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.”

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

A poll sug­gests vac­cine da­ta boost­ed Pfiz­er's pub­lic im­age, but oth­er da­ta point to long road ahead

For much of the pharmaceutical industry, the pandemic presented an opportunity: to prove their value to the world and turn public opinion around on a business much of the country had come to disdain.

That theory — that helping pull the country from a pandemic could neutralize years of anger over high drug prices — was put to its biggest test this month, as three different drugmakers announced data from their Covid-19 vaccines, offering the first major evidence that industry-built inoculations could turn the tide of the outbreak in the US.

Covid-19 roundup: Rus­sia prices vac­cine 't­wo or more times cheap­er' than mR­NA shots; Sino­vac PhI­II da­ta ex­pect­ed in ear­ly De­cem­ber

The world can now purchase its first registered vaccine — at less than $10 per dose.

RDIF, the Russian sovereign wealth fund and an avid backer of Sputnik V, said the vaccine will be available internationally starting from March 2021. A two-dose regimen of the adenovirus-based vaccine, which it has said is more than 90% effective, will cost less than $20.

And they are not shy about inserting themselves right into a rivalry with Western frontrunners, namely Pfizer/BioNTech and Moderna.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.