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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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J&J ad­comm live blog: J&J is al­so test­ing a two-dose vac­cine. What hap­pens if it's more ef­fec­tive?

J&J has spent the day touting the data behind their Covid-19 vaccine, but one advisor pointed that, in a few months, another batch of data could force them into a curious dilemma.

J&J was the only major vaccine developer to attempt to produce a single-dose vaccine, a huge asset when trying to rapidly inoculate the world against an ongoing public health threat, but they hedged their bets. Alongside their main Phase III trial, they also launched another that would test two doses, each spaced two months apart.

Genen­tech plots $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Genentech has committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Roche’s big South San Francisco hub will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

Am­gen, As­traZeneca speed to­ward fil­ing next-gen an­ti­body for asth­ma af­ter un­cork­ing full late-stage da­ta

On the hunt for a novel competitor to Sanofi and Regeneron’s Dupixent in severe asthma, Amgen and AstraZeneca posted “exciting” results from their next-gen antibody late last year. Now, the partners are showing their hands, and the results look good enough for approval.

Amgen and AstraZeneca’s tezepelumab plus standard of care cut the rate of severe asthma attacks by 56% at the one-year mark compared with SOC alone, according to full data from the Phase III NAVIGATOR study presented Friday at the virtual American Academy of Allergy, Asthma & Immunology meeting. And those significant results were consistent regardless of patients’ baseline eosinophil counts.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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