AS­CO17: As­traZeneca pumps up ex­pec­ta­tions on near-term can­cer drug wins, look­ing to shed a rep for fail­ure

CHICA­GO — As­traZeneca’s R&D team ar­rived at AS­CO on a crit­i­cal mis­sion.

With its lead­ing PARP in­hibitor outscor­ing chemo in treat­ing breast can­cer,  mak­ing the spot­light round on Sun­day, As­traZeneca was able to of­fer some fresh, sol­id ev­i­dence of its con­tin­u­ing progress on the all-im­por­tant can­cer front. And with sev­er­al key cat­a­lysts lined up over the sum­mer, ex­ecs want­ed to send a clear mes­sage that this can­cer drug roll they’re on is just be­gin­ning.

As far as Leerink’s Sea­mus Fer­nan­dez is con­cerned, As­traZeneca’s in­vestor meet­ing in Chica­go was a rous­ing suc­cess, with some sug­ges­tions that the phar­ma gi­ant is on the verge of of­fer­ing some ‘prac­tice chang­ing’ ad­vances in the field.

Sean Bo­hen, As­traZeneca

As­traZeneca ex­ecs left the crowd with the clear im­pres­sion that Lyn­parza was on its way to a broad ap­proval for sec­ond-line main­te­nance ther­a­py in treat­ing ovar­i­an can­cer. R&D chief Sean Bo­hen al­so hint­ed that the full read­out on the PA­CIF­IC tri­al — Imfinzi monother­a­py fol­low­ing chemo and ra­di­a­tion in Stage 3 non-small cell lung can­cer — will be a hit. Fer­nan­dez not­ed:

Based on this and com­ments from pre­vi­ous­ly in­ter­viewed KOLs, we be­lieve the re­sults are like­ly to be both clin­i­cal­ly mean­ing­ful and prac­tice-chang­ing. AZN es­ti­mates a Stage III NSCLC in­ci­dence of ~100k per year, with about half be­ing un­re­sectable. With oth­er PD-1/L1 in­hibitors ~2-3 years be­hind in the set­ting, this rep­re­sents a large mar­ket-ex­pand­ing op­por­tu­ni­ty for Imfinzi.

Fer­nan­dez be­lieves that the phar­ma gi­ant has al­ready filed its BTK drug acal­abru­ti­nib for an ap­proval at the FDA and is just wait­ing for con­fir­ma­tion that the pack­age has been ac­cept­ed for re­view. Peak sales es­ti­mates for this drug scrape the $1 bil­lion mark— pos­si­bly mak­ing it one of the top 10 or­phan drugs in the late-stage pipeline — which would make it a valu­able ad­di­tion to the port­fo­lio.

And then there’s MYS­TIC, As­traZeneca’s com­bo of its check­point Imfinzi (dur­val­um­ab) and treme­li­mum­ab, a CT­LA-4 drug. Over the past few months there’s been a grow­ing sense that this com­bi­na­tion could well floun­der on CT­LA-4’s well-known tox­i­c­i­ty. But As­traZeneca’s team is keep­ing their smiles in place, try­ing to as­sure every­one that this pro­gram re­mains on track, with plen­ty of po­ten­tial to im­press.

The da­ta are ex­pect­ed in a mat­ter of weeks now, so this mys­tery won’t play long. A suc­cess here would make a huge dif­fer­ence for the multi­na­tion­al, which has been dogged by clin­i­cal set­backs for years now. Some as­sur­ance that CEO Pas­cal So­ri­ot’s big bet on can­cer is pay­ing off with a multi­bil­lion-dol­lar re­turn would go a long way to as­sur­ing an­a­lysts that the long-await­ed turn­around is near.

Fail­ure would like­ly blight the process once again.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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The fu­ture of mR­NA, J&J's vac­cine ad­comm, Mer­ck­'s $1.85B au­toim­mune bet and more

Welcome to the third installment of Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If this report was helpful in recapping it all for you, please do share it with your colleagues.

Get ready for FDA’s third Covid-19 vaccine

On the heels of a ringing endorsement from FDA reviewers earlier in the week, J&J‘s single-dose vaccine — which proved 66% effective at preventing symptomatic Covid-19, and 85% effective at stopping severe disease 28 days after administration — the advisory committee convened by the agency voted unanimously to recommend its emergency use authorization. It was “a relatively easy call,” according to one of the committee members — although that doesn’t mean they didn’t have questions. Jason Mast has the highlights from the discussion, including new information from the company, on this live blog.

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Roche and Genen­tech re­searchers plot $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Roche and Genentech have committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Researchers from both Roche and its big South San Francisco hub — mixing teams from gRED and pRED this time — will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Am­gen, As­traZeneca speed to­ward fil­ing next-gen an­ti­body for asth­ma af­ter un­cork­ing full late-stage da­ta

On the hunt for a novel competitor to Sanofi and Regeneron’s Dupixent in severe asthma, Amgen and AstraZeneca posted “exciting” results from their next-gen antibody late last year. Now, the partners are showing their hands, and the results look good enough for approval.

Amgen and AstraZeneca’s tezepelumab plus standard of care cut the rate of severe asthma attacks by 56% at the one-year mark compared with SOC alone, according to full data from the Phase III NAVIGATOR study presented Friday at the virtual American Academy of Allergy, Asthma & Immunology meeting. And those significant results were consistent regardless of patients’ baseline eosinophil counts.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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