Back­ers of Tes­la and SpaceX fund 29-year-old Al­ice Zhang's AI-pow­ered neu­ro­science start­up

The bat­tered field of neu­ro­science has a dogged new play­er: a team of young, rene­gade PhDs who say they’re har­ness­ing ge­nomics and ar­ti­fi­cial in­tel­li­gence to re­vive the ail­ing in­dus­try.

Al­ice Zhang

The San Fran­cis­co ven­ture is called Verge Ge­nomics, and it’s led by a spunky 29-year-old PhD dropout — Al­ice Zhang — who says she’s de­ter­mined to push neu­ro­science R&D out of its decades-long slump. Jump­ing to her cause is DFJ Ven­tures, a tech in­vestor who has the likes of Twit­ter, SpaceX, and Tes­la in its port­fo­lio. The VC firm led Verge’s $32 mil­lion Se­ries A round, which was an­nounced this morn­ing. It’s not DFJ’s first rodeo in the field, how­ev­er, as it’s al­ready backed ge­nomics A-lis­ters like Hu­man Longevi­ty and Syn­thet­ic Ge­nomics.

But it’s not just tech mon­ey that’s back­ing Verge. Join­ing DFJ are a num­ber of biotech in­vestors, in­clud­ing WuXi and the ALS In­vest­ment Fund. That’s be­cause Verge isn’t strict­ly a tech com­pa­ny, nor is it strict­ly a drug­mak­er, Zhang tells me. “We’re ex­act­ly in the mid­dle,” she says.

Verge is build­ing a mas­sive data­base — one of the largest in the field, they say — of ge­nom­ic pa­tient da­ta from ALS and Parkin­son’s pa­tients. The start­up is team­ing up with a dozen uni­ver­si­ties and gov­ern­ment or­ga­ni­za­tions to get their hands on pa­tient brain sam­ples from those who have passed away from neu­ro con­di­tions. Once at Verge, the brain sam­ples are se­quenced and the da­ta are fed to the com­pa­ny’s ma­chine learn­ing soft­ware. The plan, of course, is to cull some in­sights from this da­ta, then de­ter­mine some promis­ing drug tar­gets and build out the com­pa­ny’s pipeline.

“Neu­ro­science is at least a decade be­hind can­cer in terms of lever­ag­ing com­pu­ta­tion­al tech­niques and da­ta,” Zhang says. “In ad­di­tion to ma­chine learn­ing, we’re go­ing through a re­nais­sance in neu­ro­bi­ol­o­gy with ad­vances in sin­gle cell se­quenc­ing and our un­der­stand­ing of the cir­cuit­ry of the brain.”

Bring­ing to­geth­er these ad­vance­ments in bi­ol­o­gy and tech­nol­o­gy, Zhang says she thinks neu­ro­science can progress to­ward pre­ci­sion med­i­cine the way can­cer al­ready has. The com­pa­ny says it al­ready has lead pro­grams in ALS and Parkin­son’s, and plans to tack­le Alzheimer’s as well.

I asked Zhang if she re­ceived any op­po­si­tion to her “AI-pow­ered drug dis­cov­ery” pitch while fundrais­ing — es­pe­cial­ly from in­vestors con­cerned about hype vs. re­al­i­ty.

“There’s a lot of AI com­pa­nies out there, and that begs the ques­tion — is it hype or is it hope?” Zhang said. “The skep­ti­cism is war­rant­ed when some­one comes along and says they can fix a prob­lem that’s been his­tor­i­cal­ly chal­leng­ing.”

The way Verge fought the op­po­si­tion was to “gen­er­ate com­pelling da­ta that sup­ports our claims,” Zhang said. “We could do that on both the tech­nol­o­gy and the life sci­ence side be­cause we had good bi­o­log­i­cal hy­pothe­ses.” In sup­port of that, Zhang points to a pa­per Verge col­lab­o­rat­ed with USC on (pub­lished in Na­ture in 2018) that de­scribed how gene mu­ta­tion caused tox­i­c­i­ty to nerve cells, lead­ing to ALS and some forms of de­men­tia.

In Zhang’s view, the soft­ware-on­ly com­pa­nies — and the com­pa­nies that aren’t build­ing their own data­bas­es — are the risky bets. The AI com­pa­nies ris­ing to the top, she says, are ones that in­te­grate drug de­vel­op­ment with the soft­ware side. Af­ter all, a suc­cess­ful drug is the big mon­ey­mak­er.

“One of the rea­sons why ma­chine learn­ing and ar­ti­fi­cial in­tel­li­gence hasn’t had more trac­tion in drug dis­cov­ery is be­cause si­los ex­ist be­tween the com­pu­ta­tion­al side and drug dis­cov­ery side. It’s im­por­tant to have an in­te­grat­ed team sit­ting side-by-side to de­vel­op a drug.”

Emi­ly Melton

That’s what they’re all about at Verge. The 14-per­son com­pa­ny has 10 PhDs in fields like ma­chine learn­ing, neu­ro­science, drug de­vel­op­ment, ap­plied math, bio­physics, and sta­tis­tics. Join­ing the team’s board of di­rec­tors is DFJ part­ner Emi­ly Melton.

“The sub­stan­tial in­crease in da­ta vol­umes com­bined with the ap­pli­ca­tion of ma­chine learn­ing tools has the po­ten­tial to trans­form drug dis­cov­ery and de­vel­op­ment,” Melton said in a news re­lease. “We were com­pelled by the high-cal­iber and mul­ti­dis­ci­pli­nary team at Verge Ge­nomics and their vi­sion to lever­age the con­ver­gence of tech­no­log­i­cal and neu­ro­bi­o­log­i­cal ad­vances to dis­cov­er new ther­a­pies for these com­plex dis­eases.”

Im­age: The team at San Fran­cis­co’s Verge Ge­nomics. VERGE GE­NOMICS

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

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But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

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It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

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We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

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Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

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Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.