Dicer­na scores broad, 'rest of liv­er' deal with No­vo Nordisk, bag­ging $225M in cash to hit some 30 tar­gets with RNAi plat­form

Turns out Dicer­na wasn’t done with deals yet af­ter lock­ing in $200 mil­lion up­front from Roche for a he­pati­tis B cock­tail two weeks ago.

Doug Fam­brough

No­vo Nordisk has signed on as the lat­est part­ner to its GalXC RNAi plat­form, hand­ing over $175 mil­lion in cash to claim any and all tar­gets of in­ter­est in liv­er-re­lat­ed car­dio-meta­bol­ic dis­eases that are not al­ready re­served in pre­vi­ous pacts. The Dan­ish drug­mak­er — which has sig­naled its in­ter­est to ex­pand con­sid­er­ably be­yond its core di­a­betes fran­chise in­to ar­eas like NASH — is al­so pur­chas­ing $50 mil­lion worth of Dicer­na’s eq­ui­ty at a 25% pre­mi­um of $21.93 per share. More re­search pay­ments and mile­stones ex­tend­ing to the bil­lions are on the line.

Dicer­na CEO Doug Fam­brough de­scribes the deal as a “cap­stone” for its part­ner­ing ef­forts in the liv­er space and a fur­ther sign that the biotech has en­tered a more ma­ture phase of part­ner­ing with in­creased scope and val­ue.

In a call with an­a­lysts and in­vestors fol­low­ing the an­nounce­ment, he adopt­ed a re­al es­tate anal­o­gy:

If you think of the liv­er as an is­land, there are in­di­vid­ual prop­er­ties on the is­land that we have part­nered — com­ple­ment with Alex­ion, a cou­ple of par­tic­u­lar tar­gets in NASH with BI, et cetera. The col­lab­o­ra­tion with No­vo has as its purview the rest of the land on the is­land that is not part­nered in any of the four ex­ist­ing col­lab­o­ra­tions and we will not be sell­ing any ad­di­tion­al re­al es­tates, so to speak, that No­vo could choose to de­vel­op. This al­lows new in­sights that come from hu­man ge­net­ics or frankly any source to in­spire No­vo to in­clude a tar­get in the col­lab­o­ra­tion.

Jim Weiss­man

Dicer­na is tasked with dis­cov­ery and pre­clin­i­cal can­di­date se­lec­tion on a num­ber of liv­er cell tar­gets for dis­or­ders span­ning chron­ic liv­er dis­ease, NASH, type 2 di­a­betes, obe­si­ty, and rare dis­eases. No­vo Nordisk has com­mit­ted to $25 mil­lion per year dur­ing the first three years. While the duo hasn’t dis­closed how many years they ex­pect the col­lab­o­ra­tion to run, the plan is to ex­plore around 30 through­out the pe­ri­od.

But Dicer­na’s am­bi­tions here go be­yond start­ing pro­grams for big­ger com­pa­nies to take over. It has ne­go­ti­at­ed an op­tion to opt in­to two drugs for more preva­lent ail­ments af­ter view­ing clin­i­cal da­ta gen­er­at­ed by No­vo — al­low­ing their clin­i­cal team to buy in­to suc­cess­es with­out bear­ing the cost, Fam­brough high­light­ed. Un­der the deal, it can al­so ini­ti­ate the de­vel­op­ment of two or­phan drugs that the big­ger part­ner can opt in to.

The “re­al­ly broad” col­lab­o­ra­tion is de­signed to fo­cus less on in­di­vid­ual genes than the po­ten­tial of dif­fer­ent com­bi­na­tion ap­proach­es in a num­ber of liv­er dis­eases, COO Jim Weiss­man said.

Bob Brown

In­ter­nal­ly, Dicer­na has been ap­ply­ing its plat­form rou­tine­ly to ex­am­ine a list of genes as­so­ci­at­ed with dif­fer­ent car­diometa­bol­ic dis­eases, ac­cord­ing to CSO Bob Brown.

“We just rou­tine­ly knock them out and then use the GalXC mol­e­cules we iden­ti­fied there to in­ter­ro­gate the gene func­tion in the rel­e­vant dis­ease mod­els that we run rou­tine­ly in house,” he said on the call. “There’s no di­rect align­ment of lists yet, but we’ve in­ter­ro­gat­ed ap­prox­i­mate­ly 40 genes this way in dif­fer­ent mod­els of car­diometa­bol­ic dis­ease.”

No­vo has yet to iden­ti­fy the genes that they would like to start with, but Fam­brough not­ed that the tar­gets they have ex­pressed in­ter­est in are still “very much avail­able.”

Adding to pre­vi­ous deals with Boehringer In­gel­heim, Alex­ion, Eli Lil­ly and Roche, the in­flux of cap­i­tal from No­vo should keep Dicer­na ful­ly fund­ed for at least a year af­ter the en­vi­sioned com­mer­cial launch of their lead pro­gram in pri­ma­ry hy­per­ox­aluria, the man­age­ment said.

So­cial im­age: AP Im­ages

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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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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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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