The biggest win­ners and losers in the 2016 game of block­busters

We’re just a lit­tle past the halfway mark in 2016, and we’ve got a mixed as­sort­ment of trends at play for FDA watch­ers in the big game of block­buster ap­provals — John Car­roll

First—the FDA’s list of new drugs which earned a green flag is now just at 15 (16 if you in­clude last night’s OK for Sanofi’s di­a­betes drug lixise­n­atide). For all of last year, the FDA OK’d a gen­er­a­tion-high record of 45 new drugs, leav­ing the in­dus­try far off pace. Any sig­nif­i­cant fall in that num­ber is like­ly to trig­ger some new ques­tions about R&D pro­duc­tiv­i­ty.

But raw fig­ures like these aren’t every­thing. More than half of these new drugs have been tapped at one time or an­oth­er as ei­ther block­busters in the mak­ing or a key play­er in a com­pa­ny’s fran­chise de­fense play­book.

The em­pha­sis these days is on big drugs that de­liv­er big rev­enue.

Sec­ond—it’s seem­ing­ly pret­ty easy to land a break­through drug des­ig­na­tion at the FDA to speed things along, es­pe­cial­ly if you have an in­ter­est­ing can­cer drug. Five of the 15 drugs on the FDA’s ap­proval list had BTD sta­tus–matched by block­buster sales pro­jec­tions–in­clud­ing both can­cer drugs. Three of those BTDs won an ac­cel­er­at­ed ap­proval, so you can see how big the pay­off can be. The agency can­cer czar Richard Paz­dur has an open door pol­i­cy for all the big can­cer pro­grams these days, which helps ex­plain why CBER cleared the way so fast for Juno to get back in­to the clin­ic with its CAR-T drug af­ter 4 pa­tients were killed by their drug com­bo.

The big pic­ture: Can­cer drug R&D goes faster, more biotechs get to play ear­ly on, more mon­ey gets pumped in­to on­col­o­gy star­tups.

Third—there are some clear big win­ners. And I’m putting the em­pha­sis on big, as the phar­ma gi­ants are dom­i­nat­ing the NDA field in 2016.

Roche is a stand­out, earn­ing the FDA’s ap­proval for its PD-L1 drug Tecen­triq. The pi­o­neer check­points have been rack­ing up multi­bil­lion dol­lar sales, and Roche plans to be a con­tender. Its big Genen­tech sub­sidiary has a long line­up of stud­ies in the clin­ic that mix and match its drug for a range of can­cers. Roche al­so scored a big gain with an OK for the BCL-2 in­hibitor Ven­clex­ta (three BTDs), an­oth­er block­buster in the mak­ing which is part­nered with Ab­b­Vie. Both of those big play­ers are fac­ing first-wave biosim­i­lars that could bad­ly dam­age their ag­ing fran­chis­es.

Just a few weeks ago the FDA al­so put Roche’s mul­ti­ple scle­ro­sis drug ocre­lizum­ab – unique­ly tapped for both pri­ma­ry pro­gres­sive as well as re­laps­ing/re­mit­ting — on its fast track with a De­cem­ber 28 PDU­FA date. (It’s an­oth­er BTD drug, natch, so an OK could come much soon­er.) Eval­u­ate Phar­ma has pegged peak po­ten­tial sales at $2.9 bil­lion, mak­ing this their num­ber 1 drug of the year, with a bul­let.

Eli Lil­ly CEO John Lech­leit­er

One rea­son why Eli Lil­ly CEO John Lech­leit­er could prep a move to stand down soon re­volves around the re­cent ap­proval of Taltz (ix­ek­izum­ab). But it al­so has the CDK 4/6 can­cer drug abe­maci­clib (an­oth­er BTD drug) and baric­i­tinib for rheuma­toid arthri­tis on tap for near-term ap­provals. Those ap­provals could hap­pen fast, as both earned ‘break­through’ des­ig­na­tions, putting them on the agency’s in­side track.

Lech­leit­er has been bat­ting back ques­tions about Lil­ly’s R&D op­er­a­tions for years. And while Lil­ly has a habit of muscling in on high­ly com­pet­i­tive drug de­vel­op­ment races, an­a­lysts like these drugs a lot.

Mer­ck is an­oth­er win­ner, with its hep C com­bo Zepati­er an­gling for a block­buster wedge of a big pie that’s start­ed to shrink around the edges as the com­pe­ti­tion hit the field. But Gilead, prob­a­bly the most com­pet­i­tive bio­phar­ma com­pa­ny on the plan­et, al­so counter punched with an ap­proval for Ep­clusa, an all-geno­type hep C drug priced at a dis­count rate of $74,000. Gilead is try­ing to pro­tect its enor­mous hep C fran­chise as well as it can.

Among the small­er com­pa­nies look­ing to con­quer big mar­kets: In­ter­cept scored with Ocali­va (obeti­cholic acid, or OCA) and Aca­dia won on Nu­plazid (pi­ma­vanserin), a Parkin­son’s drug which an­a­lysts at vary­ing times have tapped at peak sales of any­where from $640 mil­lion to more than $2 bil­lion. (Keep in mind, last year’s po­ten­tial block­busters of­ten turn in­to this year’s com­mer­cial dis­ap­point­ments.)

So who are the big losers so far in 2016?

As­traZeneca, which can ill af­ford it, con­tin­ues to dump any­thing that dis­ap­points. So lesin­u­rad went to Iron­wood, which paid a frac­tion of what As­traZeneca shelled out for it. That deal fol­lowed its move to cast off bro­dalum­ab to Valeant. And one in­di­ca­tion of tralok­inum­ab was sold off. The key reg­u­la­to­ry set­back oc­curred when the FDA put its ap­pli­ca­tion for ZS-9 on hold due to ques­tions re­gard­ing man­u­fac­tur­ing.

No list of reg­u­la­to­ry set­backs should ex­clude Clo­vis, which had to throw in the tow­el on rocile­tinib af­ter it fell em­bar­rass­ing­ly short of the com­pe­ti­tion. Clo­vis has yet to ac­knowl­edge that it has for­mal­ly been hand­ed a CRL, a sign of the times at that be­lea­guered com­pa­ny.

Bio­Marin’s dris­apersen was hand­ed a re­jec­tion for Duchenne mus­cu­lar dy­s­tro­phy in Eu­rope, fol­low­ing an FDA slap down. The com­pa­ny killed it, along with some fol­low-up pro­grams.

Fail­ure in Big Phar­ma tends to get a quick send off, es­pe­cial­ly at No­var­tis. So it was no sur­prise to see that a CRL for one of its top biosim­i­lars was rel­e­gat­ed to a one-lin­er in a re­cent fi­nan­cial re­port.

Block­buster suc­cess gets a march­ing band in the R&D in­dus­try. Fail­ure is buried qui­et­ly at night.

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

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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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[via AP Images]

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.

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Genen­tech sub­mits a plan to near­ly dou­ble 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.

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

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David Grainger [file photo]

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Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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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

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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.