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.” 

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

Jay Flatley, new Zymergen CEO

Fol­low­ing Au­gust melt­down, Zymer­gen hints at sal­vage plans — cut­ting jobs and rene­go­ti­at­ing loans

Two months after a spectacular implosion that saw its founding CEO leave his post amid customer reports its only product didn’t work, Zymergen provided the first peek behind the curtain for its plans moving forward.

In an SEC filing Wednesday, Zymergen told regulators it would slash about 100 jobs and had renegotiated a $100 million loan from Perceptive that loomed like a storm cloud over the company, moving up the maturity date 18 months to June 30, 2022. Jed Dean, one of Zymergen’s three co-founders and VP of operations, will also step down at the end of the month.

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Sanofi, Re­gen­eron etch out an­oth­er PhI­II vic­to­ry for Dupix­ent, eas­ing se­vere itch and clear­ing le­sions

Sanofi and Regeneron can boast of another inflammatory disease where Dupixent has proven effective.

The best-selling drug, which targets both IL-4 and IL-13, has delivered a clean sweep in a Phase III trial for prurigo nodularis, a chronic disease characterized by itch so intense that it can affect patients’ sleep and psychology. Thick skin lesions can cover most of the body.

On the primary endpoint, 37% of patients taking Dupixent saw a clinically meaningful reduction in itch compared to 22% of those on placebo (p=0.0216) at week 12. All secondary endpoints were also met, including clearance of skin lesions and improvement in quality of life.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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