Alex­ion preps an FDA pitch on Soliris suc­ces­sor while sig­nal­ing more deals ahead for rare dis­ease drugs

Alex­ion used its Q1 up­date Thurs­day to un­veil an­oth­er set of Phase III non-in­fe­ri­or stats for its Soliris suc­ces­sor, adding to its reg­u­la­to­ry pack­age for new ap­provals that should ship lat­er this year.

Fol­low­ing ear­li­er proof of non-in­fe­ri­or­i­ty for treat­ment-naive pa­tients, the new Phase III high­light­ed their suc­cess­ful switch­ing of Soliris pa­tients to ALXN1210, a shift from dos­ing every two weeks to eight weeks.

Lud­wig Hantson

To be sure, the in­ves­ti­ga­tors un­der­scored the same kind of im­prove­ments they found over Soliris on key mea­sures, but there was noth­ing sta­tis­ti­cal­ly sig­nif­i­cant about it. That will be good for shoring up their de­fense of the fran­chise drug for years to come — com­pa­ny ex­ecs talked of patents stretch­ing out to 2035 — as ri­vals press along with new drugs they hope can beat the stan­dard ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

Now the biotech is prep­ping reg­u­la­to­ry pitch­es that should ar­rive in a mat­ter of months.

Ear­li­er this month Alex­ion an­nounced a deal to buy a Stock­holm-based biotech for $855 mil­lion in cash, bag­ging a new drug for rare cas­es of Wil­son dis­ease. The com­pa­ny is us­ing that as a mod­el for more deals just like it.

Paul Clan­cy

CFO Paul Clan­cy not­ed:

I would char­ac­ter­ize the bias to­wards prod­ucts, not to­wards plat­forms. Nev­er say nev­er, but this is rare dis­ease. Wil­son rep­re­sents a good ex­am­ple of our bias. Rare dis­ease, dev­as­tat­ing dis­eases, a po­ten­tial prod­uct to trans­form the dis­ease, and we’re build­ing up our abil­i­ty to do that in­side the com­pa­ny.

CEO Lud­wig Hantson sought to re­as­sure an­a­lysts that they were sat­is­fied that they could con­vert a large num­ber of pa­tients to the new drug, which some an­a­lysts be­lieve will have to come with a dis­count for pay­ers. Dur­ing the call with an­a­lysts, the CEO said:

We have a dif­fer­en­ti­at­ed pro­file with 1210. I don’t think any­body will dis­pute that. And, for sure, we’re go­ing to try to get the best la­bel. And we be­lieve that the da­ta that we have will be re­flect­ed in the clin­i­cal tri­al sec­tion. The way I look at it, we don’t need the su­pe­ri­or­i­ty claim to be suc­cess­ful for a fast con­ver­sion. We have a strong, dif­fer­en­ti­at­ed clin­i­cal pro­file, a ro­bust da­ta pack­age.

It’s not ide­al, but an­a­lysts like Ge­of­frey Porges saw rea­sons for ap­plause.

Alex­ion not­ed that ‘1210 was nu­mer­i­cal­ly su­pe­ri­or on both the pri­ma­ry and all key sec­ondary end­points. We are al­so en­cour­aged by the dis­clo­sure that ze­ro sub­jects treat­ed with ‘1210 ex­pe­ri­enced break­through he­mol­y­sis ver­sus 5 sub­jects treat­ed with Soliris. In terms of safe­ty, there were no dis­con­tin­u­a­tions due to ad­verse events, neu­tral­iz­ing an­ti­bod­ies, or cas­es of meningo­coc­cal in­fec­tion.

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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