Kite Phar­ma hits its Q1 dead­line on fil­ing a BLA for its pi­o­neer­ing CAR-T, posts im­proved da­ta

Kite CEO Arie Bellde­grun

With the clock tick­ing down the fi­nal hours of Q1, Kite Phar­ma­ceu­ti­cals $KITE has kept its pledge to file an ap­pli­ca­tion for its pi­o­neer­ing CAR-T ther­a­py in the first quar­ter. The move puts Kite one step be­hind No­var­tis $NVS, which two days ago an­nounced that it had the FDA’s com­mit­ment for a speedy re­view of its own CAR-T.

The ques­tion now is whether the FDA will give the Kite’s ap­pli­ca­tion a pri­or­i­ty re­view as well, per­haps lin­ing up back-to-back ad­vi­so­ry com­mit­tee meet­ings to ex­am­ine the pros and cons of this new wave of can­cer ther­a­py.

Kite filed the BLA for axi­cab­ta­gene ciloleu­cel (KTE-C19, des­ig­nat­ed as a break­through ther­a­py by the FDA) as a treat­ment for pa­tients with re­lapsed or re­frac­to­ry ag­gres­sive non-Hodgkin lym­phoma who are in­el­i­gi­ble for au­tol­o­gous stem cell trans­plant.

Kite was held up a few months by the need to com­plete 6-month da­ta on axi­cab­ta­gene ciloleu­cel, a goal it ac­com­plished a month ago.

At six months, the ORR in dif­fuse large B-cell lym­phoma (DL­B­CL) hit 36%, down on­ly three points from month three. The CR rate was 31%, down two points. Big­ger drops were al­so record­ed for pri­ma­ry me­di­asti­nal B-cell lym­phoma (PM­B­CL) and trans­formed fol­lic­u­lar lym­phoma (TFL) en­rolled in Co­hort 2, but the six-month num­bers post­ed were all clear­ly sta­tis­ti­cal­ly sig­nif­i­cant, which helped qui­et some dogged crit­i­cism of the drug’s abil­i­ty to pro­vide a durable re­sponse.

On Fri­day, Kite al­so up­dat­ed its da­ta at AACR in an oral ple­nary pre­sen­ta­tion. And the num­bers had im­proved with more pa­tients to re­port on:

The ORR in the mITT analy­sis set of 101 pts was 82% (CR 54%, PR 28%), and was con­sis­tent across key co­vari­ates in­clud­ing dis­ease sub­type, re­frac­to­ry sta­tus, stage, and IPI score. At a me­di­an fol­low up of 8.7 m, 44% of pts were in re­sponse and 39% were in CR.

That’s up from 41% and 36% re­port­ed in Feb­ru­ary.

Kite — led by CEO Arie Bellde­grun — and No­var­tis are rac­ing to be right at the fore­front of in­tro­duc­ing per­son­al­ized cell ther­a­pies that reengi­neer pa­tients’ im­mune cells to tar­get can­cer. And it’s of­fered some sig­nif­i­cant re­spons­es for a num­ber of pa­tients with ad­vanced cas­es of blood can­cer.

Now Kite and No­var­tis will try to out­do each oth­er on man­u­fac­tur­ing and de­liv­ery, with the phar­ma gi­ant and the biotech need­ing to show that they can pro­vide quick and re­li­able treat­ment.

That may be a par­tic­u­lar­ly dif­fi­cult ques­tion for No­var­tis. As we re­port­ed Thurs­day af­ter­noon, the phar­ma gi­ant’s head of cell man­u­fac­tur­ing — Karen Walk­er, re­spon­si­ble for CAR-T — is leav­ing the com­pa­ny to­day af­ter a lengthy stint. Her de­par­ture fol­lows the un­ex­pect­ed move at No­var­tis to dis­solve its cell ther­a­py unit last sum­mer, lay­ing off staffers and ab­sorb­ing the group in its on­col­o­gy R&D di­vi­sion.

Walk­er and two oth­er ex­ecs were put in charge of No­var­tis’ ef­forts on CAR-T last fall, af­ter the com­pa­ny chopped up the sep­a­rate cell and gene ther­a­py group that had been cre­at­ed specif­i­cal­ly to dri­ve these new ther­a­pies to com­mer­cial­iza­tion. That move fol­lowed the de­par­ture of Us­man “Oz” Azam, who left No­var­tis in the wake of the re­or­ga­ni­za­tion.

Left on the side­lines to­day: Juno Ther­a­peu­tics. Once a leader in the CAR-T area, Juno was forced to shelve its lead ther­a­py af­ter it killed a string of pa­tients. Two of those pa­tients died af­ter a brief R&D halt or­dered by the FDA. But Juno nev­er was able to firm­ly es­tab­lish why the CAR-T caused lethal cas­es of brain swelling. Now the biotech has moved on to a fol­low-up pro­gram, putting the com­pa­ny well be­hind the fron­trun­ners.

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.

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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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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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.