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: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepeneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film Fantastic Voyage, the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his own account, along with some seed cash from friends and family.

Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.