8 stars: A look back at AS­CO spot­lights the top can­cer drugs to watch, and 1 to for­get

I’ve been mak­ing the trek to AS­CO for some years now, and I don’t re­call any oth­er week that ri­vals what I’ve just seen. There wasn’t just im­por­tant da­ta to re­port on, there was a lot of it.

CAR-T, with all of its splen­did ini­tial promise in liq­uid tu­mors, is reach­ing the first ma­jor com­mer­cial fin­ish line, with No­var­tis’ $NVS CTL019 out in front of Kite’s $KITE KTE-C19 by a nose. No­var­tis wait­ed to cap­ture the in­dus­try’s at­ten­tion at an­oth­er con­fer­ence mid-week with head-to-head da­ta, but you bet­ter be­lieve it fig­ured promi­nent­ly on my AS­CO in­ter­view sched­ule.

Juno $JUNO, knocked out of the first race, is mak­ing a strong come­back bid with JCAR017, which will like­ly fig­ure promi­nent­ly in the sec­ond wave ther­a­pies to get a close ex­am­i­na­tion in a piv­otal tri­al.

But what about BC­MA? Blue­bird $BLUE tru­ly wowed the field with da­ta from its close­ly-watched Cel­gene-part­nered pro­gram for bb2121 in treat­ing mul­ti­ple myelo­ma. But in this field the po­ten­tial for sur­prise un­der­lies every twist and turn of the im­muno-on­col­o­gy field. And so we find an ob­scure Chi­nese com­pa­ny called Nan­jing Leg­end — “great name, isn’t it?” said chief blue­bird Nick Leschly with a smile as we com­pared BC­MA da­ta — get­ting shoved in­to AS­CO’s mar­quee cir­cle with even bet­ter da­ta from a larg­er group.

So now we have a new drug to track.

I’ll ad­mit, Loxo ex­ecs have a weary­ing habit of tout­ing every tiny bit of new da­ta on pos­i­tive out­comes, but it $LOXO too is look­ing to make can­cer drug his­to­ry by go­ing af­ter a dual adult/pe­di­atric ap­proval based on a bio­mark­er, fol­low­ing close­ly on a ge­net­ic break­through by Mer­ck $MRK.

One sto­ry that didn’t get much trac­tion over­all, but should have, be­longed to Re­gen­eron $REGN and its Big Phar­ma part­ner Sanofi, which are mov­ing ahead with a next-gen PD-1 check­point, REGN2810. Re­gen­eron has one of the best an­ti­body de­vel­op­ment shops in the world. And if its re­searchers are right, they’ve gleaned new in­sights on the field that could have a pro­found af­fect on how these drugs are made and used. If you missed it, I’d en­cour­age you to go back for an­oth­er look.

In­cyte’s new up­dat­ed da­ta $IN­CY on epaca­dos­tat were al­so gone over with a fine tooth comb. There have been some re­cent jit­ters in the IDO1 field, but In­cyte re­mains out front as the big PD-(L)1 Phase III tri­als with Mer­ck and Bris­tol-My­ers Squibb get un­der­way.

There have been a few IDO jit­ters ev­i­dent, though, es­pe­cial­ly af­ter Roche’s team held their noses while dis­cussing their IDO1 da­ta for GDC-0919 (navox­i­mod), in-li­censed from NewLink for $150 mil­lion up front.

In­de­pen­dent can­cer drug in­vestor Brad Lon­car was on hand for the Roche pre­sen­ta­tion at AS­CO, and not­ed a de­tectable sound of skep­ti­cism.

A few days lat­er, Roche’s Genen­tech kicked it out of the pipeline. For NewLink $NLNK, which has ex­pe­ri­enced a string of pun­ish­ing set­backs, it was an­oth­er cau­tion­ary tale to re­mind ob­servers that not every­one is suc­ceed­ing in the boom­ing on­col­o­gy busi­ness. But the stand­outs mark po­ten­tial­ly new stan­dards of ther­a­py in on­col­o­gy. And the progress is gath­er­ing speed.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.