Alex­ion bags a block­buster new ap­proval for its cash cow Soliris. Now what?

Alex­ion $ALXN has won ap­proval to sell its main­stay ther­a­py Soliris for an­oth­er batch of pa­tients suf­fer­ing from an ul­tra-rare con­di­tion — an OK that could well be worth north of a bil­lion dol­lars.

Lud­wig Hantson

Last night the FDA came through with an OK for a sub­set of pa­tients suf­fer­ing from myas­the­nia gravis, set­ting the stage for a new sales ef­fort that Leerink’s Ge­of­frey Porges be­lieves will rack up $1.2 bil­lion in sales 5 years from now. That’s close to a fifth of the rev­enue he’s pro­ject­ing from Soliris — one of the world’s most ex­pen­sive ther­a­pies — for 2022.

That’s a big win for CEO Lud­wig Hantson and the new team at Alex­ion. Over the past year the biotech has un­der­gone a sea change, punt­ing the pre­vi­ous CEO and CFO in the wake of a sales scan­dal and ush­er­ing in a new regime that is en­tire­ly fo­cused on re­mak­ing the pipeline at Aex­ion as they move the com­pa­ny from Con­necti­cut to Boston’s Sea­port dis­trict.

Alex­ion, though, is dan­ger­ous­ly de­pen­dent on Soliris for its fu­ture, at a time a num­ber of small biotechs are tak­ing a shot at over­turn­ing its block­buster fran­chise and carv­ing out mar­ket share. This new ap­proval in­creas­es the drug’s im­por­tance at Alex­ion as the new crew seeks to di­ver­si­fy, like­ly through a slate of up­com­ing deals af­ter rolling out its re­cent plan to shake up the pipeline.

At $542,640 a year, Soliris re­mains one of the 10 most ex­pen­sive ther­a­pies on the plan­et.

Ge­of­frey Porges

The OK from the FDA is for an­ti-AchR an­ti­body-pos­i­tive gMG, which cov­ers about 5% to 10% of the to­tal for that con­di­tion. Porges not­ed this morn­ing:

Based on our re­cent MEDA­Corp physi­cian in­ter­ac­tions, we be­lieve most of the doc­tors treat­ing gMG pa­tients have al­ready ear­marked a sub­group of pa­tients for whom Soliris is ap­pro­pri­ate and in­tend to be­gin treat­ment with­in the next weeks-month. We be­lieve that re­cent con­sen­sus was still dis­count­ing Soliris’ ap­proval and/or com­mer­cial op­por­tu­ni­ty in gMG based on sales es­ti­mates (LP $5.3bn vs con­sen­sus $4.7bn in 2022). We es­ti­mate that gMG will pro­vide an in­cre­men­tal $1.2bn glob­al rev­enue for Soliris in 2022, rep­re­sent­ing ~23% of our to­tal $5.3bn pro­ject­ed sales for Soliris in the same year.

“This is a land­mark day for the mem­bers of the U.S. myas­the­nia gravis com­mu­ni­ty, who have not seen a ther­a­py ap­proved for gen­er­al­ized myas­the­nia gravis in more than 60 years,” said Nan­cy Law, the CEO of the Myas­the­nia Gravis Foun­da­tion of Amer­i­ca. “It is par­tic­u­lar­ly sig­nif­i­cant that this ap­proval of Soliris will pro­vide a new op­tion for those with gMG and es­pe­cial­ly for those who do not re­spond ad­e­quate­ly to or can­not tol­er­ate stan­dard treat­ment op­tions.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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