UP­DAT­ED: No­var­tis man­u­fac­tur­ing fa­cil­i­ty in North Car­oli­na gets FDA all-clear to pro­duce, ship gene ther­a­pies

Just one day af­ter Vas Narasimhan an­nounced $1 bil­lion in cuts amid a com­pa­ny-wide re­struc­tur­ing, the Amer­i­can-Swiss phar­ma re­ceived FDA clear­ance to pro­duce its spinal mus­cu­lar at­ro­phy drug Zol­gens­ma out of its North Car­oli­na man­u­fac­tur­ing fa­cil­i­ty.

The phar­ma made the an­nounce­ment ear­li­er Tues­day, which al­lows the mas­sive, 170,000-square-foot fa­cil­i­ty to make the drug — along with test­ing and ship­ping it. The com­mer­cial li­cen­sure is not just lim­it­ed to Zol­gens­ma, ac­cord­ing to No­var­tis — the plant will al­so have the abil­i­ty to pro­duce cer­tain gene ther­a­py prod­ucts for use in clin­i­cal tri­als.

“Not on­ly will this fa­cil­i­ty sup­port the No­var­tis pipeline through the man­u­fac­ture of both clin­i­cal tri­al and com­mer­cial prod­ucts, it ul­ti­mate­ly al­lows us to help more pa­tients and fam­i­lies liv­ing with rare, ge­net­ic dis­eases,” Chris­tine Fox, pres­i­dent of No­var­tis gene ther­a­pies, said in a state­ment.

The fa­cil­i­ty, lo­cat­ed in the heart of the Re­search Tri­an­gle Park in Durham, North Car­oli­na, will be the sec­ond site to pro­duce Zol­gens­ma — with the first one in Lib­er­tyville, IL, ap­proved in 2019. The North Car­oli­na fa­cil­i­ty would have been No­var­tis’ third man­u­fac­tur­ing plant for Zol­gens­ma, but the phar­ma walked away from its planned Long­mont, CO, plant last year in the face of de­clin­ing sales.

This is the lat­est de­vel­op­ment sur­round­ing the gene ther­a­py de­signed for SMA, and which was at the cen­ter of scan­dal more than two years ago af­ter safe­ty con­cerns were brought up and No­var­tis’s gene ther­a­py di­vi­sion AveX­is in­formed the FDA about a da­ta ma­nip­u­la­tion is­sue that didn’t end up af­fect­ing its ap­proval. Sev­er­al AveX­is em­ploy­ees, in­clud­ing two of its top sci­en­tists, were fired in the af­ter­math.

No­var­tis cur­rent­ly has more than 20 gene ther­a­pies in its AAV-based pipeline, and while it was ad­vanc­ing ther­a­pies for Rett syn­drome and Friedre­ich’s atax­ia, for ex­am­ple — those pro­grams have been dumped, the com­pa­ny said at an R&D event in De­cem­ber.

Ed­i­tor’s note: This ar­ti­cle has been up­dat­ed to note that No­var­tis now has more than 20 gene ther­a­pies in its AAV pipeline, and the two pro­grams pre­vi­ous­ly men­tioned were dropped four months ago.

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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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: uk-cpi.com)

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.

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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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:

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.

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.