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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.