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

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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Adding mar­quee in­vestors, Black­Thorn bags $76M to back an AI-dri­ven strat­e­gy for pre­ci­sion neu­ro med­i­cine

As ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing loom ever larg­er in drug dis­cov­ery and de­vel­op­ment, a biotech op­er­at­ing at the “nexus” of tech­nol­o­gy and neu­ro­sciences has cashed in with $76 mil­lion in fresh fi­nanc­ing.

The big idea at Black­Thorn Ther­a­peu­tics is to do for neu­robe­hav­ioral dis­or­ders what ge­net­i­cal­ly tar­get­ed ther­a­py has done for on­col­o­gy: Re­de­fine pa­tient pop­u­la­tions by the un­der­ly­ing bi­ol­o­gy — dys­reg­u­lat­ed brain cir­cuits, or neu­rotypes — in­stead of symp­toms, there­by find­ing the pa­tients who are most like­ly to ben­e­fit at en­roll­ment phase.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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