Mit­subishi Tan­abe wins the race to snag the first new ALS drug OK in more than 20 years

Af­ter be­ing re­cruit­ed by the FDA, Mit­subishi Tan­abe has won the agency’s ap­proval to start sell­ing edar­avone as a new treat­ment for ALS — the first new drug for this lethal dis­ease in more than two decades and on­ly the sec­ond now avail­able to pa­tients.

Reg­u­la­tors asked the Japan­ese phar­ma com­pa­ny to file the drug for ALS af­ter its ap­proval in Ko­rea and Japan. The drug — which has al­so been sold for acute is­chemic stroke for years in Japan — will now hit the mar­ket as Rad­i­ca­va.

For a short time at least, a new sub­sidiary group set up to sell the drug will have a clear crack at a mar­ket that was slat­ed to shrink as the sole brand­ed ALS drug lost rev­enue to gener­ics. The last new ther­a­py, Ri­lutek (rilu­zole) from Sanofi, ar­rived in the US in the mid-90s, un­der­scor­ing just how dif­fi­cult this dis­ease has been to tack­le in the clin­ic.

Rad­i­ca­va is no cure. Pa­tients con­tin­ued to de­cline in the 167-pa­tient study re­viewed by the FDA, but they weren’t so quick to lose their abil­i­ty to func­tion. And in a twist, it ar­rives af­ter reg­u­la­tors asked the phar­ma com­pa­ny to file for an OK.

Er­ic Bast­ings, FDA CDER

“Af­ter learn­ing about the use of edar­avone to treat ALS in Japan, we rapid­ly en­gaged with the drug de­vel­op­er about fil­ing a mar­ket­ing ap­pli­ca­tion in the Unit­ed States,” said Er­ic Bast­ings, deputy di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “This is the first new treat­ment ap­proved by the FDA for ALS in many years, and we are pleased that peo­ple with ALS will now have an ad­di­tion­al op­tion.”

The com­pa­ny hasn’t for­mal­ly dis­trib­uted da­ta on the drug, that’s com­ing up at a sci­en­tif­ic con­fer­ence lat­er in the year, but re­searchers have not­ed that pa­tients tak­ing a 60 mg dose of the drug de­clined at a rate rough­ly a third slow­er than the place­bo arm, com­par­ing them to base­line on the re­vised ALS Func­tion­al Rat­ing Scale.

Neil A. Shnei­der, di­rec­tor of the Eleanor and Lou Gehrig ALS Cen­ter at Co­lum­bia Uni­ver­si­ty Med­ical Cen­ter, told The New York Times that the “ef­fect is mod­est but sig­nif­i­cant.”

Mit­subishi Tan­abe is plan­ning to go af­ter the mar­ket with a price of $1,086 per in­fu­sion and an an­nu­al list price of $145,524, which could pose some trou­ble for the phar­ma com­pa­ny. In ad­di­tion to the high cost and lim­it­ed ben­e­fits, Shnei­der and oth­ers in the field are none too hap­py about the risks posed to pa­tients who will need to keep an IV line left in place for their in­fu­sions, rais­ing the risk of in­fec­tion.

In the mean­time, there are at least two oth­er biotechs look­ing to make their mark in the field. Cy­to­ki­net­ics is one, with a late-stage drug called tirasem­tiv. AB Sci­ence’s AB-1010, an oral ty­ro­sine ki­nase in­hibitor, was filed for an ap­proval in Eu­rope last fall.

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.