As­traZeneca spin­off scores maid­en FDA ap­proval, where it will field Alex­ion com­pe­ti­tion and an im­mi­nent Roche ri­val

About a year af­ter the first-ever FDA ap­proval for pa­tients with neu­romyelitis op­ti­ca spec­trum dis­or­der (NMOSD), the agency has ap­proved a sec­ond ther­a­py for the rare au­toim­mune con­di­tion.

On Thurs­day, As­traZeneca spin­off Viela Bio scored its maid­en ap­proval for its lead drug, Up­liz­na, to treat NMOSD pa­tients. It will com­pete with Alex­ion’s en­trenched key­stone ther­a­py Soliris — a mon­o­clon­al an­ti­body that tar­gets com­ple­ment pro­tein C5 — which se­cured its NMOSD ap­proval last June.

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. NMOSD can be as­so­ci­at­ed with an­ti­bod­ies that bind to a pro­tein called aqua­por­in-4 (AQP4) — bind­ing of these an­ti­bod­ies ap­pears to ac­ti­vate oth­er com­po­nents of the im­mune sys­tem, caus­ing in­flam­ma­tion and dam­age to the cen­tral ner­vous sys­tem.

Up­liz­na, known chem­i­cal­ly as inebi­lizum­ab, is an in­tra­venous an­ti-CD19 drug. Its ap­proval was based on a 230-pa­tient study, in which 213 pa­tients had an­ti­bod­ies against AQP4. The risk of re­lapse in the 161 an­ti-AQP4 an­ti­body-pos­i­tive pa­tients who were treat­ed with Up­liz­na was di­min­ished by 77% ver­sus place­bo. In ad­di­tion, 89% of pa­tients in the AQP4 an­ti­body-pos­i­tive group re­mained re­lapse-free dur­ing the six-month pe­ri­od post-treat­ment com­pared to 58% of place­bo sub­jects.

There was no ev­i­dence of a ben­e­fit in pa­tients who were an­ti-AQP4 an­ti­body neg­a­tive.

The ap­proval is large­ly in line with base case ex­pec­ta­tions — the drug has got no black box warn­ing and the ap­proval is for AQP4+ pa­tients on­ly, not­ed Stifel’s Derek Archi­la, an­tic­i­pat­ing the drug will gen­er­ate about half a bil­lion dol­lars in US sales at peak.

Viela’s shares $VIE jumped more than 6.5% to $54.82 in Fri­day pre­mar­ket trad­ing.

Soliris’ ap­proval, which came with a black box warn­ing, was based on a study that test­ed the drug in an­ti-AQP4 an­ti­body-pos­i­tive pa­tients. Da­ta showed 98% of Soliris-treat­ed pa­tients were re­lapse-free ver­sus 63% on place­bo. SVB Leerink’s Ge­of­frey Porges has fore­cast Soliris sales in NMOSD reach­ing about $700 mil­lion by 2024.

With­in the NMOSD realm is an­oth­er ri­val wait­ing in the wings: Roche and Chugai’s satral­izum­ab.  The drug in­hibits IL-6 sig­nal­ing, which is be­lieved to play a key role in the in­flam­ma­tion that oc­curs in peo­ple with NMOSD.

Da­ta from a late-stage study in the sub­group of pa­tients that are AQP4-IgG seropos­i­tive — who tend to ex­pe­ri­ence a more se­vere dis­ease course — saw a 74% re­duc­tion in the risk of re­laps­es in the satral­izum­ab group. About 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. A mar­ket­ing ap­pli­ca­tion for the ther­a­py is cur­rent­ly un­der FDA re­view.

Viela ex­ec­u­tives have un­der­scored that the fact Up­liz­na does not car­ry a black box warn­ing should help it achieve first line sta­tus, and that the la­bel re­flects the drug’s abil­i­ty to re­duce hos­pi­tal­iza­tion rates could prove to be a dif­fer­en­tia­tor against oth­er agents, no­tably satral­izum­ab, Stifel’s Archi­la not­ed.

“While we agree with the lat­ter, we do be­lieve the ex­ten­sive ex­pe­ri­ence physi­cians have with rit­ux­imab in NMOSD and rit­ux­imab’s large safe­ty data­base from its many years on the mar­ket will like­ly be a chal­lenge for first-line us­age of Up­liz­na,” Archi­la said.

While Viela has not dis­closed Up­liz­na’s list price so far, they have gen­er­al­ly laid out a range of $200,000-$280,000/year as fea­si­ble. That range is still far be­low Soliris, which at a stag­ger­ing cost of more than $500,000 per year is one of the most ex­pen­sive drugs in the world.

“Our es­ti­mate for the an­nu­al cost of treat­ment for NMOSD with biosim­i­lar or brand­ed rit­ux­imab is around $33,000-$37,000 and is con­sid­er­ably less than Viela’s pro­posed pric­ing for Up­liz­na, which could cre­ate an­oth­er chal­lenge for adop­tion,” Archi­la said.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

President Joe Biden signs the Democrats' landmark climate change and health care bill. From L-R: Sen. Joe Manchin (D-WV), Senate Majority Leader Chuck Schumer (D-NY), House Majority Whip James Clyburn (D-SC), Rep. Frank Pallone (D-NJ) and Rep. Kathy Castor (D-FL). (Susan Walsh/AP Images)

Pres­i­dent Biden signs ma­jor drug pric­ing re­forms in­to law: What's com­ing for bio­phar­ma?

President Joe Biden yesterday afternoon signed into law historic, decades-in-the-making new drug pricing reforms as part of a wider reconciliation bill that will likely take a chunk out of biopharma companies’ profits for some blockbusters just prior to generic or biosimilar competition.

The partisan bill (all Democrats in the House and Senate voted for it, and all Republicans voted against it) includes not only Medicare price negotiations — which won’t kick off until 2026, leaving ample time for a legal challenge — but mandatory inflation-related rebates, and a $2,000 annual cap on what seniors’ pay for their prescription drugs.

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