FDA stamps fast OK on Astel­las' pi­o­neer­ing FLT3 AML drug gilter­i­tinib, ex­pand­ing on a record year for new ap­provals

Astel­las has picked up boast­ing rights to gain­ing the first FDA ap­proval of a FLT3 in­hibitor for a spe­cif­ic group of FLT3 pos­i­tive acute myeloid leukemia pa­tients — which ac­counts for close to a third of the pop­u­la­tion.

Back in May the Japan­ese phar­ma re­port­ed that the agency had of­fered a snap 6-month re­view on its fast-track pro­gram for gilter­i­tinib, some­thing that’s be­come quite com­mon in the on­col­o­gy group.

That’s good news for the 30% or so of the 19,000 new­ly di­ag­nosed AML cas­es each year that will be linked to the ge­net­ic mu­ta­tion. The drug has been in a piv­otal study where in­ves­ti­ga­tors have been study­ing it’s abil­i­ty to cut re­laps­es in the pa­tient pop­u­la­tion.

The drug will be sold as Xospa­ta, and Astel­las set the whole­sale price at $22,500 for 30 days, which would trans­late to $270,000 for a year of ther­a­py — though pay­ers ne­go­ti­ate down from that fig­ure. In a state­ment, the com­pa­ny said that their in­ter­im analy­sis of the piv­otal pro­gram tracked a me­di­an du­ra­tion of re­sponse of 4.6 months.

Alexan­der Ed­ward Perl

This OK de­liv­ers a “new, high­ly-ef­fec­tive, and well-tol­er­at­ed treat­ment op­tion to the clin­ic for a group of tru­ly high-risk pa­tients who, un­til to­day, had no spe­cif­ic ther­a­pies avail­able be­yond chemother­a­py to treat their dis­ease,” says Alexan­der Perl, an as­so­ciate pro­fes­sor of Hema­tol­ogy-On­col­o­gy at the Uni­ver­si­ty of Penn­syl­va­nia. Perl has been lead­ing the clin­i­cal tri­al pro­gram for this drug.

“Al­though we’re wait­ing for the fi­nal analy­sis of (the Phase III study) AD­MI­RAL, the avail­able da­ta with gilter­i­tinib show few­er and milder side ef­fects than typ­i­cal­ly is seen with tra­di­tion­al chemother­a­py,” Perl added, not­ing that they al­so tracked a sig­nif­i­cant­ly high­er rate of com­plete re­spons­es.

The ap­proval marks the FDA’s 54th OK of the year as it goes deep­er in­to record ter­ri­to­ry af­ter pass­ing the old record set in 1996.


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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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.

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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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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.