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

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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