GSK aban­dons Ion­is' FDA-ready in­ot­ersen as new CEO sweeps out rare dis­ease ef­forts

Just three months af­ter Io­n­is spelled out pos­i­tive Phase III ef­fi­ca­cy da­ta on its top prospect in­ot­ersen, Glax­o­SmithK­line is sweep­ing their al­liance on the drug out the back door, along with rights to an­oth­er treat­ment they have rights to.

Em­ma Walm­s­ley, GSK CEO

It’s a stun­ning 11th hour re­ver­sal for Io­n­is $IONS, which says it now plans to push for an FDA ap­proval of in­ot­ersen on its own this fall as it thinks through whether it will com­mer­cial­ize or co-com­mer­cial­ize in the US on its own.

Ac­cord­ing to Io­n­is, GSK ex­ecs de­cid­ed to aban­don in­ot­ersen, de­signed to treat rare cas­es of TTR amy­loi­do­sis (AT­TR), with an ini­tial fo­cus on polyneu­ropa­thy due to hered­i­tary TTR amy­loi­do­sis (hAT­TR-PN). That drug is a close­ly-watched ri­val to Al­ny­lam’s lead drug patisir­an. Al­so out: IO­N­IS-FB-LRx, now at the thresh­old of Phase II for dry age-re­lat­ed mac­u­lar de­gen­er­a­tion, as GSK turns its back on rare dis­eases as part of its top-to-bot­tom R&D re­vamp un­der new CEO Em­ma Walm­s­ley.

“Our goals for in­ot­ersen are to max­i­mize its com­mer­cial suc­cess and op­ti­mize our com­mer­cial par­tic­i­pa­tion. To achieve these goals, we are ac­tive­ly con­sid­er­ing form­ing a com­mer­cial sub­sidiary to com­mer­cial­ize or co-com­mer­cial­ize in­ot­ersen in North Amer­i­ca, as well as oth­er op­tions. Our re­cent ex­pe­ri­ence build­ing a com­mer­cial sub­sidiary has pre­pared us for this op­por­tu­ni­ty. We have sub­stan­tial in­ter­est from po­ten­tial part­ners and are in dis­cus­sions with sev­er­al par­ties. We be­lieve that, to­geth­er with the right com­mer­cial part­ner, we can max­i­mize the com­mer­cial suc­cess of the drug world­wide,” said Sarah Boyce, chief busi­ness of­fi­cer of Io­n­is Phar­ma­ceu­ti­cals.

Sarah Boyce, Io­n­is CBO

Fil­ing for ap­proval and get­ting it are two dif­fer­ent things, though. Io­n­is’ late-stage ef­fort has al­so been flagged for se­ri­ous safe­ty con­cerns which could al­so ham­per its quest for an OK, as well as pos­si­bly sign­ing up a new com­mer­cial­iza­tion part­ner. Paul Mat­teis at Leerink swift­ly not­ed:

Nor­mal­ly in such a sit­u­a­tion like this in­vestors might wor­ry that there’s some not-yet seen-el­e­ment of the full da­ta that may be prob­lem­at­ic or dis­con­cert­ing. To this point — giv­en the well doc­u­ment­ed safe­ty is­sues as­so­ci­at­ed with In­ot­ersen (i.e., throm­bo­cy­tope­nia), GSK’s de­ci­sion may in­crease in­vestor scruti­ny on TTR­rx’s safe­ty pro­file as it is de­pict­ed in ad­di­tion­al full da­ta pre­sen­ta­tions this Fall (Amer­i­can Neu­ro­log­i­cal As­so­ci­a­tion, Oct. 15-17, oth­ers). Our mod­el cur­rent­ly as­sumes a 75%/25% AL­NY (MP) ver­sus IONS split, and while we are en­cour­aged that IONS’s plans for a YE17 MAA fil­ing are still on track, we are a bit con­cerned that GSK’s de­ci­sion may al­so put IONS be­hind the curve with re­spect to launch pre­pared­ness.

Stan­ley Crooke, Io­n­is CEO

Two oth­er al­liances will con­tin­ue. GSK is cur­rent­ly in Phase II with IO­N­IS-HB­VRx and IO­N­IS-HBV-LRx.

CEO Stan­ley Crooke not­ed that Io­n­is is now “ac­cel­er­at­ing the ex­pan­sion of our TTR pro­gram for pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis and the de­vel­op­ment of our LI­CA fol­low-on drug. Our ex­pe­ri­ence in the com­plet­ed Phase 3 NEU­RO-TTR study pro­vides im­por­tant in­for­ma­tion to aid in de­sign of a study in pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis. We have al­ready iden­ti­fied a more po­tent and con­ve­nient LI­CA fol­low-on and we ex­pect de­vel­op­ment of the LI­CA drug to al­so pro­ceed rapid­ly.”

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a plan to near­ly dou­ble its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.