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.”

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; 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.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Eu­ro­pean study finds that Gilead­'s Covid-19 an­tivi­ral remde­sivir shows no clin­i­cal ben­e­fit

Gilead’s remdesivir — or Veklury, as it’s marketed in the US — raked in around $2.8 billion last year as the only FDA-approved antiviral to treat Covid-19. But new data from a European study suggest the drug, which has been given to about half of hospitalized Covid patients in the country, has no actual benefit.

The open-label DisCoVeRy trial enrolled Covid-19 patients across 48 sites in Europe to test a handful of treatments, including remdesivir, lopinavir–ritonavir, lopinavir–ritonavir and interferon beta-1a, and hydroxychloroquine. To participate, patients had to show symptoms for seven days and require oxygen support. A total of 429 patients were randomized to receive remdesivir plus standard of care, while 428 were assigned to standard of care alone.

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Covid-19 roundup: FDA re­veals boost­er ad­comm ques­tion; Eli Lil­ly's an­ti­body cock­tail cleared for pre­ven­tion

The FDA released briefing documents this week from the agency and Pfizer each outlining their arguments for today’s Covid-19 booster shot adcomm, but one thing conspicuously missing was the question on which panel members would be voting. But late Thursday night, regulators published that question.

Adcomm members will be asked whether or not the safety and efficacy data from Pfizer/BioNTech’s original Phase III study “support approval” of a booster shot at least six months after the second dose in individuals older than 16. The question notably excludes the real-world data from Israel and other analyses that Pfizer and the Biden administration had said would be a centerpiece of their arguments for boosters.