GSK aban­dons Ion­is' FDA-ready in­ot­ersen as new CEO sweeps out rare dis­ease ef­forts

Just three months af­ter Io­n­is spelled out pos­i­tive Phase III ef­fi­ca­cy da­ta on its top prospect in­ot­ersen, Glax­o­SmithK­line is sweep­ing their al­liance on the drug out the back door, along with rights to an­oth­er treat­ment they have rights to.

Em­ma Walm­s­ley, GSK CEO

It’s a stun­ning 11th hour re­ver­sal for Io­n­is $IONS, which says it now plans to push for an FDA ap­proval of in­ot­ersen on its own this fall as it thinks through whether it will com­mer­cial­ize or co-com­mer­cial­ize in the US on its own.

Ac­cord­ing to Io­n­is, GSK ex­ecs de­cid­ed to aban­don in­ot­ersen, de­signed to treat rare cas­es of TTR amy­loi­do­sis (AT­TR), with an ini­tial fo­cus on polyneu­ropa­thy due to hered­i­tary TTR amy­loi­do­sis (hAT­TR-PN). That drug is a close­ly-watched ri­val to Al­ny­lam’s lead drug patisir­an. Al­so out: IO­N­IS-FB-LRx, now at the thresh­old of Phase II for dry age-re­lat­ed mac­u­lar de­gen­er­a­tion, as GSK turns its back on rare dis­eases as part of its top-to-bot­tom R&D re­vamp un­der new CEO Em­ma Walm­s­ley.

“Our goals for in­ot­ersen are to max­i­mize its com­mer­cial suc­cess and op­ti­mize our com­mer­cial par­tic­i­pa­tion. To achieve these goals, we are ac­tive­ly con­sid­er­ing form­ing a com­mer­cial sub­sidiary to com­mer­cial­ize or co-com­mer­cial­ize in­ot­ersen in North Amer­i­ca, as well as oth­er op­tions. Our re­cent ex­pe­ri­ence build­ing a com­mer­cial sub­sidiary has pre­pared us for this op­por­tu­ni­ty. We have sub­stan­tial in­ter­est from po­ten­tial part­ners and are in dis­cus­sions with sev­er­al par­ties. We be­lieve that, to­geth­er with the right com­mer­cial part­ner, we can max­i­mize the com­mer­cial suc­cess of the drug world­wide,” said Sarah Boyce, chief busi­ness of­fi­cer of Io­n­is Phar­ma­ceu­ti­cals.

Sarah Boyce, Io­n­is CBO

Fil­ing for ap­proval and get­ting it are two dif­fer­ent things, though. Io­n­is’ late-stage ef­fort has al­so been flagged for se­ri­ous safe­ty con­cerns which could al­so ham­per its quest for an OK, as well as pos­si­bly sign­ing up a new com­mer­cial­iza­tion part­ner. Paul Mat­teis at Leerink swift­ly not­ed:

Nor­mal­ly in such a sit­u­a­tion like this in­vestors might wor­ry that there’s some not-yet seen-el­e­ment of the full da­ta that may be prob­lem­at­ic or dis­con­cert­ing. To this point — giv­en the well doc­u­ment­ed safe­ty is­sues as­so­ci­at­ed with In­ot­ersen (i.e., throm­bo­cy­tope­nia), GSK’s de­ci­sion may in­crease in­vestor scruti­ny on TTR­rx’s safe­ty pro­file as it is de­pict­ed in ad­di­tion­al full da­ta pre­sen­ta­tions this Fall (Amer­i­can Neu­ro­log­i­cal As­so­ci­a­tion, Oct. 15-17, oth­ers). Our mod­el cur­rent­ly as­sumes a 75%/25% AL­NY (MP) ver­sus IONS split, and while we are en­cour­aged that IONS’s plans for a YE17 MAA fil­ing are still on track, we are a bit con­cerned that GSK’s de­ci­sion may al­so put IONS be­hind the curve with re­spect to launch pre­pared­ness.

Stan­ley Crooke, Io­n­is CEO

Two oth­er al­liances will con­tin­ue. GSK is cur­rent­ly in Phase II with IO­N­IS-HB­VRx and IO­N­IS-HBV-LRx.

CEO Stan­ley Crooke not­ed that Io­n­is is now “ac­cel­er­at­ing the ex­pan­sion of our TTR pro­gram for pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis and the de­vel­op­ment of our LI­CA fol­low-on drug. Our ex­pe­ri­ence in the com­plet­ed Phase 3 NEU­RO-TTR study pro­vides im­por­tant in­for­ma­tion to aid in de­sign of a study in pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis. We have al­ready iden­ti­fied a more po­tent and con­ve­nient LI­CA fol­low-on and we ex­pect de­vel­op­ment of the LI­CA drug to al­so pro­ceed rapid­ly.”

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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