Pumped by an ear­ly glimpse of ef­fi­ca­cy, Newron charts a piv­otal course for schiz­o­phre­nia drug

Ravi Anand, Newron

Just a few days af­ter Newron fi­nal­ly land­ed an FDA ap­proval for Parkin­son’s add-on ther­a­py Xada­go af­ter a 4-year reg­u­la­to­ry odyssey, the biotech is back with a snap­shot of ear­ly da­ta on a new treat­ment for schiz­o­phre­nia. And while they were able to pull out pos­i­tive da­ta from the Phase IIa study of eve­namide, in­ves­ti­ga­tors plan to make some sig­nif­i­cant changes to the next study that could pro­vide more plain­ly promis­ing piv­otal re­sults.

Pre­sent­ing at the 16th Con­gress on Schiz­o­phre­nia Re­search in San Diego over the week­end, Newron con­clud­ed that their drug eve­namide did help a large group of pa­tients.

The mean change for the Pos­i­tive and Neg­a­tive Syn­drome Scale (PANSS) score over­all was -5.1 for the add-on com­pared to -3.7 for the place­bo, which Newron Chief Med­ical Of­fi­cer Ravi Anand de­scribed as “very close” to sta­tis­ti­cal sig­nif­i­cance. If you just looked at the pos­i­tive side of the scale, he added, the re­sults were sta­tis­ti­cal­ly sig­nif­i­cant.

It’s im­por­tant to note that Newron and Anand de­cid­ed to amp up the study so they could go be­yond the safe­ty da­ta they were orig­i­nal­ly look­ing for — the drug proved tol­er­a­ble, they say — and start to see how this ther­a­py could work as an add-on.

The com­pa­ny re­cruit­ed 89 schiz­o­phre­nia pa­tients with break­through psy­chot­ic symp­toms while be­ing treat­ed with risperi­done or arip­ipra­zole.

“Com­pound A or B have the same char­ac­ter­is­tics,” says Anand. “All of them work to a cer­tain ex­tent.”

The two main ther­a­pies in use for schiz­o­phre­nia do es­sen­tial­ly the same thing, Anand tells me. And the re­sults are fre­quent­ly the same as well. Pa­tients of­ten re­spond ini­tial­ly and then switch af­ter the re­sponse winds down. And then the re­sponse winds down again.

In a wide range of an­i­mal tests, he adds, eve­namide ap­peared lim­it­ed to sodi­um chan­nel block­age and glu­ta­mate re­lease, which is what they were look­ing for as a sign of its on-tar­get speci­fici­ty, re­duc­ing the odds of an off-tar­get hit.

While the place­bo re­sponse may look large to the unini­ti­at­ed, Anand says it’s ac­tu­al­ly low. That’s be­cause re­searchers specif­i­cal­ly looked for pa­tients with a con­strained lev­el of symp­toms. In the piv­otal tri­als to come, he says, they’ll be look­ing for pa­tients ex­pe­ri­enc­ing el­e­vat­ed symp­toms of schiz­o­phre­nia, where the drug’s im­pact could be more clear­ly dis­tin­guished from a place­bo. And if it works in the next tri­al in­volv­ing about 360 pa­tients, to get start­ed next year, he be­lieves it will be straight­for­ward to repli­cate the re­sults in a sec­ond con­fir­ma­to­ry tri­al re­quired by the FDA.

Anand adds that the drug’s “ef­fect ap­pears much bet­ter in younger pa­tients.” That’s be­cause “old­er pa­tients have lost the ca­pac­i­ty to re­spond. The dis­ease de­stroys cog­ni­tion” and as pa­tients age, there are neu­ro­chem­i­cal changes in the brain due to their long run­ning use of an­ti-psy­chotics that make their re­sponse to an add-on like eve­namide less like­ly.

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.