Eiger chalks up one win, one loss be­tween a pair of PhII rare dis­ease drugs, reshuf­fling pri­or­i­ties

Months af­ter Eiger Bio­phar­ma­ceu­ti­cals con­ced­ed uben­imex had no ef­fect in pul­monary ar­te­r­i­al hy­per­ten­sion, the drug has proven to be a flop again — this time in low­er leg lym­phede­ma.

David Cory

When com­pared to place­bo in a Phase II study, pa­tients on uben­imex showed no im­prove­ment in the swelling on their limbs, whether mea­sured by skin thick­ness (pri­ma­ry end­point) nor limb vol­ume and bioim­ped­ance (sec­ondary end­point).

While in­ves­ti­ga­tors in the clin­ic may fur­ther an­a­lyze in­di­vid­ual pa­tient re­spons­es that “war­rant fur­ther ex­plo­ration,” the Pa­lo Al­to-based biotech says it is not plan­ning any ad­di­tion­al clin­i­cal work un­less a part­ner comes along, pre­fer­ring in­stead to fo­cus on a sec­ond set of Phase II re­sults al­so an­nounced to­day.

In­vestors are ap­par­ent­ly hap­py with the glass half full, send­ing shares $EIGR up 11%.

With avex­i­tide, Eiger is tar­get­ing the dan­ger­ous­ly low blood glu­cose lev­els that some pa­tients ex­pe­ri­ence af­ter un­der­go­ing weight loss surgery, known as post-bariatric hy­po­glycemia (PBH). The drug is a glucagon-like pep­tide-1 an­tag­o­nist — in the same class as the GLP-1 drugs mak­ing waves in di­a­betes — dosed sub­cu­ta­neous­ly, de­signed to be ad­min­is­tered by pa­tients them­selves.

Lisa Porter

“We are very pleased by the re­sults from PRE­VENT, our first out­pa­tient study of avex­i­tide in pa­tients suf­fer­ing from PBH,” said Lisa Porter, CMO of meta­bol­ic dis­eases at Eiger. “Avex­i­tide treat­ment led to clin­i­cal­ly mean­ing­ful im­prove­ments con­sis­tent­ly through­out 28-days of treat­ment, re­duc­ing post­pran­di­al hy­per­in­su­line­mic hy­po­glycemia and as­so­ci­at­ed signs and symp­toms.”

The whole study last­ed for 42 days for the 18 pa­tients en­rolled: Af­ter 14 days of place­bo in­jec­tions, the pa­tients shift­ed to 30 mg avex­i­tide twice dai­ly for 14 days, and then re­ceived 60 mg dai­ly in­jec­tions for an­oth­er 14 days. Both dos­ing reg­i­mens hit the pri­ma­ry end­point of im­prov­ing post­pran­di­al glu­cose nadir — the low­est blood sug­ar point af­ter meals — at 57.1 mg/dL (p=0.001) and 59.2 mg/dL (p=0.0002) ver­sus the place­bo num­ber of 47.1 mg/dL. The p val­ues were a bit more shaky for the sec­ondary end­point of re­duced post­pran­di­al in­sulin peak, though Eiger claims it’s al­so “sta­tis­ti­cal­ly sig­nif­i­cant with avex­i­tide 30 mg BID (349.5 vs 454.5 μIU/mL; p < 0.03) and 60 mg QD (357.2 vs 454.5 μIU/mL; p = 0.04).”

“Eiger is ad­vanc­ing on­ly the most promis­ing pro­grams in our pipeline for rare dis­eases,” said David Cory, pres­i­dent and CEO. “The com­pa­ny is now fo­cused on ad­vanc­ing plans for a new drug ap­pli­ca­tion (NDA) in Prog­e­ria, en­roll­ment in the first-ever Phase 3 study in he­pati­tis delta virus (HDV) in­fec­tion, and reg­u­la­to­ry guid­ance in post-bariatric hy­po­glycemia (PBH) in 2019.”

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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