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

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.


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.


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.


  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.


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.


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.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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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.”

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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.

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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.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.