We de­stroyed near­ly 8,000 packs of ex­pired Orkam­bi stock last year, Ver­tex tells UK MPs

To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed on Wednes­day that last year close to 8,000 packs (each con­tain­ing a 28-day sup­ply) of Orkam­bi were de­stroyed af­ter cross­ing their ex­piry date. The US drug­mak­er has been locked in ne­go­ti­a­tion with NICE, which has re­fused to al­low the drug in to Eng­land’s Na­tion­al Health Ser­vice un­til Ver­tex of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness.

In a stand­off with UK par­lia­ment ear­li­er this month, Ver­tex chief Jeff Lei­den stood his ground, de­spite be­ing chas­tised by a pletho­ra of MPs for Ver­tex’s pric­ing strat­e­gy, busi­ness mod­el and ethics.

“The prob­lem is we’ve been paint­ed as I think not be­ing will­ing to take the of­fer, the 90% dis­count that Eng­land has made to us. I would ac­tu­al­ly say it dif­fer­ent­ly. We can­not take that of­fer. Not that we won’t take it; we can’t,” he said, em­pha­siz­ing Ver­tex’s ‘ex­tra­or­di­nary’ in­vest­ment in CF R&D — and the promise of its cur­rent and fu­ture med­i­cines.

Im­age: Jeff Lei­den tes­ti­fy­ing in the Com­mons.

Last year, Ver­tex $VRTX raked in $1.26 bil­lion in net Orkam­bi rev­enue.

As part of the pro­ceed­ings, the Com­mons health and so­cial care com­mit­tee chair Sarah Wol­las­ton asked Ver­tex ex­ec­u­tives how much Orkam­bi stock had gone out of date over the past year.

Stu­art Ar­buck­le

“I do not be­lieve that we have had any stock go out of date and be de­stroyed…we do not pack­age it up un­til we are due to be sell­ing it,” Ver­tex chief com­mer­cial of­fi­cer Stu­art Ar­buck­le re­spond­ed. “I would be sur­prised if it was very much.”

On Wednes­day, in a let­ter to the com­mit­tee Ver­tex said 7,880 packs of Orkam­bi man­u­fac­tured in 2015 and 2016 to sup­ply 13 coun­tries — in­clud­ing the UK — ex­ceed­ed their best be­fore date and were ac­cord­ing­ly dis­card­ed.

“More than 80,000 packs of Orkam­bi have been dis­trib­uted for com­mer­cial sale in the EU since li­cens­ing in 2015, and more than 11,000 packs of Orkam­bi have been sup­plied free of charge…to ap­prox­i­mate­ly 600 pa­tients in Eng­land,” Ver­tex said in the state­ment.

NICE, along with NHS Eng­land, met with Ver­tex last week and agreed to con­tin­ue dis­cus­sions, a NICE spokesper­son told End­points News.

“It is un­eth­i­cal and un­law­ful to sup­ply ex­pired prod­ucts to pa­tients, and we will not do so ir­re­spec­tive of the ac­cess sit­u­a­tion in a giv­en coun­try. We re­main com­mit­ted to work­ing with NHS Eng­land and NICE to reach an ac­cess agree­ment and are con­fi­dent that we will be able to sup­ply ap­proved prod­uct to all el­i­gi­ble Eng­lish pa­tients with­in a few weeks should the gov­ern­ment de­cide to pro­vide ac­cess to our med­i­cines. In the mean­time, we will con­tin­ue to pro­vide free med­i­cines to the sick­est CF pa­tients in Eng­land,” a Ver­tex spokesper­son said in an emailed state­ment on Thurs­day.

The UK has more than 10,400 cys­tic fi­bro­sis pa­tients – the largest CF pop­u­la­tion out­side the US – and rough­ly half the pa­tients car­ry the ge­net­ic mu­ta­tion that could ben­e­fit from Orkam­bi, ac­cord­ing the Cys­tic Fi­bro­sis Foun­da­tion.

“It’s heart­break­ing that pack­ets of life­sav­ing drugs have been thrown away be­cause they’re out of date – what a des­per­ate waste. Thou­sands of peo­ple have suf­fered while these drugs have been sit­ting on the shelf. It is vi­tal the talks that have start­ed de­liv­er a re­al re­sult now. All par­ties in­volved must reach a deal im­me­di­ate­ly so that we can put an end to any fur­ther un­nec­es­sary death and de­te­ri­o­ra­tion,” a spokesper­son told End­points News.

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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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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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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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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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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.