Janet Woodcock, acting FDA commissioner (AP Images)

Janet Wood­cock slams calls for FDA 'fire­wal­l' amid ad­u­canum­ab de­ba­cle, cit­ing need for col­lab­o­ra­tive drug de­vel­op­ment

The FDA has long tak­en a col­lab­o­ra­tive ap­proach to work­ing with drug­mak­ers on ap­proval ap­pli­ca­tions, all in the in­ter­est, it says, of bring­ing the best mol­e­cules to mar­ket. But when the drug is a lemon, the cozy re­la­tion­ship be­tween reg­u­la­tors and in­dus­try courts easy blame.

Could a “fire­wall” be­tween reg­u­la­tors and drug­mak­ers pre­vent lousy drugs from get­ting the agency’s back­ing? Act­ing com­mis­sion­er Janet Wood­cock says no way.

In De­cem­ber, the ad­vo­ca­cy group Pub­lic Cit­i­zen is­sued a scathing state­ment on the agency’s re­view of Bio­gen’s snakebit Alzheimer’s med ad­u­canum­ab, say­ing neu­ro chief Bil­ly Dunn and his crew formed an “in­ap­pro­pri­ate close col­lab­o­ra­tion with Bio­gen.” The group pro­posed cre­at­ing a fire­wall be­tween agency staff to pre­vent sim­i­lar re­la­tion­ships from oc­cur­ring in the fu­ture, but Wood­cock shut down that idea in a Feb. 11 let­ter to Michael Car­ome, di­rec­tor of Pub­lic Cit­i­zen’s health re­search group.

“The fire­wall you pro­pose would sig­nif­i­cant­ly re­duce the ef­fi­cien­cy of FDA’s re­view process and cause de­lays in drug de­vel­op­ment,” Wood­cock wrote in part. “Nor is a fire­wall nec­es­sary to en­sure the in­tegri­ty of FDA’s de­ci­sion-mak­ing.”

Not on­ly would a fire­wall be need­ed, Pub­lic Cit­i­zen said at the time, but al­so a com­plete in­ves­ti­ga­tion of the ad­u­canum­ab re­view process by HHS. That in­clud­ed scru­ti­niz­ing the re­la­tion­ship be­tween Dunn and Bio­gen ex­ecs and ask­ing then-FDA com­mis­sion­er Stephen Hahn to re­move Dunn from his role un­til an in­spec­tor gen­er­al probe could be com­plet­ed.

Wood­cock did not ad­dress any oth­er spe­cif­ic de­mands or sug­ges­tions made by Pub­lic Cit­i­zen, but she em­pha­sized that the de­lays caused by such a fire­wall could se­ri­ous­ly ham­per the de­vel­op­ment process as well as pre­vent pa­tients from get­ting time­ly ac­cess to the drugs:

Not on­ly do FDA’s in­ter­ac­tions with spon­sors help en­sure that pre-clin­i­cal and clin­i­cal de­vel­op­ment pro­grams are ap­pro­pri­ate­ly de­signed to yield the da­ta that would be need­ed to sup­port an ap­pli­ca­tion, but al­so these in­ter­ac­tions re­duce the po­ten­tial for du­plica­tive or oth­er­wise un­nec­es­sary test­ing in hu­mans and an­i­mals. For FDA to make its ex­pec­ta­tions clear, staff in­volved in the re­view of ap­pli­ca­tions must have a thor­ough un­der­stand­ing of the de­vel­op­ment pro­gram, from the pre-clin­i­cal phase through the clin­i­cal phase.

Car­ome called Wood­cock’s let­ter “very de­fen­sive” in an emailed state­ment to End­points News. He said Wood­cock “of­fers full-throat­ed sup­port for the FDA’s in­ter­ac­tions with phar­ma­ceu­ti­cal com­pa­nies dur­ing the drug de­vel­op­ment process. She ex­tols the ben­e­fits of these in­ter­ac­tions and ig­nores their po­ten­tial down­sides, which were read­i­ly ap­par­ent in the agency’s re­view of ad­u­canum­ab as pre­sent­ed at the FDA ad­vi­so­ry com­mit­tee meet­ing for the drug last No­vem­ber.”

He went on:

Her re­jec­tion of our pro­pos­al that a fire­wall be cre­at­ed be­tween FDA staff in­volved in any pre-sub­mis­sion in­ter­ac­tions with spon­sors and FDA staff in­volved in the post-sub­mis­sion new drug ap­pli­ca­tion (NDA) or BLA re­view and de­ci­sion-mak­ing is not sur­pris­ing giv­en that she has fos­tered the ever clos­er re­la­tion­ship be­tween the FDA and the phar­ma­ceu­ti­cal in­dus­try since the ear­ly 1990s when PDU­FA was first en­act­ed.

Wood­cock, who ran CDER dur­ing the ad­u­canum­ab re­view, is now con­sid­ered a front-run­ner to take the reins at FDA on a per­ma­nent ba­sis. In an End­points poll con­duct­ed in mid-Jan­u­ary, rough­ly two-thirds of our read­ers want her to stay in her po­si­tion.

This all start­ed back in No­vem­ber when an ad­comm re­view­ing ad­u­canum­ab sur­pris­ing­ly vot­ed against the ex­per­i­men­tal drug by a near unan­i­mous mar­gin. The pan­elists al­so took the un­usu­al step of crit­i­ciz­ing the FDA and Dunn, say­ing their pre­sen­ta­tion was heav­i­ly weight­ed in Bio­gen’s fa­vor and ex­clud­ed dis­cus­sion of the harsh­ly crit­i­cal sta­tis­ti­cal re­view from FDA staffers.

Bil­ly Dunn

Dunn of­fered a full-throat­ed en­dorse­ment of the drug de­spite the sta­tis­ti­cal re­view con­clud­ing that the da­ta were con­flict­ing and made the case that the drug didn’t work. That en­dorse­ment, how­ev­er, out­raged some on the pan­el in­clud­ing Johns Hop­kins pro­fes­sor Caleb Alexan­der, who said the FDA pre­sen­ta­tion was “strik­ing­ly in­con­gru­ous” and open to elic­it­ing se­lec­tive in­for­ma­tion.

Ad­comm mem­bers al­so want­ed to know why Dunn was will­ing to ig­nore the typ­i­cal stan­dard and en­thu­si­as­ti­cal­ly back a drug that failed one of two piv­otal stud­ies.

Ad­u­canum­ab has long been one of the most con­tro­ver­sial pro­grams in phar­ma, with an­a­lysts go­ing back and forth over its pur­port­ed ef­fi­ca­cy. Bio­gen has main­tained that the drug would be the first to re­duce cog­ni­tive de­cline in Alzheimer’s pa­tients, but the com­pa­ny’s ap­proach to the da­ta — in­clud­ing co-au­thor­ing FDA re­view doc­u­ments — dis­turbed many sci­en­tists and out­side ob­servers.

The can­di­date’s PDU­FA date was de­layed last month to June 7, a move that sent Bio­gen $BI­IB shares spik­ing about 13% at the time.

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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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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Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slapdown, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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An Ar­ray co-founder re-emerges as CEO of a small aca­d­e­m­ic spin­out, look­ing to re­make an old class of can­cer drugs

Tony Piscopio hadn’t worked as a bench scientist in years when, around 2011, he got put in touch with a team at the University of Colorado trying to revitalize an old approach to treating cancer.

Piscopio, who had co-founded Array Biopharma before heading to South Korea to launch a new company, was back in the states, unattached and intrigued. He founded a three-person company with two professors, Xuedong Liu and Gail Eckhardt, and while they worked on the biology side, he returned to his old chemist chair and began drawing up potential compounds on a computer, along with manufacturing processes to make them. Outsourcing companies synthesized or analyzed the results.

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