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.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Gallia Levy, Spark CMO (Roche)

Spark Ther­a­peu­tics nabs new CMO from Genen­tech, fill­ing a ma­jor post-merg­er de­par­ture

Spark Therapeutics is getting a new CMO from their new owners.

The gene therapy company-turned-subsidiary has named Gallia Levy, who had been running rare blood disorders — including clinical development for their blockbuster-potential hemophilia antibody Hemlibra for Roche’s big biotech sub Genentech — to run medical affairs.

The appointment is a fitting one. Roche spent $4.8 billion to acquire Spark last year in large part to get their hands on their hemophilia gene therapy, SPK-8011, and expand the toe-hold Hemlibra gave them in an crowded hemophilia space.  It’s also a somewhat ironic appointment: The FTC held up the Spark acquistions for nearly a year, reportedly over concerns about the anti-trust implications of Roche owning both a top chronic treatment in Hemlibra and a top one-time treatment in Spark’s gene therapy.

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