Ax­o­vant races to PhI­II de­men­tia study as nelotanserin clears an ear­ly hur­dle, but ques­tions linger

Vivek Ra­maswamy

Ax­o­vant says that its drug nelotanserin has cleared an ear­ly hur­dle in a Phase II study for Lewy body de­men­tia, trig­ger­ing a de­ci­sion to ex­pand the mid-stage tri­al and hit­ting the green light for a Phase III to get start­ed lat­er this year. But in­vestors are like­ly to ques­tion a stum­ble on key sec­ondary goals.

A snap­shot of re­sults for the first tiny group of 11 pa­tients con­clud­ed that they were demon­strat­ing sta­tis­ti­cal­ly sig­nif­i­cant re­spons­es to the drug on the pri­ma­ry end­point. These pa­tients in­clud­ed peo­ple suf­fer­ing from Parkin­son’s as well as Lewy body de­men­tia. They were re­cruit­ed af­ter suf­fer­ing from fre­quent vi­su­al hal­lu­ci­na­tions.

“The pri­ma­ry out­come mea­sures of the study were ex­trapyra­mi­dal signs as as­sessed by Uni­fied Parkin­son’s Dis­ease Rat­ing Scale (UP­DRS) Parts II + III and safe­ty,” the com­pa­ny re­port­ed.

There was a big hitch, though. The tri­al did not hit sec­ondary end­points laid out by in­ves­ti­ga­tors to track the fre­quen­cy of vi­su­al hal­lu­ci­na­tions, in­clud­ing both a stan­dard SAPS as­sess­ment as well as an end­point they cre­at­ed in­ter­nal­ly at Ax­o­vant. And that may have weighed in on in­vestors’ re­sponse to the re­sults, with Ax­o­vant’s stock up on­ly 5% in pre-mar­ket trad­ing.

Ax­o­vant, the first of Vivek Ra­maswamy’s biotechs to get start­ed, plans to hus­tle in­to the ex­pand­ed Phase II for the drug — a 5HT2A an­tag­o­nist — as it wastes no time in get­ting the Phase III up and run­ning. Ra­maswamy has made no se­cret of his plans to ac­cel­er­ate R&D at his com­pa­nies. And Ax­o­vant is the most ad­vanced with a big tri­al un­der­way for Alzheimer’s.

Ax­o­vant al­ready faces an es­tab­lished mar­ket play­er. Aca­dia’s ri­val drug Nu­plazid won FDA ap­proval for Parkin­son’s de­men­tia last spring, but on­ly af­ter reg­u­la­tors ex­pressed their safe­ty con­cerns, not­ing a dis­tinct in­crease in the num­ber of deaths as well as the rate of ad­verse events among the pa­tients tak­ing the drug com­pared to the con­trol arm of the study — even if there was no ob­vi­ous clue what was trig­ger­ing those events.

Ax­o­vant’s hope is that it can come out of clin­i­cal tri­als with a bet­ter suc­cess rate and a clean­er safe­ty pro­file as both com­pa­nies tar­get a broad pa­tient pop­u­la­tion suf­fer­ing from dif­fer­ent forms of de­men­tia.

“I am in­trigued by the ben­e­fits ob­served on the UP­DRS in this study,” stat­ed Dr. James Lev­erenz, Chair, Sci­en­tif­ic Ad­vi­so­ry Coun­cil of the Lewy Body De­men­tia As­so­ci­a­tion and Di­rec­tor of the Cleve­land Lou Ru­vo Cen­ter for Brain Health at the Cleve­land Clin­ic. “If a sin­gle drug could si­mul­ta­ne­ous­ly ad­dress the mo­tor and neu­ropsy­chi­atric symp­toms of Lewy body de­men­tia, which the pub­lished lit­er­a­ture sug­gests may be the case for 5HT2A an­tag­o­nists, it would rep­re­sent a unique and im­por­tant ad­vance for the treat­ment of this con­di­tion. I look for­ward to re­view­ing the full dataset lat­er this year.”

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.

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.

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.