Scott Got­tlieb has some ideas about block­ing any more would-be Mar­tin Shkrelis from price goug­ing

FDA com­mis­sion­er Scott Got­tlieb wants to stop the next Mar­tin Shkre­li be­fore he, or she, gets start­ed on the next drug price goug­ing scan­dal. And shin­ing a bright light on the drugs most vul­ner­a­ble to pric­ing abuse will be one way to get start­ed.

In tes­ti­mo­ny be­fore the House ap­pro­pri­a­tions sub­com­mit­tee Thurs­day, the new com­mis­sion­er high­light­ed sev­er­al meth­ods that could pre­vent bio­phar­ma com­pa­nies from gam­ing the sys­tem, fo­cus­ing specif­i­cal­ly on “sit­u­a­tions where off-patent drugs lack an ap­proved gener­ic com­peti­tor.” Got­tlieb will get the FDA to pub­lish a list of drugs that are off patent “for which FDA has not ap­proved a sin­gle gener­ic ap­pli­cant.”

Scott Got­tlieb

Got­tlieb wants to flag the po­ten­tial for a re­peat of Shkre­li’s de­ci­sion to buy an old gener­ic, Dara­prim, and then rais­ing the price of the drug overnight by more than 5000% at his com­pa­ny, Tur­ing. Dara­prim had no gener­ic com­pe­ti­tion on the mar­ket, giv­ing Shkre­li a clear and le­gal path to jack­ing up the price.

There are var­i­ous ways to im­prove the gener­ic drug ap­proval process to in­crease com­pe­ti­tion and pre­vent play­ers from gam­ing prices, he says. But it’s clear­ly an is­sue he wants to tack­le ear­ly on.

“We do have sit­u­a­tions where spec­u­la­tors, for lack of a bet­ter word, can come in and buy a low vol­ume gener­ic, jack up the price know­ing that it will take po­ten­tial­ly years for gener­ic com­pe­ti­tion to come on to the mar­ket so they have that sort of an ex­clu­siv­i­ty pe­ri­od,” said the com­mis­sion­er.

Got­tlieb out­lined a three-prong plan to speed up gener­ic drug ap­provals and com­plete­ly elim­i­nate the back­log of ap­pli­ca­tions for knock­offs, hur­ry­ing along cheap­er al­ter­na­tives to brand ther­a­pies that could go a long way to re­duc­ing the coun­try’s spend­ing on phar­ma­ceu­ti­cals.

He told law­mak­ers that he plans to:

— “Cur­tail gam­ing by in­dus­try of our reg­u­la­tions which can ex­tend mo­nop­oly pe­ri­ods be­yond the time frame Con­gress in­tend­ed, hin­der­ing com­pe­ti­tion.”

— “Im­prove the process­es that en­able gener­ic ver­sions of com­plex drugs to be ap­proved for mar­ket­ing.”

— “In­crease the over­all ef­fi­cien­cy of the gener­ic drug re­view process while com­plete­ly elim­i­nat­ing the back­log of gener­ic ap­pli­ca­tions.”

The REMS pro­gram (risk eval­u­a­tion and mit­i­ga­tion strate­gies), he not­ed, has been used to slow gener­ic com­pe­ti­tion, and stream­lin­ing the process by waiv­ing the re­quire­ment that a gener­ic and brand man­u­fac­tur­er share a sin­gle sys­tem for as­sur­ing safe use could be used to elim­i­nate one tool used to slow gener­ics.

In the past, gener­ic man­u­fac­tur­ers have had to sue brand op­er­a­tions which re­fused to ne­go­ti­ate a shared safe­ty sys­tem as a way to block cheap­er com­pe­ti­tion.

Rep. Robert Ader­holt

Got­tlieb al­so sin­gled out the on­col­o­gy di­vi­sion at the FDA for em­brac­ing the break­through ther­a­py pro­gram, speed­ing along new ap­provals. Un­for­tu­nate­ly, he added, not every part of the agency does as well as the on­col­o­gy group, and he in­tends to bring the lag­gards up to speed. In ad­di­tion, he added, the FDA could do more by tak­ing a more pro­gres­sive view of some new tech­nolo­gies, like bio­mark­ers, in as­sess­ing drugs, look­ing at tri­al de­signs to find more ef­fi­cient ways to de­vel­op drugs and adding new tools for ther­a­peu­tic re­views.

It was im­me­di­ate­ly clear that the law­mak­ers of all stripes didn’t care for the Trump ad­min­is­tra­tion’s plans for the 2018 FDA bud­get.

De­moc­rats and Re­pub­li­cans alike wast­ed lit­tle time in crit­i­ciz­ing the Trump ad­min­is­tra­tion’s plan to dou­ble up on in­dus­try user fees while slash­ing the fed­er­al gov­ern­ment’s con­tri­bu­tion to the FDA’s bud­get.

“The agency’s chances of off­set­ting bud­get au­thor­i­ty with user fees face a very stiff up­hill bat­tle in the fu­ture,” not­ed Al­aba­ma Re­pub­li­can Robert Ader­holt. He al­so cit­ed the FDA’s rapid progress in hus­tling along new ap­provals of Keytru­da, which he should help, “uti­liz­ing the lat­est tech­nol­o­gy, make drugs ac­ces­si­ble be­fore a lengthy drug tri­al.”

One plus for the FDA: Got­tlieb not­ed that the FDA is no longer in the grip of a fed­er­al hir­ing freeze im­posed by Pres­i­dent Trump, ac­cord­ing to RAPS. The freeze, he told law­mak­ers, has been lift­ed.

The home run count: The $100M+ mega-round boom in biotech in­spired a $6.7B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.

Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMedO

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Covid-19 roundup: CDC de­bat­ing who should get first avail­able vac­cines; EU in Gilead talks af­ter US gob­bled first remde­sivir dos­es

The federal government has now spent billions of dollars accelerating the development of a Covid-19 vaccine, and yet they’ve remained hush-hush on who, precisely, would actually get inoculated once the first doses are approved and available. Internally, though, they have been debating it.

The CDC and an advisory committee of outside health experts have been working since April to devise a ranking system that would determine who receives a vaccine and when, The New York Times reported. The question of who is first in line for inoculation is important because no matter how many doses developers can make or how quickly they can make them, doses will still come out in batches; 300 million inoculations will not appear overnight.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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