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.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.