Bio­gen hy­pes a fresh set of ear­ly, up­beat re­sults for Alzheimer’s drug

New da­ta from an ear­ly-stage study of Bio­gen’s Alzheimer’s drug ad­u­canum­ab has demon­strat­ed a clear im­pact on de­posits of amy­loid be­ta, the tox­ic tan­gles in the brain sus­pect­ed of trig­ger­ing the mem­o­ry-wast­ing ail­ment. And while they couldn’t pre­cise­ly prove it, the re­searchers spot­light­ed ad­di­tion­al ev­i­dence that the drug might al­so be slow­ing the steady cog­ni­tive de­cline of the peo­ple tak­ing the drug.

Al San­drock, Bio­gen Chief Med­ical Of­fi­cer

The ev­i­dence — a de­tailed re­tread of re­sults that have al­ready been re­viewed — im­me­di­ate­ly trig­gered the kind of re­lent­less hype that has sur­round­ed ad­u­canum­ab from the first ear­ly signs of its abil­i­ty to have an im­pact on the dis­ease. But there are some im­por­tant caveats that en­thu­si­asts nev­er­the­less need to keep in mind.

First, while amy­loid be­ta de­posits are a well de­fined tar­get, it’s al­so not uni­ver­sal­ly ac­cept­ed as the sole cause of Alzheimer’s. There are some who be­lieve it may not be in­volved at all, point­ing to pa­tients who have the clus­ters in their brains but no symp­toms of de­men­tia. In ad­di­tion, the re­searchers care­ful­ly not­ed that the study with 165 pa­tients was not pow­ered to pro­vide clear ev­i­dence of ef­fi­ca­cy.

The en­thu­si­asm among the ex­ecs and re­searchers in­volved, though, was pal­pa­ble. And Bio­gen, which bad­ly needs to whip up some ex­cite­ment for the com­pa­ny’s pipeline, was not in the least bit re­luc­tant to pump the lat­est da­ta set that has now been pub­lished in Na­ture. Chief Med­ical Of­fi­cer Al San­drock re­marked:

“These ear­ly stud­ies of ad­u­canum­ab show its ef­fec­tive­ness in re­mov­ing amy­loid plaque from the brain as well as its po­ten­tial ef­fect on the slow­ing of cog­ni­tive de­cline in pa­tients suf­fer­ing from Alzheimer’s dis­ease. Pub­li­ca­tion in Na­ture is an achieve­ment we share with the many sci­en­tists and clin­i­cal in­ves­ti­ga­tors who con­duct­ed this re­search as well as the pa­tients who vol­un­teered to par­tic­i­pate in our clin­i­cal tri­al; we are grate­ful to all of them.”

There is, how­ev­er, a long way to go from a suc­cess­ful Phase Ib study to a piv­otal win, which is where Bio­gen is now fo­cused.

Shares of Bio­gen re­mained down slight­ly for the day.

In ad­di­tion to the pos­i­tive signs that the high dose was most ef­fec­tive, there were al­so some con­cerns.

Check­ing on safe­ty, 38% of the pa­tients in the high, 10 mg kg arm of the study dropped out, many cit­ing side ef­fects, com­pared to 25% of the place­bo arm.

Bio­gen’s progress on Alzheimer’s has helped whip up fresh en­thu­si­asm for the amy­loid be­ta hy­poth­e­sis, even spurring Roche to keep one of its failed drugs, gan­tenerum­ab, in the pipeline. Sev­er­al late-stage drugs, in­clud­ing solanezum­ab at Eli Lil­ly, al­so at­tack a-be­ta, look­ing to prove their ef­fec­tive­ness in very ear­ly-stage pa­tients, be­fore the dis­ease caus­es se­ri­ous brain dam­age. New imag­ing tech­niques are al­so bet­ter able to iden­ti­fy pa­tients who are most like­ly to ben­e­fit.

Lil­ly is ex­pect­ed to de­liv­er piv­otal da­ta from its sec­ond Phase III pro­gram lat­er in the year. But it’s al­so been back­ing away from a key end­point on dai­ly func­tion, sig­nal­ing that it ex­pects to be able to prove an im­pact on cog­ni­tion.

Bio­gen, mean­while, is see­ing off CEO George Scan­gos at a time the biotech com­pa­ny has been un­der heavy pres­sure to do some­thing now to prove it has im­por­tant new drugs in the pipeline that can make up for flag­ging sales of its flag­ship drug, Tec­fidera. Still some ways from their piv­otal read out, and un­able so far to come up with the kind of deal that can gen­er­ate some cheers from Wall Street an­a­lysts, Bio­gen will set­tle for trum­pet­ing an­oth­er ear­ly snap­shot for ad­u­canum­ab.

Jef­feries’ Bri­an Abra­hams not­ed:

Much of the clin­i­cal de­tails, sub-analy­ses and pre­clin­i­cal da­ta have all been ex­ten­sive­ly pre­sent­ed, and we didn’t de­tect any­thing ma­te­ri­al­ly new in the ar­ti­cle; it did cite cau­tion in in­ter­pret­ing cog­ni­tive end­points giv­en lack of pow­er­ing for these mea­sures and no im­pu­ta­tion for miss­ing val­ues, and point­ed out the pos­si­bil­i­ty the need for MRIs in pa­tients de­vel­op­ing ARIA could have un­blind­ed the study. Still, the dose-de­pen­dent plaque re­duc­tions and cor­re­la­tion be­tween plaque clear­ance and cog­ni­tive ben­e­fits con­tin­ue to look most com­pelling to us, and we con­tin­ue to see a 45% prob­a­bil­i­ty of ul­ti­mate­ly gen­er­at­ing $2B+ rev­enues to BI­IB by 2025.

 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

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.

Mike Grey. Mirum

In $86M IPO pitch, Mirum spells out plans to turn Shire dis­cards in­to or­phan liv­er drug suc­cess­es

Mike Grey doesn’t have any time to waste. Hav­ing re­gained con­trol of two liv­er dis­ease drugs from Shire and po­si­tioned them for piv­otal stud­ies — five years af­ter first hand­ing them off in a deal to sell Lu­me­na, where he was CEO — Grey is steer­ing Mirum straight in­to an IPO with a $86 mil­lion ask.

Not that Mirum has spent much of its $120 mil­lion Se­ries A cash since launch­ing last No­vem­ber. Ac­cord­ing to the S-1, the Cal­i­forn­ian biotech has burned through $23.3 mil­lion as of March, but ex­pects ex­pens­es to pick up once their clin­i­cal work gath­ers steam.

Evotec CEO Werner Lanthaler, File Photo

Ox­ford, Evotec ramp up LAB10x with AI ex­perts at Sen­syne — fo­cused on biotech spin­outs

Ox­ford is al­ly­ing it­self with Evotec and ar­ti­fi­cial in­tel­li­gence out­fit Sen­syne Health to ramp up some new biotech spin­outs while look­ing to “ac­cel­er­ate da­ta-dri­ven drug dis­cov­ery and de­vel­op­ment.”

The big idea here is that Ox­ford sci­en­tists — some of the best drug hunters in the world — can uti­lize Sen­syne’s AI plat­form for their work, re­ly­ing on the chemists and hands-on de­vel­op­ers at Evotec to push ahead to a crit­i­cal proof of con­cept mo­ment. And they’ll do it through a project leader called LAB10x, which gets £5 mil­lion over the next three years to fund the work.

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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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.

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.