Pos­i­tive piv­otal da­ta in hand, GBT is hus­tling to the FDA in search of a quick OK for sick­le cell drug -- but that's no easy task

Glob­al Blood Ther­a­peu­tics sur­prised some ob­servers this morn­ing with their plan to take the first round of pos­i­tive late-stage da­ta for the sick­le cell dis­ease drug vox­elo­tor straight to the FDA in search of an ac­cel­er­at­ed ap­proval.

Ted Love

The reg­u­la­to­ry strat­e­gy is based on their da­ta for the pri­ma­ry out­come of the study, with 58% of the pa­tients in the high dose of the arm demon­strat­ing a 1 g/dL-plus in­crease in he­mo­glo­bin at 12 weeks com­pared to on­ly 9% of pa­tients tak­ing a place­bo. In the low­er-dose arm there was a 38% re­sponse rate, pass­ing the bar set by the FDA.

El­liott Vichin­sky, the di­rec­tor of the Com­pre­hen­sive Cen­ter for Sick­le Cell Dis­ease, UCSF Be­nioff Chil­dren’s Hos­pi­tal Oak­land, said that “in­creas­ing he­mo­glo­bin lev­els by re­duc­ing he­mol­y­sis in SCD pa­tients is like­ly to de­crease mor­bid­i­ty and mor­tal­i­ty.”

“Based up­on vox­elo­tor’s ro­bust im­pact on he­molyt­ic ane­mia, we be­lieve it meets the stan­dard for ac­cel­er­at­ed ap­proval,” said CEO Ted Love, “and we look for­ward to pro­vid­ing fur­ther up­dates on our reg­u­la­to­ry dis­cus­sions as soon as pos­si­ble, but no lat­er than year-end.”

But some in­vestors were clear­ly not ready to go so fast, mov­ing past the tri­umphal­ism in the biotech’s re­marks to some dis­cor­dant notes in the pre­sen­ta­tion this morn­ing.

Re­searchers failed to see a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in rates for va­so-oc­clu­sive cri­sis (VOC), which they at­trib­uted to “lim­it­ed pa­tient fol­low-up.” And their at­tempt to gain a big thumbs-up from pa­tients by track­ing pa­tient re­port­ed out­comes proved a com­plete bust, forc­ing ex­ecs to try and put some dis­tance be­tween them­selves and the PRO re­sults by drop­ping it as a key sec­ondary.

Those dis­tinc­tions caused some hedg­ing on the stock re­ac­tion, with a brief surge fol­lowed by a dip in­to the red as the share price waf­fled. But by mid-af­ter­noon, the op­ti­mists ap­peared to have won out, with the share price surg­ing 20%.

El­liott Vichin­sky

The biotech’s move to win a quick OK on the Part A da­ta fits in with the cur­rent flex­i­ble ap­proach that the FDA un­der Scott Got­tlieb has promised as reg­u­la­tors hus­tle to bring more drugs to the mar­ket. GBT now has to see just how flex­i­ble reg­u­la­tors are.

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.