Clin­i­cal tri­al spon­sors have to dis­close decade’s worth of un­re­leased da­ta, fed­er­al judge rules

A decade’s worth of un­re­leased tri­al da­ta may soon see the light of day.

A New York fed­er­al judge ruled this week that the FDA and the NIH have for years mis­in­ter­pret­ed a law that would re­quire com­pa­nies, uni­ver­si­ties and oth­er clin­i­cal tri­al spon­sors to re­lease tri­al da­ta from stud­ies com­plet­ed be­tween 2007 and 2017. The rul­ing cov­ers drugs and med­ical de­vices that were ex­per­i­men­tal when the study was com­plet­ed but have since been ap­proved, po­ten­tial­ly putting hun­dreds of spon­sors out of com­pli­ance if they don’t put their re­sults on clin­i­cal­tri­

The FDA had been in­ter­pret­ing a ‘fi­nal rule’ added to 2007 law, known as the Food and Drug Ad­min­is­tra­tion Amend­ments Act, to mean that spon­sors on­ly had to re­port re­sults for tri­als that were com­plet­ed af­ter the law rule was pro­mul­gat­ed in 2017. Plain­tiffs said it was retroac­tive.

“The court has set aside that er­ro­neous in­ter­pre­ta­tion of the law and has said that the statute means what it has al­ways said,” Christo­pher Morten, the lawyer for the plain­tiffs, told End­points News. “So our hope here is that tri­al spon­sors are go­ing to start, fi­nal­ly, af­ter years of non­com­pli­ance, re­port­ing some of that miss­ing da­ta to the pa­tients.”

The FDA and the NIH did not put out state­ments fol­low­ing the rul­ing and did not im­me­di­ate­ly re­spond to re­quests for com­ment.

FDAAA was the law that re­quired spon­sors to reg­is­ter most tri­als on clin­i­cal­tri­, and in 2018 the change went in­to ef­fect re­quir­ing that com­pa­nies post re­sults with­in a year of the tri­al’s com­ple­tion. The gov­ern­ment in­ter­pret­ed it to mean on­ly the re­sults of tri­als com­plet­ed af­ter the law went in­to ef­fect.

The FDA and NIH’s ex­e­cu­tion of that rule, though, has been the sub­ject of ma­jor scruti­ny in the last few months. In­ves­ti­ga­tions in The Lancet and in Sci­ence pub­lished in Jan­u­ary found that tri­al spon­sors have wide­ly ig­nored the re­port­ing re­quire­ments since 2018. The Lancet study, by Ben Goldacre, found on­ly 41% of over 4,700 tri­als re­port­ed on time.  An­a­lyz­ing a sim­i­lar dataset, Sci­ence’s Charles Piller found 45% non­com­pli­ance and iso­lat­ed a group of 30 “ha­bit­u­al of­fend­ers” who col­lec­tive­ly failed to re­port the re­sults 67% of the time.

Ad­vo­cates for greater trans­paren­cy say this kind of ob­fus­ca­tion hurts the field by giv­ing doc­tors and re­searchers on­ly a par­tial glimpse at the over­all re­sults. It can even be dan­ger­ous, Morten ar­gued. He cit­ed Vioxx, the painkiller Mer­ck pulled off the mar­ket in 2004 af­ter stud­ies showed an in­creased risk of stroke. Had Mer­ck dis­closed pre-ap­proval tri­al re­sults soon­er, Morten said, the risk could have been caught soon­er.

The case was brought by for­mer as­so­ciate FDA com­mis­sion­er Pe­ter Lurie and NYU jour­nal­ist Charles Seife, who ar­gued a mis­in­ter­pre­ta­tion of the rule has im­ped­ed their work more broad­ly. The rul­ing takes the re­port­ing re­quire­ments pre­vi­ous­ly ap­plied on­ly to tri­als com­plet­ed since 2018 and ap­plies them to those com­plet­ed since 2007.

“That’s hurt pa­tients who lack the op­por­tu­ni­ty to learn about the drugs they take, it’s hurt doc­tors that lack in­for­ma­tion on the drugs they pre­scribe, and it’s hurt re­searchers like Charles and Pe­ter,” Morten said.

It’s not clear, though, how the rul­ing will be en­forced. The plain­tiffs were over­ruled on a sep­a­rate is­sue, in which they ar­gued the FDA and NIH were not abid­ing by a statute re­quir­ing them to give pub­lic no­tice of non­com­pli­ance to com­pa­nies not abid­ing by the FDAAA rules.

The gov­ern­ment could yet ap­peal the rul­ing but it’s not yet clear if they will.

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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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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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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CMO Levi Garraway (Genentech)

Fo­cus­ing on the bright side, FDA OKs Roche's Actem­ra for rare lung dis­ease de­spite PhI­II flop

Actemra’s failure to hit the primary endpoint in a Phase III study didn’t stop the FDA from granting Roche priority review. And it’s certainly not standing in the way of a sixth approval for Roche’s IL-6 drug.

Regulators have cleared Actemra, or tocilizumab, for systemic sclerosis-associated interstitial lung disease in adult patients. Roche’s big Genentech subsidiary notes that it is the first biologic approved for this rare disease.

Af­ter three years of courtship (and turn­downs), Mer­ck pounced on the first glance of clin­i­cal da­ta in $1.85B Pan­dion takeover

It’s almost become cliché for biotech executives to talk about the importance of keeping your options open and being prepared to go all the way. But when it comes to negotiating with a giant like Merck, a little patience can indeed go a long way.

Just ask Pandion Therapeutics.

Days ago we already learned that Merck is shelling out $1.85 billion to pick up the biotech and its slate of autoimmune hopefuls. What we didn’t know until the SEC disclosure dropped Thursday is that the deal comes after Pandion turned down two other proposals from Merck over the past three years and held out until the last minute for a sweetened deal.

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