Watch out Ab­b­Vie and J&J, As­traZeneca just took an­oth­er big swing at one of your star can­cer drug fran­chis­es

As­traZeneca re­port­ed Tues­day that its BTK con­tender Calquence (acal­abru­ti­nib) aced a Phase III tri­al as a sec­ond-line ther­a­py for chron­ic lym­pho­cyt­ic leukemia pa­tients, al­low­ing re­searchers to call it ear­ly as they po­si­tion this drug for green­er pas­tures. 

José Basel­ga

We won’t get the da­ta un­til lat­er in the year, but the UK phar­ma gi­ant $AZN says they nailed sta­tis­ti­cal­ly sig­nif­i­cant re­sults on pro­gres­sion-free sur­vival com­pared to a con­trol group get­ting a com­bo of rit­ux­imab plus physi­cian’s choice of ide­lal­is­ib or ben­damus­tine. And José Basel­ga, the com­pa­ny’s on­col­o­gy re­search chief, says this is the first time a monother­a­py has proven su­pe­ri­or to a stan­dard of care in this seg­ment of the pop­u­la­tion.

Lat­er this year we’ll get the Phase III da­ta for front­line CLL as As­traZeneca pa­tient­ly builds its case for a ri­val fran­chise op­er­a­tion. The com­pa­ny won its first OK for Calquence in late 2017 for man­tle cell lym­phoma, which marked their first en­try for this drug. And if As­traZeneca CEO Pas­cal So­ri­ot gets his way, they’ll soon be go­ing head-to-head on CLL with J&J $JNJ and Ab­b­Vie’s $AB­BV Im­bru­vi­ca, the pi­o­neer­ing BTK on the mar­ket.

Im­bru­vi­ca earned $3.6 bil­lion for J&J and Ab­bie last year, up 40% over the year be­fore.

The Phase III win is the lat­est score for As­traZeneca since it bought this drug in the $7 bil­lion Ac­er­ta Phar­ma ac­qui­si­tion back in 2015. 

Ex­pand­ing the mar­ket for Calquence has been part of So­ri­ot’s on­col­o­gy game plan for some time. Just as he built Lyn­parza and Tagris­so in­to ma­jor new rev­enue streams for the com­pa­ny, the R&D group has been steadi­ly plug­ging away at new can­cer in­di­ca­tions for its oth­er big play­ers.

As is typ­i­cal­ly true for all can­cer drug fran­chis­es, As­traZeneca and the Ab­b­Vie/J&J op­er­a­tion aren’t work­ing in a vac­u­um. BeiGene $BGNE, the Chi­na-based biotech which is an­gling to prove that it can make bet­ter can­cer drugs than the ones al­ready on the mar­ket, has its own BTK drug — zanubru­ti­nib — which it is lin­ing up in ear­ly-stage stud­ies for CLL and more. Much more. Here’s BeiGene’s break­down of the stud­ies:

Phase 3 clin­i­cal tri­al in pa­tients with Walden­ström macroglob­u­line­mia (WM) com­par­ing zanubru­ti­nib to ibru­ti­nib,  cur­rent­ly the on­ly ap­proved BTK in­hibitor for WM; a glob­al Phase 3 clin­i­cal tri­al in pa­tients with pre­vi­ous­ly un­treat­ed chron­ic lym­pho­cyt­ic leukemia (CLL)/small lym­pho­cyt­ic lym­phoma (SLL); a piv­otal Phase 2 tri­al in pa­tients with re­lapsed/re­frac­to­ry (R/R) fol­lic­u­lar lym­phoma in com­bi­na­tion with Gazy­va (obin­u­tuzum­ab); glob­al Phase 2 tri­al in pa­tients with R/R mar­gin­al zone lym­phoma (MZL), a Phase 3 tri­al com­par­ing zanubru­ti­nib to ibru­ti­nib in pa­tients with R/R CLL/SLL; and a glob­al Phase 1 tri­al. In Chi­na, BeiGene has com­plet­ed two piv­otal Phase 2 clin­i­cal tri­als of zanubru­ti­nib in pa­tients with MCL and CLL/SLL and the en­roll­ment in the piv­otal Phase 2 clin­i­cal tri­als in pa­tients with WM.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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