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.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Covid-19 roundup: Left out no longer, No­vavax se­cures largest Warp Speed deal yet: $1.6B

It looks like Novavax won’t be left out of Operation Warp Speed after all.

A month after the Gaithersburg, MD biotech saw its shares tumble when it was left off the first reported list of finalists for the White House’s Covid-19 vaccine accelerator, HHS and the Department of Defense have announced a $1.6 billion deal to scale up their Covid-19 candidate. It is the largest deal HHS has announced yet, eclipsing the $1.2 billion deal the administration reached with AstraZeneca in May.

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In­vestors give ail­ing Unum a lease on life and a whole new suite of ex­per­i­men­tal can­cer drugs

Investors, it seems, are willing to give Unum Therapeutics one last shot — or at least one last shot to a company of that name.

The ailing cancer biotech, beset by a series of clinical holds and multiple failed lead programs, announced today that they’ve acquired Kiq LLC and that investors are putting in $104 million to advance Kiq’s pipeline of kinase inhibitors. Unum shareholders will now own only 16.2% of the company and CEO Chuck Wilson indicated that the cell therapies the biotech has worked on since its founding may be on their way out, saying Unum will “explore strategic options” for those products.

RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Sec­ond PhI­II study for Idor­si­a's sleep drug re­turns pos­i­tive re­sults, but al­so rais­es new ques­tions

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.

UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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