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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.