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.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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