Eli Lil­ly jumps in­to a $473M hunt with Sig­ilon for the Holy Grail in di­a­betes cell ther­a­pies

Stem cell ther­a­pies have gone through every stage of the long and painful hype cy­cle for emerg­ing drug tech­nolo­gies: Start­ing with wild­ly in­flat­ed ear­ly ex­pec­ta­tions through bit­ter dis­il­lu­sion­ment, a trough of de­spair and on to a slow and pa­tient re­cov­ery brought about by years of dili­gent re­search work in the lab.

Paul Wot­ton, who’s seen it all, be­lieves he can now see the off-the-shelf cell prod­ucts of the fu­ture — en­cap­su­lat­ed in what they’ve dubbed Afi­bromer plat­form tech — tak­ing shape at the end of the new­ly ris­ing curve.

As the found­ing CEO at Flag­ship-seed­ed Sig­ilon, a start­up launched last year in Cam­bridge, MA with a $23.5 mil­lion round, he’s now un­veil­ing one of the fi­nal re­main­ing pieces nec­es­sary for a come­back: An al­liance with a Big Phar­ma part­ner.

To­day Sig­ilon is an­nounc­ing a $473 mil­lion pact with Eli Lil­ly — which is step­ping out un­der a bold­er R&D chief in Dan Skovron­sky — to dri­ve their stem cell work on Type 1 di­a­betes in­to the clin­ic, with $63 mil­lion of that in an up­front and the rest divvied up with a goal of ful­ly fund­ing the de­vel­op­ment of a durable new ther­a­py for the dis­ease. Eli Lil­ly is al­so mak­ing an undis­closed eq­ui­ty in­vest­ment in the com­pa­ny.

“I think we’ve kind of dis­cov­ered the Holy Grail to make cell ther­a­py a re­al­i­ty,” says Wot­ton. 

While re­searchers fig­ured out a long time ago how to get stem cells to trans­form in­to in­sulin pro­duc­ing pan­cre­at­ic be­ta cells for di­a­bet­ics, get­ting them in­to the body to do their work with­out trig­ger­ing a se­vere im­mune re­ac­tion has been an in­sur­mount­able ob­sta­cle.

Bob Langer

But af­ter test­ing new tech out of the labs of MIT’s Bob Langer and Daniel An­der­son in ro­dents as well as non-hu­man pri­mates, Sig­ilon be­lieves it’s with­in a cou­ple of years of start­ing work on a hu­man clin­i­cal tri­al — a ma­jor step to­ward ob­tain­ing clear proof-of-con­cept da­ta. They be­lieve they can de­liv­er islet cells to the body, and keep it hid­den from the im­mune sys­tem, pre­vent­ing fi­bro­sis that can choke cells of nu­tri­tion and oxy­gen. And while they are now pi­o­neer­ing a ground­break­ing field, there are sol­id pre­clin­i­cal rea­sons to be­lieve that they can make the hur­dle in hu­mans.

“Bob Langer’s team screened 600 mol­e­cules that could evade the im­mune sys­tem,” says Wot­ton. And they be­lieve they are on to the kind of en­cap­su­lat­ed cell-based ther­a­pies that can be de­liv­ered in wa­ter sol­u­ble poly­mers — Langer’s sweet spot in the lab — nec­es­sary for de­vis­ing a ther­a­py that could cre­ate a re­li­able sup­ply of in­sulin for pa­tients.

They’re not alone here. Har­vard spin­out Sem­ma re­cent­ly gar­nered a $114 mil­lion megaround to see if their “tea bag” tech for pro­duc­ing in­sulin can do the same thing.

It won’t be quick. There’s no hard time­line on the di­a­betes side, but Sig­ilon is with­in a cou­ple of years of mov­ing a pro­gram in­to the clin­ic us­ing the same tech plat­form to spawn fac­tor VI­II and IX for he­mo­phil­ia pa­tients. And that could help pave the way to a clin­i­cal study in di­a­betes soon af­ter.

“What we have is plat­form tech­nol­o­gy that you can ap­ply to any al­lo­gene­ic (off the shelf) cell line,” says Wot­ton, “pro­gram cells to man­u­fac­ture fac­tor VI­II or IX, or man­u­fac­ture an­ti­bod­ies, with an abil­i­ty to avoid de­tec­tion of the im­mune sys­tem.”

One way to think of it, he says, is as a kind of “gene ther­a­py in a box,” able to ex­press two pro­teins with an op­tion of top­ping up the dose if need­ed lat­er.

How long will that kind of ther­a­py last? Wot­ton doesn’t know the an­swer to that yet, but he’s gam­bling that it’s years rather than months. And Eli Lil­ly, which is in­creas­ing their bet on bold re­search gam­bles, wants to come along for the ride.

This is a big deal for the cell ther­a­py field in re­gen­er­a­tive med­i­cine, as the big play­ers large­ly bowed out of the are­na years ago, sat­is­fied that they were far, far away from any com­mer­cial pro­grams. Eli Lil­ly’s re­turn, along­side a same-day move by Roche we are re­port­ing now, could be an­oth­er sign that the hype cy­cle is fi­nal­ly en­ter­ing the be­gin­ning of the end.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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