Hori­zon Phar­ma folds its cards on Friedre­ich’s Atax­ia af­ter Ac­tim­mune flunks PhI­II

Tim­o­thy P. Wal­bert, Hori­zon

A year-and-a-half af­ter launch­ing their Phase III study of their lead drug Ac­tim­mune for Friedre­ich’s Atax­ia, Hori­zon Phar­ma has had to call it quits af­ter rack­ing up a full slate of end­point fail­ures.

Not on­ly did the drug (in­ter­fer­on gam­ma-1b) flunk the pri­ma­ry on the mod­i­fied Friedre­ich’s Atax­ia Rat­ing Scale at 26 weeks com­pared to a place­bo, it al­so failed the sec­ondary guide posts as well. The Dublin-based com­pa­ny is now shut­ter­ing the pro­gram and walk­ing away.

Trad­ing of Hori­zon’s shares $HZNP was halt­ed ahead of the an­nounce­ment ear­ly Thurs­day, then plunged 22% when trad­ing was re­sumed.

Hori­zon Phar­ma has turned to buy­outs to ex­pand its port­fo­lio. The com­pa­ny bagged Rap­tor Phar­ma­ceu­ti­cals $RPTP and a pair of rare dis­ease drugs on the mar­ket — along with a mul­ti-drug pipeline — in an $800 mil­lion cash deal two months ago. Hori­zon paid $9 a share for Rap­tor, which al­so has been pur­su­ing a trou­bled de­vel­op­ment path for a new drug for Hunt­ing­ton’s drug, now in a piv­otal tri­al.

“A well-de­signed, rig­or­ous study like STEAD­FAST would not have been pos­si­ble with­out the ex­tra­or­di­nary dri­ve of the FA com­mu­ni­ty, par­tic­u­lar­ly the peo­ple who en­rolled in the study, the clin­i­cal tri­al in­ves­ti­ga­tors and the Friedre­ich’s Atax­ia Re­search Al­liance,” said Tim­o­thy P. Wal­bert, chair­man, pres­i­dent and chief ex­ec­u­tive of­fi­cer, Hori­zon Phar­ma.  “While the re­sults were not what we hoped for, this is the very rea­son why re­search and de­vel­op­ment is im­por­tant – to find an­swers that may help in­form fu­ture re­search.”

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.

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.

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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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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Re­gen­eron/Sanofi's an­ti­body un­der­whelms in asth­ma study — shares of ri­val Anap­tys­Bio pay the price

Al­though ex­pec­ta­tions were mut­ed, Re­gen­eron $REGN and Sanofi’s $SNY ex­per­i­men­tal IL-33 an­ti­body has un­der­whelmed in a proof-of-con­cept mid-stage asth­ma tri­al. Al­though the drug sig­nif­i­cant­ly im­proved the loss of asth­ma con­trol and lung func­tion as a monother­a­py com­pared to a place­bo, its ef­fect was nei­ther su­pe­ri­or to the es­tab­lished Dupix­ent, nor of val­ue when com­bined with the IL-4/IL-13 treat­ment.

Green-light­ed in Japan, FDA quick­ly spurns Dai­ichi Sanky­o's flawed ap­pli­ca­tion for AML drug

Three days af­ter win­ning Japan­ese ap­proval for its acute myeloid leukemia drug quizar­tinib, Dai­ichi Sankyo is be­ing forced to en­dure an em­bar­rass­ing re­jec­tion at the hands of the FDA.

US reg­u­la­tors wast­ed no time in bat­ting back quizar­tinib af­ter first high­light­ing the messy da­ta in its ap­pli­ca­tion in an in­ter­nal re­view, that in turn per­suad­ed a large ma­jor­i­ty of out­side ex­perts to rec­om­mend a re­jec­tion for the drug, which tar­gets FLT3-ITD–pos­i­tive AML cas­es.

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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HIV, pneu­mo­coc­cal — and what? Mer­ck­'s un­ex­pect­ed pipeline high­light ex­cites a lit­tle biotech

In an R&D update dominated by oncology — mostly Keytruda, followed by Lynparza and Lenvima — Merck chose to highlight a program in sensory pathology, an HIV drug, and a group of pneumococcal vaccines. And that has made at least one biotech very happy.

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