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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Sanofi and GSK reach deal with Cana­da for 72 mil­lion vac­cine dos­es

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to announce their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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