Scott Got­tlieb will push to pub­lish CRLs; Marathon's demise of­fers the in­dus­try a cau­tion­ary tale

End­points News as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.


Scott Got­tlieb did the right thing of­fer­ing to pub­lish those CRLs

Dur­ing Scott Got­tlieb’s last round of writ­ten an­swers to the Sen­ate com­mit­tee re­view­ing his nom­i­na­tion as FDA com­mis­sion­er, I was pleased to see him vol­un­tar­i­ly pro­pose that the agency start pub­lish­ing com­plete re­sponse let­ters. Got­tlieb has done that be­fore, but so far we haven’t seen a recom­mit af­ter his nom­i­na­tion.

Why is this so im­por­tant?

We know that there have been a num­ber of in­stances when drug com­pa­nies re­ly on the agency’s si­lence to put a PR spin on their CRLs. Or they just don’t say any­thing at all, which is just as com­mon.

Frankly, af­ter com­pa­nies have had a chance to talk up their prospects — of­ten for years — for drugs on­ly to see them turned back at the fin­ish line with­out any hon­est ac­count­ing for the re­jec­tion is un­ac­cept­able. Physi­cians, pa­tients, in­vestors, tri­al sub­jects (and, yes, jour­nal­ists) have a right to find out what trig­gers a reg­u­la­to­ry boo­by trap.

No one forced Got­tlieb’s arm on this. There was no spe­cif­ic query on CRLs. He pro­posed it, with a caveat on redact­ing pro­pri­etary in­fo, which is the right thing to do — one rea­son why we’ve been sup­port­ive of his nom­i­na­tion. It’s al­so a prac­ti­cal way to dis­tance him­self from ac­cu­sa­tions from some De­moc­rats his past busi­ness ties leave him hope­less­ly con­flict­ed.

The com­mit­tee vote send­ing the nom­i­na­tion to the Sen­ate floor will hope­ful­ly be act­ed on in the next cou­ple of weeks. The soon­er the bet­ter. It’s time to get start­ed on the next chap­ter on the FDA and bio­phar­ma.


As PTC preps a con­tro­ver­sial launch of de­flaza­cort, Marathon is wind­ing down

In a Sun­day news scoop, we re­vealed that Marathon Phar­ma­ceu­ti­cals is be­ing wound down now that PTC has ac­quired US rights to its steroid de­flaza­cort.

These days, get­ting held up as an ex­am­ple of ram­pant price goug­ing can cre­ate an en­vi­ron­ment that is too tox­ic to deal with.

The next step in this process will be to see how much of the tox­i­c­i­ty is ab­sorbed by PTC, which bought the drug and has the un­en­vi­able task now to try and come up with an ac­cept­able price af­ter Marathon failed spec­tac­u­lar­ly at $89,000 a year. For par­ents who had been buy­ing it from the UK’s Mas­ters at a lit­tle more than $1,000, that was a shock. How PTC hopes to make cash now is hard to un­der­stand.

You can be sure that PTC had a firm price in mind when they bought this drug. But it ap­pears we’ll have to wait in­to ear­ly May to get a look at the stick­er.  We’ll be wait­ing.

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.

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.