Roche racks up an­oth­er check­point win as Tecen­triq scores in lat­est PhI­II lung can­cer study

Com­ing fast on the heels of Bris­tol-My­ers Squibb’s sting­ing set­back with its star can­cer ther­a­py Op­di­vo, Roche says that its PD-L1 check­point drug Tecen­triq (ate­zolizum­ab) hit its key goals in a Phase III study for non-small cell lung can­cer, ex­tend­ing the lives of pa­tients in the tri­al.

San­dra Horn­ing, Roche CMO

The phar­ma gi­ant’s big Genen­tech unit re­port­ed that Tecen­triq met its co-pri­ma­ry end­points on im­prov­ing sur­vival rates for the over­all pop­u­la­tion of lung can­cer pa­tients as well as a spe­cif­ic sub­group se­lect­ed by PD-L1 ex­pres­sion, a cru­cial bio­mark­er for this treat­ment. In­ves­ti­ga­tors gath­ered the da­ta among 1,225 pa­tients whose lung can­cer had spread on or af­ter chemo.

The phar­ma gi­ant was stick­ing with top-line da­ta on­line in this first analy­sis, hold­ing back the spe­cif­ic re­sults for an up­com­ing sci­en­tif­ic con­fer­ence — stan­dard prac­tice in R&D, at least for pos­i­tive stud­ies.  And Roche says it plans to hus­tle the new da­ta to the FDA as it works in­tent­ly on grow­ing the mar­ket.

The suc­cess lands just weeks af­ter Bris­tol-My­ers man­aged to stun just about every­one in the on­col­o­gy field with the news that its Phase III for its PD-1 drug Op­di­vo in first-line lung can­cer pa­tients had failed, in­stant­ly giv­ing Mer­ck a big edge in a huge mar­ket. Op­di­vo’s fail­ure star­tled a big crowd of in­vestors who had be­come used to con­sid­er­ing Op­di­vo as the dom­i­nant play­er, dis­rupt­ing a field that is de­liv­er­ing megablock­buster re­turns.

Roche has been play­ing catch-up with Bris­tol-My­ers — the dom­i­nant play­er — and Mer­ck. But it has at­tract­ed con­sid­er­able at­ten­tion for its com­ple­men­tary PD-L1 ap­proach, which is carv­ing out its own place in the mar­ket af­ter gain­ing its first ap­proval for blad­der can­cer back in May.

Bris­tol-My­ers’ set­back in the check­point field — where drugs dis­man­tle a mech­a­nism that hides can­cer cells from an im­mune sys­tem at­tack — spurred some sig­nif­i­cant crit­i­cism that the com­pa­ny had tried to tack­le a pa­tient pop­u­la­tion too big to de­liv­er pos­i­tive re­sults, forc­ing in­ves­ti­ga­tors to re­think how they de­sign and ex­e­cute Phase III stud­ies for a check­point pro­gram. Re­searchers on the Bris­tol-My­ers study had re­cruit­ed a broad pop­u­la­tion of 541 pre­vi­ous­ly un­treat­ed first line pa­tients whose tu­mors ex­pressed PD-L1 at a low lev­el of ≥ 5%.

Genen­tech, mean­while, has eight late-stage stud­ies on­go­ing for var­i­ous stages of lung can­cer, high­light­ing its sig­nif­i­cance in a boom­ing field. And with break­through sta­tus at the FDA, it ex­pects to hus­tle up a whole slate of ap­pli­ca­tions to ex­pand its use.

Sea­mus Fer­nan­dez at Leerink sees tougher head­winds ahead for Op­di­vo now:

At a min­i­mum, we have to as­sume that this re­sult will in­crease the view that PD-1 & PD-L1 an­ti­bod­ies have very sim­i­lar ef­fi­ca­cy pro­files, re­sult­ing in in­creased com­pe­ti­tion in 2L lung can­cer – Op­di­vo’s cur­rent strong­hold. In our view, this places even greater im­por­tance on a pos­i­tive out­come in the Check­Mate-227 (CM-227) tri­al of Op­di­vo + Yer­voy (ip­il­i­mum­ab; CT­LA-4 in­hibitor) in 1L NSCLC. While full re­sults of the OAK tri­al will de­ter­mine if physi­cians will dis­tin­guish Tecen­triq com­mer­cial­ly, we be­lieve the top line re­sults sug­gest less ef­fi­ca­cy dif­fer­en­ti­a­tion be­tween Tecen­triq, Op­di­vo, and Mer­ck’s (MP) Keytru­da (pem­brolizum­ab; PD-1 in­hibitor), and near-term, MRK is like­ly to main­tain the most unique op­por­tu­ni­ty in 1st line NSCLC.

San­dra Horn­ing, Roche’s chief med­ical of­fi­cer and head of Glob­al Prod­uct De­vel­op­ment, had this to say:

“These re­sults add to the grow­ing body of ev­i­dence that sup­ports the role of Tecen­triq as a po­ten­tial new treat­ment for spe­cif­ic types of ad­vanced NSCLC. This is very en­cour­ag­ing news for peo­ple liv­ing with this dis­ease be­cause lung can­cer is the lead­ing cause of can­cer deaths around the world. We hope to bring this treat­ment op­tion to pa­tients as soon as pos­si­ble.”

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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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

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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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

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It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

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

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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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Bob Smith, Pfizer

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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

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

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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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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

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