Amarin takes ap­pli­ca­tion to ex­pand Vas­cepa la­bel to the FDA as M&A chat­ter heats up

Hav­ing un­veiled an un­ex­pect­ed set of heart pro­tec­tive re­sults for their fish oil-de­rived pill Vas­cepa, Amarin is march­ing ahead to ful­fill block­buster ex­pec­ta­tions for its cho­les­terol-low­er­ing drug by sub­mit­ting an ap­pli­ca­tion to ex­pand Vas­cepa’s la­bel to in­clude the re­duc­tion in car­dio­vas­cu­lar risk.

The com­pa­ny sparked a flur­ry of M&A chat­ter af­ter re­veal­ing da­ta from the RE­DUCE-IT study last year — a 25% re­duc­tion in the risk for the first oc­cur­rence of a ma­jor car­dio event, and a 26% re­duc­tion for 3-point MACE, a com­pos­ite of car­dio­vas­cu­lar death, non­fa­tal heart at­tack and non­fa­tal stroke — al­though con­cerns about the im­pact of the min­er­al oil place­bo on the re­sults prompt­ed lin­ger­ing ques­tions. Ear­li­er this month, Amarin de­buted its ex­plorato­ry analy­sis of the tri­al, with re­searchers sug­gest­ing a 30% re­duc­tion in car­dio­vas­cu­lar events com­pared to the place­bo arm.

John Thero

“The RE­DUCE-IT re­sults sup­port that ap­prox­i­mate­ly 1 few­er ma­jor car­dio­vas­cu­lar ad­verse event would oc­cur on av­er­age for every 6 pa­tients treat­ed with Vas­cepa for 5 years on top of statin ther­a­py com­pared to place­bo,” Amarin chief John Thero said in a state­ment.

On Thurs­day, Amarin said it was op­er­at­ing un­der the as­sump­tion that the sN­DA will be re­viewed over a stan­dard ten months re­sult­ing in a PDU­FA date near the end of Jan­u­ary 2020, and that it ex­pects the reg­u­la­tor with or­ga­nize an ad­vi­so­ry com­mit­tee meet­ing of out­side ex­perts to de­lib­er­ate and rec­om­mend whether Vas­cepa’s la­bel should be ex­pand­ed be­fore the FDA makes its fi­nal de­ci­sion.

Ear­li­er in the week, the Amer­i­can Di­a­betes As­so­ci­a­tion is­sued fresh “stan­dards of care” guide­lines to in­clude Vas­cepa. They rec­om­mend­ed  that Vas­cepa “be con­sid­ered for pa­tients with di­a­betes and ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease or oth­er car­diac risk fac­tors on a statin with con­trolled LDL-C, but with el­e­vat­ed triglyc­erides to re­duce car­dio­vas­cu­lar risk.”

Vas­cepa, which gen­er­at­ed about $229 mil­lion in 2018 sales, com­pris­es omega-3 acid called EPA de­rived from fish — was orig­i­nal­ly ap­proved in 2012 for pa­tients with se­vere hy­per­triglyc­eridemia. It is rea­son­ably priced with an an­nu­al price tag of rough­ly $2400, Amarin con­tends, adding that the ma­jor­i­ty of in­sured pa­tients in­sur­ance who ob­tain Vas­cepa pre­scrip­tions pay a month­ly co-pay charge of $9.99 or less.

If and when Vas­cepa’s la­bel is ex­pand­ed, the com­pa­ny aims to keep Vas­cepa “af­ford­ably priced” Thero told End­points News in an emailed state­ment.

Mean­while, PC­SK9 in­hibitors that were ap­proved in 2015 car­ried a price close to $14,000 and were pegged to at­tain block­buster sta­tus for their abil­i­ty to dra­mat­i­cal­ly low­er lev­els of LDL cho­les­terol, fac­ing push­back from in­sur­ers for their high stick­er prices that led to low­er adop­tion than ex­pect­ed, de­spite lat­er tri­als that demon­strat­ed they al­so sig­nif­i­cant­ly cut the risk of heart at­tacks and stroke. Fol­low­ing Am­gen’s de­ci­sion to slash the price of its PC­SK9 drug Repatha by 60% to $5,850 last year, the team be­hind their main ri­val treat­ment, Pralu­ent — Re­gen­eron $REGN and Sanofi $SNY — have fol­lowed suit with the same dis­count, be­gin­ning ear­ly March.

In 2016, Amarin $AM­RN won a land­mark rul­ing against the FDA, which al­lowed the drug­mak­er to ex­er­cise its first amend­ment rights by pro­mot­ing Vas­cepa for off-la­bel us­es as long as it does so ‘truth­ful­ly.’ The com­pa­ny was al­so seek­ing to mar­ket the drug to pa­tients with not just se­vere triglyc­eride lev­els, but those con­sid­ered to have ‘high’ lev­els of the blood fat.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

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John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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