Cel­gene's dis­clo­sures about ozan­i­mod this week spur an­a­lyst­s' fears of a lengthy de­lay for a key drug

Re­searchers for Cel­gene $CELG turned up at the Amer­i­can Acad­e­my of Neu­rol­o­gy meet­ing this week with some news about the mul­ti­ple scle­ro­sis drug ozan­i­mod that quick­ly cap­tured an­a­lysts’ at­ten­tion.

In a pre­sen­ta­tion on ozan­i­mod Phase III da­ta, an in­ves­ti­ga­tor spot­light­ed an ac­tive metabo­lite pro­duced by the drug, spurring an ah-ha mo­ment for an­a­lysts who have been puz­zling out why the FDA re­cent­ly is­sued a stun­ning refuse-to-file no­tice on the drug.

Jef­feries’ Michael Yee not­ed this morn­ing:

We and many ob­servers be­lieve the is­sue re­lates to this metabo­lite that hasn’t been suf­fi­cient­ly char­ac­ter­ized by CELG and may not have suf­fi­cient in­for­ma­tion and safe­ty da­ta to per­mit FDA to ful­ly re­view the drug for ap­proval which is a prob­lem.

There’s two ways to look at this, he adds, de­pend­ing on whether you’re a bull or a bear on the suf­fer­ing stock. The bull ar­gu­ment would pose that a pre­sen­ta­tion at a con­fer­ence like this would mean that Cel­gene couldn’t be all that con­cerned by the metabo­lite is­sue, while the bear view would be: 

(I)nsuf­fi­cient tox cov­er­age for this metabo­lite which is the ac­tu­al ac­tive moi­ety, FDA guid­ance sug­gests needs 1-2 years more pre­clin­i­cal stud­ies, brings big un­cer­tain­ty even if NDA fil­ing – whether Ozan­i­mod would ac­tu­al­ly get ap­proved and/or now has hair on it.

In­vestors now want to know if the com­pa­ny plans to re-file soon or will look to go back to the draw­ing board with new, and po­ten­tial­ly dam­ag­ing, de­lays to dis­close. But there’s no sign of an ear­ly re­ac­tion on the stock price, which is slight­ly in the green in pre-mar­ket trad­ing. The stock is down 18%, though, from the be­gin­ning of the year.

Mark Alles

The biotech re­port­ed at the end of Feb­ru­ary that the RTF came through be­cause the FDA de­ter­mined “that the non­clin­i­cal and clin­i­cal phar­ma­col­o­gy sec­tions in the NDA were in­suf­fi­cient to per­mit a com­plete re­view” for mul­ti­ple scle­ro­sis, leav­ing plen­ty of unan­swered ques­tions about a drug that Cel­gene ex­ecs had con­fi­dent­ly pre­dict­ed would bring in $4 bil­lion to $6 bil­lion a year. Com­ing on the heels of the im­plo­sion of its $710 mil­lion cash roll of the dice on the in­flam­ma­to­ry bow­el dis­ease drug mon­gersen (GED-301), in­vestors start­ed won­der­ing why the com­pa­ny was sud­den­ly lurch­ing from dis­as­ter to dis­as­ter.

CEO Mark Alles fol­lowed up with a man­age­ment shake­up that left him with wider di­rect con­trol over op­er­a­tions at the com­pa­ny.

I’ve sent out a mes­sage to Cel­gene, but the com­pa­ny isn’t not­ed for quick trans­paren­cy.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

Israel Lowy (Regeneron)

#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Regeneron entered the PD-(L)1 game late, so they devised a two-pronged strategy to catch up with Big Pharma rivals: They would push it into cancers where PD-1s had yet been tested, and they would prove that it’s as powerful in the big indications as any other on the market.

They cleared a hurdle on the first goal Friday, showing a 31% response in patients with the rare skin cancer basal cell carcinoma. And with the data they’re rolling out Monday, Regeneron cancer chief Israel Lowy is ready to declare success on the second.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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