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.”

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two American drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend. The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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