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

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Pfiz­er re-ups on Mis­sion Ther­a­peu­tics col­lab­o­ra­tion, lead­ing a $15M round and grab­bing first dibs on DUBs

Seven years after Pfizer first invested in Mission Therapeutics, a biotech that researches selectively inhibiting deubiquitylating enzymes (DUBs), the pharma giant is re-upping its commitment to the company in another sign of confidence in the field of protein degradation.

Pfizer’s VC arm is heading up a $15 million round, announced Monday morning, and increasing its overall stake in Mission. Pfizer is also entering into a licensing agreement that would give it first dibs at negotiating exclusivity after accessing certain DUB inhibitors and screening them for their potential as drugs.

Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? Ac­tu­al­ly, An­nex­on was al­ready prepped and primed to toss its S-1 to Wall Street as in­vestors ral­lied

The Wall Street IPO shuffle generally calls for a little distance between the crossover ante and the Wall Street double, but with the window on the street wide open and biotech sizzling hot, who’s waiting?

The crew at Annexon didn’t leave anyone in suspense for long about their IPO plans. A day after the Bay Area biotech with clinical plans to target neurodegeneration quietly unveiled a $100 million raise, they were back with an S-1 outlining a pitch to double that — or more.

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Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Sec­ond PhI­II study for Idor­si­a's sleep drug re­turns pos­i­tive re­sults, but al­so rais­es new ques­tions

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.

UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.