In a bat­tered field, As­traZeneca's lu­pus drug clears piv­otal study — but can they get it ap­proved now?

A year af­ter As­traZeneca ad­mit­ted de­feat in a late-stage study test­ing its lu­pus drug an­i­frol­um­ab, the British drug­mak­er has cleared their sec­ond piv­otal tri­al, hit­ting the pri­ma­ry end­point and leav­ing their re­search team hope­ful that they have enough da­ta in hand to get an ap­proval.

Last year, an­i­frol­um­ab failed to meet the main goal of di­min­ish­ing dis­ease ac­tiv­i­ty in the 460-pa­tient TULIP 1 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 — and the study met the main goal of re­duc­ing dis­ease sever­i­ty.

Both tri­als mea­sured dis­ease ac­tiv­i­ty us­ing the British Isles Lu­pus As­sess­ment Group based Com­pos­ite Lu­pus As­sess­ment (BI­CLA) — which re­quires im­prove­ment in all or­gans with dis­ease ac­tiv­i­ty at base­line with no new flares — at week 52. In typ­i­cal fash­ion, the com­pa­ny is hold­ing the da­ta back for a sci­en­tif­ic con­fer­ence.

Reg­u­la­tors, though, won’t have to wait.

Usu­al­ly, a drug de­vel­op­er needs pos­i­tive da­ta from two piv­otal tri­als to se­cure ap­proval, but reg­u­la­tors have proved in­creas­ing­ly like­ly to over­look that stan­dard — par­tic­u­lar­ly if they’re up against a big chal­lenge like lu­pus. As­traZeneca’s Mene Pan­ga­los is look­ing to cap­i­tal­ize on that, in a state­ment on Thurs­day he said the com­pa­ny is ex­plor­ing path­ways to get an­i­frol­um­ab on the mar­ket.

The drug 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 $AZN.

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 $BI­IB an­ti-CD40L drug failed in a late-stage study, months af­ter Xen­cor $XN­CR and Sanofi’s $SNY Abl­ynx al­so con­ced­ed de­feat in their pro­grams.

Mean­while, there is cause for some cau­tious op­ti­mism. Some bi­o­log­ics that are ap­proved for oth­er au­toim­mune dis­ease are be­ing test­ed for use in lu­pus — in­clud­ing Eli Lil­ly’s $LLY Olu­mi­ant and J&J’s $JNJ 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 $GSK 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.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Pro­tect­ing its megablock­buster, Janssen chal­lenges Am­gen's Ste­lara biosim­i­lar ahead of planned 2023 launch

Johnson & Johnson unit Janssen on Wednesday sued Amgen over the company’s proposed biosimilar to its megablockbuster Stelara (ustekinumab), after Amgen said it was ready to launch next May or as soon as the FDA signs off on it.

If Amgen carries through with that plan, Janssen told the Delaware district court that the Thousand Oaks, CA-based company will infringe on at least two Janssen patents.

Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Digital render of CPI's Medicines Manufacturing Innovation Centre in Glasgow, Scotland (Image:

CPI opens the doors to a new $100M+ man­u­fac­tur­ing fa­cil­i­ty in Scot­land

A manufacturing site that has received interest and investments from large pharma companies and the UK government is opening its doors in Scotland.

The manufacturer CPI (Centre for Process Innovation) has opened a new £88 million ($105 million) “Medicines Manufacturing Innovation Centre” in Glasgow, Scotland, to accelerate the development of manufacturing tech and solve longstanding challenges in medicine development and manufacturing.

Lex­i­con slams FDA over hear­ing de­nial fol­low­ing a CRL for its SGLT2 in­hibitor can­di­date

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.

Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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