Aerie shares shoot up as in­ves­ti­ga­tors take an­oth­er big step to an NDA with 2nd pos­i­tive glau­co­ma PhI­II

Aerie Phar­ma­ceu­ti­cals is now two for two on its piv­otal Phase III stud­ies for Ro­cla­tan, a com­bo drug that com­bines the gener­ic la­tanoprost to its in-house drug Rho­pres­sa.

In­ves­ti­ga­tors re­port­ed top line da­ta to­day, say­ing that the com­bo beat each of the two drugs sep­a­rate­ly in low­er­ing in­traoc­u­lar pres­sure, the key gauge for suc­cess here.

Once again, Aerie shares $AERI took off on the news, spik­ing more than 30% Thurs­day as in­vestors pon­dered the im­pli­ca­tions. The biotech promised to file for an ap­proval in the first half of 2018, pro­vid­ed an on­go­ing safe­ty study ends suc­cess­ful­ly.

Aerie CEO Vi­cente Anido

The IOP-low­er­ing ef­fect of Ro­cla­tan, says Aerie, was 1 to 3 mmHg (mil­lime­ters of mer­cury) greater than monother­a­py with ei­ther la­tanoprost or Rho­pres­sa — which is up for an FDA re­view with a Feb­ru­ary 28, 2018 PDU­FA date — in the study. The study eval­u­at­ed pa­tients with max­i­mum base­line in­traoc­u­lar pres­sures rang­ing from above 20 to be­low 36 mmHg. The com­bo al­so re­duced mean di­ur­nal IOPs to 16 mmHg or low­er for 56% of the pa­tients tak­ing it, bet­ter than both of the oth­er drug arms. In the first Phase III the com­bo ex­ceed­ed that of la­tanoprost by an av­er­age across the study du­ra­tion of 1.9 mmHg and ex­ceed­ed Rho­pres­sa by 2.6 mmHg.”

Once again, though, the com­bo was al­so linked with a high rate of eye red­ness among pa­tients, a trait al­so tied to Rho­pres­sa. There was a 10% dis­con­tin­u­a­tion rate in the study, and to be fair to com­pa­ny crit­ics, this drug will get some very close scruti­ny from reg­u­la­tors be­fore it gets to the mar­ket.

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.

“With this pos­i­tive Mer­cury 2 da­ta, we now have two suc­cess­ful piv­otal tri­als for Ro­cla­tan. The topline ef­fi­ca­cy re­sults demon­strat­ed in Mer­cury 2 are con­sis­tent with Mer­cury 1, con­firm­ing the po­ten­tial for Ro­cla­tan to be­come the most ef­fi­ca­cious IOP-low­er­ing ther­a­py to en­ter the mar­ket, if ap­proved. Now that the ef­fi­ca­cy re­sults for both Mer­cury 1 and 2 have proven suc­cess­ful, and if the Mer­cury 1 12-month safe­ty re­sults are al­so suc­cess­ful, we ex­pect to sub­mit our Ro­cla­tan NDA (new drug ap­pli­ca­tion) in the first half of 2018,” said Aerie CEO Vi­cente Anido.

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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Paul Hudson, Getty Images

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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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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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)

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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