SteadyMed shares slammed as FDA toss­es its NDA for PAH drug-de­vice

When SteadyMed $STDY out­lined its IPO plans in ear­ly 2015, the biotech as­sured would-be in­vestors that it could be ready to file an ap­pli­ca­tion for its drug-de­vice ther­a­peu­tic Trevyent a year lat­er.

Things haven’t worked out as planned for the biotech, though. The NDA ar­rived at the agency in June of this year, and has now been hand­ed back as in­com­plete.

This morn­ing the biotech re­port­ed that the FDA has is­sued a refuse-to-file let­ter on Trevyent — the FDA’s sec­ond RTF this week — which us­es a pump to de­liv­er a re­for­mu­lat­ed ver­sion of Unit­ed Ther­a­peu­tics’ main­stay drug Re­mod­ulin for pul­monary ar­te­r­i­al hy­per­ten­sion.

That’s not the news in­vestors were look­ing for. Its shares plunged 44%, tak­ing a big bite out of its mi­cro mar­ket cap.

The ac­tive in­gre­di­ent in Unit­ed’s Re­mod­ulin is tre­pros­tinil, and SteadyMed — based in San Ra­mon, CA with R&D in Is­rael — has been fight­ing to can­cel Unit­ed’s patents on that drug. It has some im­me­di­ate con­cerns to deal with at the FDA, though.

Ac­cord­ing to the biotech:

FDA has re­quest­ed fur­ther in­for­ma­tion on cer­tain de­vice spec­i­fi­ca­tions and per­for­mance test­ing and has re­quest­ed ad­di­tion­al de­sign ver­i­fi­ca­tion and val­i­da­tion test­ing on the fi­nal, to-be-mar­ket­ed Trevyent prod­uct. With­in the next 30 days, the Com­pa­ny will re­quest a Type A meet­ing with the FDA to gain fur­ther clar­i­fi­ca­tion on the ad­di­tion­al in­for­ma­tion re­quired for re­sub­mis­sion and ac­cep­tance of the NDA. The Com­pa­ny will pro­vide fur­ther guid­ance af­ter the an­tic­i­pat­ed meet­ing with FDA.

Jonathan Rig­by

The FDA al­so is­sued a rare RTF ear­li­er in the week to Acor­da, which has been at­tempt­ing a high wire act to hus­tle CVT-301 to the FDA as it looks to re­place Ampyra as gener­ic com­pe­ti­tion looms.

As for SteadyMed, the com­pa­ny plans to re­group and get back in front of reg­u­la­tors.

“We be­lieve that the is­sues raised in the let­ter from FDA can be suf­fi­cient­ly ad­dressed. Our next step is to work with the FDA to ad­dress the open is­sues and iden­ti­fy a path to a suc­cess­ful re­sub­mis­sion and ac­cep­tance of our ap­pli­ca­tion,” said SteadyMed CEO Jonathan Rig­by. “We be­lieve that Trevyent holds the po­ten­tial to sig­nif­i­cant­ly im­prove the lives of pa­tients suf­fer­ing from PAH com­pared to the cur­rent stan­dard of care, and re­main com­mit­ted to bring­ing the prod­uct to pa­tients in need.”

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.