Bio­Marin says the FDA game clock now tick­ing on their block­buster hope­ful peg­valiase

Bio­Marin $BM­RN has tak­en a big step to­ward a fi­nal FDA de­ci­sion on its lat­est rare dis­ease drug to reach reg­u­la­tors. The agency ac­cept­ed the biotech’s ap­pli­ca­tion for peg­valiase, hand­ed it a pri­or­i­ty re­view to speed the process, and then like­ly shoved it back down the line with some spe­cial re­quests on CMC.

Of­fi­cial­ly, the PDU­FA date for the drug is Feb­ru­ary 28, but the new CMC in­for­ma­tion de­mand­ed by reg­u­la­tors will most like­ly cause a de­lay of a few months, says the com­pa­ny, putting a de­ci­sion back to the end of May.

Peg­valiase is a new drug aimed at phenylke­tonuria, or PKU. The drug is de­signed to low­er blood pheny­lala­nine (Phe), the key bio­mark­er in play. And that’s just what re­searchers proved in a Phase III that read out in the spring of 2016.

Ac­cord­ing to Bio­Marin, their drug main­tained mean blood Phe lev­els at 527.2 umol/L against a base­line set at 503.9 umol/L. The place­bo arm re­port­ed an in­crease over eight weeks from 536 umol/L to 1385.7. That’s good enough for Bio­Marin’s ex­ec­u­tive crew to con­fi­dent­ly pre­dict block­buster sales at $1 bil­lion, but a num­ber of an­a­lysts have been hedg­ing their bets, low­er­ing their peaks to around $500 mil­lion.

Bio­Marin’s stock didn’t fare well in ear­ly 2016, though, be­cause the drug al­so failed to demon­strate a sig­nif­i­cant im­prove­ment in cog­ni­tion among pa­tients.

Phe is typ­i­cal­ly man­aged by a strict di­et, with pa­tients on Bio­Marin’s Ku­van of­ten able to sub­stan­tial­ly rein it in. This new in­jectable, AKA PEG-PAL, is in­tend­ed to elim­i­nate the di­etary el­e­ment, pos­si­bly aim­ing at the most se­vere cas­es.

Leerink’s Joseph Schwartz gives Bio­Marin good odds — 60% — of an ap­proval, but rec­og­nizes that in­vestors have been skit­tish in light of the fail­ure on neu­rocog­ni­tion. The pri­or­i­ty re­view des­ig­na­tion, giv­en the track record for fol­low-up ap­provals, could be telling.

We be­lieve FDA’s de­ci­sion to grant pri­or­i­ty re­view could calm in­vestor fears/skep­ti­cism as the des­ig­na­tion con­veys the Agency’s op­ti­mism and pos­i­tive view on the drug’s ac­crued da­ta thus far. Fur­ther­more, giv­en Bio­Marin’s ex­pe­ri­ence with Ku­van in PKU, the pos­i­tive trend may con­tin­ue for peg­valiase.

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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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.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.