Dems fire off a sil­ly pric­ing bill; What's wrong with No­var­tis?

End­points as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

The De­moc­rats field few good ideas in a drug pric­ing bill that is DOA

Any Sen­ate bill that re­lies on a long list of De­moc­rats for its sup­port doesn’t have much chance of be­com­ing law. But the Dems’ pro­posed law does help il­lus­trate what Par­ty lead­ers are think­ing these days, and where Pres­i­dent Trump may find com­mon ground if the ul­tra con­ser­v­a­tive wing of the Re­pub­li­can Par­ty can’t be brought back in­to the fold.

At the top of the list of things-phar­ma-com­pa­nies-hate is a re­quire­ment to break out de­vel­op­ment costs for each new drug. The un­end­ing de­bate over the cost of R&D gen­er­al­ly re­volves around glob­al R&D bud­gets com­pared to ap­provals. The De­moc­rats want to see some hard num­bers for each new pro­gram, prob­a­bly so they can start sham­ing man­u­fac­tur­ers for ex­ces­sive pric­ing.

Medicare ne­go­ti­a­tions are back, of course. Al­ready sup­port­ed by Trump, gov­ern­ment an­a­lysts have con­clud­ed that Medicare price ne­go­ti­a­tions as such wouldn’t like­ly cre­ate much in the way of sav­ings. That would take a for­mu­la­ry, and the threat of kick­ing off drugs priced too high, an idea which caus­es angst in phar­ma cir­cles.

In this new bill Medicare would be al­lowed to use VA prices or a price list from some oth­er agency if of­fi­cials can’t ne­go­ti­ate a “fair” price. We al­ready know from the Cal­i­for­nia bill that drug­mak­ers would fight this to the last lob­by­ist. There’s al­so a spe­cial ex­cise tax in the event a phar­ma com­pa­ny push­es up the price of drug more than med­ical in­fla­tion.

In­ter­est­ing­ly, there’s $2 bil­lion “prize” mon­ey for new an­tibi­otics. Giv­en the need here, and the low cost, it’s un­like­ly to spur much op­po­si­tion. And there’s $10 bil­lion to pay for drug tri­als at the NIH. Al­so, not so con­tro­ver­sial, but right now the fo­cus in the new ad­min­is­tra­tion is on cut­ting the NIH bud­get, not adding to it.

Bot­tom line: Aside from Medicare ne­go­ti­a­tions, there isn’t much here for a bi­par­ti­san ap­proach. It’s DOA, like most every­thing else in the Capi­tol these days.

Num­bers be­fore peo­ple. It’s the in­dus­try stan­dard.

Over the past week we’ve cov­ered sev­er­al new re­or­ga­ni­za­tion sto­ries. Am­gen led the way, with its planned move push­ing about 10% of its Thou­sand Oaks staff out of HQ. The cuts or re­lo­ca­tions in­volve mov­ing po­si­tions and re­searchers to the two big hubs of the Bay Area and Cam­bridge/Boston. Ver­tex, we learned from an SEC fil­ing, is shut­ter­ing its Cana­di­an R&D fa­cil­i­ty in a con­sol­i­da­tion. R&D sources tell us they’ve al­so been root­ing out pock­ets of the “Old Guard” in what are re­ferred to as “stealth lay­offs.” And Take­da gave us an ex­am­ple of the M&A va­ri­ety of job cuts, with their de­ci­sion to ax 180 of about 300 jobs at the new­ly ac­quired Ari­ad (with some axed staffers go­ing to a CRO).

With biotech val­u­a­tions be­ing what they are, it shouldn’t sur­prise any­one to see buy­ers cut as deep as they pos­si­bly can af­ter an ac­qui­si­tion. It’s one way to make num­bers work. That’s a sim­ple, though painful, re­al­i­ty.

Am­gen, for all its biotech his­to­ry, is now a Big Phar­ma and it will look for ef­fi­cien­cies wher­ev­er it can find them. Like all the Big Phar­mas, the on­ly con­stant in R&D is change.

Ver­tex gets a black star next to its name. Not for the Cana­di­an re­treat — every­body is con­sol­i­dat­ing in des­ig­nat­ed hubs — but for its re­fusal to be hon­est about the stealth lay­offs. You don’t have to be down­siz­ing to note when you are lay­ing peo­ple off from their jobs.

I was asked by a Roche em­ploy­ee at Nut­ley a few years ago what I thought about job se­cu­ri­ty in R&D. I told her I didn’t see much of it. The fact is com­pa­nies will do what’s in the best in­ter­est of their share­hold­ers every time. For rank-and-file em­ploy­ees, that takes a will­ing­ness to stay flex­i­ble and move as need­ed and de­sired.

The con­stant churn helps with new com­pa­ny cre­ation, but the me­chan­ics of this new re­al­i­ty are un­for­giv­ing at a per­son­al lev­el.

You can’t fight a trend like this. But you can de­ter­mine the best way to man­age a ca­reer in which job se­cu­ri­ty is a thing of the past — if it ever ex­ist­ed.

What’s wrong with No­var­tis?

I have no in­side knowl­edge why Karen Walk­er is leav­ing her se­nior po­si­tion in charge of cell ther­a­py man­u­fac­tur­ing for No­var­tis. Seat­tle is a nice place to live, or so I hear, and for all I know Seat­tle Ge­net­ics made her an of­fer she couldn’t refuse. That’s her busi­ness.

But her tim­ing, leav­ing two days af­ter the FDA ac­cept­ed No­var­tis’ ap­pli­ca­tion for its pi­o­neer­ing CAR-T, couldn’t have been worse for the com­pa­ny. Man­u­fac­tur­ing is a crit­i­cal el­e­ment in this field, more than most oth­ers, and she would have played a key role in its re­view.

It’s an­oth­er ex­am­ple of the ex­o­dus of tal­ent from No­var­tis over the past year. Some of that is self-in­flect­ed, like No­var­tis’ ques­tion­able de­ci­sion to dis­solve an in­de­pen­dent cell and gene ther­a­py unit, which led Oz Azam to leave the com­pa­ny. But bleed­ing tal­ent the way No­var­tis has is in­dica­tive of deep prob­lems.

The up­side here is that lots of these ex­pe­ri­enced ex­ecs — like Azam and Walk­er — are tak­ing new jobs in a boom­ing biotech sec­tor. But what’s wrong with No­var­tis?

Third Rock’s lat­est start­up launch­es in style, with an am­bi­tious set of goals

While turnover re­mains high, the in­dus­try re­mains vi­brant be­cause new com­pa­ny cre­ation in biotech is strong. One of our top sto­ries this week fo­cused on a clas­sic Third Rock start­up, Tan­go Ther­a­peu­tics. I en­joyed cov­er­ing the sci­ence they will be ex­plor­ing, as the team grows in­to the mid-20s.

This com­pa­ny has every­thing a start­up wants: Brains, mon­ey and am­bi­tion. And it’s carv­ing out new ter­ri­to­ry in drug de­vel­op­ment, go­ing big.

It’s com­pa­nies like these that make my job fun. Whether it works or not, it cer­tain­ly looks like they de­serve a shot. And it keeps the big hubs like Boston/Cam­bridge teem­ing with new ideas for old, im­pos­si­ble tar­gets. Thumbs up, in­deed.

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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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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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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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

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.