Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bris­tol My­ers Squibb is turn­ing to one of the star up­starts in the ma­chine learn­ing world to go back to the draw­ing board and come up with the dis­ease mod­els need­ed to find drugs that can work against two of the tough­est tar­gets in the neu­ro world.

Daphne Koller’s well-fund­ed in­sitro is get­ting $70 mil­lion in cash and near-term mile­stones to use their ma­chine learn­ing plat­form to cre­ate in­duced pluripo­tent stem cell-de­rived dis­ease mod­els for ALS and fron­totem­po­ral de­men­tia.

Then they’ll use those in­sights to start build­ing new drugs for those two ail­ments; a com­plex, ground-up ap­proach that has al­ready won a close al­liance with Gilead.

Suc­cess would trig­ger up to $2 bil­lion in mile­stones, run­ning a gamut of re­search and com­mer­cial goals.

Richard Har­g­reaves

“We be­lieve that ma­chine learn­ing and da­ta gen­er­at­ed by nov­el ex­per­i­men­tal plat­forms of­fer the op­por­tu­ni­ty to re­think how we dis­cov­er and de­sign nov­el med­i­cines,” said Richard Har­g­reaves, the chief of the neu­ro group at Bris­tol My­ers, who made the leap from Cel­gene.

Koller’s been mak­ing great strides with a new tech­nol­o­gy that has gained im­mense in­ter­est, but still has a long way to go to prove it­self as the ma­jor play­ers start fig­ur­ing out how to in­te­grate ar­ti­fi­cial in­tel­li­gence and ML in­to their game plans for drug dis­cov­ery and de­vel­op­ment.

As she told me in in­ter­views for the End­points 11 awards, the field has been in­tense­ly hyped by a slew of new play­ers. From her per­spec­tive, the tech holds lots of promise, but there’s a tremen­dous amount of ground­work that has to be done to glean the nec­es­sary da­ta for it to work. And it starts with the right dis­ease mod­els.

I’ve looked for in­stance at cel­lu­lar phe­no­types and I’ve built a mod­el that aligns those with hu­man clin­i­cal out­come: Can I take a group of ge­net­ic back­grounds that I’ve nev­er seen be­fore and pre­dict clin­i­cal out­comes for those pa­tients? That is a con­fi­dence build­ing mea­sure. It says I built a dis­ease mod­el that is ac­tu­al­ly pre­dic­tive of what we see in hu­mans. By the way, an­i­mal mod­els, they don’t even get asked that ques­tion in a lot of cas­es, which I find rather shock­ing.

On top of that, the ex-Stan­ford pro­fes­sor has been frank about un­der­stand­ing the tech bet­ter than drug de­vel­op­ment, a weak­ness she’s been work­ing on by re­cruit­ing top tal­ent from that oth­er side of the R&D di­men­sion. That strat­e­gy re­cent­ly led to the ar­rival of Mer­ck R&D chief Roger Perl­mut­ter on the board.

Koller told me:

The ear­ly days were very chal­leng­ing, I won’t lie. I was al­so ac­tu­al­ly new to the space…To build some­thing from scratch you need a net­work, you need to be con­nect­ed to peo­ple that you can re­cruit, you need to ask ad­vice. When I came in­to this I had none of that. So it was an in­cred­i­bly chal­leng­ing thing to just start from a blank slate in a space where you were a new­com­er and fig­ure out even how to go about build­ing a wet lab that I had nev­er built be­fore.

Now she’s work­ing with Har­g­reaves, one of the top neu­ro­sci­en­tists in the field, as Koller and her team con­tin­ue to build up the com­pa­ny.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Scoop: Google’s GV spear­heads the Spot­light syn­di­cate — back­ing an up­start biotech aimed at ‘de­moc­ra­tiz­ing’ gene edit­ing

CRISPR had no sooner started to shake the very foundations of drug development before its limitations began to loom large. Gene editing could change the world — if only you could get around the hurdles that threatened to trip up every program.

So it’s only natural to see CRISPR 2.0 taking shape before the pioneers can get the lead therapies through development. And who better than Google’s GV venture arm to take the lead spot in a small syndicate backing some scientists with their own unique twist on a solution?

FDA gives Rhythm the green light for set­melan­otide, a drug aimed at re­duc­ing obe­si­ty in cer­tain ge­net­ic dis­or­ders

A little over a year after completing successful pivotal trials, Rhythm Pharmaceuticals $RYTM has its first drug approval on its hands.

The Boston-based biotech announced Friday that the FDA gave setmelanotide the thumbs-up for three rare genetic disorders that result in obesity in patients six and older. It’s the agency’s first such approval, Rhythm said, with the indicated deficiencies being the POMC, PCSK1 and LEPR genes. Rhythm will market the drug as Imcivree, and plans to have it on the shelves in the first quarter of 2021.

Pascal Soriot (Getty)

As­traZeneca CEO So­ri­ot plans new study to test that con­tro­ver­sial 90% ef­fi­ca­cy fig­ure, wait­ing for US da­ta be­fore go­ing to FDA

Pascal Soriot spent the long Thanksgiving weekend digging AstraZeneca out of a hole, promising to put an end to the questions around its interim Phase III vaccine data by conducting a new study while going to regulators with a large part of what it already has.

AstraZeneca and its partners at Oxford had initially touted high-level results from two studies conducted in the UK and Brazil as positive. But the enthusiasm was soon shadowed by confusion as observers probed into how the highest, 90% efficacy was seen in a dosing regimen given to a small group of volunteers due to an error. Among a larger cohort given the intended shots, the vaccine was only 62% effective, a rate that would’ve been respectable had Pfizer/BioNTech and Moderna not posted efficacy rates of 94%, 95% for their mRNA candidates. And many weren’t sure what to make of the average 70% number that AstraZeneca ran in headlines.

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Robert Clarke (Kinaset)

Ki­naset launch­es with $40M and a JAK in­hibitor from Vec­tura's old pipeline

Kinaset Therapeutics is joining the search for a better severe asthma treatment, picking up where Vectura left off when it decided to clear house last year.

UK-based Vectura — which took a big hit when its most advanced candidate flopped in a Phase III asthma trial back in 2018 — recently shifted to a CDMO model, offloading all of its R&D programs. Robert Clarke, who’s worked on inhalable therapeutics for 21-plus years, had close contacts at the company and took a look at what they were offering. After doing some research, he was attracted by VR475, a pan-JAK inhibitor.

Stephané Bancel (Endpoints at JPM20)

Mod­er­na cal­i­brates fi­nal Covid-19 vac­cine ef­fi­ca­cy at 94.1% — and to­day it's gun­ning for the EUA

Nearly a year ago, as the coronavirus emerged in China, the NIH and four major companies bet on an unproven genetic technology as the best tool for developing a vaccine to stem the outbreak. Today, a second such vaccine is heading to the FDA.

Moderna said Monday that they will request an emergency use authorization from the FDA after a final analysis showed their mRNA vaccine was 94.1% effective at preventing symptomatic Covid-19. The data confirm the results from an interim analysis and matches efficacy Pfizer and BioNTech showed in a Phase III study, setting the biotech up to potentially nab one of the first two Covid-19 vaccine OKs.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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