New PhI­II gives Es­pe­ri­on's CEO a chance to push back against a le­gion of crit­ics — but can he win back in­vestors?

Es­pe­ri­on $ES­PR would like to have back its rep for own­ing a cho­les­terol drug de­signed to hit a sweet spot in the mar­ket — with­out any un­sa­vory safe­ty is­sues that could per­suade reg­u­la­tors to re­quire an ex­pen­sive and lengthy car­dio out­comes study ahead of a mar­ket­ing ap­proval.

To that end, CEO Tim Mayleben is rolling out a fresh bat­tery of da­ta from their third piv­otal study of be­mpe­doic acid. And they want you to be­lieve as they do: That the con­sis­tent abil­i­ty to cut LDL — in this case by demon­strat­ing a fa­mil­iar 23% drop, with a 25% low­er­ing in high-sen­si­tiv­i­ty C-re­ac­tive pro­tein — tied in to no new fa­tal­i­ties (whew) and what they in­sist is a clean safe­ty pro­file should be wide­ly ap­plaud­ed.

Whether they can achieve their goal in a mar­ket that has sharp­ened its knives for biotech stocks and kept on the look­out for any weak­ness­es, though, is still open to ques­tion. But they’re go­ing to try.

“We’re try­ing to pro­vide more con­text than we did ear­li­er this month,” CEO Tim Mayleben told me in a pre­view of to­day’s num­bers. Mayleben has seen his stock price gy­rate be­fore on the way an in­di­vid­ual da­ta point is in­ter­pret­ed, but now he has re­port­ed da­ta on 4,000 pa­tients all to­geth­er — 1,500 who have been on the drug for more than a year.

“That’s what reg­u­la­tors will look at, the pooled da­ta,” says the CEO. “When you look at it over­all, you see a very bal­anced safe­ty and tol­er­a­bil­i­ty pro­file.” And he still isn’t wor­ried about the prospect of head-to-head com­pe­ti­tion with the PC­SK9s. 

Part of that pooled da­ta that will get close scruti­ny to­day is a fa­tal­i­ty rate of 0.5 per­cent in the drug arm and 0.2 per­cent for the place­bo group.

“I would say that with re­spect to pric­ing, one of the things we have seen con­stant­ly is con­fu­sion, mis­un­der­stand­ings about the net price,” the CEO adds. Once you fac­tor in the usu­al dis­counts ex­pect­ed in this mar­ket, he says, you can ex­pect a net price of less than half the $5,000 (or so) that the PC­SK9s will go for. And for mil­lions of pa­tients who are not con­trolled on statins and don’t need a dra­mat­ic cut in LDL, he be­lieves he’ll have a clear­ly de­fined mar­ket of­fer­ing a big ad­van­tage to pay­ers.

Ul­rich Laufs

The team at Es­pe­ri­on has good rea­son to push back af­ter their stock was pum­meled ear­li­er this month in the af­ter­math of its last Phase III read­out, when an­a­lysts and in­vestors re­coiled from an im­bal­ance of deaths in the study and asked some sharply point­ed ques­tions about the fu­ture of a drug that has a pro­ject­ed price just un­der what Re­gen­eron and Sanofi re­cent­ly set as their low mark for an ap­proved PC­SK9 ri­val that every­one agrees is much, much bet­ter at low­er­ing LDL, with ri­vals on the mar­ket and in the pipeline that could do just as well — or bet­ter.

That prospect of low­er­ing prices on the PC­SK9 class could crush a play­er like Es­pe­ri­on, a sen­ti­ment that helped trig­ger a 30% land­slide in the share price.

De­spite the ab­sence of deaths, the be­mpe­doic acid arm did see a 6% rate of se­ri­ous ad­verse events, com­pared to 3.6% for the place­bo group. Dis­con­tin­u­a­tions were 18.4% for the drug arm ver­sus 11.7% for the place­bo — but the study in­ves­ti­ga­tor ruled out any con­nec­tion be­tween the drug and the SAEs.

Ul­rich Laufs, a mem­ber of Es­pe­ri­on’s Phase III ex­ec­u­tive com­mit­tee and di­rec­tor of the de­part­ment of car­di­ol­o­gy at Leipzig Uni­ver­si­ty, called the da­ta “amaz­ing­ly con­sis­tent” and added that “the med­ical com­mu­ni­ty is in need of a new oral ther­a­py which is ef­fec­tive, well tol­er­at­ed and con­ve­nient for this com­plex pa­tient pop­u­la­tion who may have run out of oth­er op­tions.”  

Es­pe­ri­on has two more Phase III read­outs, this sum­mer and then in Sep­tem­ber. No lat­er than Q1, says Mayleben, they’ll have the NDA com­plete. And then reg­u­la­tors can have the last say on ef­fi­ca­cy and safe­ty.

In the mean­time, the in­vestors are still see­ing more red than green in the up­dates from Es­pe­ri­on. The stock was down 3% by mid-day Wednes­day.

Im­age: Tim Mayleben

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.