No­var­tis gets a speedy re­view for its CAR-T pi­o­neer as FDA lines up a mar­ket­ing de­ci­sion

Vas­ant Narasimhan, No­var­tis

No­var­tis $NVS has pulled in­to the in­side track at the FDA with its CAR-T CTL019, pick­ing up a pri­or­i­ty re­view from the agency that will land a mar­ket­ing de­ci­sion for re­lapsed and re­frac­to­ry (r/r) pe­di­atric and young adult pa­tients with B-cell acute lym­phoblas­tic leukemia.

The pi­o­neer­ing ap­pli­ca­tion comes as we wait for Kite to make its move at the agency af­ter be­ing forced to wait for 6-month da­ta on its ri­val CAR-T. Kite $KITE has promised to fin­ish its rolling ap­pli­ca­tion by the end of Q1, now just days away. And a spokesper­son for Kite tells me now that the biotech is on track to wrap the sub­mis­sion by Fri­day.

The pri­or­i­ty re­view leaves No­var­tis in shoot­ing range of the world’s first ap­proval for a CAR-T, which takes T-cells from pa­tients and then en­gi­neers them to at­tack can­cer cells. Blood can­cers proved to be the best, ear­ly tar­gets in the field, which has seen a line­up of com­pa­nies jump in on the promise of dra­mat­ic re­spons­es for some pa­tients.

No­var­tis pushed ahead in CAR-T af­ter stun­ning the in­dus­try with its de­ci­sion last sum­mer to dis­solve its cell and gene ther­a­py unit, a sto­ry we broke just af­ter launch­ing End­points News. The phar­ma gi­ant in­sist­ed that the sud­den sharp turn, which prompt­ed the unit’s chief, Us­man “Oz” Azam, to join an ex­o­dus of ex­ecs out of No­var­tis over the past year, wouldn’t side­track its pro­grams.

But it’s def­i­nite­ly in a tight race for the fin­ish line.

The progress of these ear­ly lead­ers hasn’t come eas­i­ly. Juno was once in the lead pack, un­til its fron­trun­ner was side­tracked twice by pa­tient deaths which it nev­er ful­ly ex­plained. Now that lead pro­gram has been shelved as Juno switch­es fo­cus to the next ther­a­py in the pipeline, hand­ing the lead to No­var­tis and Kite.

No­var­tis says it’s prep­ping an EMA ap­pli­ca­tion and plans ad­di­tion­al fil­ings for CTL019 in the US and EU mar­kets lat­er this year, in­clud­ing a BLA with the FDA for treat­ment of adults with r/r dif­fuse large B-cell lym­phoma (DL­B­CL) and ap­pli­ca­tions for mar­ket­ing au­tho­riza­tion with the EMA in r/r B-cell ALL and r/r DL­B­CL.

Wed­bush’s David Nieren­garten re­cent­ly spec­u­lat­ed that Kite’s da­ta up­date put it in line for a late 2017 ap­proval, shift­ing the fo­cus to pric­ing. He not­ed:

“(W)e note to jus­ti­fy the cur­rent mar­ket cap­i­tal­iza­tion, we would re­quire ei­ther a price point of $500,000 per course or up to 50% mar­ket share (or some com­bi­na­tion), both of which, while pos­si­ble, we be­lieve is less like­ly.”

But some an­a­lysts start this bid­ding at $250,000.

Who­ev­er gets on­to the mar­ket first will have a big edge in set­ting the price for these first wave drugs.

“With CTL019, No­var­tis is at the fore­front of the sci­ence and de­vel­op­ment of im­muno­cel­lu­lar ther­a­py as a po­ten­tial new in­no­v­a­tive ap­proach to treat­ing cer­tain can­cers where there are lim­it­ed op­tions,” said Vas Narasimhan, Glob­al Head of Drug De­vel­op­ment and Chief Med­ical Of­fi­cer, No­var­tis. “The pri­or­i­ty re­view and file ac­cep­tance of CTL019 by the FDA brings us one step clos­er to de­liv­er­ing this nov­el treat­ment op­tion to chil­dren and young adults with r/r B-cell ALL in the Unit­ed States.”

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.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.