Drug Development

The biopharma week in review: Thumbs Up, Thumbs Down

My weekly take on the top news of the week to hit the biopharma world. We might be in the middle of a long, hot summer, but there’s been plenty of news to liven things up. — John Carroll

thumb BioMarin turns in jaw-dropping gene therapy results

Kudos to BioMarin, which just handed in some startling good efficacy data on their gene therapy for hemophilia A. We’re still talking small numbers, but as proof-of-concept trials go, it’s an eye opener. Several of the patients involved responded very well to the once-and-done approach to this rare disease. They are clearly in the lead in hemophilia A gene therapies, but look for lots of activity from rivals interested in moving ahead on their own. Gene therapy could transform lives. It’s great to see it.

thumb Eli Lilly turns a corner, and a new CEO takes the helm

True, I haven’t been a big fan of Eli Lilly in the past. It is rigidly conservative in its R&D ops and often well behind rivals when it brings a new drug to the market. But after a miserable record on drug development, the company has some bright prospects at the FDA. Their top late-stage drugs are as close as you get to derisked; Lilly may be slow but it does not mess around when it comes to designing and executing studies. And the new CEO, David Ricks, looks eager to maintain some old traditions while loosening the reins on the BD team. Lechleiter may not be going out on top, but he’s moved the company past the bottom. Lilly, a top 10 R&D player, is now looking up. That deserves some credit.

thumb down Hey, TauRx: You experiment with Alzheimer’s drugs, not trial designs

You can excuse any company for wanting to move the goal posts in order to get a win in Phase III. TauRx shifted them down to the red zone, then called a play and declared a touchdown. But their failed Phase III effort on Alzheimer’s doesn’t look any better after a subgroup analysis persuaded them to make a last minute switch up to focus on a small bunch of patients. New Phase III? Maybe, but it’s going to take a brave set of investors to put up the money on a long shot like this.

thumb down thumb Blockbusters still sizzle, but the R&D productivity pace slows

The pace of new drug approvals at the FDA is off to a slow start in 2016 (boo), but the short list of successful new therapies includes some remarkable blockbusters-to-be (yay). Roche has already made a big mark. And the FDA’s commitment to follow through on the recent breakthrough drug designation with accelerated approvals is on full display now. R&D success is clearly measured by potential revenue for new drugs. Biopharma isn’t messing around any more.

thumb Before cell therapies arrive on the market, the pioneers look to third-gen tech

We’ve clearly been in a mostly bullish mode this week, judging by this week’s largely positive set of thumbs up. So let’s keep it up. We’re adding new T cell tech to the package of interesting stories for the week. There was the way cool new science being used to build bulletproof CARs, as well as Kite’s move to get in line with some off-the-shelf remedies. There’s a long way to go in the clinic, but this kind of scientific progress bodes well for a booming field.

thumb down Biotech IPOs in general, Kadmon in particular

Looking back on the Q2 slate of biotech IPOs? Well, don’t, unless you’re a glutton for punishment. All but 2 of 12 of the current class of newly public biotechs are trading at a lower price than what the stock was issued at. And Kadmon didn’t help the 2016 stats at all after braving the sneers and tears about its founder Sam Waksal’s sordid past and pushing through an IPO this week. Ex-con Waksal had to leave ahead of the IPO, grabbing a multimillion-dollar package on the way out. We’ll see how well investors do as it makes its way through Phase II work.

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John Carroll, Editor and Co-Founder

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