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

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.

Ab­b­Vie, Scripps ex­pand part­ner­ship, for­ti­fy fo­cus on can­cer drugs

Scripps and AbbVie go way back. Research conducted in the lab of Scripps scientist Richard Lerner led to the discovery of Humira. The antibody, approved by the FDA in 2002 and sold by AbbVie, went on to become the world’s bestselling treatment. In 2018, the drugmaker and the non-profit organization signed a pact focused on developing cancer treatments — and now, the scope of that partnership has broadened to encompass a range of diseases, including immunological and neurological conditions.

South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

Cel­lar­i­ty, Flag­ship's $50M bet on net­work bi­ol­o­gy, mar­ries ma­chine learn­ing and sin­gle-cell tech for drug dis­cov­ery

Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team were floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a bottom-up approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.