Es­pe­ri­on strikes Japan deal for 'goldilock­s' cho­les­terol drug, bags $60M cash as pan­dem­ic forces vir­tu­al US roll­out

Es­pe­ri­on has forged its first re­gion­al deal for Nexle­tol and Nexl­izet since win­ning US and EU ap­provals for the cho­les­terol fight­ing drugs, bag­ging $60 mil­lion up­front while se­cur­ing a phar­ma part­ner in Japan.

Not on­ly is Ot­su­ka promis­ing $450 mil­lion in to­tal mile­stones and 15% to 30% roy­al­ties, it’s al­so pick­ing up the tab for all de­vel­op­ment, reg­u­la­to­ry and sales in Japan — which Es­pe­ri­on es­ti­mates would amount to $100 mil­lion over the next few years.

“We had orig­i­nal­ly set our­selves a goal to get some­thing done by the end of last year,” Es­pe­ri­on CEO Tim Mayleben told End­points News. “Then as the ap­provals were com­ing in­to fo­cus and we knew from our dis­cus­sions with reg­u­la­tors that those were like­ly to hap­pen in the first part of this year, we were hear­ing from po­ten­tial part­ners at the same time that our ne­go­ti­a­tions would im­prove once we had the ap­provals in hand. So we again made the ex­plic­it de­ci­sion to push this out a few months and so that def­i­nite­ly paid off.”

By their count, there’s nev­er been a Japan deal with that big of an up­front. Their new Japan­ese part­ner al­so brings es­tab­lished re­la­tion­ships with health­care providers to its long his­to­ry of com­mer­cial­iz­ing car­dio­vas­cu­lar med­i­cines.

Tim Mayleben

By hit­ting the goldilocks zone be­tween old statins and ex­pen­sive an­ti-PC­SK9 ther­a­pies — with all the prop­er­ties that re­sem­ble “the old com­fort­able shoe” as chief com­mer­cial of­fi­cer Mark Glick­man de­scribes it — Es­pe­ri­on promised to serve hy­per­c­ho­les­terolemia pa­tients who are ei­ther in­tol­er­ant to statins or need fur­ther cho­les­terol re­duc­tion, but are priced out of the new gen­er­a­tion of treat­ments.

Their main prod­uct, be­mpe­doic acid, is a pro­drug that in­hibits adeno­sine triphos­phate cit­rate lyase (ACL) in the liv­er. Since that en­zyme plays a role in cho­les­terol syn­the­sis with­in the cell, block­ing it re­sults in less in­tra­cel­lu­lar cho­les­terol and more LDL re­cep­tors to cap­ture cho­les­terol in the blood.

Nexle­tol is a tablet con­sist­ing just of be­mpe­doic acid. With Nexl­izet, they add in the cho­les­terol ab­sorp­tion in­hibitor eze­tim­ibe to boost the po­ten­cy.

An oral, once-dai­ly non-statin ther­a­py would be a wel­come ad­di­tion to any com­pa­ny with a car­dio­vas­cu­lar fo­cus, Mayleben said, hint­ing that they are in the fi­nal stages of clos­ing a “true rest of world” deal, in­clud­ing Chi­na, with a multi­na­tion­al phar­ma com­pa­ny. That would give Es­pe­ri­on three part­ners, a num­ber that he be­lieves is man­age­able for his biotech.

“Those trav­el ex­tra­or­di­nar­i­ly well across dif­fer­ent ge­o­gra­phies,” he said. “Some of these new fanci­er tech­nolo­gies don’t trav­el as well — mon­o­clon­als and what not, be­cause there’s an even greater aver­sion to in­jectable ther­a­pies or just be­cause of the cost.”

Dai­ichi Sankyo signed up for Eu­rope and Switzer­land rights back in Jan­u­ary 2019, a year be­fore the EMA gave the green­light, with $150 mil­lion up­front in a deal worth $900 mil­lion. The Eu­ro­pean Com­mis­sion ce­ment­ed that with a fi­nal OK un­der the brand names Nilem­do and Nus­ten­di. The first sale would yield an­oth­er $150 mil­lion in mile­stones.

Mean­while in the US, Es­pe­ri­on has al­ready launched Nexle­tol to a pan­dem­ic-strick­en coun­try, set­ting up a re­mote cam­paign to en­cour­age adop­tion of its $10 per day reg­i­men. The Nexl­izet roll­out is sched­uled for Ju­ly.

“In re­sponse to the spread of SARS-CoV-2 and COVID-19, our com­mer­cial and med­ical or­ga­ni­za­tions have sus­pend­ed per­son­al in­ter­ac­tions with physi­cians and cus­tomers and will be con­duct­ing ac­tiv­i­ties vir­tu­al­ly,” it wrote in a re­cent SEC fil­ing.

Hav­ing com­plet­ed two and a half weeks of in­ten­sive train­ing in both the new dig­i­tal tools and vir­tu­al ways of mar­ket­ing, the field team has just be­gun mak­ing calls last Thurs­day — with ex­tra sen­si­tiv­i­ty to lo­cal sit­u­a­tions.

“None of our sales rep­re­sen­ta­tives are call­ing any­body in the New York area,” Mayleben said. But places like Flori­da, where he is based, of­fer a lit­tle more room to be­gin push­ing a new drug. “We’ll start to see the re­sults of that in our script da­ta per­haps by the end of the month.”

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA plans new stud­ies on ac­cel­er­at­ed ap­proval dis­clo­sures in bio­phar­ma ads

When people read biopharma companies’ websites about new drugs approved via the FDA’s accelerated pathway, like Biogen’s new Alzheimer’s drug, do they understand that these drugs may only be reasonably likely to predict clinical benefit and still require confirmatory studies?

That’s what the FDA’s Office of Prescription Drug Promotion wants to firm up as an agency analysis of direct-to-consumer websites for accelerated approval drugs previously found that only 21% of the disclosures used language directly from the label.

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Siddhartha Mukherjee, AP Images

Sid Mukher­jee's Vor taps a CAR-T de­vel­op­ment part­ner to strap to its at­tempt at bet­ter stem cell trans­plants

In the buzzy world of oncology, few names shine brighter than Columbia oncologist and bestselling author Siddhartha Mukherjee, whose Vor Biopharma is looking to rewrite the rules of stem cell transplants. If his team’s tech works as planned, it would also rewrite the rules on how CAR-Ts can be deployed in blood cancer — and now it’s partnering up with a little-known biotech to test that hypothesis.