News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

“(T)here is cur­rent­ly no ap­proved ther­a­py for pa­tients who progress on CAR-Ts,” not­ed Re­gen­eron’s Is­rael Lowy. “Our po­ten­tial­ly reg­is­tra­tional Phase 2 pro­gram is ini­ti­at­ing this month and will proac­tive­ly eval­u­ate ac­tive REGN1979 dos­es in in­do­lent and ag­gres­sive non-Hodgkin lym­phoma.”

Not­ed Baird’s Bri­an Sko­r­ney:

The fol­lic­u­lar lym­phoma da­ta don’t look as stel­lar as the pri­or da­ta cut but the DL­B­CL da­ta im­prove. We think this bis­pe­cif­ic pro­gram looks like a strong next-gen B-cell lym­phoma ther­a­py that could yield a fair­ly sig­nif­i­cant mar­ket op­por­tu­ni­ty.

Ar­Qule rights the ship with more proof-of-con­cept da­ta

Af­ter a less than flat­ter­ing show­ing at the last EHA, Ar­Qule is re­turn­ing tri­umphant.

Hom­ing in on the BTK-C481AS mu­ta­tion, Ar­Qule high­light­ed da­ta from a co­hort of six chron­ic lym­pho­cyt­ic leukemia pa­tients who have ei­ther not re­spond­ed to treat­ment or re­lapsed. Hav­ing re­ceived the 65 mg dose, four of them ex­pe­ri­enced a par­tial re­sponse — a sig­nif­i­cant feat giv­en they have al­ready ex­haust­ed avail­able ther­a­pies, the com­pa­ny said. Shares $AR­QL surged 47% pre-mar­ket to $9.26.

Bri­an Schwartz Ar­Qule

“We are now fo­cused on fi­nal­iz­ing the rec­om­mend­ed phase 2 dose and plan­ning for the ex­pan­sion of our clin­i­cal ef­forts with ARQ 531 in­to lat­er stage tri­als across mul­ti­ple in­di­ca­tions as a sin­gle agent and as a com­bi­na­tion ther­a­py,” CMO Bri­an Schwartz said in a pre­pared state­ment.

In ad­di­tion, in­ves­ti­ga­tors al­so not­ed a par­tial re­sponse for a pa­tient with Richter’s Trans­for­ma­tion who had pro­gressed on ibru­ti­nib and R-CHOP.

Lit­tle Ory­zon shows it’s do­ing al­right with AML drug on its own

Ory­zon has seen the first glimpse of ef­fi­ca­cy da­ta for its LSD1 in­hibitor, iadadem­stat when com­bined with chemother­a­py in el­der­ly pa­tients with acute myeloid leukemia. Clin­i­cal re­spons­es were ob­served rapid­ly with a me­di­an time of 1.5 months, and of the 5 evalu­able pa­tients, 3 achieved com­plete re­mis­sions (al­beit with in­com­plete hema­to­log­ic re­cov­ery) while 1 had a par­tial re­mis­sion.

The tri­al is be­ing con­duct­ed in Spain, where Ory­zon was found­ed. The com­pa­ny plans to re­cruit up to 36 pa­tients to set the stage for the broad­er ap­pli­ca­tion of iadadem­stat —  once part­nered but lat­er dumped by Roche — in oth­er leukemias.

BerGen­Bio spot­lights AML drug’s po­ten­tial in pa­tients un­fit for in­tense chemo

Nor­way’s BerGen­Bio is spot­light­ing 6 re­spons­es out of 14 evalu­able pa­tients on its drug, which is de­signed to pair with low-in­ten­si­ty chemother­a­py for acute myeloid leukemia pa­tients who can­not stand in­tense chemother­a­py. Their AXL in­hibitor be­m­cen­tinib helped push 4 pa­tients in­to com­plete re­mis­sion (with or with­out in­com­plete hema­to­log­ic re­cov­ery) and 2 oth­ers in­to par­tial re­mis­sion.

“Be­m­cen­tinib in com­bi­na­tion with LDAC re­sult­ed in a sub­stan­tial­ly high­er ORR than ex­pect­ed for sin­gle-agent cy­tara­bine and clear­ly war­rant fur­ther in­ves­ti­ga­tion of be­m­cen­tinib in an ex­pan­sion co­hort of AML pa­tients un­fit for in­ten­sive chemother­a­py,” pro­fes­sor Bjørn Tore Gjert­sen of Hauke­land Uni­ver­si­ty Hos­pi­tal and the Uni­ver­si­ty of Bergen said. “Our sig­nal cell-lev­el bio­mark­er analy­sis of AML blasts from pa­tients in­di­cates a sub­stan­tial ef­fect on cell sig­nal­ing and rep­re­sents a po­ten­tial new bio­mark­er strat­e­gy.”

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 help@endpointsnews.com 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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