Hillary Clin­ton's War on Phar­ma II; A call for com­mon sense re­port­ing on Alzheimer's R&D

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

Hillary Clin­ton want­ed that war with phar­ma, bad­ly

For all the back­lash around Mar­tin Shkre­li’s brief, dis­as­trous turn as CEO of Tur­ing, the Tweet from Hillary Clin­ton con­demn­ing his price goug­ing stands out for the wide­spread dam­age it did to biotech stocks. There are lots of rea­sons why the biotech stock in­dex­es have tanked over the past year. And it’s easy to see why that may have been over­due. But hav­ing a pres­i­den­tial can­di­date use Shkre­li to de­clare a “war on pharma” sets the stage for the bat­tle to con­tin­ue in DC af­ter the votes are count­ed. Try­ing to score cheap points with vot­ers in a pri­ma­ry is a bad way to es­tab­lish pol­i­cy. Yet that is what we are look­ing at if Clin­ton is elect­ed. The US — and the bad­ly frac­tured health sys­tem that needs re­form­ing — de­serve bet­ter. And biotech has enough prob­lems right now with­out get­ting caught up in the war. Let’s urge Clin­ton to take a more re­spon­si­ble stand if she wins the big elec­tion Tues­day.

The SEC sends an im­por­tant mes­sage on who biotech ac­coun­tants are work­ing for

We re­port­ed this week that the SEC wants to sanc­tion the PwC ac­coun­tant who let Steven Bur­rill go his mer­ry way for years be­fore he was caught – by oth­er means – loot­ing the biotech fund he man­aged. The SEC has al­ready dealt with Bur­rill, who was forced to re­im­burse funds used for per­son­al ex­pens­es, but en­forcers want to send a mes­sage here to oth­er ac­coun­tants do­ing the books for biotechs and all com­pa­nies. You have a pro­fes­sion­al re­spon­si­bil­i­ty to check out why ex­ecs are, say, ad­vanc­ing big­ger and big­ger sums for work not yet done. Sums that may nev­er have been war­rant­ed un­der any cir­cum­stances. And you can’t be waved away by the same ex­ecs who may have some­thing to hide. Bur­rill’s in­vestors de­served bet­ter. What­ev­er the in­di­vid­ual mer­its of these charges, let’s hope that mes­sage comes through loud and clear.

New in­ter­nal FDA doc­u­ments un­der­score Wood­cock’s un­in­formed de­ci­sion to back Sarep­ta drug

As I’ve said be­fore, there’s no rea­son to ex­pect the Sarep­ta con­tro­ver­sy to go away any­time soon. The lat­est doc­u­ment drop from the FDA on the in­ter­nal war over Janet Wood­cock’s in­sis­tence on an ap­proval now in­cludes ac­cu­sa­tions that she ig­nored in­ter­nal as­sess­ments on its Duchenne drug Ex­ondys 51 from se­nior of­fi­cials. This was noth­ing more than a gut-lev­el de­ci­sion which clear­ly de­vi­at­ed from agency stan­dards for an ap­proval. And FDA Com­mis­sion­er Robert Califf un­wise­ly de­cid­ed to let her have her way, open­ing up a new and dan­ger­ous path for oth­ers to fol­low. Let’s all con­tin­ue to hope this drug is as safe and ef­fi­ca­cious as the com­pa­ny and pa­tients ad­vo­cates in­sist it is. But ap­prov­ing an ex­per­i­men­tal drug like this was worse than a mis­take. It was bad pol­i­cy mak­ing at its worse.

Let’s re­main pro­fes­sion­al­ly skep­ti­cal about Alzheimer’s drug re­search

Look­ing over all the ways main­stream me­dia fawns over ear­ly-stage Alzheimer’s drug re­search has be­come some­thing of a hob­by of mine. And this week we were treat­ed to the full smor­gas­bord of gulli­bil­i­ty with a string of un­re­served re­views for the ear­ly an­i­mal and hu­man da­ta that’s be­come avail­able for Mer­ck’s late-stage BACE drug. Some of the UK pa­pers (hel­lo Dai­ly Mail) have a ten­den­cy to jump from the slight­est in­di­ca­tion of ef­fi­ca­cy in small groups to won­der­ing when the new cure will be ap­proved. We saw the same thing re­cent­ly when Bio­gen tout­ed its ear­ly da­ta on ad­u­canum­ab. But it’s a cru­el hoax on the pa­tients and their fam­i­lies. There are good rea­sons why Alzheimer’s has been an R&D dis­as­ter zone for more than a decade. Let’s wait for some con­vinc­ing da­ta be­fore herald­ing the next big break­through. We’re a long way from a cure. And that’s a hard truth. Alzheimer’s de­vel­op­ment has a long, long way to go, un­der the best of cir­cum­stances.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

[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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.