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

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.