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.”

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

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

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

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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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.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand 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.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

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

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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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.