In wake of a CAR-T re­struc­tur­ing, No­var­tis says it’s ready to hunt a pi­o­neer­ing OK for lead pro­gram

Bruno St­rig­i­ni, CEO No­var­tis On­col­o­gy

No­var­tis is still in the race to win the world’s first ap­proval for a pi­o­neer­ing CAR-T treat­ment.

The phar­ma gi­ant $NVS pulled back the veil from their Phase II study of their CAR-T CTL019, reg­is­ter­ing an im­pres­sive 82% com­plete re­mis­sion rate among the 50 chil­dren and young adults with B-cell acute lym­phoblas­tic leukemia. And with no deaths, though sig­nif­i­cant ev­i­dence of tox­i­c­i­ty, No­var­tis says it will be ready to file for an ap­proval ear­ly on in a loom­ing 2017.

Forty-one of 50 pa­tients achieved com­plete re­mis­sion or com­plete re­mis­sion with in­com­plete blood count re­cov­ery three months af­ter an in­fu­sion of CTL019, which reengi­neers pa­tients’ T cells in­to a can­cer ther­a­py.

Close to half of the pa­tients ex­pe­ri­enced a grade 3 or 4 case of cy­tokine re­lease syn­drome, not un­com­mon in the field. And 15% per­cent of pa­tients ex­pe­ri­enced grade 3 neu­ro­log­i­cal and psy­chi­atric events in­clud­ing en­cephalopa­thy and delir­i­um. But there were no grade 4 events and no one died.

That’s a sol­id enough ben­e­fit/safe­ty pro­file to take to reg­u­la­tors as No­var­tis hunts up an ac­cel­er­at­ed ap­proval.

No­var­tis man­aged to stun prac­ti­cal­ly every­one in the field when it abrupt­ly dis­band­ed its cell and gene ther­a­py unit three months ago, lay­ing off 120 and in­te­grat­ing an­oth­er 280 back in­to its reg­u­lar R&D ranks as CAR-T chief Us­man ‘Oz’ Azam hit the ex­it. That was wide­ly re­gard­ed as a se­ri­ous set­back that could af­fect fu­ture pro­grams, even if more ad­vanced ef­forts like this one at the Uni­ver­si­ty of Penn­syl­va­nia stayed on track.

Juno has been thrown in­to ut­ter dis­ar­ray with mul­ti­ple deaths de­rail­ing their lead ef­fort for JCAR015. And Kite re­cent­ly not­ed that it would need a lit­tle more time to com­plete a rolling sub­mis­sion for their top ther­a­py, which should now be com­plet­ed in Q1.

“This first-of-its-kind tri­al rep­re­sents ex­cit­ing progress to­ward our goal of help­ing chil­dren and young adults with re­lapsed or re­frac­to­ry B-cell ALL, a pa­tient pop­u­la­tion with an ur­gent need for new treat­ment op­tions,” said Bruno St­rig­i­ni, CEO, No­var­tis On­col­o­gy. “We are com­mit­ted to ad­vanc­ing CTL019 and look for­ward to work­ing close­ly with the FDA and EMA in the com­ing months.”

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.