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Trump needs to prove he’s serious about science; PTC’s latest move is a jaw dropper

Endpoints assesses the big biopharma stories of the week, with a little added commentary on what they mean for the industry.

thumb down Two big steps back for the Trump administration

Elias Zerhouni

With Donald Trump in the White House, it’s always one step forward and two steps back for biopharma. So last week we had Scott Gottlieb’s nom for FDA commissioner (a big step forward) followed by this week’s news that Trump’s budget plans to slash NIH spending by close to $6 billion, while doubling industry’s fees to the FDA (you guessed it.)

Both budget items are nonstarters. Blasting the research budget at the NIH would gut the world’s most promising discovery activities. In an interview with Forbes’  Matthew Herper, former NIH chief Elias Zerhouni noted that if you cut the NIH’s budget by 20%, you’d essentially close the tap on new grants, a knockout blow to a large group of investigators which would be especially devastating for young scientists.

Coming as it does after years of flat budgeting, broken only recently by an increase last year, Trump is playing irresponsible politics. The idea of simply doubling industry fees is also a stick in the eye to drug developers.

thumb And the survey says…

Speaking of Scott Gottlieb.

We ran a snap poll over the weekend to get a better sense of what biopharma was thinking in regards to his nomination. And the response was unequivocal, with 87% giving the move their approval.

For Trump, it was a savvy choice. Rather than put in someone who would like to destroy the agency, he went with a pick who would set out to make it better. On Thursday night, I met with a group of biotech execs in the Bay Area, and the same support we saw in the poll permeated the room. The FDA is a large agency and while some elements in it have enthusiastically sought to accelerate approvals, it’s also clear that others on the inside could do better.

Several in the room, though, were openly skeptical that Gottlieb or any new commissioner can achieve a quick response in a federal agency like the FDA. One suggested a timeline of one to two years. But I’ll take that. In the scheme of things, a two-year evolution would be better than trying to shove overnight changes.

Steady and intelligent will beat rash and crash every time.

thumb down PTC’s shameful decision to buy deflazacort and partner with Marathon is a telling move

For the past two years, PTC Therapeutics has been stubbornly hammering on the FDA’s doors in search of its acceptance of ataluren for review as a new therapy for Duchenne muscular dystrophy. Not unreasonably the FDA has done what it could to bar entry, refusing to accept the inadequate application. This drug, after all, has now failed three straight studies, proving over and over that it’s a dud.

So now they’re taking a back door into the market. And they seem content to make a large number of enemies in the Duchenne community to capitalize on the gambit. Their deal to buy Marathon’s rights to deflazacort for $140 million — plus a stream of royalties —i demonstrates their contempt for patients.

If PTC really cared for patients, they’d never market deflazacort. That would let Duchenne families continue to buy the exact same old steroid from overseas sources for a still hefty price of about $1,000 a year — high for a generic. There’s no question about quality or safety. It’s the exact same thing.

Any price that PTC puts on deflazacort in the US higher than $1,000 a year will be too high. They’ll only join the ranks of companies branded as price-gouging villains. That’s why their stock plunged on Thursday. It was a reckless and stupid move by a company practiced in denying reality. But shamelessness is PTC’s hallmark. This is a company that sells a drug in Europe that it has shown repeatedly can’t help patients. And it’s looking to gain patient support for the same drug in the US.

Regulators, meanwhile, seem helpless to prevent any of this.

thumb Some serious biotech money is being raised for potentially breakthrough science

Ever since the 2008 financial crisis, the biotech venture scene has emerged as a very stable, growing field. A lineup of well known VC groups staffed by experienced insiders have been pumping billions of dollars into startups.

Now, all of a sudden, we’re starting to see a few new players jump into the game. Over the course of this week I profiled three new debuts — a remarkable number for biotech VCs. There was Biomatics, which has already made its mark in the past eight months, Pivotal bioVenture Partners and a new fund from Bill Maris, the ex-Google guru. The Pivotal money is coming from a Chinese group interested in making global bets.

What is extraordinary is that all three of these new players are keen to fund startups pursuing breakthrough science. And that is helping seed another big year for some astonishing plays. I illustrated that this week in my story on eGenesis, which is out to make xenografting a reality after decades as a scifi sideshow.

It’s a great time to be covering this business. Obviously quite a lot of this will never make it into medical practice. But some razor edge science is getting financed and we’ll all benefit from that.


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RAPS Regulatory Convergence 2017