Med­i­cines Co., Al­ny­lam pre­pare to throw the dice on a late-stage ef­fort to leapfrog PC­SK9 gi­ants

While Am­gen and its ri­vals at Re­gen­eron/Sanofi strug­gle to es­tab­lish a mar­ket for their pi­o­neer­ing PC­SK9 ther­a­pies — while grap­pling over a nasty law­suit over the IP — Al­ny­lam $AL­NY and The Med­i­cines Com­pa­ny $MD­CO are hatch­ing plans for a come-from-be­hind at­tempt to leapfrog the squab­bling fron­trun­ners.

Clive Mean­well, The Med­i­cines Com­pa­ny

This morn­ing the com­pa­nies an­nounced that the FDA has signed off on their Phase III pro­gram, which will en­roll a to­tal of 3,000 sub­jects with ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease and fa­mil­ial hy­per­c­ho­les­terolemia to test the abil­i­ty of in­clisir­an to slash LDL over 18 months. And they aren’t wait­ing for the re­sults to be­gin their car­dio out­comes study.

Re­searchers plan to sweep up 14,000 pa­tients for the out­comes study, look­ing to see if this new drug can im­press pay­ers with da­ta on a com­pos­ite of coro­nary heart dis­ease death, non-fa­tal my­ocar­dial in­farc­tion and fa­tal and non-fa­tal is­chemic stroke.

That’s a tricky propo­si­tion, though.

Am­gen hit marks for sev­er­al out­comes with their car­dio fol­low-up tri­al ear­li­er this year, but was slapped with a back­lash of skep­ti­cism from an­a­lysts and pay­ers who want­ed to see bet­ter da­ta to jus­ti­fy the price of the drug — par­tic­u­lar­ly on re­duc­ing mor­tal­i­ty. Am­gen’s mon­ey-back guar­an­tee for pa­tients on their drug who go on to ex­pe­ri­ence a heart at­tack failed to win over the doubt­ful, who were quick to do the math on costs and sav­ings.

Learn­ing from the pi­o­neers, The Med­i­cines Com­pa­ny and Al­ny­lam — which de­vel­oped the RNA drug — want to nail down the right da­ta and then an­gle in with a drug that may on­ly have to be giv­en two or three times each year. Af­ter that, the whole glob­al bat­tle for mar­ket share could get down to price.

Last fall re­searchers in the Phase II study re­port­ed that a sin­gle 300 mg in­jec­tion of their RNAi drug — now dubbed in­clisir­an — reg­is­tered a mean 43% re­duc­tion in bad LDL cho­les­terol af­ter 180 days, trig­ger­ing some ex­cit­ed buzz among the an­a­lysts fol­low­ing this pro­gram.

Not­ed Med­i­cines Co. CEO Clive Mean­well:

Al­though not re­quired for the NDA, we will al­so per­form an ag­gres­sive car­dio­vas­cu­lar out­comes tri­al in high-risk sub­jects with AS­CVD and/or risk-equiv­a­lents, such as di­a­betes. We be­lieve that pos­i­tive out­comes da­ta, with pri­ma­ry out­come clin­i­cal ef­fects greater than those re­port­ed for an­ti-PC­SK9 mon­o­clon­al an­ti­bod­ies, will dri­ve a high lev­el of com­pet­i­tive­ness in the world­wide mar­ket – which we ex­pect to be­come very large.

Sanofi and Re­gen­eron, mean­while, ex­pect to have their car­dio da­ta avail­able lat­er this year, hop­ing to trump Am­gen as the com­pa­nies fight it out on the patent front.

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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