Mer­ck’s $1B cash gam­ble pays off with a sur­pris­ing PhI­II car­dio suc­cess for Bay­er’s heart drug veri­ciguat

More than 3 years af­ter Mer­ck stepped up and paid $1 bil­lion in cold, hard cash to gain the US com­mer­cial rights to Bay­er’s high-risk heart drug veri­ciguat in a broad-rang­ing car­dio al­liance, the part­ners say their Phase III study has come through with promis­ing da­ta and a date with reg­u­la­tors.

We don’t have the da­ta, and won’t un­til they put it out at an up­com­ing sci­en­tif­ic ses­sion, but Mer­ck tout­ed the re­sults, say­ing that their big Phase III VIC­TO­RIA study hit the pri­ma­ry end­point  — with veri­ciguat com­bined with avail­able ther­a­pies re­duc­ing “the risk of the com­pos­ite end­point of heart fail­ure hos­pi­tal­iza­tion or car­dio­vas­cu­lar death in pa­tients with wors­en­ing chron­ic heart fail­ure with re­duced ejec­tion frac­tion (HFrEF) com­pared to place­bo when giv­en in com­bi­na­tion with avail­able heart fail­ure ther­a­pies.”

De­pend­ing on the hard da­ta, and how it breaks out with the com­bi­na­tions used, this drug could pose a threat to No­var­tis’ block­buster drug En­tresto, cur­rent­ly at $1.6 bil­lion while an­a­lysts ex­pect peak sales to hit $4 bil­lion.

The drug is a sol­u­ble guany­late cy­clase (sGC) stim­u­la­tor, which Bay­er and Mer­ck have had high hopes for. Ev­i­dent­ly, so did car­di­ol­o­gists. Cowen’s last analy­sis set po­ten­tial sales at $400 mil­lion in 2024, but that num­ber could go up sig­nif­i­cant­ly now.

Cowen’s Steve Scala not­ed this morn­ing:

Veri­ciguat could be a lu­cra­tive prod­uct for Mer­ck, and one with po­ten­tial­ly un­der-ap­pre­ci­at­ed val­ue. At Cowen’s Ther­a­peu­tics Con­fer­ence in Sep­tem­ber 2019, 80% of spe­cial­ists an­tic­i­pat­ed a pos­i­tive re­sult from VIC­TO­RIA where­as on­ly 51% of in­vestors shared this op­ti­mism.

In­ves­ti­ga­tors re­cruit­ed more than 5,000 pa­tients at more than 600 cen­ters in 42 coun­tries for this study — one of the most ex­pen­sive propo­si­tions in R&D. Mil­lions of peo­ple in the US suf­fer from heart fail­ure with re­duced ejec­tion frac­tion when the fail­ing heart fails to con­tract prop­er­ly to eject blood in­to the sys­tem. Bay­er holds ex-US rights to the drug and al­so stands to earn cash from the $1.1 bil­lion in mile­stones Mer­ck agreed on for their col­lab­o­ra­tion.

Re­mark­ably, the drug was pushed in­to Phase III de­spite fail­ing the mid-stage tri­al — though in­ves­ti­ga­tors flagged a suc­cess at the high dose of 10 mg. In VIC­TO­RIA, re­searchers start­ed pa­tients at 2.5 mg and then titrat­ed up to 5 and then 10 mg.

The suc­cess here marks the lat­est in a se­ries of col­lab­o­ra­tive wins for Mer­ck, which en­joys these oc­ca­sion­al splurges. One of their biggest suc­cess­es has been tied to As­traZeneca’s Lyn­parza, where they’ve been pur­su­ing an ac­tive sched­ule of new tri­als aimed at ex­pand­ing use.

“VIC­TO­RIA is the first large con­tem­po­rary out­comes study to fo­cus ex­clu­sive­ly on a pop­u­la­tion with wors­en­ing chron­ic heart fail­ure who have a high risk for car­dio­vas­cu­lar mor­tal­i­ty and re­peat­ed heart fail­ure hos­pi­tal­iza­tions. We are pleased veri­ciguat met this pri­ma­ry end­point and look for­ward to shar­ing the de­tailed find­ings of the study,” said Mer­ck CMO Roy Baynes in a state­ment.

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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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.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.