#ASH17: No­var­tis’ 6-month Kym­ri­ah da­ta in DL­B­CL sets up a head-to-head fight with Gilead

No­var­tis $NVS has the kind of ev­i­dence of dura­bil­i­ty for its CAR-T drug Kym­ri­ah at 6 months it needs to win a quick shot at com­pet­ing with Gilead on dif­fuse large B-cell lym­phoma (DL­B­CL) cas­es.

Just a day af­ter com­plet­ing their fil­ing with the FDA, No­var­tis re­port­ed Wednes­day that among 81 pa­tients at month 3, the com­plete re­sponse rate hit 32% with the par­tial re­sponse rate com­ing in at 6%. Their ab­stract on Kym­ri­ah (CTL019) for ASH al­so shows that among 46 pa­tients evalu­able at 6 months, the CR rate was 30% — with 95% re­main­ing con­sis­tent­ly free of any sign of their can­cer — and the PR rate was 7%. Re­searchers said that re­sponse rates were con­sis­tent across sub­groups (in­clud­ing those with dou­ble-hit lym­phoma). Me­di­an du­ra­tion of re­sponse was not reached and the 6-month prob­a­bil­i­ty of be­ing re­lapse free was 73.5% — main­tain­ing the kind of ef­fi­ca­cy for this can­cer that has made CAR-T a new fac­tor in com­bat­ing blood can­cers.

That is right in line with Kite’s re­port­ed 31% com­plete re­sponse rate at 6 months, which al­so pro­vid­ed ev­i­dence of dura­bil­i­ty that sug­gests a block­buster fu­ture for these drugs.

The lat­est da­ta puts No­var­tis on a short track to go­ing head-to-head with Gilead, which paid close to $12 bil­lion to land Kite and its new­ly ap­proved CAR-T drug Yescar­ta. The FDA has proven to be more than will­ing to speed up their OKs on can­cer drugs, es­pe­cial­ly when they are work­ing with al­ready ap­proved ther­a­pies.

Kym­ri­ah was ap­proved last Au­gust for acute lym­phoblas­tic leukemia (ALL), the first such OK for any CAR-T af­ter years of ri­val­ry in the clin­ic.

To be sure, there are dan­gers for pa­tients to con­sid­er — with cy­tokine storms and neu­ro­log­i­cal tox­i­c­i­ty play­ing a big role — but No­var­tis’ ab­stract al­so spells out how an ag­gres­sive re­sponse can help man­age side ef­fects, with no deaths from side ef­fects to re­port for an ad­vanced, heav­i­ly pre­treat­ed group of pa­tients.

States the ab­stract:

Over­all, 86% of pa­tients had grade 3 or 4 ad­verse events (AEs). Cy­tokine re­lease syn­drome (CRS) oc­curred in 58% of in­fused pa­tients, with 15% grade 3 and 8% grade 4 us­ing the Penn grad­ing scale and man­aged by a pro­to­col-spe­cif­ic al­go­rithm. 15% of pa­tients re­ceived an­ti-IL6 ther­a­py, tocilizum­ab, for CRS man­age­ment with good re­sponse and 11% of pa­tients re­ceived cor­ti­cos­teroids. Oth­er grade 3 or 4 AEs of spe­cial in­ter­est in­clud­ed neu­ro­log­ic AEs (12%, man­aged with sup­port­ive care), cy­tope­nias last­ing >28 days (27%), in­fec­tions (20%), and febrile neu­trope­nia (13%). Three pa­tients died with­in 30 days of in­fu­sion, all due to dis­ease pro­gres­sion. No deaths were at­trib­uted to CTL019. No CRS or neu­ro­log­ic event as­so­ci­at­ed deaths oc­curred.

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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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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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.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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