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

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.