Ned Sharpless. NCI

Sharp­less out­lines FDA’s key pri­or­i­ties for ad­vanc­ing pub­lic health

Four months in­to his tenure, Act­ing FDA Com­mis­sion­er Ned Sharp­less sent an email to FDA staff on Tues­day out­lin­ing four main pri­or­i­ty ar­eas: as­sur­ing pub­lic health through emerg­ing tech­nolo­gies, in­vest­ing in the FDA’s next gen­er­a­tion ex­pert work­force and in­fra­struc­ture, mod­ern­iz­ing frame­works and pro­mot­ing and pro­tect­ing con­sumer and pa­tient safe­ty.

On the emerg­ing tech­nolo­gies front, Sharp­less dis­cussed how the FDA is try­ing to stay ahead of the curve while en­cour­ag­ing in­dus­try to adopt less cost­ly and more re­li­able ad­vanced man­u­fac­tur­ing plat­forms.

“We see the promise of tech­nolo­gies like 3D-print­ed de­vices, cell-cul­tured food, cell-based ther­a­peu­tics, and in­ten­tion­al ge­nom­ic al­ter­ations to an­i­mals and plants to de­liv­er enor­mous ben­e­fits to pub­lic health. As these fas­ci­nat­ing tech­nolo­gies are de­vel­oped, how­ev­er, the FDA will al­so need to aug­ment its ex­per­tise in these ar­eas to af­ford op­ti­mal re­view and reg­u­la­tion of prod­ucts de­rived from cut­ting-edge tech­nol­o­gy,” he wrote.

Sharp­less al­so de­clared hir­ing as an “im­me­di­ate pri­or­i­ty,” as oth­er of­fi­cials have lament­ed the gaps in the FDA’s work­force.

“We will be de­ploy­ing the 21st Cen­tu­ry Cures hir­ing au­thor­i­ty at much greater scale. Sim­ply put, we need to re­cruit, de­vel­op, and re­tain world- class peo­ple, and will be an­nounc­ing more spe­cif­ic plans in these ar­eas soon,” he wrote.

On mod­ern­iz­ing frame­works, Sharp­less touched on the FDA’s work to speed gener­ic drugs and biosim­i­lars to mar­ket to low­er drug prices and not­ed work to try to help in­dus­try with “the most ex­pen­sive part of drug dis­cov­ery and de­vel­op­ment,” which is the clin­i­cal test­ing.

“To ex­pe­dite de­vel­op­ment of ef­fec­tive ther­a­peu­tics and di­ag­nos­tics, we will con­tin­ue to pro­mote the use of in­no­v­a­tive clin­i­cal tri­al de­signs such as plat­form tri­als, bas­ket stud­ies, adap­tive tri­als, and prag­mat­ic ran­dom­ized con­trolled tri­als that are more ef­fi­cient and will low­er costs of run­ning tri­als,” he wrote.

Re­al-world ev­i­dence, a top­ic dis­cussed last week at an FDA event, will al­so help with pre- and post­mar­ket ad­verse event de­tec­tion and ef­fi­ca­cy analy­ses for clin­i­cal sit­u­a­tions where ran­dom­ized clin­i­cal tri­als may not be fea­si­ble.

“En­hanced use of re­al-world ev­i­dence for reg­u­la­to­ry de­ci­sion mak­ing will im­prove the gen­er­al­iz­abil­i­ty of ev­i­dence to pa­tient pop­u­la­tions that may be ex­clud­ed from tra­di­tion­al clin­i­cal tri­als,” he added.

As far as pa­tient safe­ty, Sharp­less re­it­er­at­ed the FDA’s in­ter­est in strength­en­ing post-mar­ket sur­veil­lance.

“For in­stance, nov­el ma­chine learn­ing al­go­rithms can al­low us to pri­or­i­tize in­ter­na­tion­al mail in­spec­tions to bet­ter de­tect il­lic­it opi­oid ship­ments; blockchain can help us track and trace food out­breaks with greater speed; and aug­ment­ed track and trace tech­nolo­gies can help us im­prove the safe­ty of drugs, de­vices, and vet­eri­nary prod­ucts,” he wrote.


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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Pfiz­er's Xalko­ri fol­low-up, which smoked the old­er drug in 1st-line pa­tients, scores dou­ble win at the FDA

Despite being the first to market in ALK-positive non-small cell lung cancer, Pfizer’s Xalkori languished on the shelf as other competitors outpaced it. Now, after Pfizer’s follow-up drug absolutely smoked Xalkori in first-line patients, the FDA is taking notice.

In one fell swoop, the agency approved Lorbrena as a first-line treatment for patients with anaplastic lymphoma kinase (ALK)-positive NSCLC and extended the drug’s 2018 accelerated approval to a full approval, Pfizer announced on Wednesday.

In the lat­est big in­vest­ment in gene ther­a­py man­u­fac­tur­ing, Bio­gen com­mits $200M to a ma­jor new fa­cil­i­ty in NC

You’d be forgiven for thinking that the only R&D effort of any consequence at Biogen belongs to aducanumab, its controversial Alzheimer’s drug. But behind the uproar around that drug, the big biotech has a full scale pipeline in play that includes a growing focus on developing gene therapies.

Now Biogen plans to build up the kind of manufacturing muscle that will give it an advantage in gaining FDA approvals — where CMC is always key — and then marketing them around the world.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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