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.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Don't let Ab­b­Vie fool FTC with an easy di­vesti­ture, plead crit­ics in lat­est at­tack on $63B Al­ler­gan buy­out

If the FTC must let AbbVie and Allergan go ahead with their merger, at least make them divest their latest blockbuster on the market, a chorus of unions, consumer groups and public interest organizations plead in a new attempt to rein in the megamerger.

There’s a second part to their argument: If the antitrust watchdog does greenlight the divestiture AbbVie wants, then at least ensure the pharma giant cannot corner its future rivals with its exclusionary tactics.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

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Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Bris­tol-My­ers shrugs off an­oth­er Op­di­vo com­bo flop as $1.74B deal goes pfffft

Bristol-Myers Squibb’s enthusiasm for immuno-oncology was at fever pitch back in 2015, when it struck one of its classic tie-ups with little Five Prime Therapeutics for its colony-stimulating factor 1 receptor drug. The Big Pharma giant plunked down $350 million in cash and committed itself to $1.4 billion in milestones as it laid out plans for 6 tumor type programs in combo with Opdivo, its big new PD-1 breakthrough.

Today, it’s another I/O bust.

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Heron's Ground­hog Day; Vi­for forms JV with Fre­se­nius Kabi in Chi­na

→ Another delay has blighted Heron Therapeutics’ quest to get its long-acting non-opioid painkiller for post-surgical analgesia on to the US market. The company, whose product is meant to rival Pacira’s J&J-partnered, long-acting post-op painkiller Exparel, was handed an unexpected CRL last year after the FDA asked for more data relating to chemistry, manufacturing, and controls as well as non-clinical information.

Matt Roe (Verana)

Fresh off $100M round, GV-backed re­al-world da­ta start­up woos Duke re­searcher as CMO

Days ago Verana Health closed $100 million to expand its real-world data system and get ready for “regulatory-grade.” What’s left unsaid was that they’ve also found the guy to help them do it.

The San Francisco startup has recruited Matt Roe from Duke University to be its chief medical officer, overseeing the iterative process of generating clinical data and deriving scientific insight from prospective research. His job is also to work with other stakeholders such as academic centers and the FDA to map out more use cases for real-world data.

Auste­do, Teva's bright spot as it ex­e­cutes turn­around, fails two Touret­te's tri­als

It was all going so well. Last week, Teva investors saw the light at the end of the tunnel, after Israel’s largest drugmaker handsomely beat Wall Street sales expectations by $100 million — following years of decline, precipitated by falling revenue, legal costs and mounting debt.

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