Was PTC Ther­a­peu­tics play­ing games when it tried to defuse the de­flaza­cort con­tro­ver­sy with a $35,000 an­nu­al price?

PTC Ther­a­peu­tics CEO Stu­art Peltz

When PTC Ther­a­peu­tics $PTCT CEO Stu­art Peltz rolled out the biotech’s Q1 re­port this morn­ing, he not­ed that com­pa­ny ex­ecs came up with what they be­lieve is a “sus­tain­able” price for the cheap, old steroid ac­quired from the con­tro­ver­sial crew at Marathon Phar­ma­ceu­ti­cals for $140 mil­lion up front and tar­get­ed at the Duchenne com­mu­ni­ty in the US.

The net price, he said, would av­er­age $35,000 a year for a pa­tient that weighed 25 kilo­grams, or 55 pounds.

“We be­lieve this rep­re­sents sus­tain­able pric­ing which bal­ances pro­vid­ing ac­cess to all el­i­gi­ble pa­tients in the US in an ul­tra or­phan pop­u­la­tion while main­tain­ing suf­fi­cient in­fra­struc­ture and pro­grams, in­clud­ing con­tin­ued in­vest­ment in Duchenne,” Peltz said.

That $35,000 net — the price pay­ers could ex­pect af­ter dis­counts — sound­ed like quite a drop from the $89,000 an­nu­al price point that trig­gered a storm of protest for Marathon, un­til you start to do the math on what these pa­tients ac­tu­al­ly weigh.

Matthew Eck­ler, RBC

RBC’s Matthew Eck­ler did the math, and came up with a much high­er num­ber. If you con­sid­er that most of the boys are in the 35- to 40-ki­lo range, he says, you’re re­al­ly talk­ing about an av­er­age range more like $45,000 to $50,000 a year.

That’s 45 to 50 times more than the $1,000 av­er­age price Duchenne par­ents have been pay­ing to bring in de­flaza­cort, a gener­ic out­side the US which has been avail­able for decades at a low price. But now that it’s ap­proved and avail­able in the US, those for­eign sup­pli­ers are be­ing closed to Amer­i­can fam­i­lies. And PTC’s “net” price may ac­tu­al­ly wind up high­er than the $54,000 price Marathon field­ed, but failed to sell.

Ac­cord­ing to com­mu­ni­ty ac­tivist Chris­tine Mc­Sh­er­ry of the non­prof­it Jett Foun­da­tion, many of the old­er pa­tients like her son now weigh 60 to 100 ki­los, and at that range the price — pro­vid­ed PTC sticks to the weight/cost ra­tio — par­ents would ac­tu­al­ly be forced to buy the steroid with a net price high­er than Marathon’s $89,000 tag.

So, I asked Eck­ler, for boys weigh­ing 75 ki­los this would mean a price of $105,000?

Eck­ler replied:

Based on what they have told us yes, but that as­sumes the cost per mg is the same for all size boys.

It’s pos­si­ble that they could work out agree­ments with pay­ors where the cost per mg is not sta­t­ic based on the weight of boys. At this point (we) will have to wait and see. Re­gard­less, there will be boys who are much heav­ier than the 25mg guide­line and thus (their) cost is go­ing to be more.

PTC did not ex­plain what it’s whole­sale ac­qui­si­tion cost, or WAC, was or how the weight/price for­mu­la would work. It’s ex­treme­ly un­usu­al for com­pa­nies to avoid the high WAC price tag en­tire­ly. Most be­gin with that and then ne­go­ti­ate pri­vate­ly with pay­ers.

Even at $35,000, an­a­lysts like Eck­ler ex­pect the head­lines to con­tin­ue to run against PTC, now that the com­pa­ny has picked up the price goug­ing ti­tle that Marathon has been try­ing so hard to shed. And that could play hav­oc with PTC’s plans to start work­ing with the US Duchenne com­mu­ni­ty as it tries to force the FDA to re­verse its po­si­tion on ataluren — a drug that failed its last three stud­ies in a row — and ap­prove it for US mar­ket­ing. The drug is avail­able in Eu­rope.

Notes Eck­ler:

Drug pric­ing has re­mained in the head­lines as a non-par­ti­san po­lit­i­cal ral­ly­ing cry, and we don’t an­tic­i­pate this will change in the near term. Ul­ti­mate­ly, we see this as­set as neg­a­tive on an NPV ba­sis giv­en: 1) the sig­nif­i­cant up­front in­vest­ment ($140M vs. 2017 sales guid­ance of $5-$10M) for a drug which we view as hav­ing ~120M in peak sales po­ten­tial; 2) the avail­abil­i­ty of oth­er cor­ti­cos­teroids for DMD pa­tients; and 3) the like­li­hood of con­tin­ued pric­ing con­tro­ver­sy and pay­or push­back re­sult­ing in a tem­pered com­mer­cial launch.

I asked an­oth­er an­gry Duchenne par­ent what she planned to do now that PTC has post­ed a price.

“Still go­ing to Mex­i­co,” she replied, re­fer­ring to the low-cost phar­ma­cies that sell drugs cheap along the bor­der.

One big stum­bling block that PTC faces is gener­ic pred­nisone, which is shap­ing up as the pre­ferred steroid among pay­ers — for some ob­vi­ous rea­sons. I asked Ex­press Scripts about their ap­proach to de­flaza­cort (brand name Em­flaza), and they replied:

We are re­quir­ing a pri­or au­tho­riza­tion for this med­ica­tion. For the man­age­ment of DMD, our cri­te­ria re­quires pa­tients to try the low­er cost, equal­ly ef­fec­tive pred­nisone be­fore Em­flaza. Ex­cept for rare cas­es, the low­er cost pred­nisone should work for pa­tients. The price of pred­nisone is any­where from $0.08 per tablet to $1.50 per tablet.

The neg­a­tive sen­ti­ment isn’t sit­ting too well with the in­vest­ment com­mu­ni­ty, which has dri­ven PTC’s shares down by 10% so far to­day.

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.