Thumbs Up/Thumbs Down: The FDA dam­aged it­self bad­ly with the Sarep­ta OK

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

  Califf’s de­ci­sion to bow to Janet Wood­cock on Sarep­ta hurts the en­tire biotech in­dus­try.

Janet Wood­cock’s de­ci­sion to over­ride the caus­tic in­ter­nal crit­i­cism of Sarep­ta’s weak and at times mis­lead­ing case for its Duchenne drug eteplirsen has dam­aged the FDA in ways that will leave a mark for years to come. Over­rul­ing her col­leagues – in­clud­ing se­nior FDA of­fi­cials — to pro­vide an ac­cel­er­at­ed ap­proval this week for this ap­pli­ca­tion sends a clear mes­sage to biotech: If you can muster enough pub­lic sup­port for a cam­paign – which in­cludes an er­ro­neous study for bait — you should pro­ceed full steam ahead. To his cred­it, FDA com­mis­sion­er Robert Califf post­ed his own re­view of the case as well as the heat­ed ob­jec­tions of two top reg­u­la­tors, in­clud­ing the cur­rent act­ing chief sci­en­tist. Not to his cred­it, Califf chose to stand down in “def­er­ence” to Wood­cock’s in­tran­si­gence, but not be­fore chid­ing Sarep­ta for pro­mot­ing a study that raised false hopes in the pa­tient com­mu­ni­ty, which swal­lowed it all hook, line and sinker. He was al­so none too hap­py that Sarep­ta stayed fo­cused on its lob­by­ing cam­paign rather than the clin­i­cal work that need­ed to be done. I sin­cere­ly hope that eteplirsen does every­thing that ad­vo­cates de­vout­ly be­lieve it does, with­out pre­sent­ing the dan­gers cit­ed for a “sci­en­tif­i­cal­ly el­e­gant place­bo.” And let’s hope pa­tients aren’t left to cov­er any of the $300,000 an­nu­al cost. Pay­ers, and ul­ti­mate­ly so­ci­ety, can and should do that now. And what hap­pens next? Does the FDA now slap back the next com­pa­ny to use sim­i­lar tac­tics, to try and reestab­lish old bound­aries? Or does it low­er its stan­dards per­ma­nent­ly, en­cour­ag­ing more large demon­stra­tions and dodgy da­ta to back weak ap­pli­ca­tions? That’s a po­si­tion the FDA should nev­er have been put in. And that is on Janet Wood­cock and Robert Califf.

 The im­muno-on­col­o­gy rev­o­lu­tion will cause lots of pain, but it will be worth it.

We are liv­ing at the start of the im­muno-on­col­o­gy rev­o­lu­tion, and it’s been a re­al priv­i­lege cov­er­ing the ex­plo­sion of deals and col­lab­o­ra­tions that are be­ing struck now. Every day it seems brings one, or two, new I/O pacts. This week there was iOmx’s start­up round to fund the hunt for new check­point in­hibitors. Genen­tech struck a po­ten­tial­ly ground­break­ing part­ner­ship with BioN­Tech on us­ing mes­sen­ger RNA to de­vel­op per­son­al­ized can­cer vac­cines. With the three lead­ing check­points on the mar­ket and more com­ing, the po­ten­tial to part­ner on new com­bos is dri­ving bil­lions of dol­lars in fresh in­vest­ments. True, any­thing this hot will breed an ex­cess of al­ter­na­tives. In­vestors will get burned. The shake­out will have to oc­cur even­tu­al­ly and not every­one will suc­ceed. Many, most, will fail or fall by the way­side. But it’s been fun watch­ing the spot­light grow big­ger and more in­tense every week. I was chat­ting with an­oth­er CEO at a US start­up in this field ear­li­er in the week, and he be­lieves that we’re still just at the be­gin­ning of this i/o era. This will go on for an­oth­er 20 years, he says. And he’s like­ly right. What a great time to be in biotech.

 Set­backs hap­pen. It’s time for the big com­pa­nies to be more trans­par­ent about them.

Why do so many Big Phar­ma com­pa­nies have such a hard time talk­ing about a set­back? In re­cent times Am­gen nev­er missed a beat when it came to tout­ing their kid­ney dis­ease drug Parsabiv (etel­cal­ce­tide), and then the FDA un­ex­pect­ed­ly re­ject­ed it and the big biotech shut down, adopt­ing the tur­tle de­fense in re­fus­ing to even at­tempt to ex­plain it. No­var­tis likes to slip bad news in­to quar­ter­ly re­ports, in­clud­ing a line item on the re­cent re­jec­tion of one of its biosim­i­lars. And this week J&J could on­ly ac­knowl­edge, af­ter I con­tact­ed them, that the FDA had placed a hold on one of its can­cer drugs. They de­clined to ex­plain the se­ri­ous ad­verse events cit­ed on clin­i­cal­tri­ that forced reg­u­la­tors’ hands. I’ve said it be­fore and no doubt will have plen­ty of oc­ca­sions to do it again, but com­pa­nies run­ning drug stud­ies have a re­spon­si­bil­i­ty to ex­plain these sna­fus in some de­tail. They owe it to their in­vestors, the peo­ple who sign up for all of their clin­i­cal stud­ies and the sci­en­tists try­ing to make safe progress in the same field. There’s far too lit­tle trans­paren­cy in drug de­vel­op­ment. At a time when the in­dus­try is look­ing for some un­der­stand­ing from the pub­lic on the cost of in­no­va­tion, big play­ers should al­so rec­og­nize that they can be lead­ers in own­ing up to what can go wrong in de­vel­op­ing a new drug. It won’t hurt as much as they ev­i­dent­ly think it will.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.