Is sec­ond time the charm for ozan­i­mod? Cel­gene starts to find out with EMA fil­ing

Cel­gene has of­fi­cial­ly em­barked on ozan­i­mod’s come­back reg­u­la­to­ry jour­ney, sub­mit­ting an ap­pli­ca­tion to the EMA days be­fore it plans to knock on the FDA’s door — just in time for the dead­line it has set for it­self.

Jay Back­strom

Say­ing Cel­gene — and Bris­tol-My­ers, which is set to ac­quire the biotech as long as the grow­ing in­vestor op­po­si­tion doesn’t de­rail the deal — has a lot rid­ing on the de­ci­sion would be an un­der­state­ment. Fol­low­ing an em­bar­rass­ing refuse-to-file episode with the FDA last Feb­ru­ary, pos­i­tive progress here is cru­cial not just to ma­te­ri­al­ize ozan­i­mod’s block­buster promis­es as a cash cow as Revlim­id ap­proach­es a patent cliff, but to prove Cel­gene’s team still pos­sess­es the de­vel­op­ment shrewd­ness that marked the com­pa­ny for years.

For the Eu­ro­pean ap­pli­ca­tion, Cel­gene has pro­vid­ed da­ta from the SUN­BEAM and RA­DI­ANCE Part B Phase III, which of­fered en­cour­ag­ing com­par­isons with Avonex in treat­ing pa­tients with re­laps­ing mul­ti­ple scle­ro­sis.

Back when the tri­al read out in 2017, up­beat an­a­lysts al­so ran their own cross-tri­al com­par­isons with No­var­tis’ Gilenya and con­clud­ed that Cel­gene might leap out on­to the mar­ket with a sim­i­lar ef­fi­ca­cy pro­file but bet­ter safe­ty fea­tures.

One of them, Bri­an Sko­r­ney of Baird, re­mains op­ti­mistic — if slight­ly more cau­tious. From a re­cent note:

Man­age­ment cit­ed that they ex­pect to re­sub­mit the NDA for ozan­i­mod in March, file for ap­proval of liso-cel in the sec­ond half of this year, and se­cure ap­proval of bb2121 by EOY 2020. We con­tin­ue to be­lieve that the even­tu­al ap­proval of these three as­sets with­in the giv­en time­lines is more like­ly than not. Based on the avail­able clin­i­cal in­for­ma­tion and the an­tic­i­pat­ed time­lines of the on­go­ing clin­i­cal tri­al pro­grams, our es­ti­mates for the like­li­hood of suc­cess of each as­set are ~75% for ozan­i­mod, ~90% for liso-cel, and ~85% for bb2121.

Fresh from a ma­jor late-stage im­plo­sion sur­round­ing mon­gersen, Cel­gene shook in­vestors when it dis­closed the RTF more than a year ago. The FDA, it said back then, had de­ter­mined “that the non­clin­i­cal and clin­i­cal phar­ma­col­o­gy sec­tions in the NDA were in­suf­fi­cient to per­mit a com­plete re­view.”

Then the com­pa­ny man­aged to at­tract side eyes again when a top ex­ec pinned the blame for the fi­as­co on Re­cep­tos, the orig­i­nal de­vel­op­er of the drug — which was bought out by Cel­gene 2.5 years be­fore the fil­ing, its ex-CEO point­ed out in re­sponse. In its glo­ry days, Cel­gene has said ozan­i­mod is worth $4 bil­lion to $6 bil­lion a year in peak sales, but that’s be­com­ing an in­creas­ing­ly tough pitch with an­a­lysts, es­pe­cial­ly with new safe­ty tri­als now un­der way rais­ing ques­tions about po­ten­tial drug-drug in­ter­ac­tions.

Cel­gene, mean­while, still “be­lieves ozan­i­mod could be a best-in-class, oral op­tion for use ear­ly in the treat­ment of re­laps­ing forms of mul­ti­ple scle­ro­sis,” CMO Jay Back­strom said in a state­ment.

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

Genen­tech sub­mits a big plan to ex­pand 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.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

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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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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[via AP Images]

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.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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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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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.