Bio­Marin scores a quick FDA OK for its lat­est rare dis­ease en­try — flagged at $702K WAC

Jean Jacques Bi­en­aimé

The FDA has pro­vid­ed a wide­ly ex­pect­ed green light for Bio­Marin’s Brineu­ra (cer­liponase al­fa), set­ting up the roll­out for one of the most ex­pen­sive new drugs to hit the mar­ket.

Bio­Marin $BM­RN at­tract­ed at­ten­tion for its lat­est rare dis­ease drug for its ef­fi­ca­cy in treat­ing Neu­ronal Ceroid Lipo­fus­ci­nosis Type 2 dis­ease (CLN2), a form of Bat­ten dis­ease. The dis­ease pro­gress­es rapid­ly, with re­cur­rent seizures and vi­sion loss oc­cur­ring as most vic­tims are quick­ly left un­able to walk by the age of six be­fore even­tu­al­ly killing them. And just days ago the Eu­ro­pean Med­i­cines Agency’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use pro­vid­ed their thumbs up as well.

The drug was test­ed in on­ly 22 pa­tients in an ab­bre­vi­at­ed study, with a com­par­i­son on the nat­ur­al his­to­ry of sev­er­al dozen oth­ers. And that’s a mod­el that will help de­fine more such stud­ies in the fu­ture.

Bio­Marin CEO JJ Bi­en­ame not­ed in a call with an­a­lysts Thurs­day evening  that the ap­proval came “af­ter just 3 years and 8 months in the clin­ic,” with max­i­mum flex­i­bil­i­ty from the FDA for an ul­tra rare dis­ease based on one small clin­i­cal study, with a his­tor­i­cal com­par­i­son, and no ex­pert pan­el re­view. And they won a pri­or­i­ty re­view vouch­er, which could eas­i­ly be worth $125 mil­lion or more, based on re­cent sales.

The drug is de­signed to slow loss of am­bu­la­tion among pa­tients, as the da­ta in­di­cate.

The cost: $27,000 every two weeks, or $702,000 list; an av­er­age $486,000 per year af­ter Med­ic­aid dis­counts are fac­tored. That will be the sec­ond most ex­pen­sive ther­a­py avail­able in the US, right af­ter Hori­zon’s Rav­ic­ti, at $793,632 whole­sale, ac­cord­ing to a Rein­sur­ance Group of Amer­i­ca list based on WAC prices. Spin­raza from Bio­gen is now be­ing sold for $750,000 for the first year, which then drops to half that price.

Piper Jaf­fray an­a­lyst Joshua Schim­mer has al­ready not­ed that the com­pa­ny warned that its list price on this drug will fall on the high side, putting it on our lat­est list of the world’s 10 prici­est drugs, which you can see here. That list is dom­i­nat­ed by or­phan drugs de­vel­oped for small pa­tient pop­u­la­tions.

Bio­Marin’s stock, which had es­sen­tial­ly baked this ap­proval in­to the price, was up on­ly slight­ly at the end of the day.

“The FDA is com­mit­ted to ap­prov­ing new and in­no­v­a­tive ther­a­pies for pa­tients with rare dis­eases, par­tic­u­lar­ly where there are no ap­proved treat­ment op­tions,” said Julie Beitz, di­rec­tor of the Of­fice of Drug Eval­u­a­tion III in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “Ap­prov­ing the first drug for the treat­ment of this form of Bat­ten dis­ease is an im­por­tant ad­vance for pa­tients suf­fer­ing with this con­di­tion.”

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.

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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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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