Ul­tragenyx stum­bles with UX007 PhI­II, shares plunge

Rare dis­ease spe­cial­ist Ul­tragenyx Phar­ma $RARE has aban­doned its ex­per­i­men­tal drug, UX007, as a treat­ment for Glut1 DS – a de­bil­i­tat­ing dis­ease char­ac­ter­ized by seizures, de­vel­op­men­tal de­lay, and move­ment dis­or­ders. The drug­mak­er, whose stock fell sharply in ear­ly trad­ing, said the drug failed to demon­strate a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in a Phase III tri­al.

UX007, al­so dubbed tri­hep­tanoinwas de­signed to ad­dress glu­cose trans­porter type-1 de­fi­cien­cy syn­drome — or Glut1 DS — by get­ting glu­cose in­to the brains of the ge­net­i­cal­ly af­flict­ed group. With­out ad­e­quate glu­cose, pa­tients have a ten­den­cy to suf­fer from re­peat­ed seizures. Rough­ly 500 cas­es have been re­port­ed world­wide since the dis­or­der was first iden­ti­fied in 1991, ac­cord­ing to the NIH.

The late-stage, place­bo-con­trolled tri­al in­clud­ed 44 chil­dren and adults with Glut1 DS that were ex­pe­ri­enc­ing dis­tinct episodes of ab­nor­mal move­ments. The main goal was to achieve a sig­nif­i­cant re­duc­tion in the fre­quen­cy of so-called dis­abling parox­ys­mal move­ment dis­or­ders, while sec­ondary end­points in­clud­ed re­duc­ing the du­ra­tion of of the move­ment dis­or­der events; walk­ing ca­pac­i­ty and en­durance; pa­tient-re­port­ed as­sess­ments of phys­i­cal func­tion, up­per ex­trem­i­ty func­tion, fa­tigue and pain. Nei­ther the pri­ma­ry nor the sec­ondary end­points were met, Ul­tragenyx said.

The an­nounce­ment sent the biotech’s shares tum­bling about 16% in ear­ly trad­ing. Many in­vestors were cau­tious on Glut1DS prospects, so “we are sur­prised by the stock mar­ket re­ac­tion, the chal­leng­ing tape notwith­stand­ing,” Leerink’s Joseph Schwartz wrote in a note. 

But all is not lost. De­spite a failed mid-stage study, Ul­tragenyx in Au­gust said the FDA was will­ing to con­sid­er an ac­cel­er­at­ed path­way for UX007 as a treat­ment for LC-FAOD — a group of con­di­tions that pre­vent the body from break­ing down long-chain fat­ty acids in­to en­er­gy — af­ter the drug­mak­er cob­bled to­geth­er da­ta un­der­scor­ing a fall in ma­jor clin­i­cal events for LC-FAOD.

On Fri­day, the mak­er of the $200,000 rare dis­ease treat­ment buro­sum­ab said the FDA had ac­cept­ed its pro­pos­al to sub­mit an ap­pli­ca­tion to mar­ket UX007 for LC-FAOD based on ex­ist­ing da­ta. Ul­tragenyx, which al­so sells Mep­se­vii, is al­so talk­ing to reg­u­la­tors at the EMA, and will pro­vide an up­date on those dis­cus­sions lat­er this year.

“Tri­hep­tanoin seems like­ly to ob­tain FDA ap­proval in…LC-FAOD – which to­geth­er with oth­er progress at the com­pa­ny sug­gests to us down­side for RARE is lim­it­ed at cur­rent lev­els,” Schwartz added.

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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