As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

Af­ter fum­bling in its first late-stage lu­pus study, As­traZeneca dis­closed that a sec­ond piv­otal tri­al test­ing its ex­per­i­men­tal drug, an­i­frol­um­ab, had met the main goal, in Au­gust. Ear­li­er this week, the British drug­mak­er broke out the num­bers from its suc­cess­ful study.

Last year, an­i­frol­um­ab failed to meet the main goal of di­min­ish­ing dis­ease ac­tiv­i­ty in the 460-pa­tient TULIP I study, a 52-week tri­al that test­ed two dos­es of the drug ver­sus a place­bo. But in the 373-pa­tient TULIP II study, the high­er dose (300 mg) was com­pared to pa­tients giv­en a place­bo — pa­tients in both arms were on base­line stan­dard care.

An­i­frol­um­ab in­duced a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment with 47.8% of pa­tients re­spond­ing to the drug, ver­sus 31.5% of pa­tients on place­bo at week 52, as mea­sured by the British Isles Lu­pus As­sess­ment Group–based Com­pos­ite Lu­pus As­sess­ment (BI­CLA) com­pos­ite mea­sure — which re­quires im­prove­ment in all or­gans with dis­ease ac­tiv­i­ty at base­line with no new flares.

The As­traZeneca drug al­so proved sta­tis­ti­cal­ly su­pe­ri­or on a raft of sec­ondary end­points, such as its im­pact on oral cor­ti­cos­teroid (OCS) use and skin man­i­fes­ta­tions. 51.5% of pa­tients tak­ing OCS achieved a sus­tained re­duc­tion in OCS use, ver­sus 30.2% of pa­tients on place­bo; and 49% of pa­tients re­ceiv­ing an­i­frol­um­ab with mod­er­ate-to-se­vere skin dis­ease saw im­prove­ments at week 12, com­pared with 25% of pa­tients re­ceiv­ing place­bo.

Lu­pus is a drug de­vel­op­er’s night­mare. In the last six decades, there has been one FDA ap­proval. In re­cent years, the field has re­sem­bled a grave­yard. Last Oc­to­ber, UCB and Bio­gen’s an­ti-CD40L drug failed in a late-stage study, months af­ter Xen­cor and Sanofi’s Abl­ynx al­so con­ced­ed de­feat in their pro­grams.

Mean­while, there is cause for some cau­tious op­ti­mism, with a cadre of drug de­vel­op­ers work­ing on de­vel­op­ing new treat­ments. Some bi­o­log­ics that are ap­proved for oth­er au­toim­mune dis­eases are be­ing test­ed for use in lu­pus — in­clud­ing Eli Lil­ly’s Olu­mi­ant and J&J’s Ste­lara. French biotech Neo­vacs is al­so in mid-stage de­vel­op­ment with a lu­pus vac­cine.

The on­ly bi­o­log­ic so far to win ap­proval for lu­pus is GSK’s Benlysta — which was cleared for adult use in 2011 and for rare cas­es of child­hood lu­pus this year. (GSK is in the midst of test­ing Benlysta in com­bi­na­tion with Roche’s rit­ux­imab in the hope the com­bi­na­tion will have a more po­tent ef­fect on the dis­ease ver­sus Benlysta monother­a­py.)

Apart from that, pa­tients are usu­al­ly giv­en NSAIDS, an­ti­malar­i­al drugs, cor­ti­cos­teroids, and im­muno­sup­pres­sants to con­trol the symp­toms of the sys­temic au­toim­mune dis­ease, in which the body’s im­mune sys­tem launch­es an at­tack on its own tis­sues and or­gans. About 1.5 mil­lion Amer­i­cans and at least five mil­lion peo­ple glob­al­ly suf­fer from a form of lu­pus, es­ti­mates The Lu­pus Foun­da­tion of Amer­i­ca.

An­i­frol­um­ab, which is al­so be­ing eval­u­at­ed in a mid-stage tri­al in lu­pus nephri­tis, is a mon­o­clon­al an­ti­body en­gi­neered to thwart the ac­tiv­i­ty of all type I in­ter­fer­ons — cy­tokines in­volved in in­flam­ma­to­ry path­ways. Rough­ly 60% to 80% of adults with sys­temic lu­pus ery­the­mato­sus (SLE) car­ry in­creased type I in­ter­fer­on gene sig­na­ture, ac­cord­ing to As­traZeneca.

De­spite the failed TULIP 1 tri­al, As­traZeneca’s Mene Pan­ga­los in Au­gust said the com­pa­ny is ex­plor­ing path­ways to get an­i­frol­um­ab on the mar­ket. “There is now a strong body of ev­i­dence demon­strat­ing the ben­e­fit of an­i­frol­um­ab, and we look for­ward to bring­ing this po­ten­tial new med­i­cine to pa­tients with sys­temic lu­pus ery­the­mato­sus as soon as pos­si­ble,” he said in a state­ment on Mon­day.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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