Ovid's snap­shot of broad ef­fi­ca­cy da­ta for their PhII An­gel­man syn­drome tri­al can't stop a rout -- shares crushed

Ovid Ther­a­peu­tics $OVID says it picked up a snap­shot of broad, pos­i­tive ef­fi­ca­cy in its Phase II study of a new drug for rare cas­es of An­gel­man syn­drome and will now turn to the FDA to see about map­ping out a reg­is­tra­tional path­way with the FDA for a piv­otal test. But the biotech has to clear some hur­dles ahead if it ex­pects to con­vince an­a­lysts of the drug’s po­ten­tial. And its share price was crushed Mon­day morn­ing as skep­tics pounced.

Je­re­my Levin, Ovid

The drug is OV101, which passed the pri­ma­ry goal on safe­ty and tol­er­a­bil­i­ty and al­so scored what Ovid CEO Je­re­my Levin called the first clear­ly promis­ing da­ta record­ed in a Phase II study for An­gel­man syn­drome.

“This is the first time that any­thing has shown it has an im­pact,” Levin told me ear­ly Mon­day. “This is ter­rif­ic.”

The mar­ket, though, didn’t take the same view, with Ovid shares crash­ing af­ter the re­sults were met with some ini­tial skep­ti­cism about some fail­ures al­so re­port­ed by Ovid. The stock was down 30% in pre-mar­ket trad­ing.

The key ef­fi­ca­cy end­point — in Levin’s view — was based on the physi­cian-rat­ed clin­i­cal glob­al im­pres­sions of im­prove­ment (CGI-I), which clin­i­cians use to track a va­ri­ety of symp­toms for a dis­ease that can leave pa­tients un­able to com­mu­ni­cate or crip­pled with anx­i­ety. On that col­lec­tive end­point, Ovid came away with a high­ly sta­tis­ti­cal­ly sig­nif­i­cant p val­ue of 0.0006 for the once-dai­ly drug arm — which is ex­act­ly what they want­ed to see.

The twice-dai­ly drug arm, though, did not achieve sta­tis­ti­cal sig­nif­i­cance, falling well short of the mark. But Levin says that was ex­pect­ed af­ter pre­clin­i­cal stud­ies demon­strat­ed that too much drug — de­signed to clar­i­fy sig­nal from noise — would fail to rein in symp­toms. On a pooled ba­sis, he says the da­ta are still pos­i­tive, with a p val­ue of 0.02.

That po­si­tion at­tract­ed plen­ty of hos­tile re­ac­tion on Twit­ter this morn­ing, though, as some ques­tioned whether Ovid had spelled out a like­ly fail­ure for the high­er dose. Al­so, CGI was list­ed as the 16th of 17 sec­ondary end­points on clin­i­cal­tri­als.gov, rais­ing fur­ther sus­pi­cions that the da­ta were more neg­a­tive than the com­pa­ny as­sert­ed.

Ovid ex­ecs say that the drug was al­so scored on “a pre­spec­i­fied sub­set of scales across the do­mains of be­hav­ior, sleep and gait,” but failed to demon­strate sta­tis­ti­cal sig­nif­i­cance. Levin not­ed those scores re­lied on care­givers’ as­sess­ments, which aren’t al­ways the best way of gath­er­ing da­ta in a study.

The pa­tients will now be fol­lowed on an on­go­ing ba­sis.

The drug is an ex­trasy­nap­tic GA­BAA re­cep­tor ag­o­nist. There are no ap­proved ther­a­py for this con­di­tion.

 

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

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Lynn Baxter, Viiv Healthcare's head of North America

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