Al­ny­lam flags loom­ing FDA ap­pli­ca­tion for givosir­an, adding an­oth­er round of stel­lar ef­fi­ca­cy da­ta from ear­ly study

Al­ny­lam $AL­NY is buff­ing up its da­ta on givosir­an in an­tic­i­pa­tion of launch­ing an as­sault on an ac­cel­er­at­ed ap­proval lat­er in the year.

The biotech is of­fer­ing up some long-term ex­ten­sion da­ta from a small, ear­ly-stage tri­al of its num­ber two RNAi drug in the pipeline. Re­searchers are stick­ing with the low dose of the drug af­ter track­ing a big drop in the rate of acute he­pat­ic por­phyr­ia at­tacks along with lev­els of aminole­vulin­ic acid, be­lieved to be the pri­ma­ry dis­ease trig­ger for the ul­tra-rare dis­ease, and the bio­mark­er for por­pho­bilino­gen.

Akin Ak­inc

Fol­low­ing up on its first set of pos­i­tive num­bers for the Phase I tri­al, in­ves­ti­ga­tors tracked a 75% drop in the rate of at­tacks, do­ing even bet­ter with a 90% drop in an ex­ten­sion study rel­a­tive to the run-in base­line rate ahead of dos­ing, en­cour­ag­ing them to see a ris­ing rate of ef­fi­ca­cy with longterm dos­ing.

“We view these new re­sults with givosir­an as very en­cour­ag­ing, since they demon­strate ro­bust and what we be­lieve to be clin­i­cal­ly mean­ing­ful re­duc­tions in uri­nary ALA, por­phyr­ia at­tack rate, and hemin ad­min­is­tra­tion with con­tin­ued dos­ing for up to near­ly two years,” said Akin Ak­inc, who’s run­ning the pro­gram at Al­ny­lam. “We al­so be­lieve our new da­ta sup­port use of a month­ly dos­ing reg­i­men for sus­tained re­duc­tions in ALAS1 mR­NA and uri­nary ALA, with im­proved clin­i­cal ac­tiv­i­ty.”

Ak­inc al­so not­ed in a state­ment that re­searchers are close to en­rolling the 30th pa­tient in their on­go­ing Phase III study, leav­ing them with­in shot of in­ter­im da­ta that could be used to sup­port an NDA fil­ing be­fore the end of this year.

If they’re proven right, that would be the sec­ond drug Al­ny­lam has pitched to the FDA, with patisir­an cur­rent­ly un­der re­view and tipped to mus­cle aside a ri­val ther­a­py de­vel­oped by Io­n­is. Givosir­an jumped up in the queue at Al­ny­lam af­ter the biotech was forced to aban­don a pro­gram for its hered­i­tary AT­TR amy­loi­do­sis med re­vusir­an in 2016 af­ter mon­i­tors tracked a high­er rate of deaths in the drug arm. 

Al­ny­lam’s drugs have been dogged by safe­ty is­sues through its his­to­ry, but ap­pears poised to make the leap in­to com­mer­cial­iza­tion work with patisir­an pos­si­bly fol­lowed quick­ly by givosir­an.

The Al­ny­lam group has not­ed a num­ber of ad­verse events among the pa­tients treat­ed with givosir­an, adding one that they say is like­ly linked to their drug. A pa­tient with a his­to­ry of al­ler­gic re­ac­tions ex­pe­ri­enced an ana­phy­lac­tic re­ac­tion af­ter their third dose of givosir­an and dis­con­tin­ued from the study.

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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