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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.