Michael Kauffman (CEO, Karyopharm)

Karyopharm bags an up­set win at the FDA as reg­u­la­tors OK myelo­ma drug de­spite a host of ob­jec­tions

Some­body high up at the FDA must re­al­ly like Karyopharm $KP­TI.

On Wednes­day the biotech an­nounced that the agency had stamped an OK on their ap­pli­ca­tion to mar­ket se­linex­or for mul­ti­ple myelo­ma, cut­ting ahead of the late-stage re­sults — though in an in­trigu­ing note the agency said they had a look at ad­di­tion­al re­sults from an on­go­ing study.

The ap­proval came de­spite a sol­id ma­jor­i­ty of ex­perts on an out­side pan­el who vot­ed against a quick OK, pre­fer­ring to see the Phase III da­ta first. And it ar­rives de­spite an in­ter­nal re­view com­plete with a host of ob­jec­tions to the da­ta de­liv­ered to back the pitch.

The drug will be sold as Xpovio at a whole­sale price of $22,000 a month.

Pre­sent­ed with the sur­prise turn­around, in­vestors bid up the shares by 36% as a copy of the la­bel spread on Twit­ter ahead of the re­lease.

FDA rep­re­sen­ta­tives were po­lite about it, but out­lined mul­ti­ple prob­lems with the da­ta that Karyopharm pre­sent­ed: Miss­ing da­ta due to dropouts, un­ac­cept­able re­al world ev­i­dence, an ab­sence of ev­i­dence of pos­i­tive sin­gle agent ac­tiv­i­ty (there are neg­a­tive re­sults), most­ly par­tial re­spons­es and much, much more — all in­for­ma­tion that the biotech failed to spot­light in the lead-up to the NDA. In one study the FDA cit­ed, the over­all sur­vival rate was worse in the se­linex­or arm.

David Har­ring­ton [Dana Far­ber]

Then there was the tox­i­c­i­ty pro­file, with a high fre­quen­cy of treat­ment emer­gent ad­verse events among pa­tients tak­ing the drug. The agency cit­ed a 94% rate of grade 3 or grade 4 ad­verse event. 10 deaths were due to a fa­tal ad­verse event in the main sin­gle arm study used for the ac­cel­er­at­ed ap­proval. And 9 in 10 pa­tients re­quired a dose mod­i­fi­ca­tion, with a ma­jor­i­ty re­quir­ing 2 mod­i­fi­ca­tions.

That case per­suad­ed 8 of 13 ex­perts to vote against an ear­ly ap­proval. But the 5 votes in fa­vor il­lus­trat­ed the agency’s ap­petite for new drugs for pa­tients who have run out of op­tions.

Dana Far­ber’s David Har­ring­ton joined the mi­nor­i­ty in fa­vor of pro­vid­ing an ac­cel­er­at­ed ap­proval. “The da­ta are not con­clu­sive in ei­ther di­rec­tion,” he said at the time, but…”I think we do pa­tients some po­ten­tial ben­e­fit if this is used con­struc­tive­ly.”

They’ll have that now. The drug is re­served for the last line of de­fense af­ter at least 4 pri­or drugs.

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.

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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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.

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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.

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