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: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi's Paul Hud­son is bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.”

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting: