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.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Scott Struthers, Crinetics CEO

Cri­net­ics spins out ra­dio­phar­ma ef­forts in­to a new com­pa­ny, high­light­ing the grow­ing field­'s al­lure

Largely known for its nonpeptide small molecule research, Crinetics has been keeping its radiopharma work comparatively under wraps. But that changed Monday afternoon as the California biotech spun out a new company focused solely on the burgeoning field.

Crinetics launched Radionetics after the closing bell Monday, the company announced, seeding the new entity with $30 million raised from 5AM Ventures and Frazier Healthcare Partners. Radionetics will start with its own radiopharma-centric platform and a pipeline of 10 programs aimed at solid tumors.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.