Flag­ship’s Sig­ilon grabs $80M to bring Robert Langer cell ther­a­py tech in­to the clin­ic

Roge­rio Vi­val­di had nev­er giv­en much thought to in­dus­try.

A new­ly mint­ed MD, he was work­ing at a hos­pi­tal in Rio De Janeiro when one of his pro­fes­sors asked if he might take on a case that re­quired con­sis­tent fol­low-up: A 14-year-old boy with Gauch­er’s, a rare ge­net­ic dis­or­der that meant he was miss­ing a key en­zyme of­ten called the cell’s re­cy­cling sys­tem. He had come in with en­larged or­gans and stunt­ed growth.

Roge­rio Vi­val­di

But the par­ents were in­ter­est­ed in try­ing a new treat­ment in­tro­duced that year, 1991, from Gen­zyme, one of the first ma­jor rare dis­ease biotechs. It would re­place the miss­ing en­zyme with a re­com­bi­nant form. Vi­val­di took the case. The boy re­cov­ered. He’s now 41, Vi­val­di said, with two kids.

“Usu­al­ly, I de­scribe that mo­ment as trans­form­ing the pa­tient’s life,” Vi­val­di told End­points News. “What peo­ple don’t re­al­ize is that mo­ment al­so changed my life. I had no thought of go­ing in­to biotech. I was a clin­i­cian.”

Vi­val­di, though, would soon take a job at Gen­zyme and then a se­ries of biotech C-suites be­fore Flag­ship tapped him 2 years ago to lead one of its new­er biotechs: Sig­ilon Ther­a­peu­tics. And to­day, he’s help­ing piv­ot the com­pa­ny to­ward the clin­ic for the first of sev­er­al chron­ic ill­ness­es, an­nounc­ing an $80.3 mil­lion Se­ries B fi­nanc­ing that will help push their lead drug in­to hu­man test­ing.

“It’s kind of the physi­cian ex­pe­ri­ence with the pa­tient ex­pe­ri­ence: What should we do — as a new class of med­i­cines — to re­al­ly bring a func­tion­al cure for pa­tients with chron­ic dis­eases?” Vi­val­di said, cit­ing his work with en­zyme ther­a­py and his own ex­pe­ri­ence with type 1 di­a­betes.

Sig­ilon’s plat­form comes out of work from Robert Langer’s and Daniel An­der­son’s labs at MIT and sev­er­al grants from the Ju­ve­nile Di­a­betes Re­search Foun­da­tion. For years, biotech and aca­d­e­m­ic re­searchers have known that a form of cell ther­a­py known as islet cell trans­plant could of­fer a po­ten­tial func­tion­al cure to di­a­betes, al­low­ing a pa­tient’s pan­creas to once again pump out prop­er dos­es of in­sulin.

The prob­lem, Vi­val­di said, is that the body’s im­mune sys­tem tends to at­tack these new cells, cov­er­ing it in fi­brot­ic scar tis­sue and ren­der­ing it non-func­tion­al. The few hun­dred pa­tients to re­ceive the trans­plant in the last 20 years have had to take ex­ten­sive dos­es of im­muno-sup­pres­sives. One way re­searchers long the­o­rized you could avoid these is­sues was by en­cap­su­lat­ing the cells in poly­mers — like mi­cro­scop­ic space suits. In 2016, Langer and An­der­son made it work, at least in the lab, and launched Sig­ilon with Flag­ship.

“It cre­ates a sphere or cap­sule where you could put many cells — be­tween 25 and 45,000 cells in one sin­gle 1.5 mil­ligram di­am­e­ter,” Vi­val­di said. “We cre­ate a space where the cells can be pro­duc­ing what­ev­er we en­gi­neer the cells to pro­duce.”

In 2018, Eli Lil­ly signed on to Sig­ilon’s di­a­betes pro­gram for $63 mil­lion up­front and $410 mil­lion in mile­stones. Sig­ilon in­sists, though, that the plat­form is much broad­er. The first clin­i­cal in­di­ca­tion will come lat­er this year in he­mo­phil­ia A be­cause, Vi­val­di said, it’s eas­i­er to see they are get­ting ac­tiv­i­ty in the blood rather than tis­sue. Be­yond that, there are pro­grams in he­mo­phil­ia B and even a pair of lyso­so­mal dis­or­ders — the same cat­e­go­ry as Gauch­er’s — that have not proven as sus­cep­ti­ble to en­zyme re­place­ment ther­a­py.

They won’t be alone in com­pet­ing on any of those in­di­ca­tions. Oth­er com­pa­nies, such as Vi­a­cyte, are us­ing oth­er meth­ods to get islet trans­plants to work, in­clud­ing by us­ing pluripo­tent stem cells. And gene ther­a­py is in ear­ly de­vel­op­ment for he­mo­phil­ia, lyso­so­mal dis­or­ders and even di­a­betes. Vi­val­di, though, said they can use their ther­a­py in far more pa­tients. Some pa­tients can’t get AAV, the vec­tor used in most gene ther­a­pies, he not­ed, and gene ther­a­py is gen­er­al­ly for pa­tients with more dire prog­noses, as op­posed to rel­a­tive­ly healthy ones.

“The le­git­i­ma­cy of our tech­nol­o­gy is much broad­er,” Vi­val­di said.

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.

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: 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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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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A Sin­ga­pore VC rais­es $200M for a new round, but will Covid-19 pre­vent it from rais­ing the rest?

A top Singaporean biotech venture fund is nearly halfway toward its largest ever fund, but in a sign of what could be in store for VCs amid a global economic freeze, said they could face headwinds raising the other half.

Vickers Venture Partners has secured $200 million out of a targeted $500 million for its 6th fund, first announced in early 2018. They’ve given themselves 13 months to complete the financing, Vickers founder Finian Tan told Deal Street Asia, but the financial frost settling amid the Covid-19 pandemic could slow efforts.

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Strug­gling Unum ex­ecs are ready to con­sid­er a sale, merg­er or any deal that comes its way

Unum $UMRX is working its way through a survival plan of sorts.

After getting hit with a trio of FDA holds in its brief public history and triggering its second pivot to a new lead drug program while laying off 60% of the staff, the troubled penny stock biotech Unum Therapeutics has hatched new plans to secure financial backing while lining up a go-forward strategy for the company.

First, Lincoln Park Capital Fund has agreed to buy up to $25 million of the long-suffering stock, as Unum directs. And the executive team — led by CEO Chuck Wilson — has put everything on the table for consideration: a sale, acquisition, merger, licensing deal, you name it. The ACTR707 program, meanwhile, is being formally wrapped up — their second failed lead program.