How much is your vi­sion worth? Spark CEO Jeff Mar­raz­zo has a price in mind

Just how much is your vi­sion worth?

That’s the bil­lion-dol­lar ques­tion at Spark Ther­a­peu­tics now as the FDA winds up with an odds-on ap­proval for its gene ther­a­py used to fix a rare form of in­her­it­ed blind­ness called RPE65-me­di­at­ed reti­nal dy­s­tro­phy.

Jeff Mar­raz­zo

It’s ver­boten in bio­phar­ma for CEOs to dis­cuss hard prices ahead of an ap­proval, but Spark $ONCE CEO Jeff Mar­raz­zo has been do­ing the math on the eco­nom­ic val­ue of vi­sion, and came up with a sub­stan­tial 7-fig­ure num­ber: $1 mil­lion-plus for Lux­tur­na.

“While it is not our in­ten­tion to­day to guide you with the po­ten­tial price if it is ap­proved, we are en­cour­aged that by mod­el­ing rea­son­able as­sump­tions about the im­pact of Lux­tur­na on these types of in­di­rect costs, as well as on qual­i­ty of life and di­rect med­ical cost over a pa­tient’s life­time that there is sup­port for the val­ue of the ther­a­py in ex­cess of $1 mil­lion per pa­tient,” he said dur­ing Spark’s Q3 call with an­a­lysts on Tues­day.

Mar­raz­zo then care­ful­ly walked through the eco­nom­ic ar­gu­ment for a price that could land the com­pa­ny’s lead treat­ment right at the top of the list of the 10 most ex­pen­sive ther­a­pies on the plan­et.

There are sev­er­al fac­tors to con­sid­er in eval­u­at­ing their ther­a­py’s price, Mar­raz­zo said. Start with the val­ue of a job, some­thing 70% of the blind in the US don’t have. There’s the cost of a care­giv­er for blind chil­dren, of­ten a par­ent who can’t work. And there are state court de­ci­sions that place the val­ue of sight for plain­tiffs at more than $1 mil­lion.

Spark’s move here is cru­cial for the en­tire gene ther­a­py field. This is the first such treat­ment to reach the thresh­old of an FDA ap­proval, and its mar­ket­ing plan will in­flu­ence the mar­ket val­u­a­tion of every biotech in the field. And while a cou­ple of gene ther­a­pies have been sanc­tioned in Eu­rope, they’ve on­ly been rarely used, falling well short of the kind of com­mer­cial suc­cess need­ed for a vi­able mar­ket­ing ef­fort.

Over the last year there have been a va­ri­ety of wide-rang­ing dis­cus­sions about the com­ing price dis­cus­sion on gene ther­a­pies. Some have sug­gest­ed that the man­u­fac­tur­ers should spread the price over a pe­ri­od of time, to make it eas­i­er to cov­er or lim­it the price if the gene ther­a­py’s ef­fect waned over time.

Mar­raz­zo, for his part, says re­im­burse­ment rules in the US make such spread out pay­ments vir­tu­al­ly im­pos­si­ble, sug­gest­ing that Spark will wind up with a 7-fig­ure stick­er that gets paid on de­liv­ery. While he says that the fi­nal price has yet to be nailed down, at this stage of the game it’s more than like­ly that Spark has a hard price in mind for the roll­out to come soon.

And the CEO just made a block­buster eco­nom­ic case for Lux­tur­na.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

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For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

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Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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