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.

 

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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