Tout­ing new way of min­ing neu­ro tar­gets, Take­da spin­out draws $45M from GV, Bill Gates, Fore­site

More than three years af­ter Take­da spun out its top neu­ro­science team in Cam­bridge, UK in­to a transat­lantic biotech, GV, Bill Gates and Fore­site Cap­i­tal are chip­ping in $45 mil­lion to pow­er the dis­cov­ery en­gine it was found­ed on.

Brad Mar­gus

Cere­vance has moved one pro­gram — for Parkin­son’s — in­to the clin­ic; the pro­ceeds should fund oth­ers, in­clud­ing sev­er­al de­signed to ad­dress neu­roin­flam­ma­tion in Alzheimer’s dis­ease. With an ex­pect­ed sec­ond close of the Se­ries B that should rough­ly dou­ble their take, the biotech should have four more drugs in hu­man tri­als be­fore they need to raise an­oth­er round, CEO Brad Mar­gus said.

But per­haps more im­por­tant­ly, the syn­di­cate is in­ter­est­ed in find­ing more tar­gets on Cere­vance’s NETSseq plat­form. Pi­o­neered by Nathaniel Heintz at the Rock­e­feller Uni­ver­si­ty, the tech­nol­o­gy pro­files post-mortem hu­man brain tis­sue sam­ples, map­ping out tran­scrip­tion­al and epi­ge­net­ic prop­er­ties of neu­rons and glial cells.

The com­pa­ny works with 14 brain banks around the world to as­sem­ble its brain tis­sue col­lec­tion — which now stands at 7,000 sam­ples in to­tal, in­clud­ing healthy con­trols. A look at their ex­ist­ing sam­ples could of­fer a hint on their next steps: Parkin­son’s (with or with­out de­men­tia), Alzheimer’s, Hunt­ing­ton’s, amy­otroph­ic lat­er­al scle­ro­sis and es­sen­tial tremor. Ad­di­tion­al donor sam­ples are planned for a range of CNS dis­or­ders from ma­jor de­pres­sion to pro­gres­sive supranu­clear pal­sy, ac­cord­ing to Cere­vance’s web­site.

Nathaniel Heintz

It goes one step fur­ther than the mouse-based work at En­voy, the last biotech found­ed by Heintz and Mar­gus. En­voy was sold to Take­da in 2012, and from its pipeline Cere­vance picked out a pre­clin­i­cal lead as­set that it has since ush­ered in­to Phase II.

“Know­ing that tar­get, we [turned to] the Cere­vance plat­form and looked at hu­man tis­sue and con­firmed that the tar­get was re­al­ly se­lec­tive­ly ex­pressed in this one cell type in the stria­tum in the brain that’s im­por­tant for Parkin­son’s,” Mar­gus told End­points News.

Just what pre­cise pro­tein CVN424 acts on, Cere­vance isn’t dis­clos­ing, ex­cept to em­pha­size that it is present in dopamine re­cep­tor D2-ex­press­ing medi­um spiny neu­rons — mean­ing the com­pound mod­u­lates the in­hibito­ry D2-de­pen­dent in­di­rect path­way but not the ex­ci­ta­to­ry D1-de­pen­dent di­rect path­way.

Sim­i­lar­ly, its oth­er (very ear­ly) Parkin­son’s and Alzheimer’s can­di­dates hit tar­gets se­lec­tive­ly ex­pressed on a par­tic­u­lar cell type.

“The plat­form is re­al­ly play­ing out where we had hoped,” Mar­gus said. “It’s re­veal­ing tar­gets that changed in dis­ease or that are se­lec­tive­ly ex­pressed in dis­eased cell types.”

Mark Carl­ton

All but one of its six pre­clin­i­cal pro­grams should ad­dress a tar­get that’s present in the over­all pa­tient pop­u­la­tion — and not a sub­set iden­ti­fied by a bio­mark­er. In neu­rode­gen­er­a­tive dis­eases, he added, cer­tain cell types ap­pear more vul­ner­a­ble than oth­ers: With Alzheimer’s, for in­stance, the hip­pocam­pus and the mem­o­ry cor­tex lose cells much ear­li­er in the dis­ease.

While Cere­vance has on­ly small mol­e­cule dis­cov­ery ex­per­tise in­ter­nal­ly, it’s open to part­ner­ing for tar­gets where an­ti­bod­ies, an­ti­sense oligonu­cleotides or even pro­tein de­graders would make more sense. The same goes for dis­ease ar­eas the 34-per­son team doesn’t have the time or en­er­gy for — in­clud­ing one PhI drug that they li­censed from Take­da.

“We ab­solute­ly are about find­ing great tar­gets, not about lim­it­ing to one modal­i­ty,” Mar­gus said.

Take­da is both a part­ner and an in­vestor, hav­ing signed on for a re­search al­liance ap­ply­ing the tech in gas­troin­testi­nal dis­or­ders (a project over­seen by Mark Carl­ton, a vet­er­an of the Japan­ese phar­ma and now Cere­vance’s CSO). It re­turned for the Se­ries B along­side Light­stone Ven­tures and the De­men­tia Dis­cov­ery Fund.

“A few months ago we thought we were gonna have to not be able to do any­thing in Chi­na and do every­thing in the US or UK,” he said. “Now it’s the oth­er way around, where we’re push­ing more of our work to Chi­na.”

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.

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A P val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a P value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.

Noubar Afeyan, Flagship founder and CEO (Victor Boyko/Getty Images)

UP­DAT­ED: Flag­ship launch­es Sen­da Bio­sciences with $88M in back­ing, look­ing to pi­o­neer the field of 'In­ter­sys­tems Bi­ol­o­gy'

Flagship Pioneering has a fresh company out this week, one that aims to lay the groundwork for a whole new discipline.

Senda Biosciences launched Wednesday with $88 million in Flagship cash. The goal? Gain insights into the molecular connections between people and coevolved nonhuman species like plants and bacteria, paving the way for “Intersystems Biology.”

Guillaume Pfefer has been tapped to run the show, a 25-year biotech veteran who comes from GSK after leading the development of the company’s shingles vaccine.

Q32 Bio grabs $60M to kick off hu­man stud­ies for next-gen com­ple­ment drugs — with some Covid-19 tweaks along the way

For a company that launched in the early months of the pandemic, Q32 Bio had its fair share of run-ins with the new normals under Covid-19.

The original plan, for instance, was to conduct first-in-human studies of the IL-7 receptor antibody it licensed from Bristol Myers Squibb in the Netherlands. But they realized shortly after that while the country was beginning to open up clinical trials, there were additional restrictions on drugs that tampered with immunological mechanisms.

Konstantin Poukalov

Per­cep­tive re­cruits A-list in­vestors to back its in-house Chi­na start­up with a mam­moth $310M raise

It took two years for Perceptive Advisors to conceive and boot up LianBio, its big bet on a new kind of in-licensing model for China, seeding it with enough cash to set up two anchoring deals with MyoKardia and BridgeBio. The result was a startup that was all ready to go, reaping $310 million just a little over two months after official launch.

Homegrown Chinese biotechs — many of them boasting of US ties and execs with overseas credentials — have been raking in mega-venture rounds in 2020, both from influential local backers and overseas VC firms that have been loading up new cash. As with IPOs, the deal flow might be slower but the amounts are often more staggering. LianBio’s latest round, unusually, is branded both a Series A and crossover.

Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.

CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,800+ biopharma pros reading Endpoints daily — and it's free.