House lines up biotech lol­lipops as sup­port grows for an epic 21st Cen­tu­ry Cures Act

Now that Don­ald Trump is head­ed to the White House with plans to gut Oba­maCare, the House has field­ed a new ver­sion of the 21st Cen­tu­ry Cures Act in a bid to push through a bill now that in­cludes fresh promis­es of an even speed­i­er and more re­spon­sive FDA. And now that the De­moc­rats and Re­pub­li­cans have reached an agree­ment on how to fund the mea­sure, pluck­ing cash from the car­cass of the Af­ford­able Care Act, there are good odds that the House and Sen­ate could reach an agree­ment on a fi­nal bill in the not too dis­tant fu­ture.

Ac­cord­ing to Kaiser Health News, more than 1,400 lob­by­ists have tak­en a crack — for and against — var­i­ous sec­tions of this bill. And that helps ex­plain why this com­pro­mise bill weighs in at an epic 944 pages.

Rachel Sachs, a pro­fes­sor at Wash­ing­ton Uni­ver­si­ty who’s been fol­low­ing this bill close­ly, notes that some of the orig­i­nal good­ies for drug and de­vice mak­ers have been re­moved, in­clud­ing one sec­tion that would have al­lowed de­vel­op­ers to cir­cum­vent the FDA’s ap­proval process.

So what’s in? A spe­cial man­date for the NIH to sup­port young sci­en­tists look­ing for first-time grants; the pri­or­i­ty re­view vouch­er pro­gram that the FDA hates; an ex­tra $500 mil­lion in fund­ing for the FDA to move drugs and de­vices along faster over the next 10 years, and so on.

Here’s a 44-page sum­ma­ry to help di­gest what’s com­ing. You’ll find this in the sec­tion on speed­ed drug de­vel­op­ment, a promise that Trump made in his post-elec­tion game plan. The bill:

• Es­tab­lish­es a re­view path­way at FDA for bio­mark­ers and oth­er drug de­vel­op­ment tools that can be used to help short­en drug de­vel­op­ment time and re­duce the fail­ure rate in drug de­vel­op­ment.

• Al­lows spon­sors of ge­net­i­cal­ly tar­get­ed or vari­ant pro­tein tar­get­ed drugs to re­ly on da­ta for the same or sim­i­lar tech­nol­o­gy from pre­vi­ous­ly ap­proved ap­pli­ca­tions by the same spon­sor.

• Does not al­ter the ex­ist­ing ap­proval stan­dards for drugs.

There have been some high-pro­file scan­dals this year to sug­gest that the FDA may have act­ed hasti­ly in ap­prov­ing drugs or get­ting them back in­to the clin­ic af­ter a safe­ty mishap, most re­cent­ly with Juno’s new clin­i­cal hold in the wake of more pa­tient deaths. Far from slow­ing down the march at the FDA to low­er the bar for bio­phar­ma, Con­gress is in­tent on hit­ting the gas and forc­ing reg­u­la­tors to go even faster.

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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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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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.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.