BeiGene touts 'en­cour­ag­ing' re­sponse rates as the Chi­nese biotech mounts chal­lenge to BTK, PD-1 lead­ers

As BeiGene sets the stage for a piv­otal show­down with the OG BTK in­hibitor Im­bru­vi­ca, it’s ze­ro­ing in on a sub­set of pa­tients for whom their drug, zanubru­ti­nib, could be es­pe­cial­ly help­ful.

The biotech — which has ori­gins in Chi­na — al­so bran­dished the lat­est piv­otal re­sults for its PD-1 drug tislelizum­ab, an­oth­er fran­chise shak­er that’s un­der pri­or­i­ty re­view in Chi­na, at the EHA Con­gress.

Jane Huang BeiGene

In­ves­ti­ga­tors took a da­ta cut from a Phase III open-la­bel tri­al that is putting zanubru­ti­nib against Im­bru­vi­ca (ibru­ti­nib) in pa­tients with Walden­ström’s Macroglob­u­line­mia to show­case promis­ing re­sponse rates. A non-ran­dom­ized co­hort of 26 pa­tients, who has the MYD88WT geno­type of WM, all re­ceived zanubru­ti­nib. Five of them were treat­ment-naïve while the oth­ers had re­lapsed/re­frac­to­ry dis­ease.

“For these pa­tients, who typ­i­cal­ly have poor­er prog­noses with low­er re­sponse rates, we rec­og­nize the re­al need for a high­ly po­tent and se­lec­tive BTK in­hibitor that can sus­tain BTK in­hi­bi­tion and re­duce off-tar­get ef­fects,” CMO Jane Huang said in a state­ment.

At a me­di­an fol­low-up of 12.2 months, the drug scored an over­all re­sponse rate of 80.8%, with 53.8% of pa­tients ex­pe­ri­enc­ing a par­tial re­sponse or bet­ter. The very good par­tial re­sponse rate was 23.1% while ex­act­ly one pa­tient achieved a com­plete re­sponse.

Im­bru­vi­ca was first ap­proved in 2015 as a monother­a­py for WM, a rare, slow-grow­ing and in­cur­able form of non-Hodgkin lym­phoma. A com­bi­na­tion with Rit­ux­an was al­so OK’d last year based on pro­gres­sion-free sur­vival re­sults. The haz­ard ra­tio com­pared to Rit­ux­an alone was an im­pres­sive 0.20 (p<0.0001).

But BeiGene is com­ing hard at J&J and Ab­b­Vie with zanubru­ti­nib, which be­came the first drug from main­land Chi­na to win an FDA break­through ther­a­py des­ig­na­tion this Jan­u­ary. It’s al­so be­ing test­ed for man­tle cell lym­phoma.

Nei­ther PFS nor over­all sur­vival has been reached in the WM study, though Huang added up­dates from an on­go­ing Phase I/II tri­al give them rea­son to be­lieve that zanubru­ti­nib can in­duce sus­tained re­spons­es, with high rates of CR/VG­PR at 42%.

In that sin­gle arm tri­al — which start­ed off with 77 pa­tients but now has 61 — the es­ti­mat­ed PFS rate at 12 and 24 months was 90% and 81%, re­spec­tive­ly.

Mean­while, tislelizum­ab has demon­strat­ed an ORR of 87% and CR of 63% among pa­tients with clas­si­cal Hodgkin’s lym­phoma who’ve av­er­aged 3 lines of pri­or ther­a­py. Twelve-month PFS was es­ti­mat­ed at 73.8% and me­di­an PFS has not been reached at 13.9 months of me­di­an fol­low-up.

It’s a slight im­prove­ment from the da­ta they pre­sent­ed at ASH in 2018, where they saw an ORR of 86% and CR of 61% in 70 evalu­able pa­tients — more than dou­ble the com­plete re­sponse rate for a lead­ing check­point ac­cord­ing to Er­ic Hedrick, an on­col­o­gist and BeiGene’s chief ad­vi­sor on this pro­gram.

“If the ma­jor­i­ty are achiev­ing a com­plete re­sponse and they were tru­ly durable, that would be a step up in the qual­i­ty of the re­sponse that we’ve typ­i­cal­ly seen in PD-1 in Hodgkin’s dis­ease,” a cau­tious Hedrick told End­points News at the time.

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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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 with any issues.

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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

UP­DAT­ED: 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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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. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

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.

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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