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.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Linda Marbán, Capricor CEO

Capri­cor hits pri­ma­ry end­point in Duchenne ex­ten­sion study, ready­ing its FDA pitch

It has been a very bumpy ride for Capricor Therapeutics over the past several years, including a halted J&J partnership and venture into penny stock status. However, the latest news from the San Diego-based biotech shows that its experimental Duchenne drug may be a durable, long-term treatment.

The company posted the one-year results from its open-label extension study for their candidate dubbed CAP-1002. The extension from its Phase II study, named HOPE-2, was conducted in boys and young men with later-stage Duchenne muscular dystrophy who paused and then resumed treatment after a year. Researchers measured patients in the one-year follow-up against the original study’s treatment arm effect and in two subsets of the original placebo group.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.