Ab­b­Vie and Roche tout a pos­i­tive PhI­II com­bo study of Ven­clex­ta, Rit­ux­an in CLL — ahead of FDA pitch

Ab­b­Vie and Roche are get­ting a boost out of the news that their com­bi­na­tion of Ven­clex­ta and Rit­ux­an met their pri­ma­ry end­point on pro­gres­sion-free sur­vival, beat­ing out a com­bi­na­tion of chemother­a­py and Rit­ux­an for treat­ment-re­sis­tant cas­es of chron­ic lym­pho­cyt­ic leukemia.

Ab­b­Vie says they’ll be hus­tling along the da­ta — none of which was on dis­play to­day — to reg­u­la­tors af­ter pick­ing up a break­through drug des­ig­na­tion on the com­bo for CLL, which has at­tract­ed a num­ber of drugs.

Michael Sev­eri­no

The re­sults from the MU­RA­NO study have been await­ed by a cou­ple of the an­a­lysts cov­er­ing Ab­b­Vie to see how the com­pa­ny can fare this year in ex­pand­ing its prospects in on­col­o­gy. Its big can­cer drug dri­ver is Im­bru­vi­ca, with Hu­mi­ra pro­vid­ing the bulk of its rev­enue.

Leerink’s Ge­of­frey Porges counts him­self as a hap­py camper on the news. He not­ed:

 We an­tic­i­pate an ex­pe­dit­ed reg­u­la­to­ry fil­ing and ap­proval based on these da­ta, which will ex­pand the la­beled in­di­ca­tion for Ven­clex­ta from a nar­row sub­set of CLL pa­tients (17p dele­tion) to the broad­er R/R CLL pop­u­la­tion and will ac­cel­er­ate the Ven­clex­ta rev­enue op­por­tu­ni­ty by H2 2018. Ven­clex­ta’s cur­rent quar­ter­ly rev­enue run rate of $20-30mm (our es­ti­mate) should in­crease sig­nif­i­cant­ly with the la­bel ex­pan­sion that is like­ly to fol­low this re­sult.

Ex­pand­ing the num­ber of FDA OKs for this drug is cru­cial to Ab­b­Vie’s plan to ex­pand rev­enue from about $125 mil­lion this year to as much as $1.5 bil­lion to $2 bil­lion a year by the end of the decade. The drug was orig­i­nal­ly ap­proved for pa­tients with CLL with 17p dele­tion back in April of 2016.

Re­searchers are al­so stay­ing mum about the safe­ty re­sults from the study, say­ing they’re ex­am­in­ing ad­verse events reg­is­tered in the study.

CLL ac­counts for about a quar­ter of all the new leukemia cas­es di­ag­nosed every year.

“Ab­b­Vie is com­mit­ted to re­search­ing the full po­ten­tial of Ven­clex­ta both as monother­a­py and com­bi­na­tion ther­a­py in pa­tients with CLL and oth­er hema­to­log­ic ma­lig­nan­cies. The analy­sis of the MU­RA­NO tri­al showed that Ven­clex­ta in com­bi­na­tion with Rit­ux­an may of­fer an­oth­er op­tion for pa­tients with R/R CLL, po­ten­tial­ly pro­vid­ing them with a chemother­a­py-free ther­a­py,” said Michael Sev­eri­no, Ab­b­Vie’s chief sci­en­tif­ic of­fi­cer. “We are look­ing for­ward to work­ing with reg­u­la­to­ry au­thor­i­ties around the world to bring this ad­di­tion­al treat­ment reg­i­men to R/R CLL pa­tients.”

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

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

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

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

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.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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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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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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