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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

No­var­tis un­veils 'US-first' strat­e­gy ahead of San­doz spin­off

Weeks after announcing the spinoff of generics arm Sandoz, Vas Narasimhan paints a picture of the new, slimmer Novartis — with a “US-first mindset,” he said at an investor event on Thursday.

The CEO unveiled ambitious plans to become a top-five player in the US by 2027 at Novartis’ “Meet the Management” event in Basel, Switzerland, which means ramping up clinical trials in the states and “building capability and talent, among other things.” The company’s also shooting for a top-three ranking in China.

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Pfiz­er sacks phar­ma com­pe­ti­tion in ear­ly NFL TV ad­ver­tis­ing sea­son

If pharma advertising had a fantasy football league, Pfizer would be crushing the competition. A dive into the National Football League’s TV commercial buys across early season games by iSpot shows a hefty lead with its Covid-19 Comirnaty vaccine ads.

More than 175 million impressions with $9.5 million in media spending put Pfizer in the top spot with a 65% share of voice across NFL pharma spending, according to the real-time TV ad tracker. In a distant second place is Bristol Myers Squibb’s Opdivo with 44 million impressions, $5.2 million in spending and a 16% share, followed by BMS’ Zeposia with 31 million impressions, $3.3 million in media buys and an 11% share.

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