In the hunt for a megablock­buster, Cel­gene plans FDA fil­ing in wake of PhI­II win for ozan­i­mod in MS


When Cel­gene bought out Re­cep­tos and its au­toim­mune drug ozan­i­mod back in 2015 for $7.2 bil­lion in cash, the big biotech bull­ish­ly fore­cast their drug would be worth $4 bil­lion to $6 bil­lion in peak sales. To­day, the ex­ec­u­tive crew start­ed to make good on that pro­jec­tion with a pos­i­tive read­out on the top-line da­ta from their Phase III mul­ti­ple scle­ro­sis study.

The com­pa­ny says it’s on track to file for an ap­proval of the S1P1 re­cep­tor an­tag­o­nist this year.

In­ves­ti­ga­tors weren’t hand­ing out any hard da­ta to­day. But the com­pa­ny says that ozan­i­mod de­liv­ered “sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­ing­ful im­prove­ments com­pared to Avonex for the pri­ma­ry end­point of ARR (the an­nu­al­ized re­lapse rate) and the mea­sured sec­ondary end­points of the num­ber of gadolin­i­um-en­hanc­ing MRI le­sions and the num­ber of new or en­larg­ing T2 MRI le­sions at month 12.”

A sec­ond Phase III study in MS will read out in Q2.

This is a tough mar­ket, though. To get any­where close to $6 bil­lion, ef­fi­ca­cy alone won’t cut it. Cel­gene will al­so have to prove a sub­stan­tial safe­ty ad­van­tage with their drug.

Ge­of­frey Porges, Leerink

Just a few days ago Ge­of­frey Porges at Leerink out­lined the strat­e­gy for ozan­i­mod.

Re­sults from the two Ozan­i­mod phase III tri­als in mul­ti­ple scle­ro­sis are due in the first half of this year, with phase III ul­cer­a­tive col­i­tis da­ta ex­pect­ed in the first half of 2018. CELG al­ways as­sumed ozan­i­mod would get same class la­bel­ing match­ing oth­er S1P1 in­hibitors such as Gilenya, but al­so an­tic­i­pate that the phase III safe­ty da­ta would be in the la­bel so that CELG could dis­cuss the po­ten­tial dif­fer­ences with physi­cians and pay­ers. CELG stat­ed the com­pa­ny needs to see “re­al­ly good ef­fi­ca­cy” and a sim­i­lar lack of car­dio and liv­er tox­i­c­i­ty as shown in the phase II tri­al. About 15% of pa­tients dis­con­tin­ue Gilenya due to these tox­i­c­i­ty is­sues. CELG stat­ed that pric­ing will be low­er in UC when the drug is launched in that in­di­ca­tion, and the com­pa­ny plans to use dis­counts by in­di­ca­tion or oth­er meth­ods to match the ex­ist­ing pric­ing in the MS and IBD in­di­ca­tions (at a list price sim­i­lar to the ex­ist­ing $90,000 Gilenya price).

The com­pa­ny said to­day that the safe­ty pro­file of the drug is in line with their Phase II re­sults. And they sound­ed pumped about the prospects of their in­flam­ma­tion and im­munol­o­gy pipeline.

Scott Smith, Cel­gene

Jef­feries’ Bri­an Abra­hams gave the com­pa­ny a quick thumbs up, not­ing that there’s a long way to go be­fore this drug lives up to ex­pec­ta­tions.

We cur­rent­ly mod­el out-year MS sales of ozan­i­mod of ~$1B, giv­en the po­ten­tial Gilenya gener­i­ciza­tion, in­creas­ing com­pe­ti­tion, and grow­ing pric­ing pres­sure in the MS space. While it is not clear whether CELG would de­tail the agent on their own, the com­pa­ny has re­cent­ly shown more open­ness to us­ing ozan­i­mod as a cor­ner­stone to build out a broad­er neu­rol­o­gy com­mer­cial/de­vel­op­ment plat­form. In IBD, we as­sume peak sales ap­proach­ing ~$3.5B in the out-years, and while key ph.III UC da­ta is not ex­pect­ed un­til 2018, we could see some ini­tial Crohn’s dis­ease da­ta that could help clar­i­fy its pro­file and ex­pand­ed IBD po­ten­tial.

“Peo­ple liv­ing with mul­ti­ple scle­ro­sis need ad­di­tion­al ther­a­pies and we are pleased that oral ozan­i­mod showed mean­ing­ful im­prove­ments across pri­ma­ry and mea­sured sec­ondary end­points in this study,” said Scott Smith, pres­i­dent of Cel­gene In­flam­ma­tion and Im­munol­o­gy. “We look for­ward to da­ta from the con­fir­ma­to­ry phase III RA­DI­ANCE tri­al in the sec­ond quar­ter as we ad­vance to­ward planned reg­u­la­to­ry sub­mis­sions by year-end.”

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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