Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is cus­tom, an EMA pan­el on Fri­day is­sued its week­ly rec­om­men­da­tions on mar­ket­ing ap­pli­ca­tions sub­mit­ted by drug de­vel­op­ers. This week, the agency backed the use of five new ther­a­pies — in­clud­ing two No­var­tis drugs — but is­sued no neg­a­tive re­views.

No­var­tis’ S1P drug for re­laps­ing forms of mul­ti­ple scle­ro­sis (MS) drug, Mayzent (known chem­i­cal­ly as sipon­i­mod), which was ap­proved by the FDA in March — has been giv­en the nod by the EMA. The Swiss drug­mak­er al­ready sells its oth­er MS drug, Gilenya, in both re­gions.

The com­pa­ny has al­so se­cured a pos­i­tive EMA rec­om­men­da­tion for Is­tur­isa — known chem­i­cal­ly as os­ilo­dro­stat — for use in Cush­ing’s syn­drome, a dis­ease char­ac­ter­ized by the ex­ag­ger­at­ed pro­duc­tion of the hor­mone cor­ti­sol. Ear­li­er this year, No­var­tis palmed off three en­docrine drugs, in­clud­ing os­ilo­dro­stat, to Italy’s Recor­dati for $390 mil­lion up­front. It al­so stands to make more in mile­stone pay­ments tied to os­ilo­dro­stat.

Fel­low Swiss drug gi­ant Roche al­so scored the EMA en­dorse­ment for its lat­est an­ti­body-drug con­ju­gate (ADC), Po­livy. The drug, known chem­i­cal­ly as po­latuzum­ab ve­dotin, has al­ready won US ap­proval in pa­tients with dif­fuse large B-cell lym­phoma as part of a reg­i­men that in­cludes the chemother­a­py ben­damus­tine and a ver­sion of its ag­ing block­buster can­cer drug, Rit­ux­an. The com­bi­na­tion is rem­i­nis­cent of Roche’s first cleared ADC reg­i­men, Kad­cy­la, which in­cludes its best­selling can­cer drug Her­ceptin and a chemother­a­py agent.

Jazz Phar­ma­ceu­ti­cals, which scored US ap­proval for its wake-pro­mot­ing drug sol­ri­amfe­tol, chris­tened Sunosi, in March — now has the EMA’s bless­ing. Like Xyrem — the block­buster nar­colep­sy drug that ac­counts for the bulk of Jazz’s sales — the dual-act­ing dopamine and nor­ep­i­neph­rine re­up­take in­hibitor is de­signed to treat ex­ces­sive day­time sleepi­ness for pa­tients with nar­colep­sy.

Rigel’s Tavlesse, known chem­i­cal­ly as fos­ta­ma­tinib, was ap­proved last year by the FDA as a sec­ond-line ther­a­py for throm­bo­cy­tope­nia in adult pa­tients with chron­ic im­mune throm­bo­cy­tope­nia. The drug, which is de­signed to block spleen ty­ro­sine ki­nase (SYK), and re­duces an­ti­body-me­di­at­ed de­struc­tion of platelets, now has the EMA’s back­ing.

The EMA al­so backed the ap­proval of two gener­ic med­i­cines and rec­om­mend­ed ex­pand­ing the la­bels of Roche’s Kad­cy­la and Cel­gene’s Revlim­id. The Eu­ro­pean Com­mis­sion typ­i­cal­ly is­sues its de­ci­sions about two months fol­low­ing its com­mit­tee’s rec­om­men­da­tions.

The use of two ap­proved drugs was al­so re­strict­ed by the agency’s hu­man med­i­cines com­mit­tee (CHMP).

The com­mit­tee en­dorsed the de­ci­sion of the EMA’s safe­ty pan­el, con­clud­ing that the use of Pfiz­er’s block­buster JAK in­hibitor Xel­janz could in­crease the risk of blood clots in the lungs and in deep veins in pa­tients who are al­ready at high risk. Af­ter re­view­ing all the da­ta, in­clud­ing the on­go­ing study A3921133 in pa­tients with rheuma­toid arthri­tis, the agency said it was cau­tion­ing the use of the drug in all pa­tients at high risk of blood clots.

In Feb­ru­ary, Pfiz­er oust­ed the 10 mg dose from the A3921133 study, switch­ing pa­tients to the small­er 5 mg dose af­ter doc­u­ment­ing “a sta­tis­ti­cal­ly and clin­i­cal­ly im­por­tant dif­fer­ence in the oc­cur­rence of pul­monary em­bolism” as well as an im­bal­ance in mor­tal­i­ty rate.

The EMA pan­el al­so banned the use of the 10 mg twice dai­ly in pa­tients with ul­cer­a­tive col­i­tis who are at high risk of blood clots, un­less there is no suit­able al­ter­na­tive treat­ment. Fur­ther, the EMA is rec­om­mend­ing that, due to an in­creased risk of in­fec­tions, pa­tients old­er than 65 years of age should be treat­ed with Xel­janz on­ly when there is no al­ter­na­tive treat­ment, it said. These rec­om­men­da­tions fol­low a lim­it im­posed by the EMA safe­ty com­mit­tee in May.

Mean­while, Sanofi’s re­laps­ing-re­mit­ting mul­ti­ple scle­ro­sis drug Lem­tra­da was al­so re­strict­ed in a rec­om­men­da­tion by the CHMP. Re­ports of im­mune-me­di­at­ed con­di­tions and prob­lems with the heart and blood ves­sels with the med­i­cine, in­clud­ing fa­tal cas­es, trig­gered a re­view in April by the agency’s safe­ty pan­el and a rec­om­men­da­tion for lim­it­ed use.

Paul Hudson, Getty Images

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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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 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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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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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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