Sem­ma steps to­ward the clin­ic af­ter demon­strat­ing ef­fect of po­ten­tial di­a­betes cure in land­mark an­i­mal stud­ies

The re­search team at the well-fund­ed Sem­ma Ther­a­peu­tics has cleared one of the last re­main­ing hur­dles to get­ting a po­ten­tial cure for di­a­betes in­to hu­man stud­ies. And if they’re right, it marks a ma­jor pre­clin­i­cal mile­stone for a resur­gent re­gen­er­a­tive med­i­cine field fo­cused on a new gen­er­a­tion of stem cell ther­a­pies.

Fe­li­cia Pagli­u­ca

Fe­li­cia Pagli­u­ca, VP of cell bi­ol­o­gy re­search and de­vel­op­ment at the Cam­bridge, MA-based biotech, told the 2019 In­ter­na­tion­al So­ci­ety for Stem Cell Re­search meet­ing in LA Sat­ur­day that their stem cell-de­rived islets per­formed as hoped for — pro­duc­ing in­sulin — in a study in­volv­ing non-hu­man pri­mates whose im­mune sys­tems had been flat­tened to pre­vent a re­jec­tion. In a sep­a­rate study in­volv­ing two pigs, a pack­age of these en­gi­neered islets con­tained in a spe­cial­ly de­signed pack­age were used suc­cess­ful­ly to gen­er­ate in­sulin with­out need­ing an im­muno­sup­pres­sant to pro­tect against a re­ac­tion.

“For the first time ever the de­vice pro­tects the cell,” Pagli­u­ca told me in a pre­view of to­day’s ses­sion, of­fer­ing ev­i­dence from a large an­i­mal mod­el that the tech­nol­o­gy func­tions with blood glu­cose lev­els, spurring in­sulin se­cre­tion as need­ed. “They re­al­ly show quite con­sis­tent re­spon­sive­ness.”

And that’s with­out fi­bro­sis, with­out cell suf­fo­ca­tion, while watch­ing the in­te­gra­tion of cell ther­a­py in­to host tis­sue.

Bas­tiano San­na

It’s ex­cit­ing, says Sem­ma CEO Bas­tiano San­na, to see the “cu­ra­tive po­ten­tial” of this cell ther­a­py.

Re­al­ly? A cure? For a mass mar­ket dis­ease like di­a­betes?

If that all seems a bit too won­der­ful to be be­lieved, think about where Pagli­u­ca is com­ing from. Stem cell ther­a­pies had their hey­day well over a decade ago as the next big thing in med­i­cine — an overnight sen­sa­tion which sput­tered out in fail­ure as the sur­vivors went back in­to the lab to do the hard work nec­es­sary to make it a re­al­i­ty. That long pe­ri­od of qui­et bred con­sid­er­able skep­ti­cism, es­pe­cial­ly af­ter the first wave of promised cures failed to ma­te­ri­al­ize. And she’s watched it play out as re­gen­er­a­tive med­i­cine made its come­back.

One of those pi­o­neer­ing sci­en­tists who stayed in the lab was Sem­ma sci­en­tif­ic founder and Har­vard pro­fes­sor Doug Melton, who pub­lished a land­mark study 5 years ago out­lin­ing how he had suc­cess­ful­ly used stem cells to cre­ate in­sulin-pro­duc­ing pan­cre­at­ic be­ta cells that were in­sert­ed in bulk in­to mice and suc­cess­ful­ly pro­tect­ed from an im­mune re­sponse — a break­through in re­gen­er­a­tive med­i­cine. And he’d been work­ing on the cure for more than 20 years, which he start­ed fol­low­ing his son’s di­ag­no­sis of Type 1 di­a­betes. 

They’ve raised $158 mil­lion over 4 years at Sem­ma to get to this stage, stand­ing on the thresh­old of a pair of small hu­man stud­ies set to launch next year. They’ll now see if they can re­pro­duce in hu­mans what they did in the non-hu­man pri­mates and pigs: first by run­ning a small study with an im­muno­sup­pres­sant, fol­lowed by the use of their de­vice in an­oth­er study lat­er next year that will avoid im­muno­sup­pres­sants.

CAR-T and gene ther­a­pies have come along to demon­strate cu­ra­tive po­ten­tial, but the CEO says it’s been frus­trat­ing to see the first wave of these ther­a­pies tar­get­ed at tiny pa­tient pop­u­la­tions. “We’re very ex­cit­ed about bring­ing the cu­ra­tive po­ten­tial of cell ther­a­pies in­to a large in­di­ca­tion.”

They’re tak­ing it step by step.

No­body is rolling out a mass tri­al for Type 2 di­a­betes. The first goal is to go af­ter some of the Type 1 di­a­betes pa­tients who are the hard­est to treat, with nowhere left to turn to rein in the au­toim­mune dis­ease. Af­ter that, they can turn to the broad­er Type 1 pop­u­la­tion be­fore mov­ing on, per­haps in­to par­tic­u­lar sub­groups of Type 2.

Ini­tial­ly, the goal will be to get the islets to do the work need­ed to safe­ly pro­duce in­sulin for these pa­tients at a re­li­able lev­el. These stem cell-de­rived islets will have to be able to be man­u­fac­tured at scale. And there’s work un­der­way to see if there’s a uni­ver­sal cell de­sign that can be used to avoid the need for the de­vice they use — some­thing Melton com­pares to a tea bag.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.