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Ned Sharp­less is not play­ing with the FDA’s gold stan­dard — any more than Scott Got­tlieb did

Ned Sharp­less got a chance to out­line just what kind of act­ing FDA chief he’ll be in the wake of Scott Got­tlieb’s de­par­ture from the agency. And aside from a more con­ser­v­a­tive se­lec­tion of socks, he’s clear­ly plan­ning to adopt the ex­act same fash­ions laid down by his pre­de­ces­sor.

In an all-hands-on-deck ad­dress to staffers, Sharp­less promised to fit the Got­tlieb mold per­fect­ly, vow­ing to main­tain the agency’s gold stan­dard on drug ap­provals while do­ing what he can to keep the copy­cats com­ing to help tamp down on drug costs.
So let me re­as­sure you, I am not plan­ning any rad­i­cal changes from what the FDA has been try­ing to ac­com­plish.

SCO­TUS just turned its back on Al­ler­gan’s le­gal ma­neu­ver to take a blow at in­ter partes re­view. What did you ex­pect?

It’s fi­nal­ly over.

From the very be­gin­ning, Al­ler­gan’s at­tempt to safe­guard its block­buster Resta­sis fran­chise by hand­ing over the patents to a Mo­hawk tribe in New York looked like a bad par­o­dy of a le­gal loop­hole ma­neu­ver. Now it’s a dead par­o­dy, af­ter the Supreme Court on Mon­day shunned the com­pa­ny’s at­tempt to take its ar­gu­ment to the high­est court in the land with a sin­gle line.

The tribe’s im­mu­ni­ty would safe­guard the patents from the in­ter partes re­view process, they rea­soned, and they could ba­si­cal­ly lease back con­trol of the drug while steer­ing clear of a se­ri­ous threat from gener­ic drug­mak­ers.

Go­ing vi­ral? J&J tries to spark a Twit­ter­storm for the lat­est da­ta on its HIV med

When you set out to make a block­buster, can a Twit­ter­storm help?

About 9 months af­ter the FDA OK’d J&J’s 4-in-1 HIV pill Sym­tuza, the phar­ma gi­ant rolled out new Phase III da­ta Thurs­day that demon­strates how new­ly di­ag­nosed HIV pa­tients are like­ly to squelch any signs of the virus if they jump on drug with­in 14 days of di­ag­no­sis. And they of­fered canned tweets in the PR to help spread the word — fast.

An­oth­er play­er in the gene ther­a­py field is gun­ning for the Duchenne MD crown — and here’s what they're bet­ting on

Most of you gene therapy enthusiasts reading this will already know about one program out of Nationwide Children’s Hospital that’s attracted considerable attention in the industry. It’s the micro-dystrophin one at Sarepta, which has snagged a big spotlight for the early — and very exciting — human data that they’ve been rolling out.

Today, a different biotech company you most likely haven’t heard nearly as much about is rolling out a new gene therapy program for Duchenne muscular dystrophy — soon to be tested in humans — that they believe can leapfrog Sarepta straight to patients.

“It’s safer and more effective compared to other approaches, including micro-dystrophin.”

That’s the CEO of Audentes talking. Matt Patterson. He’s the company co-founder who counted himself as employee #1 for the first 6 months, now with a staff of 230 and a market cap of $1.7 billion. Audentes has some early data due out on their lead gene therapy program for AT132 in a few weeks.

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RIP amy­loid be­ta the­o­ry? Nope. Bio­gen part­ner launch­es a new PhI­II be­fore ad­u­canum­ab's corpse turned cold

A day af­ter Bio­gen rat­tled the bio­phar­ma world with the news that its lead late-stage ther­a­py ad­u­canum­ab proved worth­less in treat­ing Alzheimer’s — a dis­as­ter that may dri­ve a stake through the heart of the amy­loid be­ta the­o­ry once and for all — the big biotech’s part­ners at Ei­sai have come up with their next big move.

Right in the wake of a 35% plunge in their stock val­ue, Ei­sai $ESALY is start­ing a Phase III study in­volv­ing 1,566 Alzheimer’s pa­tients with mild cog­ni­tive im­pair­ment for the con­tro­ver­sial Alzheimer’s drug BAN2401. The an­ti-amy­loid an­ti­body was the cen­ter of a firestorm of crit­i­cism over a tardy re­veal that re­searchers had pulled high-risk pa­tients out of their last study, po­ten­tial­ly warp­ing the pos­i­tive re­sults that were claimed, leav­ing that drug un­der a dark cloud.

How do you re­place a rock star like Scott Got­tlieb at the FDA? Maybe you can't

Any­one look­ing for a con­ve­nient weath­er vane to de­ter­mine the re­ac­tion to Scott Got­tlieb’s abrupt de­par­ture from the FDA need go no fur­ther than the $XBI. The S&P Biotech ETF took a 4% hit. And it was down an­oth­er 1.8% ahead of the bell on Thurs­day.

The mar­ket and the in­dus­try loved Got­tlieb, as we un­der­scored on sev­er­al oc­ca­sions with in­dus­try sur­veys high­light­ing the in­tense en­thu­si­asm for some­one who ad­vo­cat­ed for col­lab­o­rat­ing with the drug in­dus­try. Bio­phar­ma had a part­ner in Got­tlieb, and they all knew it.

As Pfiz­er kills failed PhI­II pro­grams for Baven­cio, where do they go from here in R&D?

Pfizer managed to disappoint just about everyone this morning with its full-year outlook as generic competition eats into their big franchise revenue streams. For a company that has long prided itself on the numbers, it was a telling setback that follows a wide-ranging reorganization plan that is culling staff and eliminating segments of R&D.

Shares were down close to 3% Tuesday morning, ahead of the opening bell.

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The an­tibi­otics R&D ef­fort is bro­ken. Here's one mar­ket so­lu­tion that might fix it

Any­one even slight­ly fa­mil­iar with an­tibi­otics R&D over the last 10 years would quick­ly agree that the sys­tem is bad­ly bro­ken.

Big Phar­ma — with the ex­cep­tion of Roche — large­ly pulled out of de­vel­op­ment be­cause the mar­gins are low. The hunt now is for block­busters. There are plen­ty of cheap an­tibi­otics to cov­er most of the mar­ket; the niche for new an­tibi­otics re­mains too small to sus­tain any ma­jor ef­fort need­ed here. The lit­tle biotechs left with the li­on’s share find it’s ex­pen­sive, high­ly risky and hard to make pay. And that is not a pret­ty pic­ture.

Short sell­er Cit­ron draws fresh blood go­ing af­ter an­oth­er biotech scalp — but can it still cut that deep?

Over the past 3 years, since Cit­ron’s An­drew Left made a name for him­self as the cru­sad­ing an­a­lyst who helped take down Valeant with an ex­posé on its shady ties to the spe­cial­ty phar­ma Phili­dor, he and the firm have tack­led a se­ries of big play­ers — with a sure­fire ap­proach to carv­ing up the stock price.

The Valeant take­down be­came a mod­el for biotech short at­tacks. And Cit­ron found plen­ty of lever­age as it went af­ter some much big­ger game.

Al­ler­gan and its Mo­hawk al­lies pitch their sov­er­eign im­mu­ni­ty case to SCO­TUS. And the tim­ing could­n't be worse

Al­ler­gan {AGN} is tak­ing its case against in­ter partes re­view all the way to the Supreme Court — pro­vid­ed SCO­TUS agrees to hear their at­tor­neys.

The biotech com­pa­ny made its pitch to the Supreme Court along­side the Mo­hawk tribe that took the patents on Resta­sis, Al­ler­gan’s big block­buster, as the com­pa­ny tried to wall it­self off from gener­ic man­u­fac­tur­ers. But the courts, in­clud­ing a fed­er­al ap­peals court, have ruled against the com­pa­ny and the tribe.