Ab­b­Vie re­treats as Gala­pa­gos of­fers mixed PhII CF da­ta, leav­ing a ma­jor al­liance in per­il — Ver­tex shares surge

Gala­pa­gos $GLPG out­lined some dis­tinct­ly mixed re­sults from its Phase II study of its C2 cor­rec­tor GLPG2737 for cys­tic fi­bro­sis Thurs­day af­ter­noon, man­ag­ing to boost the prospects of ri­val Ver­tex in the process.

While Gala­pa­gos said that the drug — com­bined with a sta­ble dose of Ver­tex’s Orkam­bi — had hit a sig­nif­i­cant de­crease of 19.6 mmol/L (p=0.02) on sweat chlo­ride from base­line, there was on­ly a pos­i­tive trend in ppFEV1. “The mean ab­solute change from base­line in ppFEV1 for the GLPG2737 treat­ment arm ver­sus place­bo through day 28 was 3.4% (p=0.08).”

That’s not good. Just lis­ten to Baird’s Bri­an Sko­r­ney:

The lack­lus­ter FEV1 per­for­mance re­al­ly makes it tough to imag­ine Gala­pa­gos mount­ing a rel­e­vant com­pet­i­tive cam­paign against Ver­tex’s triple com­bi­na­tion. Re­call, each of Ver­tex’s third agents showed about a 10% im­prove­ment in FEV1 when added to Symdeko. We think Gala­pa­gos need­ed to cross 5% to even have a hope of an ar­gu­ment here but re­al­is­ti­cal­ly would need to get a ~10% ben­e­fit to re­al­ly be con­sid­ered a com­pet­i­tive threat. We think the door is clos­ing on Gala­pa­gos, which is look­ing less and less like­ly to be able to pull out a triple com­bo that could even hope to match Ver­tex’s, much less beat it.

And while Gala­pa­gos is pro­ceed­ing with a study of its triple ther­a­py (da­ta due in the next quar­ter), its big part­ner Ab­b­Vie $AB­BV has opt­ed out of a sec­ond triple study that had been con­tem­plat­ed, leav­ing Gala­pa­gos “re­view­ing the fu­ture of its CF col­lab­o­ra­tion with Ab­b­Vie.”

Ab­b­Vie al­ready pulled out of one al­liance with Gala­pa­gos, sur­ren­der­ing its in­ter­est in fil­go­tinib, which Gilead picked up. The bond can’t be too strong now. Ab­b­Vie paid Gala­pagis $40 mil­lion up­front to part­ner on CF, with more than $300 mil­lion in mile­stones on the ta­ble. And Ab­b­Vie has walked away from much more than that with­out a sec­ond thought.

An­a­lysts — par­tic­u­lar­ly some of the heavy­weights that have long been lined up on Ver­tex’s side — are like­ly to see this as an­oth­er vin­di­ca­tion of their star play­er as the un­chal­lenged leader in CF.  Just a few weeks ago Pro­teosta­sis was brushed aside as it tried to of­fer some com­pe­ti­tion.

But the mar­ket wasn’t hav­ing it — then or now.

Piet Wiger­inck

Thurs­day af­ter­noon Ver­tex shares shot up 9% in af­ter-mar­ket trad­ing as in­vestors sa­vored the idea that the big biotech had been left well out in front, with an undis­put­ed lead as it pur­sues its own triplets. Gala­pa­gos, mean­while, plunged 17% on the news.

But they’re forg­ing ahead, in a fash­ion.

“The PEL­I­CAN tri­al is the first to eval­u­ate GLPG2737 as a C2 cor­rec­tor in CF pa­tients on top of Orkam­bi and showed CFTR on-tar­get ac­tiv­i­ty with GLPG2737 in com­bi­na­tion with Orkam­bi,” said Dr. Piet Wiger­inck, chief sci­en­tif­ic of­fi­cer of Gala­pa­gos. “We have ini­ti­at­ed dos­ing in the FAL­CON tri­al, in which we aim to eval­u­ate high­er ex­po­sures of GLPG2737 in CF pa­tients and fur­ther un­der­stand the po­ten­tial syn­er­gis­tic ef­fect of GLPG2737 on top of our own dual com­bi­na­tion com­pounds.”

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 help@endpointsnews.com 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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