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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

Mark Cuban (Jed Jacobsohn/AP Images)

Mov­ing to the em­ploy­er side of health­care, Mark Cuban's Cost Plus Drugs part­ners with a PBM

From “Shark Tank” to direct-to-consumer generic drugs, Mark Cuban has made another inroad in the ongoing battle over prescription drug prices. His cost-plus-15% generic drug company, frequently undercutting many competitors, now has its sights set on the employer healthcare market.

Cost Plus Drugs, which originally pledged to cut out PBMs, has now partnered with the PBM EmsanaRx, majority owned by the Purchaser Business Group on Health, to launch a supplemental drug discount program designed specifically for self-funded employers, the company announced Thursday.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.