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.”

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Michel Vounatsos, Getty Images

UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

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Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.