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.

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.

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.

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.

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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.