GV backs new ge­nomics R&D up­start; Ver­tex ad­vances plans for next-gen CF drugs

GV — for­mer­ly known as Google Ven­tures — is join­ing a syn­di­cate pro­vid­ing a $40 mil­lion Se­ries A round to Dublin-based Ge­nomics Med­i­cine Ire­land. The com­pa­ny plans to build a world-class ge­nomics re­search ef­fort to bet­ter com­pre­hend dis­eases like Alzheimer’s. Arch and Po­laris are al­so back­ing the new com­pa­ny.

Ge­of­frey Porges, Leerink

Ver­tex out­lined plans to ad­vance its next-gen cys­tic fi­bro­sis drugs in the clin­ic, adding a new pro­gram along the way, though some an­a­lysts raised con­cerns about their chances of suc­cess. From Ge­of­frey Porges: “The com­pa­ny’s triple de­vel­op­ment plans ini­tial­ly seemed straight­for­ward and en­cour­ag­ing but be­came in­creas­ing more com­pli­cat­ed dur­ing the con­fer­ence call. Man­age­ment an­nounced that both drugs in phase Ia would ad­vance to phase II, in both het-mins and ho­mozy­gous delF508’s, in sim­i­lar two-part triple com­bi­na­tion tri­als. In ex­plain­ing why they had cho­sen a mul­ti-part for­mat for each triple drug pro­gram, man­age­ment dis­closed that one of the two drugs had been as­so­ci­at­ed with sig­nif­i­cant GI ad­verse events (Vx152) at the high­er dos­es test­ed in phase Ia, while the oth­er drug was as­so­ci­at­ed with sig­nif­i­cant ter­ato­genic­i­ty risks from pre-clin­i­cal stud­ies (Vx440), thus re­strict­ing its use in women to those on “pre-spec­i­fied, non-hor­mon­al” con­tra­cep­tion. This com­pound ap­pears to be com­pli­cat­ed by the oc­cur­rence of liv­er en­zyme in­duc­tion, which im­pairs the ef­fec­tive­ness of hor­mon­al con­tra­cep­tion, and pre­sum­ably al­so has drug-drug in­ter­ac­tion is­sues with oth­er med­i­cines al­so pre­scribed to CF pa­tients.”

Te­va and IBM are ex­pand­ing their col­lab­o­ra­tion on new drug de­vel­op­ment. The work in­cludes a more ag­gres­sive fo­cus on re­pur­pos­ing ther­a­pies along with da­ta-dri­ven man­age­ment of chron­ic dis­eases.

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.

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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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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