Cel­gene works to calm down anx­ious in­vestors, promis­ing to re-file ozan­i­mod at the FDA in ear­ly 2019

For weeks now, an­a­lysts have been fret­ting over the fu­ture of Cel­gene’s $CELG mul­ti­ple scle­ro­sis drug ozan­i­mod. Turned away at the FDA’s front door with a refuse-to-file let­ter, their at­ten­tion fo­cused on an ac­tive metabo­lite of the drug and spec­u­la­tion over whether or not the big biotech would be forced to run a new hu­man study to sat­is­fy reg­u­la­tors that would set them back years.

Mark Alles

This morn­ing, Cel­gene ex­ecs went to some pains to ex­plain that the de­lay would stretch out on­ly to Q1 2019 as re­searchers un­der­took some bridg­ing non-clin­i­cal stud­ies of the drug. No lengthy hu­man stud­ies would be need­ed, the com­pa­ny added. And they would hus­tle along a Eu­ro­pean ap­pli­ca­tion along­side the FDA pitch in the first few months of next year.

The rea­son for the de­lay: “Ozan­i­mod is me­tab­o­lized in hu­mans to form one ma­jor ac­tive metabo­lite (CC-112273) and oth­er mi­nor ac­tive metabo­lites,” the com­pa­ny re­port­ed. And that metabo­lite has a long half life of 10 to 13 days.

Ever­core ISI an­a­lyst Umer Raf­fat sees this as a pos­i­tive. He notes:

If this metabo­lite was a small % of over­all ozan­i­mod AUC, that would be a rea­son­able con­clu­sion.  How­ev­er, we learned that this metabo­lite is ~90% of AUC in hu­mans… that’s key … be­cause what that means to me is that this metabo­lite is ef­fec­tive­ly the drug as we know it.  Said an­oth­er way, all the Ph 2/Ph 3 stud­ies we saw on ozan­i­mod was ef­fec­tive­ly this metabo­lite.  For that rea­son, it seems that hu­man stud­ies should not be need­ed and that’s what CELG is guid­ing.

Of course, if you were look­ing for a quick­er turn­around, the glass at Cel­gene could look more half emp­ty right now. Not­ed Ge­of­frey Porges:

This new time­line is three quar­ters be­hind what we have fore­cast and may put the com­mer­cial launch in some un­cer­tain­ty giv­en the ex­pect­ed en­try of gener­ic Gilenya. Cel­gene an­nounced that the ozan­i­mod re­sub­mis­sion plan will in­clude bridg­ing non-clin­i­cal stud­ies and ad­di­tion­al analy­sis of ex­ist­ing PK/PD da­ta, but em­pha­sized that ad­di­tion­al hu­man clin­i­cal ef­fi­ca­cy and safe­ty stud­ies are not need­ed.

The drug is a cen­tral part of CEO Mark Alles’ case that the com­pa­ny has a bright fu­ture ahead of it, ca­pa­ble of earn­ing $4 bil­lion to $6 bil­lion. Alles has been rack­ing up a se­ries of new deals, buy­ing Juno for $9 bil­lion, and re­vamp­ing the com­pa­ny’s com­mand struc­ture as a se­ries of em­bar­rass­ing sna­fus raised ques­tions about a com­pa­ny that has long been ad­mired for its abil­i­ty to ex­e­cute quick­ly and ef­fi­cient­ly.

Cel­gene has lit­tle mar­gin for er­ror now. Any new slips will like­ly be se­vere­ly pun­ished.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future.