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.

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.

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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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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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.