Am­gen, No­var­tis ready to roll as FDA green lights block­buster cam­paign for mi­graine drug — priced at $6,900

Am­gen and No­var­tis have brought home the big Aimovig (erenum­ab) ap­proval they’ve been hunt­ing, scor­ing first-mover ad­van­tage for a block­buster con­tender that will now look to start chang­ing the stan­dard of care for de­bil­i­tat­ing mi­graines.

An­tho­ny Hoop­er

Sig­nif­i­cant­ly, the heavy­weight part­ners — who will share the US mar­ket­ing rights — are launch­ing this CGRP pi­o­neer with a list price of $6,900 a year. That num­ber comes in well be­low the $8,000 to $10,000 spread that Ex­press Scripts had al­ready warned would be un­ac­cept­able to them. It’s al­so well un­der the $8,500 fig­ure the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view pen­cilled in for their re­view, which they con­sid­ered too cost­ly for all but the most se­ri­ous­ly af­flict­ed.

That mes­sage clear­ly got through to the man­u­fac­tur­ers. Am­gen in par­tic­u­lar has had to work out some deals on its PC­SK9 drug to get that past steep pay­er hur­dles.

They’ll be sell­ing their drug at $575 list for once month­ly 70 or 140 mg sin­gle-use in­jec­tors. And some­thing to keep in mind: Dis­ease ad­vo­cates say that mi­graines aren’t un­com­mon, cost­ing the econ­o­my tens of bil­lions of dol­lars a year for the dev­as­tat­ing at­tacks.

Steve Miller, chief med­ical of­fi­cer of Ex­press Scripts, had raised a warn­ing flag on this drug, try­ing to steer the com­pa­nies away from a high whole­sale price, which would be used to set out-of-pock­et costs for con­sumers and a high-wa­ter mark for pay­ers to dis­count against.

“If your ex­pec­ta­tion is that you are not go­ing to ac­tu­al­ly get that high list price, then don’t do that to pa­tients who have high co-pays,” Miller told Reuters. “Let’s be more bal­anced. Let’s get back to where gross-to-net is not so dif­fer­ent.”

Ex­press Scripts gave the drug price a quick thumbs up this morn­ing, but that doesn’t mean that they’ll make the drug read­i­ly avail­able to every­one who asks for it. A spokesper­son tells me:

Steve Miller

“We be­lieve they are pric­ing the drug re­spon­si­bly at the low­er end of the val­ue-based price range. We be­lieve there is a se­ri­ous un­met need for pa­tients with mi­graine. How­ev­er, not all pa­tients will need this ther­a­py. We see it for peo­ple with mi­graine who have failed pre­ven­tive ther­a­py. We will en­sure ap­pro­pri­ate pa­tients have ac­cess to this med­i­cine and will put a pri­or au­tho­riza­tion pro­gram in place to help pay­ers get the most val­ue for the mon­ey they spend.”

In a sign of the times, Am­gen and No­var­tis mar­ket­ing teams are wast­ing no time set­ting up an ag­gres­sive cam­paign to fo­cus more at­ten­tion on mi­graine as they roll out a ma­jor mar­ket­ing ef­fort. And they’ve got a pa­tient sup­port group — Aimovig Al­ly — ready to roll.

Time is a cru­cial fac­tor here. Eli Lil­ly is lined up as the sec­ond en­try to the CGRP space in a mat­ter of months, with fa­mil­iar da­ta on its abil­i­ty to sig­nif­i­cant­ly cut the num­ber of mi­graines chron­ic suf­fer­ers have to en­dure. Then there’s Te­va, which has to en­dure a dam­ag­ing de­lay for its ther­a­py. And lit­tle Alder is wait­ing in the wings as Al­ler­gan press­es ahead with an oral drug they’d like to use to leapfrog every­one.

To­day, though, be­longs to Am­gen and No­var­tis, which ex­e­cut­ed well on a huge Phase III ef­fort. 

“In ad­di­tion to bring­ing a new ther­a­peu­tic op­tion to pa­tients in the U.S., Am­gen al­so has a com­mit­ment to re­shape the pub­lic’s per­cep­tion of this stig­ma­tized dis­ease,” said An­tho­ny Hoop­er, ex­ec­u­tive vice pres­i­dent of glob­al com­mer­cial op­er­a­tions at Am­gen. “We have pledged a mis­sion to help change mis­con­cep­tions, stereo­typ­ing and even judg­ment that peo­ple with mi­graine face on a dai­ly ba­sis. Through ed­u­ca­tion­al pro­grams and ini­tia­tives, we hope to pro­mote more mean­ing­ful con­nec­tiv­i­ty and di­a­logue among pa­tients, physi­cians, em­ploy­ers and pay­ers.” 

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.