Let the joust­ing be­gin: Which top bio­phar­ma com­pa­nies are look­ing to make a splash at ASH?

Any­one who fol­lows on­col­o­gy knows the field has at­tract­ed so much at­ten­tion and re­search mon­ey that it sparks a con­tin­u­al erup­tion of bio­phar­ma news as com­pa­nies joust over block­buster mar­kets. That’s kept me busy this year with a string of glob­al con­fabs — as AS­CO be­comes just one big event in a fren­zied com­pe­ti­tion.

John Car­roll

So now I’ll be at­tend­ing ASH in At­lanta in ear­ly De­cem­ber for the first time. What’s on my radar? I turned to Leerink to­day for some sug­ges­tions, and boiled them down here.

Ap­provals for No­var­tis $NVS and Gilead/Kite $GILD for their first CAR-T ap­pli­ca­tions are a his­toric first step, but this tech­nol­o­gy is just now be­gin­ning to write its first com­mer­cial chap­ter. Juno $JUNO is now back af­ter get­ting de­railed with the dis­as­ter that JCAR015 be­came. And in fig­ur­ing out what went wrong there, they are promis­ing to come up with the next big thing in CAR-T: JCAR017. The ther­a­py has pro­duced some jaw-drop­ping ef­fi­ca­cy re­sults and a much im­proved safe­ty pro­file, and now we all want to see every up­date to de­ter­mine if Juno can leapfrog the lead­ers.

No­var­tis, the big phar­ma com­pa­ny that fig­ured out how to work like a biotech on this one, will be of­fer­ing some im­por­tant de­tails on Kym­ri­ah for DL­B­CL, where it’s look­ing to go head-to-head with Gilead’s Yescar­ta. So the up­date on that front from the Gilead/Kite team will earn a close in­spec­tion.

Then there’s Pfiz­er $PFE, an­oth­er of the Big Phar­ma play­ers that has made on­col­o­gy a top pri­or­i­ty. The gi­ant bagged rights to Cel­lec­tis’ $CLLS UCART19 in ALL, and a pre­sen­ta­tion in At­lanta will be close­ly fol­lowed. Cel­lec­tis’ re­cent clin­i­cal hold for its own off-the-shelf ap­proach to CAR-T — an­oth­er po­ten­tial leapfrog ther­a­py — will keep the Paris-based biotech in the in­dus­try’s eye.

Blue­bird bio $BLUE is seem­ing­ly al­ways at ASH and al­ways has some­thing in­ter­est­ing to say. In this case it’s the “Lenti­Glo­bin gene ther­a­py pro­gram in trans­fu­sion-de­pen­dent be­ta-tha­lassemia and se­vere sick­le cell dis­ease, as well as up­dat­ed re­sults of the bb2121 an­ti- BC­MA CAR-T Phase I study in pa­tients with re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma,” to quote Leerink.

Blue­bird, by the way, just an­nounced that it has dosed the first pa­tient in its North­star-3 (HGB-212) tri­al, a Phase III study of Lenti­Glo­bin.

Cel­gene $CELG has had its ups and downs this year, with some stun­ning and un­char­ac­ter­is­tic set­backs that have trig­gered a small rout on their stock, ding­ing a large swath of big biotechs in the process. A good show­ing in At­lanta would go a long way to earn­ing back some of the re­spect it’s long en­joyed. Leerink’s as­sess­ment of what to watch for on their close­ly watched ap­pear­ance at ASH:

(O)ral pre­sen­ta­tions from the bb2121 Phase I study in mul­ti­ple myelo­ma, and from the phase I TRAN­SCEND study of JCAR017 in NHL. CELG pre­pares to ini­ti­ate a piv­otal Phase III tri­al with bb2121 in myelo­ma in the com­ing months. CELG al­so will re­port sev­er­al sets of Phase I da­ta from next-gen CelMod CC-122 in NHL in­di­ca­tions.

I’m al­so pluck­ing Alex­ion $ALXN out of the mix. Their next-gen PNH drug is about the on­ly im­por­tant pro­gram they have in the pipeline, for now, and the ear­ly-stage snap­shot on ALXN1210 will get some at­ten­tion as well.

This next part is web on­ly. I’m round­ing out my list now with sug­ges­tions com­ing in over e-mail along with some sug­ges­tions via an­a­lysts re­marks. 

Spark $ONCE has one of those land­mark drugs we all like to watch in Lux­tur­na, and an­a­lysts at Jef­feries say they’ll be on hand to make sure that the biotech demon­strates con­sis­tent­ly pos­i­tive da­ta on the lead­ing drug in the gene ther­a­py field. “At ASH, we ex­pect SPK-8011 to show gen­er­al con­sis­ten­cy of 11-14% and 20-25%, with the third co­hort ide­al­ly show­ing 50-100% high­er lev­els than the pri­or co­hort,” says an up­beat Michael Yee.

I al­so plan to talk to Ax­el Hoos at GSK about their ear­ly-stage BC­MA-tar­get­ing ther­a­py, an an­ti­body drug con­ju­gate dubbed GSK2857916. Just a few days ago GSK flagged the FDA’s break­through drug des­ig­na­tion, which will like­ly keep it on fast-track de­vel­op­ment. And the phar­ma gi­ant is like­ly to use this pro­gram to high­light a re­turn to late-stage de­vel­op­ment in on­col­o­gy.

Al­so adding Re­gen­eron, which has Phase I tri­als of their an­ti-PD-1 cemi­plimab and CD20xCD3 bis­pe­cif­ic REGN1979 in b-cell lym­phomas, specif­i­cal­ly B-cell non-Hodgkin lym­phoma.

There’s al­so Gami­da Cell, which will have the full Phase II da­ta avail­able on NiCord, now in late-stage de­vel­op­ment.

That’s it for now. These are on­ly the high­lights I’m ze­ro­ing in on. But if you have sug­ges­tions for any­thing im­por­tant I missed, let me know. You can email me at john@end­pointsnews.com.


Im­age: The Geor­gia World Con­gress Cen­ter in At­lanta Shut­ter­stock

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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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.

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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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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.