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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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