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.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.