Ver­tex sharp­ens fo­cus on gene-edit­ing in li­cens­ing deal with Mer­ck KGaA

Two years ago Mer­ck KGaA splurged $230 mil­lion to li­cense all the rights to four on­col­o­gy pro­grams, in­clud­ing a DNA-de­pen­dent pro­tein ki­nase (DNA-PK) in­hibitor, from cys­tic fi­bro­sis drug­mak­er Ver­tex. On Thurs­day, the Ger­man drug­mak­er al­lowed Ver­tex ac­cess to the com­pound via a li­cens­ing deal for use in the field of gene edit­ing.

No fi­nan­cial de­tails were pro­vid­ed for the new trans­ac­tion, which al­so en­ables Ver­tex to ac­cess to an undis­closed pre­clin­i­cal com­pound. The DNA-PK in­hibitor — M9831 (for­mer­ly known as VX-984) — and the pre­clin­i­cal drug were li­censed to Mer­ck from Ver­tex in Jan­u­ary 2017 and are now part of the Darm­stadt-based com­pa­ny’s ar­se­nal of DNA Dam­age Re­sponse (DDR) in­hibitors.

Dam­age to the DNA in cells oc­curs every day and con­se­quent­ly the DDR mon­i­tors im­pact, ini­ti­ates re­pair and halts cell growth dur­ing the re­build­ing process. But in pa­tients with can­cer there is a high fre­quen­cy of DDR de­fects which cul­mi­nate in the ac­cu­mu­la­tion of DNA dam­age, giv­ing rise to mu­ta­tions that pro­mote un­bri­dled can­cer cell growth and/or en­able nor­mal cells to evade apop­to­sis. Al­though can­cer cells may ben­e­fit from DDR de­fects, oth­er func­tion­ing DNA re­pair sys­tems are re­quired for sur­vival. DDR in­hibitors thus of­fer an op­por­tu­ni­ty to fight can­cer by se­lec­tive­ly killing can­cer cells via the sup­pres­sion of stand­by DDR mech­a­nisms and spar­ing nor­mal healthy cells that are not de­pen­dent on these path­ways.

Mer­ck’s port­fo­lio of DDR in­hibitors in­cludes two ATR in­hibitors, an ATM in­hibitor and an in­ves­ti­ga­tion­al small-mol­e­cule of DNA-PK, which is a key en­zyme that could po­ten­tial­ly en­hance the ef­fi­ca­cy of DNA-dam­ag­ing agents such as ra­dio­ther­a­py and chemother­a­py. Pre­clin­i­cal da­ta has shown DNA-PK in­hi­bi­tion can al­so aug­ment CRISPR/Cas9-me­di­at­ed gene edit­ing, the com­pa­ny said.

With this deal, Ver­tex is sharp­en­ing its fo­cus in­to gene edit­ing. The Boston biotech, which oust­ed its COO and in­ter­im CFO Ian Smith for im­prop­er per­son­al con­duct on Wednes­day, is al­ready col­lab­o­rat­ing with CRISPR Ther­a­peu­tics to eval­u­ate the lat­ter’s CRISPR gene edit­ing tech­nol­o­gy to po­ten­tial­ly fix the mu­ta­tions in the CFTR gene un­der­stood to re­sult in the de­fec­tive pro­tein that caus­es cys­tic fi­bro­sis, as well as oth­er ge­net­ic dis­or­ders. Their ex­per­i­men­tal treat­ment, CTX001, was grant­ed fast track sta­tus by the FDA ear­li­er this month for sick­le cell dis­ease.

Un­der the new Mer­ck/Ver­tex deal, Mer­ck will re­ceive an up­front pay­ment and is el­i­gi­ble to re­ceive mile­stone pay­ments and  roy­al­ties from po­ten­tial drug sales, and Ver­tex has the op­tion to add in­di­ca­tions to the li­cense grant. Over­all, Mer­ck re­tains the rights to both as­sets in all oth­er dis­ease ar­eas, in­clud­ing on­col­o­gy, and can de­vel­op both these com­pounds in-house, or li­cense them to oth­er com­pa­nies for use in the gene edit­ing field.

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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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.

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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