Bio­gen, Io­n­is win FDA OK for spinal mus­cu­lar at­ro­phy drug Spin­raza

Bio­gen $BI­IB and Io­n­is $IONS got an ear­ly Christ­mas present Fri­day evening as the FDA an­nounced that it had pro­vid­ed an ac­cel­er­at­ed ap­proval for Spin­raza (nusin­ersen), their new drug — and the first — for rare cas­es of spinal mus­cu­lar at­ro­phy.

Peak sales es­ti­mates hov­er around $2 bil­lion, with some of the new es­ti­mates com­ing in over $3 bil­lion by 2026.

“Our pre­vi­ous fore­cast in­clud­ed sales of $75mm in 2017 grow­ing to $2.8bn by 2026,” not­ed Leerink’s Ge­of­frey Porges in ear­ly No­vem­ber, with the ar­rival of more Phase III da­ta. “We now as­sume a launch in Q1 2017 from Q2 2017 pre­vi­ous­ly and our cur­rent fore­cast is for sales of $92mm in 2017 in­creas­ing to $1.56bn by 2020 and then $3.2bn by 2026.”

Bio­gen says that it will be ready to ship the drug in a week.

Shares of Io­n­is shot up 10% and Bio­gen saw its stock climb 2% on the news.

The ap­proval comes a lit­tle less than two months af­ter re­searchers post­ed a fresh round of promis­ing da­ta on the drug. Us­ing the Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed re­port card for pa­tients’ mo­tor func­tions, the drug arm wit­nessed a mean 4.0 im­prove­ment on scores while the place­bo arm dropped by 1.9 points, a 5.9 point over­all spread in the drug’s fa­vor.

Io­n­is CEO Stan­ley Crooke

Both com­pa­nies bad­ly need­ed this win af­ter a year of up­heavals. The ap­proval will help set the stage for Bio­gen’s new CEO, Michel Vounatsos, to take the helm. Io­n­is, mean­while, can boast of a year-end vic­to­ry af­ter set­backs in the clin­ic. And an­a­lysts were im­pressed by the broad la­bel ap­plied by the FDA.

“La­bel­ing is not re­strict­ed to any spe­cif­ic SMA sub­type, in­di­cat­ing that it will like­ly be broad­ly pre­scribed for type 1, 2, and 3 pa­tients and while the epi­demi­ol­o­gy in SMA is not en­tire­ly de­fin­i­tive, a la­bel mak­ing the drug avail­able to all pa­tient types rep­re­sents sig­nif­i­cant ex­pan­sion po­ten­tial to the ini­tial mar­ket op­por­tu­ni­ty (though giv­en the un­met need in SMA, this pos­i­tive de­vel­op­ment is not en­tire­ly un­sur­pris­ing),” notes Jef­feries Bri­an Abra­hams.

The ap­proval al­so comes with a rare dis­ease pri­or­i­ty vouch­er, which can be used to ac­cel­er­ate re­view of an­oth­er pro­gram, or sold on the open mar­ket.

“There has been a long-stand­ing need for a treat­ment for spinal mus­cu­lar at­ro­phy, the most com­mon ge­net­ic cause of death in in­fants, and a dis­ease that can af­fect peo­ple at any stage of life,” said Bil­ly Dunn, MD, di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “As shown by our sug­ges­tion to the spon­sor to an­a­lyze the re­sults of the study ear­li­er than planned, the FDA is com­mit­ted to as­sist­ing with the de­vel­op­ment and ap­proval of safe and ef­fec­tive drugs for rare dis­eases and we worked hard to re­view this ap­pli­ca­tion quick­ly; we could not be more pleased to have the first ap­proved treat­ment for this de­bil­i­tat­ing dis­ease.”

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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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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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.