Here's why Su­cam­po paid $200M for its PhI­II Nie­mann-Pick drug

In­ves­ti­ga­tors have out­lined the re­sults of a small, sin­gle-arm study of a new drug for Nie­mann-Pick type C dis­ease which in­spired a $200 mil­lion biotech buy­out ear­li­er this year.

The drug is cy­clodex­trin, or at least a unique mix­ture of 2-hy­drox­ypropyl-ß- cy­clodex­trins (HPßCD) that Vtesse had moved in­to a piv­otal study af­ter glean­ing pos­i­tive da­ta from a study with 14 pa­tients. Su­cam­po bagged the biotech and its drug for $200 mil­lion in April, tak­ing over con­trol of the Phase III.

Daniel Ory

Ac­cord­ing to Daniel Ory of Wash­ing­ton Uni­ver­si­ty School of Med­i­cine in St Louis — the first au­thor of a new pa­per pub­lished in The Lancet — re­searchers re­cruit­ed 14 NPC pa­tients, who typ­i­cal­ly die of the dis­ease 10 to 15 years af­ter it’s di­ag­nosed.

De­liv­ered in the spinal col­umn every two weeks for 18 months, the symp­toms of 7 of the 14 pa­tients im­proved, with some re­gain­ing an abil­i­ty to speak as re­searchers tracked scores on gait, cog­ni­tion and speech.

To be sure, this is no cure. The re­searchers say that the scores of pa­tients for the point sys­tem they used wors­ened by 1.2 per year on av­er­age. But that com­pares to the his­tor­i­cal da­ta they used from an ear­li­er study, as there was no place­bo arm, with an ex­pect­ed 2.9 point de­cline. And in­ves­ti­ga­tors say that they tracked hear­ing loss as a se­ri­ous ad­verse event in the study.

“Some of the pa­tients be­gan this tri­al with­out the abil­i­ty to speak, and now they speak,” Ory said. “There is a slow­ing of the de­cline, but we were sur­prised to see trends to­ward im­prove­ment in a few cat­e­gories. Com­pared with the his­tor­i­cal da­ta, half of the pa­tients in this study saw an im­prove­ment or no wors­en­ing in the neu­ro­log­i­cal sever­i­ty score.”

Added Ory: “A ther­a­py that caus­es hear­ing loss is not ide­al. But since the dis­ease it­self caus­es hear­ing loss, we felt that this side ef­fect may be a rea­son­able trade-off, giv­en the al­ter­na­tive de­cline and death that the dis­ease al­so caus­es.”

Re­searchers at the NIH’s Na­tion­al In­sti­tute of Child Health and Hu­man De­vel­op­ment de­signed and con­duct­ed the study. And they say that they were al­so able to track a pos­i­tive in­flu­ence on sev­er­al key bio­mark­ers for the dis­ease, which is caused by the buildup of cho­les­terol in brain cells.

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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