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

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.