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.

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

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Ven­ture Cap­i­tal as a Strate­gic Part­ner: Fu­el­ing In­no­va­tion be­yond Fi­nance

The average level of investment required for a biotech start-up to succeed is increasing every year, elevating the pressure even further on venture capital to make smart financial investments. Financial investment alone, however, does not always guarantee that exciting innovations can be transformed into real businesses that make a meaningful difference to patients.

Beyond just capital

At Astellas Venture Management (AVM) – a wholly-owned venture capital organization within Astellas, headquartered in the San Francisco Bay Area – capital is just one of the ingredients we offer to add value to our biotechnology investments and partnerships. We generally take a strategic investor approach for companies in our invested portfolio, providing access to expertise, technology and/or resources in addition to the injection of finance. An equity investment from AVM can include access to Astellas’ research and development (R&D) capabilities and expertise, and a global network of partner academic institutions and biotechnology companies, to help advance and accelerate the start-up’s innovation.

Covid-19 roundup: In­ter­cept, blue­bird and a grow­ing list of biotechs feel the pain as pan­dem­ic man­gles FDA, R&D sched­ules

Around 100 staffers at Boston area hospitals have now tested positive for Covid-19, spotlighting the growing risk that the pandemic will sideline many of the most essential workers in healthcare as caseloads peak in the US and around the globe. With more than 3,400 deaths, Spain has become the latest country to surpass the official death count attributed to the new coronavirus in China, where the outbreak originated. As of Thursday morning, confirmed global cases had crossed 470,000 and the death count eclipsed 21,000.

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Af­ter crit­ics lam­bast­ed Gilead for grab­bing the FDA's spe­cial rare drug sta­tus on remde­sivir, they're giv­ing it back

Two days after Gilead won orphan drug status for remdesivir as a potential treatment for Covid-19, they’re handing it back.

The company was slammed from several sides after Gilead reported that the FDA had come through with the special status, which comes with 7 years of market exclusivity, the waiver of FDA fees and some tax credits as well. Typically, everyone who can get orphan status lands it without much of a fuss, but Democratic presidential candidate Bernie Sanders, Public Citizen and other consumer groups were outraged.

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Mod­er­na CEO Stéphane Ban­cel out­lines a short path for emer­gency use of a coro­n­avirus vac­cine

NIAID director Anthony Fauci has left no doubts that it takes 12 to 18 months to get a new vaccine tested and in commercial use, in the best of circumstances. But in times of a global emergency — like these — maybe there’s another, faster route to follow.

In an SEC filing on Tuesday, Moderna $MRNA staked out a record-setting pathway to getting their mRNA vaccine into the frontline of the healthcare response as early as this fall. The SEC filing notes that CEO Stéphane Bancel told Goldman Sachs that an emergency use approval could allow the vaccine to go to healthcare workers and certain individuals in a matter of months — presumably provided the NIH sees the safety and efficacy data they would need from the Phase I.

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Caught in a Covid-19 mael­strom, Eli Lil­ly locks down clin­i­cal tri­als as multi­bil­lion-dol­lar R&D ops de­rail

The Covid-19 pandemic has derailed Eli Lilly’s $6 billion R&D operations.

The pharma giant reported Monday morning that it has decided to hit the brakes on most new study starts and pause enrollment for most ongoing studies. Lilly adds that it is continuing dosing for ongoing studies, “but with study-by-study consideration.”

The pandemic has severely disrupted healthcare systems around the globe, says Lilly, making it difficult or impossible to conduct studies at many research sites. And there’s no timeline for when it expects to get back on track.

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UP­DAT­ED: Ver­tex joins Mer­ck, Pfiz­er — re­vamp­ing multi­bil­lion-dol­lar tri­al strat­e­gy as biotech R&D crum­bles

You can add Pfizer, Merck and — as we found out Friday morning — Vertex to the growing list of pharma giants hitting the pause button on a range of clinical trials. But not everyone in R&D is getting a red light.

Vertex says that it’s doing its best to keep working its pipeline strategy, coming up with a plan “to enable virtual clinic visits and home delivery of study drug to ensure study continuity and medical monitoring, and to facilitate study procedures.”

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Covid-19 roundup: Bris­tol My­ers sus­pends clin­i­cal tri­als, grounds field team; Vir ush­ers an­ti­body can­di­dates to hu­man test­ing

The global nature of the Covid-19 pandemic is manifesting more profoundly every day. With Spain’s death toll now surpassing China’s and India on full lockdown, the number of confirmed cases around the world has exceeded 436,000 while recoveries edged close to 112,000.

While the outbreak derails R&D at another pharma giant, several drugmakers have some encouraging updates on both experimental and repurposed molecules. Philanthropic campaigns in anticipation of the economic fallout continue. An Australian biotech is taking extreme measures to hunker down. There’s also an alternative epidemiology model emerging out of the UK, stirring up more discussion regarding the true extent of the infections in the country.

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As share buy­backs come un­der scruti­ny, what's in store for the bio­phar­ma in­dus­try?

Stock buybacks are not to be permitted for companies that will be bailed out in the coronavirus stimulus package, Congressional leaders have signaled. To what degree the biopharma industry has relied on buybacks for earnings growth in recent years, and if the trend continues, are the big questions as scrutiny into the practice heightens and balance sheets weaken with the coronavirus pandemic wreaking havoc on global economies.

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