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.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

UP­DAT­ED: Bio­gen push­es in a fresh stack of chips and starts prep­ping a glob­al R&D game plan af­ter watch­ing the cards turn on ear­ly throm­bolyt­ic da­ta

After patiently steering through a decade-long journey for its early-stage clinical work, a small Tokyo biotech has clinched a deal to out-license its lead thrombolytic agent to US heavyweight Biogen — which sees a potentially game-changing impact on the clot-busting field after taking a careful look at some upbeat Phase IIa data.

Three years after Biogen anted up $4 million to gain an option on the drug from TMS, the big US biotech is making a small bet to beef up its stroke portfolio. The BD team inked a deal to go ahead and grab rights to the drug for $18 million, with another $335 million in milestone cash on the table for a successful outcome.

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.