Re­gen­eron, Sanofi, get ready to duke it out with Pfiz­er over a megablock­buster eczema mar­ket

An­drew Blau­velt, Ore­gon Med­ical Re­search Cen­ter

Some­time over the next few days or weeks, the FDA will like­ly hand Re­gen­eron and Sanofi an ap­proval for Dupix­ent (dupilum­ab), their ground­break­ing IL-4/IL-13 in­hibitor for eczema which a num­ber of an­a­lysts have pro­ject­ed will go on to grab $4 bil­lion-plus a year in an­nu­al sales. And they’ll hit the mar­ket with some stel­lar new 16- and 52-week da­ta from an­oth­er Phase III study as they go up against Pfiz­er’s new­ly ap­proved Eu­crisa (crisabo­role, tar­get­ing PDE4).

We knew last year that dupilum­ab had hit its marks in the piv­otal SO­LO tri­als as well as CHRONOS, set­ting it up as pos­si­bly the top drug launch slat­ed for 2017. Over the week­end, in­ves­ti­ga­tors turned out with some new goal post da­ta for CHRONOS that will do noth­ing to take the shine off of its big mar­ket pre­dic­tions.

In CHRONOS, re­searchers re­cruit­ed pa­tients whose eczema wasn’t con­trolled by top­i­cal ther­a­pies in­clud­ing cor­ti­cos­teroids. They were divvied up in­to three groups: week­ly 300 mg dos­es of dupilum­ab with top­i­cal cor­ti­cos­teroids (TCS), once every two weeks dos­es with TCS or TCS alone.

In the new CHRONOS da­ta re­viewed at the an­nu­al meet­ing of the Amer­i­can Acad­e­my of Der­ma­tol­ogy in Or­lan­do over the week­end, in­ves­ti­ga­tors not­ed that the se­vere itch­ing pa­tients are af­flict­ed by was re­duced by 55% and 58% in the two drug arms af­ter 16 weeks, com­pared to 29% of pa­tients on TCS alone. And the dis­ease score rat­ing for pa­tients dropped by 4 or more points among 77% of the pa­tients in the drug arms com­pared to 37% of the place­bo/top­i­cal cor­ti­cos­teroid group.

At 52 weeks the im­prove­ment in the itch­ing score held steady at 54% and 56% in the drug ams and 27% in the place­bo group. And the 4-point-plus im­prove­ment in dis­ease score was main­tained by 65% and 76% of the pa­tients on drug, com­pared to 26% on on­ly top­i­cal cor­ti­cos­teroids.

Com­pare that to the pri­ma­ry goal in the crisabo­role study for mild to mod­er­ate eczema:

More crisabo­role- than ve­hi­cle-treat­ed pa­tients achieved (In­ves­ti­ga­tor’s Sta­t­ic Glob­al As­sess­ment) score suc­cess (clear/al­most clear with ≥2-grade im­prove­ment; AD-301: 32.8% vs 25.4%, P = .038; AD-302: 31.4% vs 18.0%, P < .001), with a greater per­cent­age with clear/al­most clear (51.7% vs 40.6%, P = .005; 48.5% vs 29.7%, P < .001).

That’s what Pfiz­er paid $5.2 bil­lion for when it ac­quired Ana­cor.

To­geth­er, these drugs are ex­pect­ed to make a cru­cial dif­fer­ence for a big group of pa­tients who have lim­it­ed treat­ment op­tions. But Re­gen­eron and Sanofi are clear­ly gun­ning for the li­on’s share of the mar­ket with a much broad­er range of late-stage da­ta to take to pay­ers and physi­cians.

They need a clear win here. Their drug sar­ilum­ab was held up by the FDA last fall over man­u­fac­tur­ing is­sues. And their big PC­SK9 play turned in­to an em­bar­rass­ing de­fen­sive ef­fort to beat back a judge’s rul­ing that their drug should be pulled due to patent vi­o­la­tions.

“These new re­sults build up­on pre­vi­ous pos­i­tive Phase III monother­a­py da­ta. In the CHRONOS study, Dupix­ent used with top­i­cal cor­ti­cos­teroids showed sig­nif­i­cant­ly greater clear­ance of skin le­sions and over­all dis­ease sever­i­ty com­pared to  top­i­cal cor­ti­cos­teroids alone, which are com­mon­ly pre­scribed for mod­er­ate-to-se­vere atopic der­mati­tis,” said An­drew Blau­velt, pres­i­dent of Ore­gon Med­ical Re­search Cen­ter and prin­ci­pal in­ves­ti­ga­tor of the study. “This study pro­vides pos­i­tive long-term da­ta for Dupix­ent, which is im­por­tant giv­en atopic der­mati­tis is a chron­ic in­flam­ma­to­ry dis­ease. Ad­di­tion­al­ly, the pre­sen­ta­tion high­lights the crit­i­cal role of IL-4 and IL-13 as dri­vers of this atopic con­di­tion.”

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.

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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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.

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.

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).

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.