Let's re­think Clin­ton's war on phar­ma; The FDA can't or­der the ge­nie back in­to the mag­ic lamp

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

 Sur­vey says!

Ever since I first start­ed think­ing about part­ner­ing up with Ar­salan Arif on End­points News, I want­ed to do a quar­ter­ly sur­vey of a sub­stan­tial num­ber of biotech ex­ecs to keep my thumb on the in­dus­try’s pulse. Now, 4 months from our launch, we did the first E100 sur­vey that found the pulse is quite strong. Yes, there is some flut­ter­ing ev­i­dent when you bring up top­ics like fu­ture fi­nanc­ings, IPOs, ac­cess to tal­ent, and so on. But by and large, biotechs are ex­pand­ing their teams and plan­ning to go in­to 2017 with am­bi­tious plans. We’ll fol­low up with a spe­cial 2017 fore­cast in Jan­u­ary. No one knows this in­dus­try bet­ter than the E100 com­bined. And you won’t want to miss what they have to say in the fu­ture.

 Hillary Clin­ton’s war on phar­ma was no chance event.

The lat­est batch of emails from Wik­iLeaks in­cludes the back­ground dis­cus­sion that oc­curred among Hillary Clin­ton’s cam­paign staff around her at­tack of Mar­tin Shkre­li, who still wears the black hat in biotech af­ter he de­cid­ed to jack up the price of Dara­prim by more than 5,000%. Clin­ton called it price goug­ing in a Tweet, trig­ger­ing a rout in biotech shares as the im­pli­ca­tions over some kind of price con­trols sank in. “FYI—We have start­ed the war with Phar­ma!!” ex­claimed se­nior ad­vis­er Ann O’Leary. “Great!” replied Mandy Grun­wald. There are bet­ter ways to have this dis­cus­sion than sin­gling out one bad ac­tor to slime an in­dus­try. Biotech is not Mar­tin Shkre­li, and the price of re­al in­no­va­tion in drugs will not be cheap. But that’s not price goug­ing. It’s time we sep­a­rat­ed drug de­vel­op­ment and the price of new drugs from the no­to­ri­ous prac­tice of rou­tine price hikes — or overnight prof­i­teer­ing — on old ther­a­pies. Maybe Clin­ton could Tweet about it some time. There’s no ques­tion now that she’s the odds-on fa­vorite to win this elec­tion.

 Kite is fly­ing high as its pi­o­neer­ing bi­o­log­ics ap­pli­ca­tion heads to the FDA

When Kite Phar­ma’s Arie Bellde­grun took to the stage in New York ear­li­er this week to spear­head a dis­cus­sion on the com­pa­ny’s am­bi­tious plans to launch the world’s first CAR-T, you could hear plen­ty of grum­bling on the side­lines. The da­ta are ear­ly. We all know that 3 months of re­spons­es in their piv­otal study is a quick read. Some want to wait for 6 months. We’ll see how that dis­cus­sion plays out. But while the com­pe­ti­tion at Juno and No­var­tis has fall­en well be­hind, Kite con­tin­ues to ag­gres­sive­ly press for­ward, not will­ing to give an inch un­less reg­u­la­tors re­quire it. Win or lose, you have to ad­mire the spir­it and com­pe­tence be­hind that.

 The FDA can’t make the Sarep­ta fall­out just dis­ap­pear

Now that the FDA has ac­tu­al­ly ap­proved Sarep­ta’s eteplirsen for Duchenne mus­cu­lar dy­s­tro­phy, the agency is try­ing to blunt the fall­out. Step one, for se­nior FDA of­fi­cial John Jenk­ins, was to spell out all the bad things that the biotech had done and then es­sen­tial­ly in­sist that no oth­er biotech will win an OK un­der sim­i­lar cir­cum­stances. Whip up ad­vo­cates to the ex­tent they at­tack FDA re­view­ers? Bad idea, says Jenk­ins. Use sus­pect da­ta from a woe­ful­ly in­ad­e­quate study to in­sist on an ap­proval? Uh-uh. The list goes on and on. What­ev­er the FDA says now, though, will do noth­ing to dis­pel the lessons to be learned from eteplirsen. And the next time a biotech comes along with an an­gry mob of sup­port­ers be­hind it, the FDA – and Janet Wood­cock – will have no one to blame but them­selves. This sto­ry is just be­gin­ning.

CRISPR: Los­ing some of the glit­ter

It was in­evitable. The third big IPO in gene edit­ing didn’t play out all that well, for­mal­ly end­ing gene edit­ing’s rep as the hottest thing in biotech. Noth­ing, of course, stays hot. But CRISPR Ther­a­peu­tics had to be dis­ap­point­ed this week that its tim­ing was off. Ed­i­tas and In­tel­lia had both made it out in big IPOs, on­ly to see their shares fade over the year. Gene edit­ing is still a pre­clin­i­cal field, with lots of changes in the tech to come. De­vel­op­ment ef­forts will be slow and painstak­ing, and it’s ask­ing a lot of in­vestors to stick through every­thing that has to hap­pen be­fore we see any ac­tu­al prod­ucts come out of it. Rev­o­lu­tions nev­er come fast or easy in biotech. Now comes the hard part. But it’s al­so the most valu­able stage for what comes next.

Gilead miss­es, again.

Gilead has plen­ty of re­sources to do R&D right. So it was kind of odd­ly fun­ny to see its messy da­ta from a tiny mid-stage pro­gram on a slate of in­di­ca­tions for GS-4997, in­clud­ing a pos­i­tive snap­shot for NASH. The prob­lem, though, was that the pic­ture proved too fuzzy to make out any ex­act ideas about its po­ten­tial. Gilead needs to prove now that it can do some­thing dra­mat­i­cal­ly great. This lit­tle stum­ble comes at a par­tic­u­lar­ly bad time.

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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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.