Carolyn Magill, Aetion CEO (via YouTube)

Ae­tion push­es Se­ries B to $82M as pan­dem­ic puts re­al-world ev­i­dence un­der the spot­light

For­mer FDA chief Scott Got­tlieb’s fa­vorite re­al-world ev­i­dence plat­form is get­ting a new stream of cash, as it nears what will like­ly be the biggest test of the young com­pa­ny so far.

Ae­tion, the New York health tech start­up, has for the sec­ond time raised an ex­ten­sion to their Se­ries B, adding $19 mil­lion from J&J’s VC arm, Green­spring As­so­ci­ates and ED­BI to bring their round to $82 mil­lion. Got­tlieb, who cham­pi­oned re­al-world ev­i­dence as com­mis­sion­er and lat­er joined the com­pa­ny’s board, said in a state­ment that the new cash would help fu­el a glob­al ex­pan­sion.

The fund­ing comes as a pan­dem­ic has rapid­ly trans­formed ques­tions around the use­ful­ness of re­al-world da­ta from a sub­ject for bio­phar­ma wonks de­bat­ing the near-fu­ture of drug de­vel­op­ment, to an is­sue that has — of­ten du­bi­ous­ly — rapid­ly in­formed and changed treat­ment de­ci­sions around the globe. And it comes as Ae­tion, in part­ner­ship with the FDA and a long list of oth­er or­ga­ni­za­tions, looks to prove their plat­form can give ear­li­er and broad­er look at treat­ments for a nov­el virus, sep­a­rat­ing them from a field that ex­perts say has yet to turn in much use­ful in­fo and is filled with flawed ap­proach.

“You could ar­gue that nev­er have our in­sights been more im­por­tant,” CEO Car­olyn Mag­ill told End­points News. 

Re­searchers turned to re­al-world da­ta ear­ly in the pan­dem­ic for the first signs of what was work­ing and what wasn’t, but the re­sults of­ten left some­thing to be de­sired. Most no­tably, two in­flu­en­tial stud­ies that looked at hy­drox­y­chloro­quine use around the world were pulled from top jour­nals af­ter it emerged that some of the da­ta were flawed. Lat­er, Gilead used a re­al-world com­para­tor in one tri­al to try to prove remde­sivir im­proved sur­vival — a method prompt­ly pil­lo­ried by out­side ex­perts.

And just this past week, con­tro­ver­sy erupt­ed af­ter the FDA au­tho­rized con­va­les­cent plas­ma based on ob­ser­va­tion­al da­ta from tens of thou­sands of pa­tients who re­ceived it un­der an ex­pand­ed pro­to­col (which dif­fers slight­ly from re­al-world ev­i­dence plat­forms). The da­ta and analy­sis were re­gard­ed as sound, but re­searchers dif­fered on whether it met the bar for an au­tho­riza­tion.

Har­lan Krumholz

Asked about re­al-world da­ta dur­ing the pan­dem­ic, Yale car­di­ol­o­gist and health out­comes re­searcher Har­lan Krumholz said such analy­ses had “awe­some po­ten­tial” but that sci­en­tists still need­ed to agree on stan­dards that dif­fer­en­ti­ate good analy­ses from shod­dy ones.

“To date, there are few ex­am­ples [where] these types of stud­ies have tipped the bal­ance,” Krumholz, who di­rects Yale’s cen­ter for out­comes re­search and eval­u­a­tion, said via email

For Mag­ill, the hy­drox­y­chloro­quine de­ba­cle un­der­scored the need for plat­forms like Ae­tion’s, where there are guard-rails in place to en­sure da­ta is ex­tract­ed and an­a­lyzed in rig­or­ous ways.

“There are key as­pects of prin­ci­pled data­base epi­demi­ol­o­gy that we think should be in­cor­po­rat­ed in­to every study,” she said. “And I think what we saw with hy­drox­y­chloro­quine, for ex­am­ple, re­in­forces that no­tion.”

In April, Ae­tion an­nounced a part­ner­ship with HealthVer­i­ty to build a re­al-time treat­ment track­er, al­low­ing doc­tors and re­searchers to look at how Covid-19 prac­tice is chang­ing day-by-day. Re­cent­ly, Mag­ill said, the track­er spot­light­ed how quick­ly physi­cians adopt­ed dex­am­etha­sone af­ter a UK team showed it im­proved sur­vival in se­vere pa­tients.

In May, they teamed with the FDA to use re­al-world ev­i­dence to learn more about the Covid-19 dis­ease pro­gres­sion and how treat­ments and di­ag­nos­tics were be­ing used. The ef­fort emerged out of the Covid-19 Ev­i­dence Ac­cel­er­a­tor, a broad­er ini­tia­tive from the Rea­gan-Udall Foun­da­tion and Friends of Can­cer Re­search. Mag­ill said they are par­tic­u­lar­ly look­ing at dif­fer­ences in how the dis­ease pro­gress­es and how pa­tients from dif­fer­ent pop­u­la­tions are treat­ed.

So far, Ae­tion has pub­lished lit­tle on Covid-19: most no­tably a study with Brigham and Women’s Hos­pi­tal on ace in­hibitors and hos­pi­tal­iza­tion risk.  They have al­so looked at dif­fer­ences be­tween hy­drox­y­chloro­quine users and non-users, Mag­ill said. The di­rect use­ful­ness of that analy­sis is like­ly lim­it­ed — hy­drox­y­chloro­quine has been shown to have lit­tle ef­fect in mul­ti­ple tri­als and is no longer wide­ly used for Covid-19— but Mag­ill said it in­formed how they do fu­ture analy­ses and set up com­par­isons.

Now, the com­pa­ny is prepar­ing to aid vac­cine mak­ers on the post-ap­proval mon­i­tor­ing that the FDA and HHS have said will be es­sen­tial for en­sur­ing the safe­ty of a vac­cine. Mag­ill de­clined to dis­close which vac­cine pro­duc­ers it worked with, but the com­pa­ny has pre­vi­ous­ly an­nounced a part­ner­ship with Sanofi. And J&J was a con­trib­u­tor to their lat­est round.

The mon­i­tor­ing will present new chal­lenges in safe­ty mon­i­tor­ing, Mag­ill said, ones they are prepar­ing for now.

”You can imag­ine that these vac­cines are new, and any­time some­thing is new there is no prece­dent and da­ta could be col­lect­ed in dif­fer­ent ways, de­pend­ing on the health sys­tem, de­pend­ing on who’s ad­min­is­ter­ing the vac­cine, as an ex­am­ple, where you are ge­o­graph­i­cal­ly,” she said. “And all of that has im­pli­ca­tions on the re­al world da­ta.”

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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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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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.

Covid-19 roundup: RE­COV­ERY tri­al halts re­cruit­ment for colchicine study af­ter find­ing ‘no con­vinc­ing ev­i­dence’; Italy blocks As­traZeneca vac­cine ship­ment meant for Aus­tralia

It may be the end of the road for colchicine, an inexpensive oral anti-inflammatory drug commonly used to treat gout, as a potential Covid-19 treatment — at least in hospitalized patients.

The UK’s RECOVERY trial put out the word on Friday that it’s halting enrollment in its colchicine study after a data monitoring committee saw “no convincing evidence that further recruitment would provide conclusive proof of worthwhile mortality benefit either overall or in any pre-specified subgroup.”

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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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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