ICER goes af­ter No­var­tis for over­pric­ing MS drug; Val­ne­va, Glax­o­SmithK­line breaks vac­cine pact

→ ICER is com­ing out against the price of No­var­tis’ Mayzent (sipon­i­mod), ar­gu­ing the mul­ti­ple scle­ro­sis treat­ment has a low long-term val­ue for mon­ey. While the price watch­dog ac­knowl­edges there’s some ev­i­dence that the drug — a block­buster-to-be priced at $88,000 a year — can im­prove out­comes for pa­tients with ac­tive sec­ondary pro­gres­sive MS, it’s dis­sat­is­fied with its lack of ben­e­fit for the whole pop­u­la­tion test­ed in a Phase III (No­tably, the FDA ap­proval cov­ers a slight­ly dif­fer­ent group, name­ly re­lapsed/re­frac­to­ry MS). Their ad­vice to pay­ers: “Giv­en its sim­i­lar­i­ties to fin­golimod, sipon­i­mod should be con­sid­ered amongst a group of high­ly ef­fec­tive dis­ease mod­i­fy­ing ther­a­pies (DMTs) for re­laps­ing forms of MS, in­clud­ing fin­golimod, alem­tuzum­ab, na­tal­izum­ab, and ocre­lizum­ab.”

Ar­row­head Phar­ma­ceu­ti­cals $AR­WR has snagged an or­phan drug des­ig­na­tion for ARO-APOC3, which tack­les a rare lipid dis­or­der known as fa­mil­ial chy­lomi­crone­mia syn­drome. Uti­liz­ing the RNAi ap­proach, Ar­row­head wants to si­lence the gene re­spon­si­ble for apolipopro­tein C-III (apoC-III). A Phase I study be­gan in March.

→ French vac­cine de­vel­op­er Val­ne­va has closed out a part­ner­ship with Glax­o­SmithK­line, a lega­cy deal in­her­it­ed from In­ter­cell and No­var­tis, re­spec­tive­ly. The ter­mi­na­tion trig­gered a €9 mil­lion pay­ment from Val­ne­va to GSK, with an­oth­er €7 mil­lion due for any mar­ket­ing ap­provals of its Ly­me vac­cine.

Paul Hudson, Getty Images

UP­DAT­ED: 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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De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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De­sert­ed by Astel­las and Mer­ck, lit­tle Cor­re­vio still can't win over FDA pan­el con­cerned with its AFib drug's safe­ty

When the FDA spurned Astellas’ pitch for atrial fibrillation drug vernakalant in 2008, regulators made it abundantly clear that it wasn’t the efficacy they had a problem with — two Phase III trials had shown the drug successfully restored 52% of patients’ heartbeat from irregular to normal — but the cardio safety issues for a drug that was to compete with well established, low-risk options. One licensing deal, one clinical hold and several studies later, the chances of approval aren’t looking any better.

New trade deal knocks out long-sought patent pro­tec­tions for drug­mak­ers

House Democrats negotiating with the Trump Administration on a new North American trade deal settled early on four issues: enforcement, labor and environmental standards and drug pricing.

On drug pricing, Politico reports, Trump crumbled within weeks of heightened negotiations.

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With loom­ing su­per­bug cri­sis, Iterum miss­es in a key an­tibi­ot­ic tri­al — and its shares tum­ble

A raft of antibiotic makers have crashed and burned recently despite getting their drugs across the finish line. Iterum Therapeutics hopes that its lead drug sulopenem, which has an outpatient focus in addition to hospitals and a plan to target areas with the highest levels of drug-resistant infections, will avert some of those reimbursement challenges. However, the company has now stumbled in a late-stage study, diminishing its shot at FDA approval it first requires.

UP­DAT­ED: Lit­tle Leo Phar­ma en­ters the prize-fight ring with pos­i­tive PhI­II atopic der­mati­tis da­ta. Now they just have to beat Dupix­ent

A day after new Sanofi CEO Paul Hudson staked his reputation and the future of the pharma giant on making Dupixent a megasuccess story, little Leo Pharma is throwing down the gauntlet on atopic dermatitis.

Three years ago Leo paid AstraZeneca $115 million to buy up rights to use tralokinumab against atopic dermatitis — with $1 billion more on the table in milestones — the Danish company says their drug has swept up positive results for all primary and secondary endpoints in three Phase III trials. Now they plan to start the final push for regulatory approvals so they can challenge the heavyweight champions in this slugfest.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Otello Stampacchia. Omega Funds

Af­ter sev­er­al high pro­file start­up launch­es, om­niv­o­rous Omega Funds clos­es $438M fund to pur­sue more deals

Omega Funds likes to work backwards. It invests with the end game — denoted by the Greek letter in its name — in mind, and it keeps tabs on the number of marketed medical products that culminate from its ventures: 37 in 16 years.

So when founder and managing director Otello Stampacchia declares it’s the most exciting time to be investing in life sciences in a generation, it’s perhaps only natural that Omega has closed its largest fund to date. With $438 million in total commitments for Fund VI, the firm will continue injecting capital into a broad swath of companies across the US and Europe.

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