Amarin scores a big win for its car­dio out­comes study on Vas­cepa — stock races high­er as an­a­lysts ap­plaud

Amarin $AM­RN has staked its claim to the next big block­buster in the car­dio space, herald­ing a clear sta­tis­ti­cal win on the pri­ma­ry end­point for its 7-year clin­i­cal quest to prove that its in­dus­tri­al strength pu­ri­fied fish oil Vas­cepa can sig­nif­i­cant­ly re­duce ma­jor car­dio threats for a broad swath of pa­tients.

An ear­ly pre­view of the top-line da­ta rolled out at a bleary-eyed time in the morn­ing on Mon­day point­ed a di­rect ar­row at a rel­a­tive 25% drop in risk for ma­jor car­dio events — such as death, stroke and hos­pi­tal­iza­tion due to angi­na — hit­ting a high­ly sta­tis­ti­cal p=<0.001 for the pa­tients in the years-long RE­DUCE-IT study. And re­searchers al­so high-fived each oth­er for a clean safe­ty pro­file in the same head­line da­ta to make the state­ment.

The da­ta sup­port­ed “ro­bust” ev­i­dence of its ef­fect across mul­ti­ple sec­ondary end­points, ac­cord­ing to the com­pa­ny. We won’t see the par­tic­u­lars un­til lat­er, but the com­pa­ny is on track to file for ap­provals in ear­ly 2019.

An­a­lysts at Jef­feries have said that a suc­cess here could trans­form Vas­cepa in­to a $2 bil­lion-plus drug. And that num­ber res­onat­ed this morn­ing, as Amarin’s share prize zoomed up 288%. The stun­ning ad­vance fol­lowed fresh re­marks from Jef­feries about the rel­a­tive val­ue that Amarin un­der­scored this morn­ing rel­a­tive to oth­er drugs that have tried — and failed — to make much of an im­pres­sion.

The mag­ni­tude of MACE re­duc­tion is so far the great­est among all ther­a­pies on top of statins, far ex­ceed­ing the clin­i­cal­ly mean­ing­ful CV ben­e­fit ex­pec­ta­tion. Re­call two PC­SK9 in­hibitors (Repatha and Pralu­ent) and Ilaris all re­duced MACE risks by 15% in re­cent­ly com­plet­ed CVOTs (FOURI­ER, ODYSSEY, and CAN­TOS). The FDA has ex­pand­ed Repatha’s la­bel to in­clude cer­tain CV risk re­duc­tion and is cur­rent­ly re­view­ing sBLA of Pralu­ent in MACE re­duc­tion (PDU­FA on 4/28/19). Our doc sur­vey sug­gest­ed 10-15%+ risk re­duc­tion in MACE is con­sid­ered clin­i­cal­ly mean­ing­ful.

“We are de­light­ed with these topline study re­sults,” said John Thero, pres­i­dent and CEO of Amarin, in a state­ment. “Giv­en Vas­cepa is af­ford­ably priced, oral­ly ad­min­is­tered and has a fa­vor­able safe­ty pro­file, RE­DUCE-IT re­sults could lead to a new par­a­digm in treat­ment to fur­ther re­duce the sig­nif­i­cant car­dio­vas­cu­lar risk that re­mains in mil­lions of pa­tients with LDL-C con­trolled by statin ther­a­py, as stud­ied in RE­DUCE-IT.” 

None of this is go­ing to make it easy for the com­pa­ny, though, with a host of an­gry short in­vestors like­ly to try what­ev­er it takes to fo­ment a back­lash against the da­ta for the last big cat­a­lyst of Q3. Fur­ther com­pli­cat­ing their suc­cess, As­traZeneca has its own out­comes study for the ri­val Epano­va ready to read out next year, while GSK’s Lo­vaza went gener­ic sev­er­al years ago.

But as the morn­ing wore on, it be­came ap­par­ent that Amarin was hold­ing on to its big gains and sup­port­ers are swoon­ing.

Pa­tients en­rolled in RE­DUCE-IT had reg­is­tered an LDL-C lev­el be­tween 41-100 mg/dL (me­di­an base­line LDL-C 75 mg/dL) con­trolled by statin ther­a­py with a slate of car­dio­vas­cu­lar risk fac­tors in­clud­ing: per­sis­tent el­e­vat­ed triglyc­erides be­tween 150-499 mg/dL (me­di­an base­line 216 mg/dL) and ei­ther es­tab­lished car­dio­vas­cu­lar dis­ease or di­a­betes mel­li­tus and at least one oth­er CV risk fac­tor. 

That’s a big mar­ket.

This Phase III has been end­less­ly de­bat­ed as the time drew near for the read­out. Amarin’s Vas­cepa — ap­proved more than 4 years ago for hy­per­triglyc­eridemia — demon­strat­ed its abil­i­ty to sig­nif­i­cant­ly re­duce triglyc­erides in those pa­tients while in­flu­enc­ing bio­mark­ers on in­flam­ma­tion. But as we know from past big car­dio stud­ies, that cer­tain­ly doesn’t nec­es­sar­i­ly trans­late in­to the kind of hard and demon­stra­ble health ben­e­fits such that pay­ers are will­ing to cov­er much larg­er mass pop­u­la­tions of at-risk mem­bers.

RE­DUCE-IT set out to an­swer that ques­tion, with re­searchers en­rolling more than 8,000 pa­tients so they could com­pare it as an add-on to statins in re­duc­ing in­stances of ma­jor ad­verse car­dio­vas­cu­lar events (MACE) as com­pared to statin-treat­ed pa­tients plus place­bo.

We al­ready know from the re­cent AS­CEND study that pop­u­lar fish oil prod­ucts sold in every phar­ma­cy have no clear health ben­e­fits, and Amarin has been built on the no­tion that its high­ly-pu­ri­fied, omega-3 fat­ty acid prod­uct could dis­tin­guish it­self from the sup­ple­ment field. Com­pa­ny ex­ecs said that fish oil’s weak­ness would sim­ply make it eas­i­er to prove Vas­cepa’s val­ue, once the out­comes da­ta were in.

Is it good enough?

Even pos­i­tive car­dio da­ta don’t al­ways trans­late in­to com­mer­cial op­por­tu­ni­ty, and an­a­lysts now will see whether Amarin has come up with da­ta that are good enough to qual­i­fy as the prac­tice-chang­ing event that the com­pa­ny’s back­ers have in­sist­ed would now be in reach.

The dif­fer­ence amounts to a mar­ket with mil­lions of po­ten­tial pa­tients.

Sup­port­ive Jef­feries an­a­lysts who helped build sup­port for the drug’s suc­cess re­cent­ly not­ed:

Pos­i­tive RE­DUCE-IT will be prac­tice chang­ing as it will pro­vide POC that us­ing a high­ly pu­ri­fied Omega-3 drug (vas­cepa) in MD pa­tients on sta­ble statins and rel­a­tive­ly well-con­trolled LDL-C (<100mg/dL) con­tributes to a CV ben­e­fit. While LDL-C has de­creased over decades, TGs con­tin­ue to rise. The mar­ket needs this type of drug.

But for every ad­vo­cate, you could count two more who bet against the drug and any up­beat rise in the share price. The die hards won’t re­treat with­out putting up a fight, but their num­bers were def­i­nite­ly dwin­dling Mon­day.

Im­age: John Thero. AMARIN

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Top an­a­lyst of­fers a rare, up­beat in­ter­pre­ta­tion of Ab­b­Vie’s $63B Al­ler­gan deal — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took at 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.