Joanna Shields, BenevolentAI CEO (Andreas Gebert/picture-alliance/dpa/AP Images)

Af­ter nab­bing two AI-gen­er­at­ed mol­e­cules, As­traZeneca re­turns to Benev­o­len­tAI with new col­lab­o­ra­tion

Rough­ly three years ago, As­traZeneca teamed up with Lon­don’s Benev­o­len­tAI to bring new drugs in­to its port­fo­lio us­ing the biotech’s AI and ma­chine learn­ing ca­pa­bil­i­ties. Now that the orig­i­nal deal has borne fruit, the two com­pa­nies are re-up­ping their col­lab­o­ra­tion.

As­traZeneca and Benev­o­len­tAI will work to­geth­er in two more dis­ease ar­eas: sys­temic lu­pus and heart fail­ure, the com­pa­nies an­nounced Thurs­day. It’s a three-year part­ner­ship that comes af­ter two can­di­dates dis­cov­ered by Benev­o­len­tAI for chron­ic kid­ney dis­ease and id­io­path­ic pul­monary fi­bro­sis were nom­i­nat­ed to As­traZeneca’s port­fo­lio last year.

No fi­nan­cials were dis­closed, but Benev­o­len­tAI CSO Anne Phe­lan told End­points News there would be an up­front pay­ment, an eq­ui­ty in­vest­ment and mile­stones.

The orig­i­nal part­ner­ship proved a sig­nif­i­cant mile­stone for As­traZeneca, as the CKD mol­e­cule nom­i­nat­ed last Jan­u­ary was the com­pa­ny’s first gen­er­at­ed by AI. At the time, As­traZeneca iden­ti­fied and val­i­dat­ed a new way to at­tack CKD thanks to the biotech’s plat­form AI tech, and be­gan de­vel­op­ing com­pounds cen­tered on this tar­get.

The IPF com­pound fol­lowed up just last month, and now marks the “per­fect time” to ex­tend the work­ing re­la­tion­ship, Phe­lan said.

Lu­pus and heart fail­ure “fall with­in [As­traZeneca’s] pri­or­i­ty ther­a­peu­tic ar­eas,” Phe­lan said. “They’re com­plex dis­eases, dif­fi­cult spaces to work in. It’s a tricky prob­lem that lends it­self well to AI and our ma­chine learn­ing ca­pa­bil­i­ties.”

At the heart of Benev­o­len­tAI’s tech­nol­o­gy is what Phe­lan de­scribes as a “knowl­edge graph,” which es­sen­tial­ly looks like a “hair­ball” net­work of thou­sands of da­ta points, or nodes, in­ter­con­nect­ed by mil­lions of lines or re­la­tion­ships. Such re­la­tion­ships are first iden­ti­fied by the ma­chine learn­ing al­go­rithms and can con­nect many dif­fer­ent and un­re­lat­ed dis­eases.

Anne Phe­lan

Benev­o­len­tAI is able to dif­fer­en­ti­ate it­self from the buzzy and grow­ing field of AI drug de­vel­op­ment be­cause it is dri­ven by a hy­poth­e­sis-first ap­proach rather than fo­cus­ing on phe­no­typ­ic screen­ing, Phe­lan added. Where­as com­peti­tors’ AI might be mak­ing new mol­e­cules and try­ing to re­verse-en­gi­neer the process, Benev­o­len­tAI starts from scratch.

“It’s not a right and wrong sit­u­a­tion … it’s a re­al­ly mul­ti­di­men­sion­al pic­ture of hu­man bi­ol­o­gy across the com­mon­al­i­ties of hu­man dis­ease,” Phe­lan said. “For ex­am­ple, de­pend­ing on the cell type that’s af­fect­ed, the graph could end up con­nect­ing to Alzheimer’s or di­a­betes, where un­der­ly­ing phys­i­ol­o­gy could be re­al­ly quite sim­i­lar.”

For As­traZeneca, lu­pus re­cent­ly turned in­to a po­ten­tial mon­ey­mak­er, as the Big Phar­ma whisked a new drug past the FDA fin­ish line last Au­gust in Saph­ne­lo. It’s the first new lu­pus ap­proval in at least the last 10 years, As­traZeneca said at the time, and re­duces over­all dis­ease ac­tiv­i­ty across or­gan sys­tems and oral cor­ti­cos­teroid use com­pared to place­bo.

Heart fail­ure, mean­while, is an area where As­traZeneca is look­ing to stake out a block­buster with Farx­i­ga. Back in 2020, the drug was ap­proved to re­duce the risk of car­dio­vas­cu­lar death or hos­pi­tal­iza­tion in heart fail­ure pa­tients with a re­duced ejec­tion frac­tion. It al­so picked up an ap­proval in CKD last April.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”