Trump's White House pro­pos­es raft of cuts to sci­ence R&D bud­gets, but FDA scores high­er funds

Pres­i­dent Trump’s 2020 bud­get pro­pos­al is out, and it calls for a slew of cut­backs to sci­ence fund­ing at NIH and the Na­tion­al Sci­ence Foun­da­tion, while of­fer­ing to boost the cof­fers at the FDA. The plan now goes to a split Con­gress, like­ly to weigh in and sig­nif­i­cant­ly al­ter the White House’s blue­print.

Over­all, the ad­min­is­tra­tion has pro­posed a rough­ly 12% cut to the HHS bud­get to $87.1 bil­lion. The NIH bud­get has been culled even as it en­hances the agency’s re­spon­si­bil­i­ty by ad­vo­cat­ing for pub­licly-fund­ed R&D ini­tia­tives to end new HIV trans­mis­sions by 2030 and de­vel­op new treat­ments for pe­di­atric can­cer. To be sure, this White House has pro­posed cuts to the NIH for the last two years — but the agency en­joys bi­par­ti­san sup­port in Con­gress and leg­is­la­tors will like­ly bat away any sug­ges­tions to shave the in­sti­tute’s bud­get in the com­ing fis­cal year.

Among the key propo­si­tions laid out in­clud­ed cap­ping out-of-pock­et costs for the el­der­ly cov­ered by Medicare, aug­ment­ing the FDA’s au­thor­i­ty to crack down on drug­mak­ers look­ing to thwart gener­ic drug ap­provals and com­pe­ti­tion, and re­form the 340B pro­gram by ask­ing par­tic­i­pat­ing fa­cil­i­ties to re­port how they are us­ing sav­ings.

Un­sur­pris­ing­ly, the bud­get al­so re­it­er­at­ed the White House’s fo­cus on rein­ing in drug prices, in­clud­ing em­pow­er­ing state gov­ern­ments to ne­go­ti­ate prices as part of their Med­ic­aid bud­gets di­rect­ly with man­u­fac­tur­ers and shore up trans­paren­cy so pa­tients are not sad­dled with sur­prise bills.

“The pro­posed bud­get in­creas­es the drum­beat on drug pric­ing re­form, and makes the pos­si­bil­i­ty of ei­ther ex­ec­u­tive and/or leg­isla­tive ac­tion in­creas­ing­ly like­ly,” Leerink Ana Gupte wrote in a note, sug­gest­ing that the pro­pos­al’s re­peat­ed fo­cus on prices fol­low­ing the HHS pro­pos­al to elim­i­nate drug man­u­fac­tur­er re­bates in­di­cates change is in the off­ing at the end of the com­ment pe­ri­od on April 8. “Our ex­pec­ta­tion that ma­jor change will be like­ly de­layed to 2021 af­ter the elec­tion.”

A cru­cial piece of pro­posed leg­is­la­tion was the im­po­si­tion of $100 mil­lion in user fees for the e-cig­a­rette in­dus­try, one that has fre­quent­ly met the ire of out­go­ing FDA com­mis­sion­er Scott Got­tlieb, who has long ad­mon­ished man­u­fac­tur­ers for en­tic­ing youth in­to nico­tine de­pen­den­cy with ex­pert­ly mar­ket­ed prod­ucts. At the mo­ment, these fees are lim­it­ed to cig­a­rettes, cig­ars and some oth­er to­bac­co prod­ucts. The funds will be used to de­vel­op a “reg­u­la­to­ry frame­work for the ap­pro­pri­ate over­sight of e-cig­a­rettes and in tak­ing con­tin­ued steps to re­duce youth use of all to­bac­co prod­ucts,” the FDA said in a sep­a­rate state­ment on Mon­day.

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 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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