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.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepeneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film Fantastic Voyage, the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his own account, along with some seed cash from friends and family.

Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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