Ver­tex's rapid-fire PhI­II pro­gram hits a road­block at the FDA. Will reg­u­la­tors force ri­val Gala­pa­gos to slow down too?

Ver­tex’s R&D team doesn’t make many mis­takes. So dur­ing yes­ter­day’s Q1 call, a group of an­a­lysts were quick to pick up on a rare stum­ble that just might make it more vul­ner­a­ble to a ri­val like Gala­pa­gos.

Ge­of­frey Porges, Leerink

While not­ing plans for launch­ing a new Phase III ef­fort with a com­bi­na­tion drug us­ing VX-445 with Symdeko, fol­low­ing up as planned, in­ves­ti­ga­tors $VRTX al­so con­ced­ed that the FDA sees its deuter­at­ed ver­sion of Ka­ly­de­co — VX-561 — as a nov­el agent that needs some ad­di­tion­al dose-rang­ing tri­al work be­fore reg­u­la­tors will al­low a move in­to Phase III.

Ver­tex bought ‘561 from Con­cert a lit­tle more than a year ago for $160 mil­lion in cash and an­oth­er $90 mil­lion in mile­stones, tak­ing over the Phase II tri­al.

Ge­of­frey Porges at Leerink sees a de­lay on VX-561 of at least a year, forc­ing the shift of fo­cus to the VX-445 com­bo now as a kind of Plan B.

This means that the com­pa­ny is now run­ning two rough­ly equiv­a­lent hors­es in the same ex­pen­sive race, and while the risk mit­i­ga­tion from this strat­e­gy is laud­able, it does seem more ex­pen­sive, in terms of tri­al costs, time and rev­enue fore­gone, than is nec­es­sary. Ver­tex may still de­vel­op a Vx561 con­tain­ing once-dai­ly triple com­bi­na­tion, but such a prod­uct might not come to mar­ket now un­til 2022 or 2023. The FDA’s stance does pro­vide some re­as­sur­ance about Ver­tex’s com­pet­i­tive po­si­tion – the FDA is clear­ly sig­nal­ing that there are no short-cuts (at least in the US) to tra­di­tion­al good drug de­vel­op­ment prac­tices in this in­di­ca­tion.

That “com­pet­i­tive po­si­tion” he’s re­fer­ring to deals with Gala­pa­gos $GLPG, which launched an ex­plorato­ry study called FAL­CON for its triple ear­li­er this week. And what the FDA finds good for Ver­tex, they’ll like­ly find good for Gala­pa­gos, where the dose-rang­ing stan­dards may al­so like­ly to slow things down. Notes Michael Yee from Jef­feries:

Thus since GLPG has all new NCE’s they will need to do lots of dose-find­ing work re­quir­ing lots more Phase IIs than the “quick” stud­ies they’re do­ing now…so while GLPG might have “da­ta” for FAL­CON study H2-18 and more da­ta in 2019 — these are sim­ple stud­ies and will need thor­ough work per FDA read-through.

Bri­an Sko­r­ney

Baird’s Bri­an Sko­r­ney al­so took a shot at Gala­pa­gos.

(W)e be­lieve it shows clear reg­u­la­to­ry prece­dent for po­ten­tial com­peti­tors who think they can move a com­bi­na­tion of three new chem­i­cal en­ti­ties in­to late stage de­vel­op­ment with­out demon­strat­ing the clin­i­cal at­trib­ut­es and li­a­bil­i­ties of each com­po­nent (we’re look­ing at you Gala­pa­gos).

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Which top 10 big phar­mas have the most to gain — or lose — over the next 5 years?

When Evaluate Pharma crunched the likely drug sales numbers for the big 10, 2 stood out. 

Takeda, with its big Shire buyout under its belt, is set to almost double its worldwide sales record for 2018 over 5 years, putting it in the big 10 — the 9th spot, to be exact — which is exactly where CEO Christophe Weber wants to be. 

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Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

J&J's Es­ke­t­a­mine, at cur­rent price, is 'low val­ue for mon­ey' — ICER

For John­son & John­son’s $JNJ phar­ma­ceu­ti­cal ver­sion of the hal­lu­cino­genic anes­thet­ic ke­t­a­mine — es­ke­t­a­mine — to be cost-ef­fec­tive for use in treat­ment-re­sis­tant de­pres­sion in the long term, its list price must be cut by up to half, ICER con­clud­ed in its fi­nal re­port on Thurs­day.

Cog­nizant of the myr­i­ad of ap­proved an­ti­de­pres­sants that of­ten don’t work, the US reg­u­la­tor en­dorsed J&J’s es­ke­t­a­mine, brand­ed as  — Spra­va­to — in March for treat­ment-re­sis­tant de­pres­sion, con­scious that the orig­i­nal cat tran­quil­iz­er is fre­quent­ly used off-la­bel for se­vere de­pres­sion.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.

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