As­traZeneca scores new goal on the pipeline front, adding its first AI-gen­er­at­ed tar­get to the port­fo­lio

As more and more bio­phar­mas de­vel­op ar­ti­fi­cial in­tel­li­gence plat­forms, the drug dis­cov­ery process is be­ing re­shaped to fit new goals on cut­ting down the prodi­gious amount of time, en­er­gy and mon­ey that go in­to a drug pro­gram. Now one of the most am­bi­tious play­ers in the dri­ve to im­prove on ROI, As­traZeneca, is mark­ing a mile­stone on that front by adding the first tar­get gen­er­at­ed by AI to its port­fo­lio.

The tar­get comes out of a col­lab­o­ra­tion with Benev­o­len­tAI, a Lon­don-based com­pa­ny that first start­ed work­ing with the big phar­ma back in April, 2019. Thanks to the biotech’s plat­form AI tech, As­traZeneca has iden­ti­fied and val­i­dat­ed a new way to at­tack chron­ic kid­ney dis­ease and will be­gin de­vel­op­ing com­pounds cen­tered on this tar­get.

This se­lec­tion fits in­to a broad­er AI strat­e­gy at As­traZeneca, the com­pa­ny’s re­nal bio­sciences chief told End­points News. And the plan isn’t to just lim­it its ap­pli­ca­tions to R&D.

“We’re in­vest­ing in AI and see it as a clear tool that can sup­port our de­ci­sions in drug dis­cov­ery,” Pernille Hansen said. “They can be not on­ly for dis­cov­er­ing new tar­gets, it can be in chem­istry, imag­ing and so forth. So there’s many, many pos­si­bil­i­ties to work with AI.”

But they’re still a long way out from prov­ing AI can of­fer tan­gi­ble ev­i­dence of an im­pact.

Pernille Hansen

Hansen de­clined to say what specif­i­cal­ly the tar­get is, what kinds of drugs will uti­lize it to treat CKD or the time­line for such drug de­vel­op­ment. The on­ly thing she’d dis­close is that it suits their re­nal and AI ap­proach­es, and that As­traZeneca re­searchers have giv­en the OK to ad­vance.

Kid­ney dis­ease is a fa­mil­iar area for the com­pa­ny, giv­en its stud­ies to ex­pand its block­buster Farx­i­ga drug in­to CKD over the last sev­er­al years. A Phase III tri­al in the dis­ease was halt­ed in March over what As­traZeneca deemed “over­whelm­ing ef­fi­ca­cy,” and the com­pa­ny re­leased topline re­sults in Ju­ly. The FDA has set a rough re­view date for some­time dur­ing the sec­ond quar­ter.

If Farx­i­ga does cross the fin­ish line in CKD, it would rep­re­sent a sec­ond ap­proval once thought un­like­ly for a drug class de­vel­oped for di­a­betes. The drug was ap­proved in May to re­duce the risk of car­dio­vas­cu­lar death or hos­pi­tal­iza­tion in heart fail­ure pa­tients with a re­duced ejec­tion frac­tion, and those with or with­out type 2 di­a­betes.

Ivan Grif­fin

Wednes­day’s tar­get, though, comes out of Benev­o­len­tAI’s plat­form, which es­sen­tial­ly forms the shape of a “knowl­edge graph,” COO Ivan Grif­fin told End­points News. Grif­fin’s com­pa­ny has spent years feed­ing its tech with all sorts of da­ta, from pro­teins and genes to re­sults pub­lished in sci­en­tif­ic jour­nals, and train­ing its al­go­rithm to make con­nec­tions that sci­en­tists may not have no­ticed at first.

One could vi­su­al­ize such the graph as an in­ter­con­nect­ed web of “nodes and edges,” Grif­fin said. Sci­en­tists take ad­van­tage of the AI-pre­dict­ed re­la­tion­ships and then in­ter­ro­gate them to see if it holds up, which is what hap­pened in As­traZeneca’s case.

“The strengths we have in the da­ta and AI and ma­chine learn­ing tech­nol­o­gy ap­proach match­es re­al­ly nice­ly, in this case, with what As­traZeneca was able to bring,” Grif­fin said. “By com­bin­ing the two, and then set­ting off to­geth­er to try to dis­cov­er tar­gets and drugs, that was the phi­los­o­phy be­hind start­ing the col­lab­o­ra­tion, and it’s very much felt like that all the way through.

In ad­di­tion to CKD, Benev­o­len­tAI and As­traZeneca are work­ing on de­vel­op­ing drugs for id­io­path­ic pul­monary fi­bro­sis as part of their col­lab­o­ra­tion.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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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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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepreneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film “Fantastic Voyage,” the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his and his co-founders’ own accounts, along with some seed cash from friends and family.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.