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.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Af­ter near­ly a year of de­bate, the Covid-19 vac­cine chal­lenge tri­als are of­fi­cial­ly com­ing

After nearly a year of public advocacy and often rancorous ethical debate, human challenge trials for Covid-19 vaccines are getting off the ground in London.

The UK government’s Covid-19 Vaccine Taskforce and the contract research firm Open Orphan announced today £10 million ($13 million) plan to test experimental Covid-19 vaccines in volunteers whintentionally exposed to the novel coronavirus. The studies, which won’t launch until early 2021, come after 9 months of debate over whether such studies were safe and would actually hasten vaccine development, and they follow a long history of researchers using challenge models to study other respiratory viruses, including flu and the coronaviruses that cause the common cold.

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Derek Chalmers, Cara Ther

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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CAR-plus: Irish biotech re­cruits Kite alum Chris Now­ers to prep dual-tar­get­ing NK cell ther­a­py for the clin­ic

Soon after Chris Nowers left Cell Medica — freshly rebranded Kuur Therapeutics — in February, the Kite Pharma alum was introduced to another cell therapy player.

The basic idea of building an off-the-shelf allogeneic platform with a CAR-NK approach was familiar to him, riding on the same wave as Takeda, J&J-backed Fate, Nkarta and others. But then there was something else that stood out: a membrane-bound TNF related apoptosis inducing ligand variant, or TRAIL variant, that’s also engineered onto the NK cell for a dual-targeted attack.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Gilead feels the heat as close al­ly Gala­pa­gos re­ports a big set­back on one of their top ex­per­i­men­tal drugs

The bad news keeps stacking up at Galapagos — which quite likely just lost control of a billion-dollar deal — and by extension their close partners at Gilead.

The biotech $GLPG reported after the bell Thursday that GLPG1972, one of their top development programs, flat failed a mid-stage study for osteoarthritis, flunking the primary and all secondary endpoints.

Testing 3 different doses of their drug, which relies on ADAMTS-5 inhibition, investigators concluded that none of them triggered a statistically significant response — as measured by cartilage thickness.

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IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.