NICE re­jects No­var­tis' mi­graine drug Aimovig, months af­ter de­clin­ing to en­dorse pricey Kym­ri­ah for adult use

No­var­tis $NVS is will­ing to play ball with UK’s cost-ef­fec­tive­ness watch­dog NICE, who re­ject­ed their mi­graine pre­ven­tion drug in a draft guid­ance on Thurs­day, months af­ter de­clin­ing to en­dorse the Swiss drug­mak­er’s cell ther­a­py Kym­ri­ah in adult lym­phoma pa­tients.

The drug, erenum­ab, is sold as Aimovig in the Unit­ed States by No­var­tis and part­ner Am­gen $AMGN. It forms part of a new crop of bi­o­log­ics tar­get­ing the CGRP pro­tein that trans­mits pain sig­nals in­to the brain, and is con­sid­ered in­stru­men­tal in gen­er­at­ing and main­tain­ing headaches as­so­ci­at­ed with mi­graine. Erenum­ab costs around £5,000 (about $6368) per year, ex­clud­ing a con­fi­den­tial dis­count that would have been grant­ed to Eng­land’s na­tion­al health ser­vice (NHS) had NICE rec­om­mend­ed the drug.

In its re­port, NICE ac­knowl­edged that al­though the drug is a clin­i­cal­ly ef­fec­tive treat­ment, the to­tal­i­ty of the da­ta sup­port­ing the med­i­cine did not in­clude all “rel­e­vant” com­par­isons against ex­ist­ing drugs and out­comes, which prompt­ed its re­jec­tion for NHS use.

Mein­dert Boy­sen

“There was not enough ev­i­dence to sug­gest that it (erenum­ab) is more ef­fec­tive than bot­u­linum tox­in type A (Al­ler­gan’s Botox) for peo­ple with chron­ic mi­graine, which NICE al­ready rec­om­mends. And for both the chron­ic and episod­ic mi­graine pop­u­la­tions there was no ev­i­dence to show that erenum­ab is ef­fec­tive in the long-term in peo­ple for whom 3 pre­vi­ous pre­ven­tive treat­ments had failed,” said NICE’s di­rec­tor of the cen­tre for health tech­nol­o­gy eval­u­a­tion Mein­dert Boy­sen in a state­ment.

No­var­tis pledged to work with NICE to ad­dress the agency’s ques­tions, but sug­gest­ed the watch­dog had not cap­tured the broad­er so­ci­etal im­pact of its med­i­cine.

“NICE does not con­sid­er the im­pact of dis­eases on broad­er so­ci­ety when eval­u­at­ing the cost-ef­fec­tive­ness of med­i­cines. We be­lieve this fails to cap­ture the true val­ue of med­i­cines like Aimovig. As mi­graine pre­dom­i­nant­ly af­fects peo­ple of work­ing age, it costs the UK econ­o­my £8.8 bil­lion per year in lost pro­duc­tiv­i­ty alone,” the drug­mak­er said in a state­ment, un­der­scor­ing that the UK al­ready “lags sig­nif­i­cant­ly be­hind” its oth­er Eu­ro­pean coun­ter­parts in en­sur­ing ac­cess to new med­i­cines.

It is es­ti­mat­ed there are 190,000 mi­graine at­tacks ex­pe­ri­enced every day in Eng­land, ac­cord­ing to NICE. The agency con­ced­ed that ex­ist­ing mi­graine treat­ments in­clud­ing be­ta-block­ers, an­ti­de­pres­sants and epilep­sy med­i­cines can have sig­nif­i­cant side-ef­fects and don’t work for some pa­tients, but for those with chron­ic mi­graine for whom at least 3 pre­vi­ous pre­ven­tive oral treat­ments have failed, Botox re­mains an op­tion.

The pro­vi­sion­al de­ci­sion on erenum­ab comes af­ter the agency re­fused to en­dorse No­var­tis’ CAR-T drug Kym­ri­ah in adults, say­ing it was too ex­pen­sive, in Sep­tem­ber, al­though it did rec­om­mend the treat­ment’s use in pe­di­atric pa­tients in a pre­vi­ous rul­ing.

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 faces ques­tions on old­er adults, asymp­to­matic in­fec­tion, long-term im­mu­ni­ty

The FDA adcomm has advanced to the free-for-all question stage of the hearing and, as they did for Moderna and Pfizer, committee members are raising questions about the lingering issues surrounding the vaccine.

In J&J’s case, one of those unknowns is a group of participants who appeared to respond worse to the vaccine: those over 60 with comorbidities. In that group, the vaccine was only 42% effective at stopping disease starting 28 days after vaccination.

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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