As­traZeneca’s shares spike on a sur­prise coup in PhI­II lung can­cer da­ta

Sean Bo­hen, As­traZeneca

As­traZeneca $AZN shares popped more than 7% this morn­ing on pos­i­tive Phase III pro­gres­sion-free sur­vival da­ta for Imfinzi (dur­val­um­ab) in treat­ing ad­vanced, in­op­er­a­ble and chemo-re­sis­tant non-small cell lung can­cer.

An­a­lysts did a quick dou­ble take and im­me­di­ate­ly cheered it as a re­mark­able – and com­plete­ly un­ex­pect­ed – hit.

That’s the right tar­get for As­traZeneca, which re­cent­ly won its first ap­proval for the world’s fifth check­point in­hibitor. On Wednes­day Mer­ck moved to the front of that field with an ap­proval to use a com­bi­na­tion of Keytru­da with chemo as a first-line ther­a­py on NSCLC, putting it on top of the heap of ri­vals look­ing to carve out a big piece of the block­buster mar­ket.

This As­traZeneca tri­al wasn’t the big study an­a­lysts have been wait­ing for.

As­traZeneca has been bet­ting heav­i­ly on MYS­TIC, us­ing a com­bi­na­tion of dur­val­um­ab and the CT­LA-4 drug treme­li­mum­ab for NSCLC. That would cre­ate some ma­jor mar­ket op­por­tu­ni­ties for the phar­ma gi­ant, which has been mak­ing a rep for it­self in the on­col­o­gy field in re­cent years — even as it’s plagued by prat­falls and dis­ap­point­ments in oth­er ar­eas of R&D.

As­traZeneca bad­ly needs every shred of good news that it can find, and there were some high fives in the or­ga­ni­za­tion on this pos­i­tive read­out, which helps the com­pa­ny dis­tin­guish it­self in the most in­tense­ly com­pet­i­tive field in bio­phar­ma.

Sea­mus Fer­nan­dez, Leerink

Sea­mus Fer­nan­dez at Leerink gave it a big thumbs up, not­ing:

This news comes as an ear­ly up­side sur­prise, as we do not cur­rent­ly have fore­casts for this in­di­ca­tion in our mod­el nor do we be­lieve this op­por­tu­ni­ty is wide­ly ap­pre­ci­at­ed by in­vestors. Im­por­tant­ly, this puts AZN square­ly in the IO fight in with a unique NSCLC in­di­ca­tion for monother­a­py PD1/PDL1 agents not cur­rent­ly shared by oth­er mem­bers of the class. AZN es­ti­mates that it is at least 2-3 years ahead of oth­er com­peti­tors, and our search­es on­ly un­cov­ered rel­a­tive­ly small sin­gle-arm Ph 2 stud­ies un­der­way for po­ten­tial com­peti­tors.

Oth­er an­a­lysts at Deutsche Bank were al­so im­pressed, of­fer­ing some block­buster num­bers for As­traZeneca on this one in­di­ca­tion.

Look for the da­ta to make the short trip to reg­u­la­tors as As­traZeneca seeks to cap­i­tal­ize on fore­casts that this drug could be worth more than $2 bil­lion a year for the com­pa­ny.

Said R&D chief Sean Bo­hen:

We look for­ward to work­ing with reg­u­la­to­ry au­thor­i­ties around the world to bring Imfinzi to lung can­cer pa­tients as soon as pos­si­ble. Along­side this, we con­tin­ue to ex­plore Imfinzi’s full po­ten­tial as monother­a­py as well as in com­bi­na­tion with treme­li­mum­ab and oth­er med­i­cines in ar­eas of con­tin­ued un­met need across mul­ti­ple types of can­cer.

At a time when the check­point can­cer mar­ket would seem to be ripe for ma­tur­ing, with 5 ap­proved, there’s in­stead been a se­ries of star­tling re­ver­sals and ad­vances. Bris­tol-My­ers Squibb made the move to the lead, then blew it with a bad­ly de­signed Phase III on front­line use. Mer­ck leapfrogged its ri­val and then, just days ago, Roche’s Tecen­triq failed a con­fir­ma­to­ry Phase III in blad­der can­cer, af­ter it had been giv­en an ac­cel­er­at­ed ap­proval.

Big sur­pris­es are be­com­ing the or­der of the day in check­point in­hi­bi­tion, a field worth tens of bil­lions of dol­lars.

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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Hal Barron. GSK

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

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

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.

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

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.