Ver­tex CEO to UK Prime Min­is­ter: You’re de­valu­ing lives, pass­ing up a great of­fer and threat­en­ing the coun­try’s biotech in­dus­try

Ver­tex has scored big in the US with its new drugs for cys­tic fi­bro­sis, but the biotech still has to slug it out for every inch of mar­ket ter­ri­to­ry in Eu­rope — where its price for Orkam­bi is of­ten seen as way too high for the val­ue it of­fers. And af­ter get­ting snubbed in the UK last week — again — af­ter a long-run­ning cam­paign to win a re­im­burse­ment deal from the NHS, CEO Jef­frey Lei­den has de­cid­ed to pub­licly chas­tise British Prime Min­is­ter There­sa May for the stand­off.

This lat­est blast oc­curred af­ter UK health of­fi­cials de­cid­ed last week that the price Ver­tex is de­mand­ing for Orkam­bi is “un­sup­port­able,” de­spite a pro-re­im­burse­ment cam­paign backed by thou­sands of pa­tients with the dis­ease.

That de­ci­sion, Lei­den tells May, demon­strates how the gov­ern­ment puts “a low­er val­ue on the life of a CF pa­tient than oth­er coun­tries around the world.” He as­serts that the UK’s de­ci­sion amounts to “shut­ting the door” on a new gen­er­a­tion of pre­ci­sion med­i­cines. And the UK, he adds, is pass­ing up a great of­fer.

We have pro­vid­ed the most in­no­v­a­tive of­fer in the world to the NHS, yet have seen no re­cep­tiv­i­ty from NHS Eng­land. In one of the most pros­per­ous coun­tries in the world, NHS Eng­land’s lev­el of in­ter­est in our of­fer rep­re­sents a lack of com­mit­ment to chil­dren and young peo­ple with this dev­as­tat­ing dis­ease….

For the UK to de­vel­op its sci­ence and in­no­va­tion ecosys­tem post-Brex­it, these pol­i­cy state­ments and plans must trans­late to im­proved pa­tient ac­cess to in­no­v­a­tive ther­a­pies, such as the new gen­er­a­tion of pre­ci­sion med­i­cines which work on dif­fer­ent geno­types of rare dis­eases. This re­quires ap­praisal process­es to keep pace with the sci­ence; cur­rent process­es have sig­nif­i­cant lim­i­ta­tions in how they cap­ture and val­ue the full med­ical, so­ci­etal, eco­nom­ic and in­no­va­tion ben­e­fits of such pre­ci­sion med­i­cines. With­out this, any fu­ture biotech in­vest­ment in the UK is at sig­nif­i­cant risk.

Ver­tex took the gloves off with re­cal­ci­trant Eu­ro­pean sin­gle-pay­er groups long ago. In France, the biotech re­cent­ly goad­ed the lo­cal pa­tient pop­u­la­tion with its de­ci­sion to scrap any plans to re­cruit French pa­tients in­to a study, in light of the gov­ern­ment’s re­fusal to cov­er their drug at what they felt was an ap­pro­pri­ate amount. 

We’ll see if lec­tur­ing the British prime min­is­ter works any bet­ter.

Ver­tex Let­ter to Prime Min­is­ter 6 Ju­ly 2018 by Am­ber on Scribd


Im­age: Jef­frey Lei­den. VER­TEX

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.

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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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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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