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.

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.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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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.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.