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.”

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.

Ab­b­Vie, Scripps ex­pand part­ner­ship, for­ti­fy fo­cus on can­cer drugs

Scripps and AbbVie go way back. Research conducted in the lab of Scripps scientist Richard Lerner led to the discovery of Humira. The antibody, approved by the FDA in 2002 and sold by AbbVie, went on to become the world’s bestselling treatment. In 2018, the drugmaker and the non-profit organization signed a pact focused on developing cancer treatments — and now, the scope of that partnership has broadened to encompass a range of diseases, including immunological and neurological conditions.

South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

Cel­lar­i­ty, Flag­ship's $50M bet on net­work bi­ol­o­gy, mar­ries ma­chine learn­ing and sin­gle-cell tech for drug dis­cov­ery

Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team were floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a bottom-up approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.