Ju­ly 12 FDA pan­el re­view looms as a wa­ter­shed mo­ment in No­var­tis’ cam­paign to win an his­toric new drug OK

If you have any in­ter­est in the CAR-T field, mark down Wednes­day, Ju­ly 12 as a red let­ter day.

The FDA post­ed a note on Tues­day say­ing that its group of out­side can­cer ex­perts will gath­er that day to dis­cuss the phar­ma gi­ant’s ap­pli­ca­tion and vote on whether No­var­tis $NVS should get a mar­ket­ing ap­proval for its lead­ing CAR-T CTL019 (ti­s­agen­le­cleu­cel-T) as a new treat­ment for re­lapsed and re­frac­to­ry pe­di­atric B-cell acute lym­phoblas­tic leukemia. And on Mon­day pre­ced­ing the pan­el re­view the FDA will be ex­pect­ed to de­liv­er its in­ter­nal re­view of the ther­a­py, which will fur­ther tip its hand on what reg­u­la­tors are think­ing about the da­ta for a drug award­ed its break­through ther­a­py des­ig­na­tion.

The lat­est twist in this close­ly-watched saga comes a lit­tle more than two months since No­var­tis won a pri­or­i­ty re­view for its po­ten­tial­ly rev­o­lu­tion­ary CAR-T, which ex­tracts T cells from pa­tients, reengi­neers them and in­fus­es them by the mil­lions to swarm can­cer cells. The pri­or­i­ty re­view lopped two months off the re­view process, hit­ting fast for­ward on the agency’s call.

Right on its heels, two months be­hind in the process, is Kite Phar­ma $KITE with its ap­pli­ca­tion to mar­ket KTE-C19 (axi­cab­ta­gene ciloleu­cel) for non-Hodgkin lym­phoma, al­so new­ly of­fered a pri­or­i­ty re­view of its own.

No­var­tis and Kite have both of­fered some com­pelling da­ta for these CAR-Ts. And while they are not cur­rent­ly go­ing head-to-head on the same in­di­ca­tion, No­var­tis will be of­fer­ing what could be a di­rect chal­lenge when it rolls out its JULI­ET da­ta mid-week.

I’ll be fol­low­ing that too.

In ad­di­tion to ef­fi­ca­cy, you can ex­pect plen­ty of dis­cus­sion about the safe­ty pro­file of this drug. Un­like its ri­vals, No­var­tis and its part­ners at the Uni­ver­si­ty of Penn­syl­va­nia have large­ly avoid­ed the safe­ty crises that af­flict­ed Juno $JUNO and blew up its lead ther­a­py. Kite al­so has had to deal with the death of one pa­tient from a case of cere­bral ede­ma while cy­tokine re­lease syn­drome con­tin­ues to hov­er as clear threat that physi­cians have to guard against in many cas­es.

If ap­proved, No­var­tis will al­so be ex­pect­ed to com­pete ef­fec­tive­ly on the man­u­fac­tur­ing side, work­ing to re­duce the time it takes to get this drug from pa­tients, pre­pare it and then get it back in­to the vein.

Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.

A USP­TO le­gal ad­vis­er is off con­tro­ver­sial Gilead HIV case af­ter ac­tivists al­lege tweets show bias

Last week, a top legal adviser in the US Patent and Trademark Office working on the high-profile Gilead HIV PrEP case tweeted at Sen Bernie Sanders (I-VT) “What proof????” and then at activists “Do facts even matter to you?”

Now, STAT reports, she’s off of the case.

Activists in the coalition PrEP4All filed a petition to the USPTO on December 9 asking longtime senior legal advisor Mary Till be removed from the Gilead case, saying her tweets showed a bias toward Gilead. PrEP4All requested earlier this month the agency reject Gilead’s three-year patent extension for TAF (tenofovir alafenamide), a component of one of the HIV prevention regimens often referred to as PrEP. They allege the pharma giant delayed developing the drug in order to “game” the system and hold off generics.

What's next for Sarep­ta? A third DMD ap­proval, an­a­lysts pre­dict

What Sarepta wants, Sarepta usually gets.

In dramatic fashion on Thursday, the approval of Vyondys 53 — to treat a subset of Duchenne muscular dystrophy (DMD) patients — was unveiled, four months after the FDA’s initial rejection. With two drugs now approved on the basis of ~1% expression of dystrophin — the missing protein that causes DMD — Sarepta is in a prime position to take its third DMD drug to the regulator, analysts said, predicting healthier odds of success.

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