Thumbs Up/Thumbs Down: No­var­tis's re­treat leaves plen­ty of ca­su­al­ties; Bio­gen or­ches­trates a shame­ful me­dia show, and more


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


No­var­tis ex­e­cutes a messy with­draw­al on CAR-T

What­ev­er No­var­tis’s PR Team has come up with to ra­tio­nal­ize its de­ci­sion to dis­band its 400-mem­ber cell and gene ther­a­py unit, make no mis­take that this move marks a sig­nif­i­cant pipeline re­treat on the phar­ma gi­ant’s part. Two years ago the com­pa­ny couldn’t say enough about its com­mit­ment to CAR-T de­vel­op­ment, but these new ac­tions speak much loud­er than words. That’s not to say that No­var­tis is out of the game, but it is clear­ly falling by the way­side as a much faster set of play­ers at Kite is left in sole pos­ses­sion of front place. (Juno’s re­cent prat­fall se­ri­ous­ly de­layed its en­try.)

Bio­gen is feast­ed af­ter re­heat­ing some old Alzheimer’s da­ta

Bio­gen ex­ecs knew ex­act­ly what they were do­ing when they her­ald­ed the pub­li­ca­tion of ear­ly Alzheimer’s da­ta for ad­u­canum­ab. By push­ing some very thin re­sults from a small study back in­to the spot­light, old claims about cog­ni­tive trends were trans­formed in­to new head­lines about a cure. The com­pa­ny, which has been strug­gling to spark fresh en­thu­si­asm for its risky pipeline, wasn’t able to get any boost out of it from Wall Street, where an­a­lysts were acute­ly aware of the sham. Pa­tients and fam­i­lies, though, were like­ly tricked by the dog and pony show in­to be­liev­ing some new won­der drug lay just on the hori­zon. The whole thing was shame­ful, and Bio­gen is com­plic­it in spurring the me­dia show.

Medi­va­tion’s David Hung makes out like a ban­dit – and that’s a good thing

When you look for an ac­coun­tant to han­dle the books, chances are you ex­pect to save more on your tax­es than what you’re pay­ing your num­ber crunch­er. The same prin­ci­ple ap­plies to what you pay your CEO when it comes time to sell the com­pa­ny. For Medi­va­tion’s David Hung, that’s a prince­ly sum of $354 mil­lion. Sound ex­pen­sive? Per­haps. But when you dig in­to the auc­tion he per­formed for Medi­va­tion be­fore sell­ing to Pfiz­er for $14 bil­lion, you get the im­pres­sion that he earned it. Hung set a stan­dard on deal­mak­ing in biotech M&A. Let’s all raise our glass­es and make a toast. A win is a win.

Mar­tin Shkre­li makes his fi­nal ex­it at Kalo­Bios

Mar­tin Shkre­li has spe­cial­ized in defin­ing the worst about biotech. Greed al­ways trumped the pub­lic in­ter­est. Con­fused ep­i­thets were coined and tossed out to the mass­es via Twit­ter. And his let-the-in­sur­ers-pay-for-it de­fense — trot­ted out once again in an ef­fort this week that would ul­ti­mate­ly help vil­i­fy My­lan — in­cit­ed a mob of an­gry crit­ics. His char­ac­ter is so tox­ic that now that he’s cut his fi­nal ties to Kalo­Bios, the biotech has re­spond­ed by vow­ing to be a mod­el of trans­paren­cy while it seeks to ham­mer out a fair way to price drugs. We could on­ly hope that the rest of the in­dus­try is shamed in­to the same po­si­tion. Be­cause the next drug pric­ing scan­dal is right around the cor­ner. In­no­va­tors have to learn how to sep­a­rate them­selves from the prof­i­teers, or every­one will be “re­formed” the same way in the back­lash to come.

No­var­tis proves it can still be the leader in biosim­i­lars

This week, No­var­tis’s biosim­i­lars crew at San­doz won a key FDA ap­proval on its biosim­i­lar of En­brel, Am­gen’s $5 bil­lion drug. Once again, the agency was ready to ex­trap­o­late the da­ta for one in­di­ca­tion and ap­ply it across the board. That will all help blaze a clear trail for oth­ers to fol­low at the agency. Of course, as the FDA be­comes part of the so­lu­tion, the courts re­main ready to throw up road­blocks for long and tor­tu­ous patent lit­i­ga­tion. And there’s no sign of that end­ing any­time soon.

RIP Roger Tsien [1952-2016]

By all ac­counts, UC San Diego’s Roger Tsien il­lu­mi­nat­ed many lives through his work, and not just by his ground­break­ing work on flu­o­res­cent pro­teins. His un­time­ly death is a cause of sad­ness, tru­ly, but we should all cel­e­brate a life well lived and the fact that his work will con­tin­ue to ben­e­fit peo­ple for some time to come.

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.