Hit-and-miss PhI­I­Is leave Lund­beck and Ot­su­ka’s lat­est Alzheimer’s pro­gram in a pre­car­i­ous po­si­tion

Sev­en months af­ter Lund­beck and Ot­su­ka re­port­ed a Phase III flop for their Alzheimer’s drug idalopir­dine, the part­ners are back with a hit-and-miss score on eas­ing Alzheimer’s ag­i­ta­tion for their block­buster ther­a­py Rex­ul­ti (br­ex­pipra­zole).

Al­ready ap­proved for schiz­o­phre­nia and ma­jor de­pres­sion, the col­lab­o­ra­tors were look­ing for the first ap­proval of an an­tipsy­chot­ic specif­i­cal­ly for ag­i­ta­tion trig­gered by Alzheimer’s de­men­tia, a con­di­tion that af­flicts mil­lions of peo­ple in the US and mil­lions more world­wide. Ag­i­ta­tion can be ex­pressed in many ways, run­ning from wan­der­ing to ag­gres­sive be­hav­ior.

But in try­ing to gain pos­i­tive da­ta reg­u­la­tors want to see from two big late-stage stud­ies, re­searchers scored a hit on the pri­ma­ry — change from base­line in the Co­hen-Mans­field Ag­i­ta­tion In­ven­to­ry to­tal score — in one study but missed in the sec­ond, though the drug did achieve the key sec­ondary end­point as­sess­ing the sever­i­ty of ag­i­ta­tion.

Is it enough for an ap­proval?

Lund­beck didn’t spell out the da­ta, which makes this too tough to call. Reg­u­la­tors have been sig­nal­ing their flex­i­bil­i­ty when it comes to Alzheimer’s, a field which has seen noth­ing but fail­ure for dis­ease mod­i­fy­ing and symp­to­matic drugs over the last 15 years. So it’s a pos­si­bil­i­ty. With Rex­ul­ti on the mar­ket al­ready, and like­ly in use for de­men­tia-re­lat­ed ag­i­ta­tion, an­a­lysts were ex­pect­ing a clear win. But it may just come down to the safe­ty pro­file.

“Im­por­tant­ly, in the two Rex­ul­ti stud­ies over­all mor­tal­i­ty was low (0.86%) with no deaths con­sid­ered to be treat­ment re­lat­ed,” not­ed Jef­feries’ Pe­ter Welford. He added:

Lund­beck and part­ner Ot­su­ka plan to meet with the FDA to dis­cuss these Rex­ul­ti da­ta. Typ­i­cal­ly we be­lieve reg­u­la­to­ry au­thor­i­ties re­quire two pos­i­tive stud­ies to sup­port ap­proval but we note equiv­o­cal study out­comes are com­mon in CNS in­di­ca­tions. Fur­ther­more, no drugs are US ap­proved specif­i­cal­ly to treat ag­i­ta­tion as­so­ci­at­ed with de­men­tia, de­spite the wide­spread use of an­tipsy­chotics. Rex­ul­ti’s com­par­a­tive­ly clean safe­ty pro­file could po­ten­tial­ly al­lay class-wide con­cerns and nu­mer­ous ini­tia­tives are on­go­ing to de­crease the off-la­bel use of an­tipsy­chotics in nurs­ing homes.

One like­ly line that the com­pa­nies will take fo­cus­es on their take­away that the da­ta var­ied by coun­try. The da­ta sep­a­ra­tion be­tween place­bo and drug was par­tic­u­lar­ly poor in Rus­sia, they said, which could be due to stan­dard of care rather than the qual­i­ty of the med­i­cine. Rus­sia and East­ern Eu­rope have been reg­u­lar­ly held out as prob­lem­at­ic lo­ca­tions for drug tri­als, which makes you won­der why Lund­beck and Ot­su­ka’s team de­cid­ed to in­clude the coun­try in a world­wide tri­al in the first place.

It will be up to the FDA now to sig­nal which way this ap­pli­ca­tion could go.

The two part­ners have been vexed by Alzheimer’s, like most oth­er com­pa­nies in this field. They re­cent­ly wrote off their 5HT6 symp­to­matic drug af­ter it flopped in stud­ies. An­tipsy­chotics, though, are not your run-of-the-mill kind of Alzheimer’s drug, open­ing up some new op­por­tu­ni­ties to fol­low.

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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