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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 119,800+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

Endpoints Premium

Premium subscription required

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

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

Endpoints Premium

Premium subscription required

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

Jacob Van Naarden, Senior VP, CEO of Loxo Oncology at Lilly; President, Lilly Oncology

Eli Lil­ly bags FDA nod for Verzenio in ear­ly breast can­cer, but a con­tro­ver­sial di­ag­nos­tic could dog its roll­out

As Eli Lilly works to consolidate its internal and Loxo teams into an oncology powerhouse, the drug giant is putting high hopes on CDK 4/6 inhibitor Verzenio to help drive the portfolio into the future. Now, the drug has scored a paradigm-altering win in early breast cancer — but will a controversial companion diagnostic hamstring Lilly’s market plans?

The FDA on Wednesday approved CDK 4/6 inhibitor Verzenio in combination with physician’s-choice endocrine therapy to cut the risk of relapse in patients with high-risk HR-positive, HER2-negative breast cancer, Lilly said in a release.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 119,800+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

FDA ad­comm to de­cide on mol­nupi­ravir EUA; Can­cer at­las un­veils new po­ten­tial drug tar­get

The FDA has another adcomm coming down the pipeline — this time on Covid-19 oral antiviral molnupiravir.

The federal agency’s advisory committee will meet on November 30th to go over Merck and Ridgeback’s EUA request for their investigational antiviral drug, and discuss the available data supporting its use in Covid-19 patients.

This comes two weeks after Merck claimed that their antiviral pill reduced the chance that newly diagnosed Covid-19 patients would be hospitalized or die by 50%. The pharma made the announcement after interim data on 775 patients in their clinical trial showed the antiviral’s potential.