De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When Re­genxBio dis­closed that the FDA had placed a par­tial clin­i­cal hold on one of its lead gene ther­a­pies, ex­ecs out­lined sev­er­al cus­tom­ary next steps: con­tin­u­ing as­sess­ment and mon­i­tor­ing, de­lay­ing a re­lat­ed IND fil­ing, and work­ing with the FDA to ad­dress the mat­ter.

As it turned out, they were plan­ning some­thing much less mun­dane. Two days af­ter an­nounc­ing the hold in its Q3 up­date, Re­genxBio filed a law­suit seek­ing to set it aside, the FDA Law Blog not­ed.

The law­suit shed light on the in­ter­ac­tions be­tween the biotech and reg­u­la­tors, re­veal­ing that there was ac­tu­al­ly a full clin­i­cal hold on the di­a­bet­ic retinopa­thy tri­al in ad­di­tion to the par­tial hold on wet age-re­lat­ed mac­u­lar de­gen­er­a­tion and Re­genxBio with­drew the IND.

Ac­cord­ing to the com­plaint, the com­pa­ny had dis­cussed re­sults from its Phase I/IIa tri­al with the FDA and was on track to be­gin the next phase be­fore the end of 2019.

But on Oc­to­ber 18, 2019, with­out no­tice or ex­pla­na­tion, FDA placed RGX-314 on a clin­i­cal hold, ef­fec­tive­ly halt­ing RE­GENXBIO’s de­vel­op­ment of this po­ten­tial­ly life-al­ter­ing treat­ment for reti­nal dis­eases that are lead­ing caus­es of adult blind­ness. Since is­su­ing the clin­i­cal hold or­der, FDA has re­buffed RE­GENXBIO’s re­peat­ed at­tempts to ob­tain an ex­pla­na­tion of the ba­sis for the clin­i­cal hold.

Fail­ing to pro­vide ad­vance warn­ing or ex­pla­na­tion for the hold, Re­genxBio claims, is in vi­o­la­tion of the FDA’s own reg­u­la­tions. And that led to an “ar­bi­trary and capri­cious” fi­nal de­ci­sion, they wrote.

But that’s not it.

Since they didn’t get a chance to re­view or re­but the rea­sons for the hold, which harmed their rep­u­ta­tion and prop­er­ty in­ter­est in RGX-314, Re­genxBio be­lieves the FDA vi­o­lat­ed the Fifth Amend­ment’s Due Process Clause. Fur­ther­more, they charged a par­tic­u­lar sec­tion of the Food, Drug, and Cos­met­ic Act rep­re­sent­ed an un­con­sti­tu­tion­al vest­ing of leg­isla­tive pow­er in the Sec­re­tary of Health and Hu­man Ser­vices.

Along with the agency, the fed­er­al gov­ern­ment, HHS Sec­re­tary Alex Azar, act­ing FDA com­mis­sion­er Brett Giroir (who has tak­en over from Ned Sharp­less while Stephen Hahn goes through the con­fir­ma­tion process), and FDA reg­u­la­to­ry project man­ag­er Ed­ward Thomp­son were al­so named as de­fen­dants. Thomp­son al­leged­ly first no­ti­fied Re­genxBio of the holds cit­ing “is­sues as­so­ci­at­ed with [RGX-314’s] de­liv­ery sys­tems.”

Fol­low­ing mul­ti­ple ex­changes, the FDA ap­par­ent­ly told Re­genxBio that it would pro­vide a writ­ten ex­pla­na­tion of the ba­sis for the hold by this Fri­day, No­vem­ber 15.

Why risk the ire of reg­u­la­tors when an up­date is due so soon? As a pro­ce­dur­al mat­ter to pre­serve their rights, the com­pa­ny said.

“This ac­tion was tak­en on the rec­om­men­da­tion from coun­sel as we con­tin­ue to work with the FDA to ad­dress this mat­ter, and we hope this step will help en­sure the FDA will pro­vide Re­genxBio with their spe­cif­ic con­cerns about the un­spec­i­fied de­vice,” it wrote in an email to End­points News.

At­tor­ney Deb­o­rah Livor­nese of­fered this take on the FDA Law Blog:

While it seems un­like­ly that the mer­its of the clin­i­cal hold will be re­solved through the ju­di­cia­ry process in a help­ful time­frame, the com­plaint has like­ly in­creased the chances that FDA will re­spond with a thor­ough ex­pla­na­tion of its rea­son for the hold when it does pro­vide the writ­ten ba­sis.

The biotech added that the plan is still to start the wet AMD tri­al and file an IND for di­a­bet­ic retinopa­thy in Q2 2020, “as we be­lieve that there are read­i­ly avail­able and suit­able al­ter­na­tives for all of the de­vices used in our stud­ies.”

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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