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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

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Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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