Amarin gets a boost from the FDA as reg­u­la­tors promise speedy Vas­cepa de­ci­sion — but what about that pan­el re­view?

There’s plen­ty of buzz to­day about Amarin $AM­RN and its quest to get car­dio out­comes da­ta added to the la­bel for Vas­cepa, its ther­a­peu­tic strength ver­sion of fish oil.

John Thero Amarin Cor­po­ra­tion

The FDA has giv­en their sN­DA pri­or­i­ty re­view sta­tus, leav­ing a de­ci­sion on open­ing up the mar­ket for this ther­a­py just 4 short months away on Sep­tem­ber 28. The move lops months off the reg­u­la­to­ry process and moves a po­ten­tial ap­proval in­to Q3 of this year — one of the biggest cat­a­lysts on the H2 cal­en­dar.

That’s a very big deal for Amarin, which man­aged to wow just about every­one with its 25% re­duc­tion in the risk for the first oc­cur­rence of a ma­jor car­dio event in their tar­get pop­u­la­tion, the pri­ma­ry end­point that has sparked buzz about a po­ten­tial takeover — though one has yet to ma­te­ri­al­ize.

As an ex­cit­ed Michael Yee at Jef­feries not­ed to­day, Amarin CEO John Thero is stay­ing con­ser­v­a­tive in an­tic­i­pat­ing a pan­el re­view. But as Am­gen got a pass on PC­SK9 CV out­comes, he’s 50% to 60% cer­tain there won’t be a pan­el — which would be the fi­nal de-risk­ing event that would pave a like­ly ap­proval here.

Here’s the biotech’s rather dry sum­ma­ry:

Amarin pre­vi­ous­ly ex­pressed that it be­lieves an Ad­Com meet­ing or­ga­nized by the FDA in con­junc­tion with its re­view of the ex­pand­ed la­bel for Vas­cepa is like­ly, as this will be the first ever drug ap­proved for the large pa­tient pop­u­la­tion stud­ied in the RE­DUCE-IT tri­al.  It is not un­com­mon for the FDA to pro­vide clar­i­fi­ca­tion lat­er in the process on whether an Ad­Com will be held.

Set­ting aside some of the wilder pro­jec­tions, the new peak sales es­ti­mates for Vas­cepa are now in block­buster ter­rain, which is why so many in­vestors have bet on a buy­out. On the oth­er hand, this is a tough mar­ket to cap­i­tal­ize on, as Am­gen and Re­gen­eron learned the hard way with PC­SK9. And that could give buy­ers cold feet in any ne­go­ti­a­tions tied to big sums in a sell­er’s mar­ket when it comes to late-stage and ap­proved drugs.

In­vestors liked the sounds of progress to­day, though, bid­ding up shares by 5% while bet­ting on the like­li­hood of an ap­proval. And Yee is al­so count­ing the mon­ey as they wait on the FDA. He notes:

The scripts con­tin­ue to go high­er each week de­spite any la­bel ex­pan­sion yet. We be­lieve this is a tes­ta­ment to the strong CVOT da­ta pub­licly pre­sent­ed and pub­lished al­ready, and the de­mand by pa­tients and docs and ease in re­im­burse­ment – per our nu­mer­ous doc checks. We think Q2 is like­ly to be a strong quar­ter (in­to the $90M+ and up from $77M in Q1) and this strong tra­jec­to­ry would put them pos­si­bly on track to beat 2019 guid­ance of $350M this year.

Im­age: An­drew Harnik AP

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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Cue the M&A chat­ter: UniQure is scout­ing for a buy­out deal as gene ther­a­py field siz­zles — re­port

All the en­thu­si­asm that’s been whipped up in the gene ther­a­py field this past year has helped stoke the ru­mor mill about all sorts of pos­si­bil­i­ties for uniQure $QURE, which has seen a quick run-up on its share price. And now the biotech’s back­ers are get­ting a big boost from Bloomberg to keep the run go­ing.

There’s no deal to re­port, but sources are telling the busi­ness news ser­vice that uniQure has brought in ad­vis­ers to see what might be done — in­clud­ing a sale — with the phar­ma gi­ants now prowl­ing the clin­i­cal play­ers for part­ners and ac­qui­si­tions.

'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).