Thumbs Up/Thumbs Down: The new buzz in R&D, block­buster work on a bud­get and why what’s dead may nev­er die

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Bio­elec­tron­ics gets a star turn

Let’s get it out front. I have a weak­ness for big think­ing about new R&D fields. So when I got wind of the tie-up be­tween Glax­o­SmithK­line and Ver­i­ly (Lord Google’s life sci­ence arm), I got up at 3 am and wrote the sto­ry. The idea that nan­otech based de­vices can be used to or­ches­trate a se­ries of elec­tric puls­es to treat a dis­ease can on­ly thrill a re­al R&D en­thu­si­ast. And at this stage, every­thing has great po­ten­tial. We’re a cou­ple of years out from proof of con­cept, so this is far from a com­mer­cial sto­ry. (Which, let’s face it, is what GSK re­al­ly needs now.) Sci­ence lovers will fol­low it as close­ly as they can. Count me in.

Bio­gen is do­ing a deal? Re­al­ly?

There’s been no doubt at all, for more than a year now, that Bio­gen needs to get se­ri­ous and do some big deals. But in­stead of pacts that can grab Wall Street’s at­ten­tion with some late-stage piz­zazz, Bio­gen has been deal­ing with a man­age­ment change­up. Doug Williams and Steve Holtz­man left last year, both find­ing new biotechs to run, and Bio­gen couldn’t get its act to­geth­er in time to save George Scan­gos’ perch at the top. The ear­ly-stage work it com­mit­ted to in gene ther­a­py is fas­ci­nat­ing, but it lacks the kind of near term cat­a­lysts that Bio­gen hungers for. Alzheimer’s is a huge, ul­tra-risky op­por­tu­ni­ty. Man­age­ment told us again this week that great deals are com­ing. We’re still wait­ing. And that’s not a good thing.

Re­gen­eron’s stel­lar Q2 up­date

This is how you set up a late-stage pipeline, on a bud­get. At $1.45 bil­lion, Re­gen­eron’s 2016 re­search bud­get won’t make the top 20. But its late-stage pipeline has sev­er­al po­ten­tial block­busters in it. I just saw a new an­a­lyst take on the com­pa­ny, not­ing that the ap­proval of drugs like dupilum­ab is baked in now. That’s an in­di­ca­tion that Re­gen­eron not on­ly knows a sol­id med­ical ad­vance, it plans and ex­e­cutes on clin­i­cal tri­als like few com­pa­nies can. Sanofi al­so gets cred­it for par­tic­i­pat­ing in its work, where it has seen far more suc­cess than its in-house ef­fort has pro­duced. Fail­ures are so fre­quent in bio­phar­ma R&D, it’s great to ap­plaud Re­gen­eron. I’m look­ing for­ward to this com­pa­ny’s third act in the clin­ic.

  Shire fol­lows Take­da in re­or­ga­niz­ing R&D.

Last week, Take­da made it clear that it is cut­ting staffers in places like Cam­bridge, UK so it can con­cen­trate its re­search forces in the Cam­bridge/Boston and Japan. This week Shire out­lined the first cut in its three-year plan: to re­duce R&D costs $210 mil­lion af­ter ac­quir­ing Bax­al­ta. Bax­al­ta, of course, was just get­ting start­ed with its so­lo act when Shire stepped in. These are all re­al-world re­sults when you are A) grow­ing through ac­qui­si­tions and B) chang­ing the cul­ture. They are painful and of­ten nec­es­sary al­ter­ations in the land­scape. But you can’t help but feel for the peo­ple whose jobs are ly­ing in the way of per­pet­u­al change. R&D has been a vol­canic field for the past decade. Job se­cu­ri­ty is a thing of the past. This has like­ly helped pro­duc­tiv­i­ty, at the cost of play­ing hell with peo­ple’s lives.

Resver­a­trol: Show me the da­ta.

Some re­search sto­ries won’t die. But truth­ful­ly, why bring back resver­a­trol from the dead at this point? The Wall Street Jour­nal made the un­for­tu­nate de­ci­sion to do that, based on some mighty thin pick­ings from a few on­go­ing re­search projects in odd pock­ets of the world. You’ll re­call GSK’s en­thu­si­asms men­tioned at the top? They got a look at resver­a­trol’s an­i­mal da­ta at Sir­tris, which turned out to be more than a lit­tle con­tro­ver­sial, and paid $720 mil­lion for it. Sir­tris nev­er got very far and GSK wound it up and shipped the re­main­ing projects south to Philly. One day, some­body may ac­tu­al­ly land re­al ev­i­dence that they found a way to turn this mir­a­cle drug in­to a re­al­i­ty. Un­til then, let’s leave it as a side show.

Deci­bel hears the call of a new biotech neigh­bor­hood.

Biotech, of course, can be pur­sued just about any­where on the plan­et, but grav­i­tates to the most ex­pen­sive places in the world. One of those places is Kendall Square in Cam­bridge, where Big Phar­ma has been mov­ing in and gen­tri­fy­ing the neigh­bor­hood with big bucks for new re­search cen­ters. They have an un­der­stand­able de­ter­mi­na­tion to learn new things from the movers and shak­ers of the sci­ence world. But as Deci­bel’s Steve Holtz­man made clear, they’re pric­ing star­tups like his out of the mar­ket. So he’s jump­ing the Charles and mak­ing his new home in Fen­way. Boston/Cam­bridge will long re­main one of the most vi­tal re­search hubs in the world. But on­ly if you think out­side the bound­aries, and that in­cludes town lines.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

Drug com­pa­nies reach $260M set­tle­ment just ahead of opi­oid tri­al; Oys­ter Point set terms for $85M IPO

→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

Sofinno­va-backed Abi­vax touts longer term mid-stage da­ta in ul­cer­a­tive col­i­tis

Two months after Abivax convinced Sofinnova to bankroll several mid-stage studies of its lead drug — ABX464 — with a €12 million stock purchase, the Paris-based biotech has rolled out more data on the anti-inflammatory molecule for all investors to see.

In a Phase IIa maintenance study involving 22 patients with moderate to severe ulcerative colitis who have been failed by previous treatments, 12 achieved clinical remission as assessed by endoscopy. But since only 19 completed the full one-year trial, 16 of whom had an endoscopy, investigators scored the remission rate at 75%. Although there’s no comparator arm, execs were pleased with improvements over an initial two-month, placebo-controlled induction phase by a number of measures ranging from remission to Mayo score and a fecal biomarker.

Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.