Roche CMO Sandra Horning speaks at an Endpoints News breakfast, January 2019 [Jeff Rumans for Endpoints News]

Look out Alex­ion, Roche breaks out the de­tails on its ri­val rare CNS drug

Months af­ter Alex­ion scored the first-ever ap­proval for pa­tients with neu­romyelitis op­ti­ca spec­trum dis­or­der (NM­SOD) — re­searchers at Roche have come out with de­tailed da­ta on their ri­val ex­per­i­men­tal drug satral­izum­ab for use in the rare cen­tral ner­vous sys­tem dis­or­der.

The 95-pa­tient study dubbed SAkuraS­tar test­ed satral­izum­ab against a place­bo. Da­ta showed satral­izum­ab monother­a­py con­ferred a 55% re­duc­tion in the risk of re­laps­es com­pared to place­bo in the over­all pop­u­la­tion, rep­re­sen­ta­tive of NMOSD pa­tients (p=0.0184). In the over­all satral­izum­ab-treat­ed pop­u­la­tion, 76.1% were re­lapse-free at 48 weeks, and 72.1% re­lapse-free at 96 weeks, ver­sus 61.9% and 51.2% with place­bo, re­spec­tive­ly.

In ad­di­tion, in the sub­group of pa­tients that are AQP4-IgG seropos­i­tive — who tend to ex­pe­ri­ence a more se­vere dis­ease course — the ef­fect of satral­izum­ab was more stark. Pa­tients on the drug saw a high­er 74% re­duc­tion in risk of re­laps­es. Da­ta showed that 82.9% were re­lapse-free at 48 weeks and 76.5% re­lapse-free at 96 weeks when treat­ed with satral­izum­ab, com­pared to 55.4% and 41.1% with place­bo, re­spec­tive­ly.

Roche’s out­go­ing chief med­ical of­fi­cer San­dra Horn­ing added: “We are en­cour­aged by these re­sults and look for­ward to work­ing with reg­u­la­tors over the com­ing months to bring satral­izum­ab to peo­ple liv­ing with NMOSD as soon as pos­si­ble.”

The dis­or­der is of­ten con­fused with mul­ti­ple scle­ro­sis as the im­mune sys­tem at­tacks its host, dam­ag­ing healthy tis­sue in the spine and eyes. Al­though the ma­jor­i­ty of NMOSD pa­tients are well man­aged on Rit­ux­an, there are still about a third of pa­tients who re­lapse and are at risk for se­vere dis­abil­i­ties and even death, if left un­treat­ed. Satral­izum­ab in­hibits IL-6 sig­nal­ing, which is be­lieved to play a key role in the in­flam­ma­tion that oc­curs in peo­ple with NMOSD.

Da­ta from SAkuraS­tar adds to the re­sults of the SAkuraSky study, which test­ed satral­izum­ab in com­bi­na­tion with base­line ther­a­py for peo­ple with NMOSD. The da­ta showed a 62% re­duc­tion in the risk of re­laps­es when used in com­bi­na­tion with base­line ther­a­py com­pared to place­bo (p=0.0184).  The drug al­so con­ferred a 79% re­duc­tion in the risk of re­laps­es in AQP4-IgG seropos­i­tive pa­tients (p=0.0086).

In a note pub­lished fol­low­ing the Alex­ion’s Soliris ap­proval in NMOSD, SVB Leerink’s Ge­of­frey Porges sug­gest­ed that while there is no head-to-head com­par­i­son be­tween Soliris and Roche’s drug, Soliris ap­pears to have an edge on ef­fi­ca­cy in re­lapsed pa­tients.

Soliris was ap­proved on the ba­sis of the PRE­VENT study which test­ed the drug in an­ti-AQP4 an­ti­body-pos­i­tive pa­tients. Da­ta showed 98% of Soliris-treat­ed pa­tients were re­lapse-free ver­sus 63% on place­bo, at 48 weeks.

Porges fore­cast Soliris sales in NMOSD reach­ing about $700 mil­lion by 2024.

Viela’s CD-19 in­hibitor, inebi­lizum­ab, al­so re­cent­ly re­port­ed pos­i­tive da­ta from its piv­otal NMOSD study.

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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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.

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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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.