Sarep­ta crit­ic Jenk­ins is ex­it­ing the FDA, and Wood­cock takes di­rect charge of the OND af­ter a painful rup­ture

John Jenk­ins

A few weeks ago, John Jenk­ins, the long­time di­rec­tor of the FDA’s Of­fice of New Drugs, caused quite a stir with some ad­vice for biotechs to avoid the same reg­u­la­to­ry path­way adopt­ed by Sarep­ta, the new win­ner of a con­tro­ver­sial FDA ap­proval for Ex­ondys 51 for Duchenne mus­cu­lar dy­s­tro­phy.

“Path tak­en by Sarep­ta NOT a good mod­el for oth­er de­vel­op­ment pro­grams,” de­clared one of Jenk­ins’ talk­ing points un­der a fi­nal sec­tion marked “lessons learned” from eteplirsen and oth­er cas­es.

In the fu­ture, he not­ed in his pre­sen­ta­tion, de­vel­op­ers would be held to a high stan­dard when it came to new mar­ket­ing ap­provals; Sarep­ta was the ex­cep­tion that proved the rules. And any­one who thought about fol­low­ing Sarep­ta’s path at the FDA, he added, was go­ing down the wrong trail.

This morn­ing, though, CDER di­rec­tor and Sarep­ta cham­pi­on Janet Wood­cock spread the word that Jenk­ins is re­tir­ing from the FDA in ear­ly Jan­u­ary — and she’s tem­porar­i­ly as­sum­ing his po­si­tion.

Janet Wood­cock

“We will con­duct a na­tion­al search to fill John’s po­si­tion,” Wood­cock notes, af­ter a lengthy sum­ma­ry of his work at the agency. “Dur­ing this time of tran­si­tion, I will serve as act­ing di­rec­tor of OND. I will con­tin­ue to en­gage in dis­cus­sions with OND staff and work close­ly with OND lead­ers as we work to­geth­er to ad­dress sci­en­tif­ic ad­vances that will im­pact the new drug re­view pro­gram in the com­ing years.”

Jenk­ins’ de­par­ture comes af­ter a bru­tal show­down be­tween Wood­cock and top of­fi­cials at the FDA who fought hard against the ap­proval of Ex­ondys 51. Sarep­ta, its crit­ics say, nev­er came close to pro­vid­ing clear ev­i­dence of ef­fi­ca­cy and safe­ty for eteplirsen. But in the end, Com­mis­sion­er Robert Califf opt­ed to de­fer to the pow­er­ful Wood­cock, over­rul­ing the ob­jec­tions and deep split in­side the FDA about its role in su­per­vis­ing this ap­proval process.

Wood­cock has sig­naled her sup­port of tak­ing a more flex­i­ble ap­proach to drug ap­provals, leav­ing some tra­di­tion­al­ists aghast. But no mat­ter how peo­ple sided in the civ­il war that di­vid­ed reg­u­la­tors on Sarep­ta, there’s no doubt that Wood­cock’s al­ready con­sid­er­able clout at the FDA just grew by sev­er­al de­grees, leav­ing her in a po­si­tion to re­form the OND on her own terms as the FDA preps for the ar­rival of Don­ald Trump at the White House.

The most im­me­di­ate im­pact of Jenk­ins’ de­par­ture will be Wood­cock’s in­creased au­thor­i­ty at the FDA, says Michael Ga­ba, the fed­er­al pol­i­cy leader of the firm’s life sci­ences team at Hol­land & Knight. But the longterm fall­out may have pro­found im­pli­ca­tions for the bio­phar­ma busi­ness.

“There’s an in­ter­est­ing con­flu­ence of events tak­ing place that may very well shape the FDA’s fu­ture un­der the next Ad­min­is­tra­tion,” he notes in an email mes­sage. “I would agree that the Jenk­ins de­par­ture cer­tain­ly sig­nals a near-term con­sol­i­da­tion of Janet Wood­cock’s au­thor­i­ty at CDER. How that plays out will be a func­tion of who is se­lect­ed to serve as the next FDA Com­mis­sion­er.”

But, he adds, if the FDA al­lows more com­pa­nies to fol­low Sarep­ta’s lead, they could be set­ting the stage for a dam­ag­ing ero­sion of pub­lic con­fi­dence in the FDA — which the in­dus­try ben­e­fits from.

“I ex­pect that the op­po­si­tion ex­pressed by Jenk­ins and oth­ers with­in the FDA lead­er­ship around the Sarep­ta ap­proval will con­tin­ue among drug re­view­ers as they are bound to en­sure prod­ucts are safe and ef­fec­tive for their in­tend­ed use as sup­port­ed by sci­en­tif­ic da­ta,” Ga­ba adds. “And I think the drug in­dus­try has to be care­ful here. I can ap­pre­ci­ate that any one drug com­pa­ny may want to press its prod­uct through with fast track au­thor­i­ty when it can, and do so with the help of pa­tient ad­vo­cates. But as more drug de­vel­op­ers are suc­cess­ful in this man­ner, then the val­ue of an FDA ap­proval could very well be di­min­ished as post-mar­ket ad­verse events in­crease in the ab­sence of sol­id up-front end-point based sci­en­tif­ic da­ta. This could, over time I sus­pect, tar­nish the FDA’s ‘good house­keep­ing seal of ap­proval’ that in­dus­try as a whole should want to up­hold.”

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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