New drug price con­tro­ver­sy looms as Bio­gen stuns pay­ers with Spin­raza­'s $750K stick­er — an­a­lyst

Ge­of­frey Porges, Leerink

One promi­nent an­a­lyst be­lieves we may soon see a new drug price con­tro­ver­sy erupt, on­ly this one will have noth­ing to do with price goug­ing on an old ther­a­py.

The drug is Spin­raza, the new­ly ap­proved pi­o­neer­ing ther­a­py for rare cas­es of spinal mus­cu­lar at­ro­phy from Bio­gen and its part­ners at Io­n­is. Ear­li­er this week Bio­gen priced the drug at about $750,000 for the first year and half that for each year af­ter, or $1.5 mil­lion over three years.

And a pres­i­den­tial-lev­el Tweet storm may not be far be­hind, says Leerink’s Ge­of­frey Porges. He notes:

“The stick­er-shock pre­sent­ed in the me­dia could turn Spin­raza in­to the So­val­di of rare dis­ease drugs, or the straw that breaks the camel’s back in terms of the US mar­ket’s tol­er­ance for rare dis­ease drug pric­ing. At the very least, and im­plied in our up­dat­ed mod­el, the price is go­ing to force pay­ers to close­ly scru­ti­nize which pa­tients re­ceive ac­cess and lim­it the over­all ac­cess pro­vid­ed. To us it seems cer­tain that pa­tients who have the less se­vere Type III and IV forms of the dis­ease or who are old­er with rel­a­tive­ly milder symp­toms will find it dif­fi­cult to ob­tain treat­ment.”

The price is sub­stan­tial­ly above what Porges had fore­cast, but it isn’t nec­es­sar­i­ly way out of line, he notes. Oth­er rare dis­ease drugs have a built-in price es­ca­la­tion mod­el as pa­tients start on a drug as in­fants and then in­crease their dosage as they grow old­er. Bio­gen won’t be able to hide any price hike un­der its flat price, and over­all the Spin­raza price won’t be that far off from oth­er rare dis­eases.

In the mean­time, look for Bio­gen to ben­e­fit from a sud­den surge in rev­enue once the new drug be­comes avail­able.

Its in­ter­est­ing to note on our end that drug price con­tro­ver­sies come in sev­er­al mod­els. Spin­raza and So­val­di may be sub­ject to a pay­er back­lash as cer­tain new drugs com­mand jaw-drop­ping prices that can stun in­sur­ers. Rare dis­ease drugs, though, have gen­er­al­ly not at­tract­ed the kind of at­ten­tion that So­val­di gar­nered, with its fo­cus on a ma­jor dis­ease like he­pati­tis C.

Then there are the Tur­ings and Valeants of the world, which jacked up prices on old drugs and at­tract­ed the at­ten­tion of law­mak­ers who re­spond­ed to the on­line lynch mobs that formed to de­mand a re­sponse. But even with pub­lic hear­ings and reg­u­lar con­dem­na­tion, Tur­ing didn’t budge on its price for Dara­prim.

Fi­nal­ly, there’s the reg­u­lar price es­ca­la­tion that can add up to big mon­ey over the years, a busi­ness mod­el fol­lowed by most of the bio­phar­ma world. Now that strat­e­gy is al­so com­ing un­der pres­sure as in­dus­try in­sid­ers like Al­ler­gan CEO Brent Saun­ders chal­lenge the in­dus­try to self-reg­u­late prices, lim­it­ing an­nu­al price hikes to mod­est sin­gle-dig­it in­creas­es.

The ju­ry is still out on that one, but the in­dus­try is far from rush­ing in­to a whole­heart­ed em­brace of price dis­ci­pline, es­pe­cial­ly as R&D con­tin­ues to dis­ap­point most ma­jor man­u­fac­tur­ers.

How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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J&J’s Rem­i­cade — the poster child for how to block biosim­i­lars — fi­nal­ly set­tles Pfiz­er suit

Biosimilars have proven time and again (although mostly in Europe) that competition works to bring down the cost of a once-pricey biologic, and can even expand its use.

J&J’s Remicade, however, has always proven to be an outlier.

Back in 2016, Pfizer won FDA approval for its infliximab biosimilar, known as Inflectra, but when the launch foundered, the company sued J&J, claiming that the company’s plan to block biosimilar competition worked incredibly well. Pfizer even went on to win FDA approval for a second infliximab biosimilar in 2017, known as Ixifi, but decided to never launch it.

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