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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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In at least one life-sci hub, gen­der and di­ver­si­ty ini­tia­tives haven’t made a dent

Gender and racial diversity at the top of UK life science companies has hardly budged over the last seven years despite repeated advocacy efforts, according to a new report.

The report, from the recruiting firm Liftstream, found that 14.8% of directors on life sciences boards were women and 21.1% of top executives were women. That’s a modest bump from the 9.8% of directors and 18.1% of executives Liftstream identified in their last report from 2014. The percentage of women CEOs moved from 8% to 9.8%.

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Will a 'risk-of­f' mind­set has­ten cell ther­a­py M&A? Io­vance surges on buy­out chat­ter

Is it time for some cell therapy M&A?

Investors of Iovance Biotherapeutics certainly thought so, sending its stock $IOVA up as much as 40% after Bloomberg reported that the cancer-focused biotech is talking to potential buyers.

While 2019 saw a number of high-profile gene therapy company takeovers — led by Roche’s $4.3 billion bid of Spark as Astellas went for Audentes, Biogen snapped up Nightstar and Vertex absorbed Exonics — large players appeared to prefer partnering on the cell therapy front, particularly when it comes to cancer. Hal Barron put his weight behind Rick Klausner’s startup as he rebuilt GlaxoSmithKline’s cancer pipeline. Takeda turned to MD Anderson to license their natural killer cell therapy.

One less ri­val for Im­muno­vant, as Alex­ion aban­dons FcRn in­hibitor

Less than one year after Alexion parted with $25 million upfront to secure access to a second anti-FcRn asset, it is abandoning the experimental drug. The discontinuation, disclosed at the SVB Leerink Global Healthcare Conference in New York during a fireside chat, bodes well for rival Immunovant.

The drug (ABY-039), partnered for development with Sweden’s Affibody, was forsaken on the basis of early-stage data that was not viewed favorably, Baird and SVB Leerink analysts noted.

Clin­i­cal tri­al spon­sors have to dis­close decade’s worth of un­re­leased da­ta, fed­er­al judge rules

A decade’s worth of unreleased trial data may soon see the light of day.

A New York federal judge ruled this week that the FDA and the NIH have for years misinterpreted a law that would require companies, universities and other clinical trial sponsors to release trial data from studies completed between 2007 and 2017. The ruling covers drugs and medical devices that were experimental when the study was completed but have since been approved, potentially putting hundreds of sponsors out of compliance if they don’t put their results on clinicaltrials.gov.

Laurie Glimcher and Ansbert Gadicke (Justin Knight, Dana-Farber Cancer Institute)

Ty­ing ba­sic sci­ence to spin­outs, Dana-Far­ber de­buts sis­ter funds to­tal­ing $126M with MPM Cap­i­tal

As one of the most prestigious cancer institutes in the US, Dana-Farber has enjoyed considerable support for its entrepreneurial pursuits, spinning out about 30 companies in the past 12 years.

“Now where we’ve always struggled — where every cancer center struggled — is support of basic science,” Barrett Rollins, chief scientific officer emeritus, told Endpoints News.

And then two of its trustees had an idea. What if they tied philanthropy to investment in Dana-Farber startups, requiring a donation to basic science as a condition for accessing its brightest biotech venture ideas?