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.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.