Re­gen­eron’s star bis­pe­cif­ic is linked to 2 deaths in a small study — which was no help for its Q1 call

One of Re­gen­eron’s star can­cer drugs in the pipeline has run in­to trou­ble, and that’s con­tribut­ing to some tur­bu­lence for the share price on Tues­day.

Is­rael Lowy

Re­gen­eron ex­ecs chose to dis­close in their Q1 call with an­a­lysts to­day that their close­ly watched bis­pe­cif­ic REGN1979 was linked with two deaths from cy­tokine re­lease syn­drome in a small PD-1 com­bo study in­volv­ing 30 pa­tients. 

This bis­pe­cif­ic hunts the B cell mark­er CD20 as well as the CD3 com­po­nent of the T cell re­cep­tor, and Re­gen­eron can­cer re­search chief Is­rael Lowy spot­light­ed a 100% over­all re­sponse rate as a monother­a­py at ASH late last year, with 8 com­plete re­spons­es and 2 par­tials among pa­tients with fol­lic­u­lar lym­phoma. That’s very ear­ly-stage da­ta from a tiny tri­al, but Phase I is the new Phase II in can­cer re­search, and those kinds of da­ta al­ways at­tract at­ten­tion in the field — par­tic­u­lar­ly when it comes from a high-pro­file out­fit like Re­gen­eron.

Ge­of­frey Porges

SVB Leerink’s Ge­of­frey Porges was on the call, and re­port­ed back:

The tri­al had on­ly en­rolled 30 pa­tients. Re­gen­eron sug­gest­ed the high­er CRS tox­i­c­i­ty was like­ly due to a height­ened im­mune re­sponse, and not­ed the CRS was al­so seen in con­junc­tion with in­creased tu­mor re­sponse. While the tox­i­c­i­ty is a pre­lim­i­nary set­back for the com­bi­na­tion, Re­gen­eron be­lieve they can in­di­vid­u­al­ly titrate dos­es and tune the se­quence of drugs to help con­trol the im­mune ac­ti­va­tion in a man­ner that cap­tures the in­creased an­ti­tu­mor ac­tiv­i­ty while lim­it­ing po­ten­tial CRS. Man­age­ment be­lieve this tun­abil­i­ty of dos­ing and se­quenc­ing is an ad­van­tage for their ther­a­peu­tic ap­proach ver­sus CAR-T ther­a­pies.

But don’t look for Re­gen­eron to back off. This com­pa­ny doesn’t back away from any­thing.

From Jef­feries’ Biren Amin:

An up­date on REGN1979 CD20xCD3 bis­pe­cif­ic is ex­pect­ed at 2 EU hema­tol­ogy mt­gs in Jun ’19, in­clud­ing da­ta on high­er dos­es, LT fol­low-up, and ef­fi­ca­cy in CAR-T fail­ures. Mgmt in­tends to ini­ti­ate 2 PII reg­is­tra­tional stud­ies for ‘1979, 1st in FL by mid-’19 and 2nd in DL­B­CL by YE ’19. A com­bo study of ‘1979 w/ Lib­tayo an­ti-PD-1 in 30 ad­vanced lym­phoma pts was al­so start­ed….

Re­gen­eron shares were down 6.5% late af­ter­noon Tues­day, but that wasn’t just about any pos­si­ble jit­ters about its bis­pe­cif­ic. In­creased R&D costs com­bined with flat Eylea sales — around the same time No­var­tis is ex­pect­ed to launch its ri­val brolu­cizum­ab — and a re­duc­tion in cash flow from its for­mer part­ner Sanofi helped dri­ve a miss on earn­ings per share and rev­enue. 

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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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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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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