The FDA's in­sid­er view on Aerie’s glau­co­ma drug: Yes on ef­fi­ca­cy, but…

Aerie CEO Vi­cente Anido

The FDA had some en­cour­ag­ing words for Aerie’s new glau­co­ma drug Rho­pres­sa, but there’s a sig­nif­i­cant caveat that might come back to haunt the biotech as it hunts an ap­proval.

In the in­ter­nal re­view of­fered up by the agency two days ahead of the mis­sion-crit­i­cal pan­el dis­cus­sion and vote, FDA in­sid­ers give the da­ta a thumbs up on ef­fi­ca­cy, but note that the com­para­tor ther­a­py was bet­ter than the drug in the more se­vere­ly af­flict­ed pa­tients in­clud­ed in their Phase III stud­ies.

Look­ing over the da­ta from three Phase III stud­ies, the FDA re­view­ers stat­ed:

(W)e con­cur with the Ap­pli­cant’s over­all ef­fi­ca­cy con­clu­sion that ne­tar­sudil 0.02% once-dai­ly (QD) (al­so re­ferred to as ne­tar­sudil QD) is ef­fi­ca­cious for the re­duc­tion of el­e­vat­ed in­traoc­u­lar pres­sure (IOP). How­ev­er, ne­tar­sudil QD is less ef­fi­ca­cious com­pared to the com­para­tor, tim­o­lol oph­thalmic so­lu­tion 0.5% twice dai­ly (BID), for sub­jects with high­er max­i­mum base­line IOP (≥ 25 mmHg).

In­vestors didn’t seem to mind the is­sue, at least at first glance. Aerie’s stock shot up 14% as the re­view made the rounds.

The FDA’s note on ef­fi­ca­cy im­me­di­ate­ly drew the at­ten­tion of Mizuho’s Difei Yang. But Yang says the sug­gest­ed la­bel doesn’t re­strict an OK to mild to mod­er­ate pa­tients, paving the way to wide­spread use. Yang notes:

We view FDA’s mod­i­fi­ca­tions to Aerie’s pro­posed lan­guage to be very mi­nor. Ad­di­tion­al­ly, we are en­cour­aged by the lan­guage used in the in­di­ca­tion and us­age sec­tion where there is no men­tion of Rho­pres­sa be­ing used for mild to mod­er­ate glau­co­ma pa­tients on­ly. The cur­rent pro­posed la­bel reads as fol­lows, “RHO­PRES­SA (ne­tar­sudil oph­thalmic so­lu­tion) 0.02% is a Rho ki­nase in­hibitor in­di­cat­ed for the re­duc­tion of el­e­vat­ed in­traoc­u­lar pres­sure in pa­tients with open-an­gle glau­co­ma or oc­u­lar hy­per­ten­sion.” We be­lieve this to be a best case sce­nario for Rho­pres­sa.

Aerie, though, still has to clear a bar on the man­u­fac­tur­ing side. Its drug is man­u­fac­tured at the Bausch + Lomb man­u­fac­tur­ing fa­cil­i­ty in Tam­pa, which re­cent­ly failed to mea­sure up to fed­er­al stan­dards for the sec­ond time. The Valeant com­pa­ny, though, fol­lowed up with a vow that it will soon have its house in or­der, en­cour­ag­ing in­vestors as short sell­ers hov­ered about in hopes of a set­back.

A bull­ish Aerie CEO Vi­cente Anido has al­ready tout­ed Rho­pres­sa as a like­ly block­buster able to earn more than $1 bil­lion a year, even though the biotech had to get the FDA’s per­mis­sion to change the end­point for its sec­ond late-stage study of Rho­pres­sa in or­der to avoid back-to-back fail­ures.

Aerie is al­so two for two on its piv­otal Phase III stud­ies for Ro­cla­tan, a com­bo drug that com­bines the gener­ic la­tanoprost to Rho­pres­sa.

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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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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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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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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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.

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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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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