Matthias Schroff, Exicure CEO

In sur­vival mode, Ex­i­cure cuts to the bone — paus­ing all R&D and chop­ping head­count by 66%

The dras­tic re­struc­tur­ing that Ex­i­cure un­der­took at the end of last year couldn’t save the biotech af­ter all.

In fact, Ex­i­cure — whose take on gene ex­pres­sion once drew the back­ing of Bill Gates and a slate of tech in­vestors — is go­ing for an­oth­er round of lay­offs, this time cut­ting to the bone with 66% of the re­main­ing staffers be­ing let go, while halt­ing all R&D ac­tiv­i­ties and look­ing to di­vest its en­tire pipeline.

To help keep the lights on longer, an ex­ist­ing in­vestor — CBI USA — al­so agreed to buy more shares for $5.4 mil­lion. As a re­sult, it now com­mands a con­trol­ling stake of a com­pa­ny whose mar­ket cap sits at un­der $9 mil­lion.

Ex­i­cure sug­gest­ed it was in deep trou­ble when it part­ed ways with found­ing CEO David Giljo­hann and chopped half of its work­force. At the time, it mapped out a plan to wind down two pro­grams — the im­muno-on­col­o­gy as­set cavro­tolimod and an­oth­er pre­clin­i­cal drug for Friedre­ich’s atax­ia — and fo­cus its re­sources on a pre­clin­i­cal pro­gram tar­get­ing SCN9A for neu­ro­path­ic pain as well as part­ner­ships with Ipsen and Ab­b­Vie. That left the com­pa­ny with 31 em­ploy­ees.

Both deals were an­chored on Ex­i­cure’s spe­cial oligonu­cleotides, or syn­thet­ic struc­tures of nu­cle­ic acids that can be used to mod­u­late gene ex­pres­sion.

But in the lat­est up­date, the com­pa­ny said it will al­so sus­pend all part­nered pro­grams and ex­plore di­vesti­ture of the SCN9A prod­uct can­di­dates, which tar­get the Nav1.7 chan­nel, for neu­ro­path­ic pain.

While those com­pounds had promis­ing ac­tiv­i­ty in pre­clin­i­cal stud­ies and spurred a sig­nif­i­cant lev­el of knock­down of the SCN9A mR­NA tran­script, Ex­i­cure said the ef­fect didn’t pan out in lat­er stud­ies.

Re­sults from a re­cent in vi­vo an­i­mal study in non-hu­man pri­mates did not meet de­sired tar­get en­gage­ment lev­els as ob­served in pre­vi­ous in vit­ro pre­clin­i­cal stud­ies. Ad­di­tion­al pre­clin­i­cal stud­ies would be re­quired to un­der­stand re­cent find­ings, like­ly de­lay­ing the tim­ing of IND-en­abling work. As a re­sult, Ex­i­cure has sus­pend­ed fur­ther pre­clin­i­cal ac­tiv­i­ties for the SCN9A pro­gram as it as­sess­es strate­gic al­ter­na­tives for all its as­sets, in­clud­ing its plat­form tech­nol­o­gy, with the goal of max­i­miz­ing stock­hold­er val­ue.

The ex­ec­u­tive team is ex­pect­ed to re­main in place fol­low­ing the re­org, Ex­i­cure added.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

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:

Lil­ly's Covid-19 mAb no longer au­tho­rized due to Omi­cron sub­vari­ants, FDA says

The FDA on Wednesday announced that Eli Lilly’s Covid-19 drug bebtelovimab is no longer authorized to treat Covid-19 because of the rising numbers of two new subvariants that the drug does not work against.

The Centers for Disease Control and Prevention last week published new estimates that the combined proportion of Covid-19 cases caused by the Omicron subvariants BQ.1 and BQ.1.1 are greater than 57% nationally, and already above 50% in all individual regions but one.

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.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,800+ biopharma pros reading Endpoints daily — and it's free.

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.