Flanked by ri­vals and skep­tics, Es­pe­ri­on flips pos­i­tive da­ta on first PhI­II for a po­ten­tial­ly game-chang­ing cho­les­terol pill

Es­pe­ri­on $ES­PR is rolling out its first pos­i­tive da­ta set from a slate of piv­otal late-stage stud­ies of its LDL-low­er­ing drug be­mpe­doic acid this morn­ing — one of the most close­ly fol­lowed pro­grams in the busi­ness.

Fac­ing a skep­ti­cal au­di­ence of pay­ers and ri­vals — which have al­ready shown they can do much, much bet­ter with the same tar­get — Es­pe­ri­on post­ed a 28% spread against a place­bo, with a 23% drop in LDL-c for the drug plus eze­tim­ibe against a 5% in­crease for pa­tients in the place­bo group.

In­ves­ti­ga­tors al­so tracked an­oth­er plus for statin in­tol­er­ant pa­tients, with a 32% drop in high-sen­si­tiv­i­ty C-re­ac­tive pro­tein, a mark­er for dam­ag­ing in­flam­ma­tion which can have se­ri­ous longterm con­se­quences for car­dio health.

“In both cas­es,” CEO Tim Mayleben tells me as he set the stage for to­day’s an­nounce­ment, “this study ex­ceed­ed ex­pec­ta­tions.”

But is that enough?

It’s all sta­tis­ti­cal­ly sig­nif­i­cant, giv­ing sup­port­ive an­a­lysts the da­ta they were look­ing for to cel­e­brate the first of 5 Phase III stud­ies due out by the fall, set­ting up a mar­ket­ing pitch to the FDA in Q1 2019. Based on the da­ta, there are good rea­sons to be­lieve that the biotech can come through with more pos­i­tive ef­fi­ca­cy da­ta and a clean bill of health on liv­er tox.

But there’s a lot more in­volved here than demon­strat­ing that a new drug can beat out a place­bo. That’s been done be­fore. Es­pe­ri­on has to con­vince every­one that its new LDL low­er­ing pill can suc­ceed where oth­er drugs — in­jecta­bles — with much bet­ter re­sults have fared poor­ly thus far. In the car­dio field, as Mer­ck demon­strat­ed with its re­cent de­ci­sion to jet­ti­son a CETP ther­a­py in the wake of a win against a place­bo, pos­i­tive da­ta is no guar­an­tee of suc­cess. It al­so has to be com­mer­cial­ly vi­able, able to con­vince the mar­ket that this drug can break in­to block­buster ter­ri­to­ry.

Be­mpe­doic acid in dif­fer­ent com­bi­na­tions has al­so scored high­er in mid-stage stud­ies. But Es­pe­ri­on de­signed this first, crit­i­cal study to add their drug af­ter a round of eze­tim­ibe, mim­ic­k­ing the re­al world en­vi­ron­ment where doc­tors will be look­ing to fol­low up on statin re­sis­tance af­ter first try­ing a gener­ic at a very, very low cost.

Mayleben has al­ready bro­ken the car­di­nal rule of drug de­vel­op­ment, nam­ing a price of about $9 to $10 a day — or in the $3,500 per year range — well ahead of a launch.

That just doesn’t hap­pen in bio­phar­ma, where the vir­tu­al law of drug mar­ket­ing re­quires that the price be kept un­der lock­down un­til af­ter a mar­ket­ing OK comes through for a new drug — es­pe­cial­ly if de­vel­op­ers know per­fect­ly well what they will sell it for.

The Es­pe­ri­on CEO, though, can’t play by those rules. He has to steer a course around PC­SK9 drugs from Re­gen­eron/Sanofi and Am­gen, which eas­i­ly outscore his ther­a­py on low­er­ing LDL. Mayleben’s tar­get will be a wide range of pa­tients who need to see a big drop in LDL, but don’t nec­es­sar­i­ly re­quire the axe PC­SK9 brings to the ta­ble — and who don’t want any part of the $14,000 an­nu­al price tag at­tached that has spurred pay­ers to throw up a com­plex set of hur­dles to pre­vent wide­spread use.

That re­quires a will­ing­ness to up­set some ap­ple carts along the way. Stick­ing with the in­dus­try stan­dard here won’t help the roll out he’s plan­ning, with some an­a­lysts set­ting peak sales es­ti­mates deep in­to block­buster ter­rain.

“Why should this not be the way things are?” asks the CEO.

He cit­ed the out­cry that met Gilead when they priced their pi­o­neer­ing hep C cure at $84,000 as an ex­am­ple of what can go wrong.

“The is­sue was not just the price, but it was above ex­pec­ta­tions,” he notes. Flag­ging the price and start­ing that dis­cus­sion well ahead of a launch just makes sense.

Mayleben adds: “I don’t think I re­al­ized that we were set­ting any sort of prece­dent with what we were do­ing.”

Es­pe­ri­on, though, doesn’t have the lux­u­ry of a break­through drug, in the same way that the PC­SK9s or So­val­di were able to en­joy. To com­pete will take some dis­rup­tive rule break­ing.

And Mayleben plans to do just that.

There are 4 more piv­otal stud­ies to come. And Es­pe­ri­on is bring­ing on a chief com­mer­cial of­fi­cer soon to start lay­ing the foun­da­tion for a com­mer­cial wing. At some point, Mayleben isn’t say­ing when, the com­pa­ny al­so wants to bring in com­mer­cial part­ners to help roll out a mass mar­ket ef­fort in ear­ly 2020. And then they’ll still have to wait for car­dio out­comes da­ta in 2022.

The 2018 da­ta sea­son has be­gun for Es­pe­ri­on, and there’s vir­tu­al­ly no wig­gle room in the course the biotech has to steer now.

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.