Intra-Cellular will have to hope for positive results of a third study that will read out next year. At that point, the company can point to the entirety of its dataset in hopes of convincing the FDA. It worked for J&J, but when Alkermes took the same approach, things didn’t go so well.

How Sean Parker’s biotech education is shaking up science

So what if Trump is serious about 'most favored nation'?

Gene therapy has already changed medicine. Now it might get some competition

Just how understanding can the FDA be?

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