Third Rock, GV back Broad spin­out Cel­sius in bid to de­vel­op pre­ci­sion meds for au­toim­mune dis­ease

Third Rock is team­ing up with Google’s ven­ture arm to back a gene ther­a­py start­up step­ping out to­day with $65 mil­lion in new mon­ey. As you might ex­pect with GV’s in­volve­ment, the new ven­ture has a tech­nol­o­gy twist, and it might have its hands on some­thing big for the au­toim­mune world.

Vi­jay Kuchroo

Are you ready for a game of buzz­word bin­go? The start­up, called Cel­sius Ther­a­peu­tics, is us­ing new tech in ge­net­ic se­quenc­ing (check) — and a pro­pri­etary ma­chine learn­ing (check) al­go­rithm (check) — to tack­le pre­ci­sion meds (check) for can­cer (check) and au­toim­mune dis­ease. I’ll break that down for you lat­er.

The tech­nol­o­gy the com­pa­ny is built on was li­censed from the Broad In­sti­tute based on the work of Aviv Regev and Vi­jay Kuchroo (both found­ing sci­en­tists at Cel­sius), in­clud­ing non-ex­clu­sive li­cens­es to sin­gle-cell se­quenc­ing tech and ex­clu­sive li­cens­es to ear­ly-stage drug pro­grams.

The com­pa­ny’s co-founder and pres­i­dent, Christoph Lengauer (a Third Rock ven­ture part­ner), tells me Cel­sius has a few things that give it an edge: a mas­sive amount of an­i­mal and hu­man da­ta to work with, a ma­chine-learn­ing al­go­rithm to make sense of that da­ta, and a new tech­nol­o­gy to se­quence sin­gle cells rather than whole genomes.

Our body has tril­lions of cells, each with ge­net­ic in­for­ma­tion stored in­side. The sets of genes vary in dif­fer­ent cell types, de­ter­min­ing a cell’s func­tion — and some­times — the code be­hind dis­ease. Hom­ing in on sin­gle cells could help re­searchers bet­ter un­der­stand the in­di­vid­ual cells and their in­ter­ac­tions that cause dis­ease, per­haps lead­ing to bet­ter ther­a­pies down the road.

When com­par­ing sin­gle cell se­quenc­ing with whole genome se­quenc­ing, Lenguaer used an anal­o­gy about mak­ing smooth­ies with fruit.

“If you put straw­ber­ries and ki­wis in a blender, the col­or of your smooth­ie will be pink be­cause it’s the dom­i­nant cell pop­u­la­tion,” Lengauer said. “Any­thing re­lat­ed to the ki­wi would be lost. That’s whole genome se­quenc­ing. With this anal­o­gy, sin­gle cell se­quenc­ing would al­low you to see the in­di­vid­ual fruits in the smooth­ie. And if some­thing was rot­ten, you could pin­point the in­di­vid­ual fruit that’s gone bad.”

Of course, we’re not talk­ing of straw­ber­ries and ki­wis, but of cells that are caus­ing dis­ease and the genes that trig­ger their mal­func­tion. Cel­sius be­lieves this tech could be the key to bring pre­ci­sion med­i­cines to au­toim­mune dis­eases for the first time.

“Many dis­eases are dri­ven by the com­bined dys­func­tion of sev­er­al spe­cif­ic cell types, and the in­ter­ac­tions be­tween them,” said Regev, the MIT pro­fes­sor who co-found­ed Cel­sius, in a state­ment. “With tra­di­tion­al ge­nom­ic se­quenc­ing, we can­not iden­ti­fy these in­di­vid­ual con­tri­bu­tions — we on­ly see the av­er­age and can miss out key crit­i­cal caus­es. But for the first time, with the ap­proach­es dis­cov­ered by our team, where we com­bine mas­sive datasets of un­prece­dent­ed size and com­plex­i­ty with so­phis­ti­cat­ed ma­chine learn­ing al­go­rithms, we are able to dis­tin­guish the spe­cif­ic cells, among many oth­ers, that play a key role in dis­ease and iden­ti­fy the genes that are trig­ger­ing their mal­func­tion. We be­lieve our ap­proach will al­low us to more ef­fi­cient­ly iden­ti­fy spe­cif­ic tar­gets for treat­ing dis­eases in spe­cif­ic pa­tients and ul­ti­mate­ly de­vel­op med­i­cines for those tar­gets.”

Us­ing this ap­proach, the Broad In­sti­tute has un­cov­ered “a hand­ful” of tar­gets in au­toim­mune con­di­tions and can­cer. Lengauer said Cel­sius hopes to reach proof of con­cept in the next five years, and the Se­ries A should take them at least part­way to­ward that goal.

While Third Rock led the re­cent round, GV par­tic­i­pat­ed along with Her­itage Provider Net­work, Cas­din Cap­i­tal, Alexan­dria Ven­ture In­vest­ments and oth­ers.

The com­pa­ny, which has been op­er­at­ing in stealth mode for the past two years, em­ploys 15 peo­ple at its Cam­bridge head­quar­ters in Kendall Square.

Im­age: Aviv Regev, Third Rock part­ner Alex­is Borisy, Christoph Lengauer. Cel­sius Ther­a­peu­tics

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Image: Chris Varma. Frontier

UP­DAT­ED: Chris Var­ma un­veils MP­M's lat­est start­up — eye­ing 'un­drug­gable' can­cer tar­gets and pow­ered by ma­chine learn­ing, $67M

Two years af­ter MPM Cap­i­tal en­list­ed Chris Var­ma on its busy on­col­o­gy team, the for­mer en­tre­pre­neur-in-res­i­dence is un­veil­ing his first ven­ture project out of his new stomp­ing grounds in the Bay Area: Fron­tier Med­i­cines.

For Var­ma, who’s al­so co-found­ed Blue­print Med­i­cines and built com­pa­nies at Third Rock and Flag­ship, this marks an­oth­er op­por­tu­ni­ty to ap­ply some cut­ting-edge sci­ence to “sev­er­al of the most im­por­tant and dif­fi­cult tar­gets in can­cer” — tar­gets that oth­ers have tried to tack­le with more clas­si­cal meth­ods and failed. The launch round comes in at $67 mil­lion, which should go some way in scaf­fold­ing a pre­clin­i­cal pipeline and push one or more as­sets in­to the clin­ic three years from now, he tells me.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.