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.

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.