Syn­cona dives deep in­to dry AMD R&D, up­ping Gy­ro­scope bet to $100M

The ex­plo­sion of gene ther­a­pies in the clin­ic promis­es to trans­form the treat­ment of eye dis­eases. And Syn­cona thinks one of their port­fo­lio com­pa­nies may have found the an­swer to dry AMD.

The life sci­ences in­vest­ment group sunk an ad­di­tion­al $57.7 mil­lion in Se­ries B fund­ing in­to the reti­nal gene ther­a­py group Gy­ro­scope Ther­a­peu­tics, bring­ing their to­tal in­vest­ment to just un­der $100 mil­lion. Syn­cona main­tains an 80% stake in the UK-based out­fit. An ad­di­tion­al $2.9 mil­lion will be pro­vid­ed by Cam­bridge In­no­va­tion Cap­i­tal.

A host of gene ther­a­pies have been re­leased or are in de­vel­op­ment for rare eye dis­or­ders – most promi­nent­ly Spark’s cost­ly Lux­tur­na, the first gene ther­a­py for in­her­it­ed blind­ness. But no ef­fec­tive treat­ment ex­ists for dry, age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD) — the most com­mon cause of blind­ness in the in­dus­tri­al world. AMD af­fects 170 mil­lion peo­ple glob­al­ly, 85-90% of whom have the dry type.

The new dol­lars will al­low Gy­ro­scope to com­plete its Phase I/II study of its GT005 ther­a­py, which works by in­ject­ing a virus-em­bed­ded so­lu­tion be­neath the reti­na to de­liv­er a gene se­quence cod­ing a pro­tein that pre­vents “com­ple­ment” cells from at­tack­ing healthy reti­nal cells. The mon­ey will al­so al­low it to start Phase II and ex­pand its sur­gi­cal plat­form to en­sure safe de­liv­ery of the gene to the reti­na.

In Feb­ru­ary, Gy­ro­scope dosed its first pa­tient, an 80-year-old Ox­ford woman, with the ther­a­py.

Two forms of AMD ex­ist: “dry” and “wet.” Sev­er­al drugs for the wet type ex­ist and more are un­der de­vel­op­ment, but be­cause the dry form re­mains un­treat­able by con­ven­tion­al means, it presents a ma­jor op­por­tu­ni­ty for de­vel­op­ers. A Ju­ly re­port from mar­ket in­tel­li­gence firm In­fini­ti Re­search es­ti­mates that “huge un­met med­ical need” for dry-AMD will dri­ve the mar­ket by 8% CA­GR.

Khurem Fa­rooq

The mar­ket’s first big dry-AMD hope, Roche’s lam­pal­izum­ab was dropped af­ter fail­ing both Phase III tri­als in 2017. Gy­ro­scope is com­pet­ing against J&J, Parisian biotech Bio­phytis and Ken­tucky-based biotech Apel­lis, among oth­ers, to be the first to in­tro­duce a dry–AMD ther­a­py. Re­searchers at NIH and in Chi­na are al­so test­ing stem-cell-based mod­els, while in Ju­ly the bio­elec­tron­ics com­pa­ny Pix­i­um Vi­sion an­nounced suc­cess in the 12-month fea­si­bil­i­ty study for their reti­nal im­plant.

Gy­ro­scope ac­quired US-based de­vice mak­er Or­bital Bio­medic in April. It is run by for­mer Genen­tech ex­ec­u­tive Khurem Fa­rooq, who over­saw com­mer­cial ac­tiv­i­ties for the wet-AMD drug Lu­cen­tis and pre-launch work for Roche’s lam­pal­izum­ab.

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.