Ul­tragenyx lands first FDA OK, with an­oth­er on the hori­zon in shift to com­mer­cial ops

Ul­tragenyx CEO Emil Kakkis is now op­er­at­ing a com­mer­cial op­er­a­tion, just as he dreamed he would when he start­ed the com­pa­ny in 2010.

The FDA has ap­proved Mep­se­vii (ve­stron­idase al­fa-vjbk) for an ul­tra-rare ge­net­ic en­zyme dis­or­der called MPS VII, and the OK comes with a pri­or­i­ty re­view vouch­er that could eas­i­ly be worth $125 mil­lion.

Emil Kakkis

The biotech $RARE has been sig­nal­ing this ad­vance for some time now, ea­ger­ly await­ing a green light along with a big ap­proval in line for buro­sum­ab, which has a much larg­er mar­ket in view. Mep­se­vii — a re­place­ment of the miss­ing be­ta-glu­curonidase en­zyme that caus­es skele­tal de­for­ma­tion as tox­ic ma­te­r­i­al builds up in cells — was test­ed in 23 pa­tients rang­ing from 5 months to 25 years, demon­strat­ing a sig­nif­i­cant gain in walk­ing dis­tance for the pa­tients in the drug arm.

Leerink’s Joseph Schwartz has high­light­ed the hand­ful of pa­tients who have MPS VII.

Giv­en the low preva­lence of MPS7 pts. WW (~200 es­ti­mat­ed), pt. iden­ti­fi­ca­tion, dis­ease aware­ness, and named pt. sales ac­tiv­i­ties could lay an im­por­tant ground­work for ve­stron­idase al­pha once it’s ap­proved.

And it won’t be cheap. In a call with an­a­lysts Wednes­day evening, Ul­tragenyx set the price at an av­er­age of $375,000 a year, well be­low the $550,000 price Schwartz had bet on. But it’s im­por­tant to re­mem­ber that the com­pa­ny price is based on weight — 4mg/kg Q2W ($2,115/vial) — with the av­er­age pegged at 55 pounds.

No­va­to, CA-based Ul­tragenyx has had its set­backs along the way, but it’s al­so been mak­ing steady progress to­ward the time when it can start field­ing a 30-per­son US sales team to mar­ket a port­fo­lio of rare dis­ease drugs. Kakkis al­so re­cent­ly de­cid­ed to buy out the sur­viv­ing gene ther­a­py port­fo­lio at the trou­bled Di­men­sion Ther­a­peu­tics, which ran in­to a thick­et of trou­ble with weak he­mo­phil­ia B da­ta for the lead pro­gram.

“This ap­proval un­der­scores the agency’s com­mit­ment to mak­ing treat­ments avail­able to pa­tients with rare dis­eases,” said Julie Beitz, di­rec­tor of the Of­fice of Drug Eval­u­a­tion III in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search (CDER). “Pri­or to to­day’s ap­proval, pa­tients with this rare, in­her­it­ed con­di­tion had no ap­proved treat­ment op­tions.”

This ap­proval al­so un­der­scores the huge pop­u­lar­i­ty of rare dis­eases for star­tups, a field where re­searchers can make re­al progress with small pa­tient groups and pay­ers are will­ing to cov­er big an­nu­al costs so long as the pa­tient num­bers are con­tained.

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.

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

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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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.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.