Is Am­gen still shoot­ing blanks in its war with pay­ers over Repatha re­im­burse­ment?

Sean Harp­er, Ex­ec­u­tive Vice Pres­i­dent, Re­search and De­vel­op­ment

With on­ly $40 mil­lion in ane­mic Q3 Repatha sales, Am­gen $AMGN has been qui­et­ly seething over the slight mar­ket trac­tion it’s see­ing for a cho­les­terol drug that promised to help rewrite pre­scrib­ing habits in the field. But pay­ers have been loathe to cov­er the pi­o­neer­ing PC­SK9 drug — as well as a ri­val from Re­gen­eron/Sanofi.

Now, they’re hop­ing that ev­i­dence of its im­pact on an im­por­tant bio­mark­er for car­dio­vas­cu­lar dis­ease will per­suade pay­ers to start tear­ing down the walls they built to keep pa­tients away from the drug. But once the da­ta gets a thor­ough re­view, Am­gen and its ri­vals may find them­selves still far from the promised land of block­buster sales.

Here’s the pos­i­tive as­pect of what they re­port­ed at the big AHA meet­ing in New Or­leans:

Pa­tients in their Repatha/statin arm ex­pe­ri­enced a 0.95% de­crease in what’s called the per­cent athero­ma vol­ume (PAV), a mea­sure of plaque in ar­ter­ies, which has a well known link to car­dio­vas­cu­lar dis­ease. Pa­tients in the place­bo/statin arm had a 0.05% in­crease in PAV. Al­so sig­nif­i­cant­ly, 64.3% of Repatha pa­tients ex­pe­ri­enced plaque re­gres­sion in PAV com­pared to 47.3% of the place­bo arm.

By one mea­sure of plaque vol­ume, there was a mean de­crease of 5.8mm in the drug arm com­pared with 0.9mm seen in the place­bo arm. And the drug clear­ly slashed lev­els of ‘bad’ LDL.

These are all sta­tis­ti­cal­ly sig­nif­i­cant in­di­ca­tions of the drug’s im­pact on dis­ease pro­gres­sion along with fresh ev­i­dence of its im­pact on LDL. That’s all good.

The study, though, was not de­signed to pro­vide ev­i­dence of the drug’s abil­i­ty to im­prove out­comes for pa­tients. But they did an “ex­plorato­ry analy­sis” in any case and found ma­jor car­dio­vas­cu­lar events oc­curred in 12.2% of pa­tients re­ceiv­ing Repatha and 15.3% in those re­ceiv­ing place­bo.

Again, the da­ta were pos­i­tive, but they were al­so not con­clu­sive — and no huge gap ap­peared, rais­ing ques­tions over whether the pre­lim­i­nary num­ber may have fall­en in­to a mar­gin of er­ror.

That didn’t stop Steve Nis­sen at the Cleve­land Clin­ic from tout­ing the car­dio ben­e­fits they saw.

“This is the first time any­one has shown these drugs do any­thing oth­er than low­er cho­les­terol,” Nis­sen told re­porters, ac­cord­ing to a Bloomberg piece. And he hit on a low­er num­ber of heart at­tacks and artery-clear­ly surg­eries as proof — though he al­so ac­knowl­edged that it wasn’t con­clu­sive.

You could hear the frus­tra­tion at Am­gen, where R&D chief Sean Harp­er had this for his pre­pared state­ment:

We re­main con­cerned that many pa­tients are ex­pe­ri­enc­ing bar­ri­ers to ac­cess­ing Repatha, de­spite their physi­cian’s treat­ment rec­om­men­da­tions. We look for­ward to our out­comes study, FOURI­ER, and will con­tin­ue to work with pay­ers to im­prove ac­cess for pa­tients who need ad­di­tion­al LDL-C low­er­ing.

Pay­ers, though, are like­ly go­ing to con­tin­ue to wait for re­al proof, not in­con­clu­sive trends. Am­gen still has plen­ty of work ahead in con­vinc­ing them to cov­er the pre­scrip­tions that are be­ing writ­ten. And the ju­ry is still out on that score, leav­ing Am­gen’s big bet on PC­SK9 up against some daunt­ing odds.

Am­gen’s shares edged up yes­ter­day on the news, then edged back down in pre-mar­ket trad­ing. In­vestors haven’t been im­pressed, ei­ther.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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