Alice Zhang (Verge Genomics)

Eli Lil­ly makes mod­est bet on 'all-in-hu­man' dis­cov­ery out­fit for ALS drugs, deep­en­ing bid in pre­ci­sion neu­rol­o­gy

For years, Big Phar­ma has re­treat­ed from neu­rol­o­gy amid a se­ries of high-pro­file fail­ures in the big-tick­et in­di­ca­tions such as Alzheimer’s and ALS. But the tide has turned in re­cent years, and big drug­mak­ers like Eli Lil­ly have dou­bled down on their in­vest­ments in the space.

Now, look­ing to crack a new path to “pre­ci­sion neu­rol­o­gy,” Lil­ly is putting its name be­hind a ge­nomics play­er aim­ing for a nov­el path to neu­ro­log­i­cal dis­ease ther­a­pies.

Lil­ly will pay $25 mil­lion up­front and up to $694 mil­lion in biobucks for li­cens­ing rights to four po­ten­tial pro­grams from Verge Ge­nomics, a San Fran­cis­co-based dis­cov­ery out­fit boast­ing an “all-in-hu­man” dis­cov­ery and de­vel­op­ment plat­form us­ing AI to crunch through da­ta from the biotech’s pro­pri­etary brain tis­sue data­base, the part­ners said.

It’s a mod­est up­front in­vest­ment for Lil­ly and one with a game clock: The dis­cov­ery pact comes with a three-year term, adding some near-term stakes for Verge’s work.

The biotech is helmed by CEO Al­ice Zhang, who co-found­ed the ge­nomics play­er at the ten­der age of 26 back in 2015. She told End­points News the Lil­ly col­lab­o­ra­tion was a nat­ur­al off­shoot of Verge’s strat­e­gy to take as many of its in-house mol­e­cules through de­vel­op­ment as pos­si­ble. The ben­e­fit of hav­ing Lil­ly and its deep ex­pe­ri­ence in neu­rol­o­gy is an ob­vi­ous boost.

Where Verge seeks to dif­fer­en­ti­ate it­self is in its dis­cov­ery and pre­clin­i­cal ap­proach, which doesn’t re­ly on an­i­mal mod­els but rather us­es AI-de­vel­oped mol­e­cules test­ed in ex vi­vo hu­man neu­rons pri­or to clin­i­cal tri­als. Lean­ing on a grow­ing dataset of neu­ro ge­net­ic da­ta, Zhang en­vi­sions a fu­ture in which neu­rol­o­gy looks a lot like on­col­o­gy, with a se­ries of high­ly val­i­dat­ed path­ways of­fer­ing a re­al chance at de­vel­op­ing pre­ci­sion med­i­cines for pa­tients.

“One of the main val­ue propo­si­tions Lil­ly saw when they chose us as a part­ner is we had the abil­i­ty to ac­tu­al­ly iden­ti­fy ge­net­ic path­ways rather than in­di­vid­ual ge­net­ic mu­ta­tions that are shared across larg­er and more com­mer­cial­ly vi­able pa­tient pop­u­la­tions,” she said. “So we can find oth­er ‘nails’ for this kind of new ‘ham­mer’ of ge­net­ic ther­a­pies that we have.”

In terms of how Verge got on Lil­ly’s radar, CBO Jane Rhodes ex­plained the con­ver­sa­tion start­ed af­ter BIO 2020 with the drug­mak­er ex­press­ing in­ter­est in Verge’s dis­cov­ery plat­form and then its po­ten­tial in drug de­vel­op­ment. That se­ries of chit-chats, which in­clud­ed hash­ing out ex­act­ly how a deal would look, even­tu­al­ly turned in­to the pact you see to­day.

“We be­gan dis­cus­sions and quick­ly honed in a few key dis­eases, ul­ti­mate­ly fo­cus­ing on ALS,” Rhodes said. “We spent a lot of time get­ting to know each oth­er and re­al­ly fine tun­ing the agree­ment so it re­al­ly de­liv­ers on what Lil­ly needs and what is a strong strate­gic ad­van­tage for Verge as well in that it un­locks val­ue and tar­gets that we wouldn’t nec­es­sar­i­ly have tar­get­ed our­selves.”

Verge will not on­ly pur­sue the four can­di­dates along­side Lil­ly but al­so has a lead can­di­date in ALS the biotech hopes to hit the clin­ic by the end of the year, Zhang said. That drug in­hibits the PIK­fyve gene, which Verge said has shown a con­nec­tion in ALS. Verge will hold on­to rights for its lead pro­grams with Lil­ly hold­ing li­cens­ing rights for the four sep­a­rate can­di­dates.

Rhodes said Verge is look­ing to strike a bal­ance be­tween ad­vanc­ing its own in-house can­di­dates and bring­ing in out­side part­ners to bring oth­er mol­e­cules through the pipeline. In the mean­time, that could mean oth­er Big Phar­ma part­ners, but the sci­en­tif­ic fit is the ul­ti­mate de­cid­ing fac­tor.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.