Alicia Secor (Atalanta)

Genen­tech and Bio­gen roll the dice on an up­start biotech with a new siR­NA ap­proach — go­ing deep in­to the brain for tar­gets in neu­rode­gen­er­a­tion

Ata­lan­ta isn’t your av­er­age stealth biotech mak­ing a de­but. They have some his­to­ry and deals to sketch out in rough out­lines.

The found­ing ex­ecs date the silent launch all the way back to mid-2019, with the pub­li­ca­tion of a sem­i­nal study look­ing at a new siR­NA strat­e­gy for neu­rode­gen­er­a­tion. And in­stead of scout­ing for their first part­ners, the fledg­ling biotech can al­ready count 2 promi­nent play­ers as al­lies in the ex­ten­sive pre­clin­i­cal work that’s been done to es­tab­lish the plat­form.

Just be­cause they’ve been as­sem­bling the pieces for well over a year doesn’t mean they’re ready to spill all the tech beans they’re work­ing with. But enough is on dis­play to arouse plen­ty of ad­di­tion­al in­ter­est in what hap­pens from here.

Aimee Jack­son

“What was ob­served in the pre­clin­i­cal re­search that was done in the lab of our founder Anas­ta­sia Khvoro­va and pub­lished in 2019 was the abil­i­ty of this unique branched siR­NA struc­ture now to over­come that bar­ri­er and get ac­cess to the deep brain re­gions,” says Aimee Jack­son, who worked on RNAi oligonu­cleotide ther­a­pies at mi­Ra­gen and has stepped in as CSO at the start­up. “So we think now we fi­nal­ly have the op­por­tu­ni­ty to un­lock these ar­eas and now ac­tu­al­ly ap­proach so­lu­tions to neu­rode­gen­er­a­tive in­di­ca­tions dri­ven by de­gen­er­a­tion in those deep­er brain struc­tures.”

Khvoro­va, who runs an epony­mous­ly named lab at UMass Med­ical School, not­ed in her ab­stract that gene si­lenc­ing, RNAi’s big promise, had nev­er been achieved in the brain us­ing small in­ter­fer­ing RNA, or siR­NA. Her team, how­ev­er, had cre­at­ed an ar­chi­tec­ture that had a “ro­bust” af­fect in the brain, achiev­ing “sus­tained gene si­lenc­ing in the cen­tral ner­vous sys­tem of mice and non­hu­man pri­mates fol­low­ing a sin­gle in­jec­tion in­to the cere­brospinal flu­id.”

Anas­ta­sia Khvoro­va

Us­ing two linked siR­NAs, the “branched” tech Ata­lan­ta boasts of, the re­searchers “in­duced the po­tent si­lenc­ing of hunt­ingtin, the causative gene in Hunt­ing­ton’s dis­ease, re­duc­ing mes­sen­ger RNA and pro­tein through­out the brain. Si­lenc­ing per­sist­ed for at least 6 months, with the de­gree of gene si­lenc­ing cor­re­lat­ing to lev­els of guide strand tis­sue ac­cu­mu­la­tion. In cynomol­gus macaques, a bo­lus in­jec­tion of di-siR­NA showed sub­stan­tial dis­tri­b­u­tion and ro­bust si­lenc­ing through­out the brain and spinal cord with­out de­tectable tox­i­c­i­ty and with min­i­mal off-tar­get ef­fects. This siR­NA de­sign may en­able RNA in­ter­fer­ence-based gene si­lenc­ing in the CNS for the treat­ment of neu­ro­log­i­cal dis­or­ders.”

You can learn more about her work on the UMass web­site on in­ven­tions, il­lus­trat­ing how the tech in­volv­ing “branched oligonu­cleotide struc­ture im­proves the lev­el of tis­sue re­ten­tion in brain by more than 100 fold com­pared to non-branched com­pounds of iden­ti­cal chem­i­cal com­po­si­tion. The in­ven­tion dis­clos­es branched oligonu­cleotides, specif­i­cal­ly di-branched as­sy­met­ric ful­ly mod­i­fied siR­NAs, ex­hibit­ing great uni­form dis­tri­b­u­tion through­out the CNS and oth­er tar­get tis­sues, en­hanced cel­lu­lar up­take, min­i­mal im­mune re­sponse and off-tar­get ef­fects, with­out for­mu­la­tion.”

Craig Mel­lo

Khvoro­va is a key sci­en­tif­ic founder of this new biotech, along with Craig Mel­lo, her fa­mous col­league at UMass who won the No­bel Prize for his foun­da­tion­al work in RNAi and Neil Aronin, an ex­pert in Hunt­ing­ton’s dis­ease.

That last ex­pert ad­vis­er puts the spot­light on one of the lead­ing pro­grams at Ata­lan­ta. Bio­gen signed up to part­ner on that tar­get, while Genen­tech aligned it­self with the biotech on Parkin­son’s and the Big Kahu­na of neu­rode­gen­er­a­tion: Alzheimer’s.

Neil Aronin

F-Prime has been foot­ing the bill on the ear­ly work — all by it­self — with Stephen Knight join­ing the board. Knight and the old Fi­deli­ty team that joined to­geth­er in F-Prime have a long his­to­ry in Alzheimer’s in­vest­ment — none of it suc­cess­ful. They backed Deb­o­rah Dun­sire on her work at Fo­rum, which went bel­ly up be­fore they all went their sep­a­rate di­rec­tions.

F-Prime and the start­up’s 2 big part­ners have put up the first $110 mil­lion to get the com­pa­ny rolling, but the founders — led by Ju­niper vet Ali­cia Sec­or as CEO and em­ploy­ee #1 at Ata­lan­ta — aren’t telling me who did what.

Are there mile­stones at­tached? Yes. We can imag­ine the num­bers are large, but the CEO won’t say how large.

Sta­cie Weninger

Ac­cord­ing to Sec­or, it was Sta­cie Weninger, a neu­ro ex­pert who heads up the F-Prime Bio­med­ical Re­search Ini­tia­tive, who first came across the tech. She’s the one who brought in Knight, whose am­bi­tions in the field have widened con­sid­er­ably from the ini­tial fo­cus on Alzheimer’s.

There are a num­ber of star­tups that have come along to take a new crack at these big neu­rode­gen­er­a­tive tar­gets, dri­ven in part by the wide­spread car­nage in the clin­ic for every­thing that came ear­li­er. Now we can add Ata­lan­ta, at a time some of the ma­jors are lin­ing them­selves up for the sec­ond, or third waves.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Northway Biotech's new manufacturing facility in Greater Boston

North­way Biotech sets up shop in Boston hub, look­ing to court more cus­tomers with bi­o­log­ics-fo­cused plant

Getting a foot in the door in Boston’s bustling biopharma hub is a rite of passage for many companies, but it comes with a steep price tag. Lithuanian CDMO Northway — now with a new moniker — will set up a new plant in close proximity, and it’s hoping its biologics focus will find a willing customer base.

Northway Biotech (formerly Northway Biotechpharma) on Wednesday held a virtual grand opening ceremony for its $40 million Waltham, MA facility — a 30,000 square-foot cGMP manufacturing and process development plant that will widely expand on the company’s previous capabilities.

News brief­ing: Five pub­lic biotechs, over 2 days, raise $883M from fresh of­fer­ings; Bel­gian biotech ex­pands Se­ries B fund­ing

The wave of biotech IPOs we’ve been seeing in the last few days underscores that the public markets remain one of the key channels for fresh investments in drug R&D. And that trend was in full view this week as a slate of biotechs nailed down hundreds of millions of dollars in fresh funds.

One of the big winners of the week is Editas $EDIT, which nailed $231 million to back its pioneering work on a gene editing platform. The biotech sold 3.5 million shares at $66 each.

Mike Grey, Plexium chairman (Horizon Therapeutics)

Plex­i­um adds in­dus­try vet Mike Grey to the brain trust with new in­vestor cash fund­ing its pro­tein degra­da­tion play

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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