Sanofi be­lieves it’s still not too late to be­come a top play­er in can­cer drug mar­ket

A lit­tle more than two years ago, Sanofi R&D chief Elias Zer­houni sig­naled the phar­ma gi­ant’s sore dis­ap­point­ment in its can­cer pipeline by ax­ing group leader Tal Zaks and 100 in­ves­ti­ga­tors who failed to de­liv­er the kind of ex­cit­ing new on­col­o­gy pro­grams the phar­ma gi­ant had promised to in­vestors.

At the time, Sanofi said it was re­or­ga­niz­ing to cut back ex­pens­es and ab­sorb­ing the rest of the can­cer R&D group in­to the glob­al re­search struc­ture. Then it turned to Re­gen­eron to go to work on a block­buster PD-1 part­ner­ship, which now is mov­ing di­rect­ly in­to piv­otal stud­ies. And Sanofi, which has strug­gled to find a ma­jor ac­qui­si­tion deal it can com­plete, has been ea­ger­ly sig­nal­ing its in­ten­tion to be­come a play­er in im­muno-on­col­o­gy, cer­tain that the big sur­pris­es of the past year — like Bris­tol-My­ers’ big set­back on first-line lung can­cer — in­di­cate that there’s still plen­ty of tro­phies to hunt in the game of block­busters played by the lead­ers in bio­phar­ma.

“It’s not a sprint to one tar­get and that’s it, you’re the win­ner,” Zer­houni told Bloomberg in a new up­date. “The im­muno-on­col­o­gy field will be go­ing through up­heaval and changes in po­si­tion for the next 20 years.”

Sanofi — like Glax­o­SmithK­line, which has al­so been sig­nal­ing a big move back in­to on­col­o­gy — finds it­self way be­hind lead­ers like Mer­ck and Roche and Bris­tol-My­ers Squibb. As­traZeneca has been mak­ing a con­cert­ed ef­fort to be a big play­er in can­cer, and has drugs like Tagris­so, Lyn­parza and its PD-L1 drug Imfinzi to boast about — when they aren’t try­ing to ex­plain away ma­jor set­backs like the failed first-round on the MYS­TIC com­bo in first-line lung can­cer.

While Zer­houni won’t come out and say so, its part­ners at Re­gen­eron have been as­sert­ing for months now that PD-1 is a much bet­ter tar­get than PD-L1 for im­muno-on­col­o­gy. Both dis­man­tle a mech­a­nism that can­cer cells use to di­vert at­ten­tion from im­mune cells, but there’s been a grow­ing dis­cus­sion that PD-1 could prove bet­ter than the oth­ers.

PD-L1 play­ers like Roche would beg to dif­fer, so ex­pect plen­ty of more dis­cus­sion on that point in the years ahead. Iron­i­cal­ly, this de­bate is al­so com­ing at a time that there’s been grow­ing dis­cus­sion around the idea that I/O could be­come com­modi­tized, with a grow­ing sec­ond wave of check­points com­ing from BeiGene/Cel­gene, No­var­tis, In­cyte and more that could wreck pric­ing.

If Sanofi is right, the phar­ma gi­ant could steal a march in a huge and grow­ing new on­col­o­gy mar­ket. But prac­ti­cal­ly all the an­a­lysts have been wait­ing for Sanofi to fol­low up on its failed Medi­va­tion bid and go af­ter a can­cer com­pa­ny that would pro­vide a pipeline at a time the com­pa­ny is fo­cus­ing more on in­ter­nal pro­grams.

“I see lit­tle op­por­tu­ni­ty for late en­trants,” Nick Turn­er, an an­a­lyst at Mirabaud Se­cu­ri­ties in Lon­don, told Bloomberg. “I find it dif­fi­cult, with­out an ac­qui­si­tion, to see how any­body else is go­ing to build a com­pet­i­tive po­si­tion in the PD-1 space.”

That won’t come cheap, es­pe­cial­ly if Em­ma Walm­s­ley at GSK brings out the check­book to mount their own come-from-be­hind strat­e­gy. GSK swapped their late-stage and com­mer­cial can­cer pro­grams to No­var­tis, and now want to be a play­er in the hottest field in bio­phar­ma R&D. Like GSK, Sanofi al­so has to over­come a his­to­ry of mis­er­able in­ter­nal R&D re­sults, which has been aug­ment­ed on­ly through pacts with com­pa­nies like Re­gen­eron or its ac­qui­si­tion of Gen­zyme.


Elias Zer­houni, Sanofi pres­i­dent, glob­al R&D, speak­ing at Sanofi’s gen­er­al share­hold­ers meet­ing last year in Paris. Vin­cent Isore, IP3, Get­ty

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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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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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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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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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.