#AACR: Mer­ck spot­lights piv­otal Keytru­da/chemo da­ta, hop­ing to break down skep­ti­cism to its front­line lung can­cer duo

CHICA­GO — Mer­ck wowed the im­muno-on­col­o­gy mar­ket ear­ly this year with the head­line news that its piv­otal KEYNOTE-189 study demon­strat­ed a clear suc­cess for Keytru­da com­bined with chemo in treat­ing front­line lung can­cer cas­es. And at AACR in Chica­go to­day, its in­ves­ti­ga­tors spelled out the promis­ing re­sults — demon­strat­ing that the com­bo re­duced the risk of death by half with a haz­ard ra­tio of 0.49 — as Mer­ck looks to hold back a wave of ma­jor league com­peti­tors as it carves out a big­ger share of the block­buster lung can­cer mar­ket.

In the first look at hard da­ta for non­squa­mous non-small cell lung can­cer — which will be care­ful­ly com­pared with the da­ta from Bris­tol-My­ers — re­searchers say that their com­bo clear­ly beat out chemo alone on over­all sur­vival, though the fi­nal OS rate for the com­bi­na­tion has not yet been reached. 

Leena Gand­hi

The great­est ben­e­fit among these pa­tients was among the high PD-L1 ex­pressers, but chief in­ves­ti­ga­tor Leena Gand­hi at the Perl­mut­ter Can­cer Cen­ter at NYU Lan­gone Health al­so says that the sur­vival ben­e­fit could be seen across the board on PD-L1 ex­pres­sion.

Mer­ck’s piv­otal da­ta from the -189 study are cru­cial to their cam­paign to be the leader in check­point in­hi­bi­tion. Ap­proved by the FDA for front­line cas­es on Phase II da­ta — but re­ject­ed in Eu­rope — Mer­ck says that physi­cians have been slow to change the way they treat lung can­cer. Now Mer­ck be­lieves this da­ta should re­solve any doubts as Keytru­da be­comes the stan­dard of care in this crit­i­cal­ly im­por­tant set­ting.

At 12 months, Gand­hi tells me, 69.2% of the com­bo arm was still alive, com­pared to 49.4% in the con­trol arm. 

Ac­cord­ing to Mer­ck, pa­tients in the drug arm were 51% less like­ly to die than the con­trol group, and those in the high­est PD-L1 score group were 58% less like­ly to die. The rates held up de­spite a high num­ber of pa­tients who crossed over to the com­bo af­ter their dis­ease pro­gressed.

“The great­est ben­e­fit was seen in those with high lev­els of PD-L1 ex­pres­sion, sug­gest­ing that PD-L1 does have some pre­dic­tive val­ue even in this com­bi­na­tion set­ting,” Gand­hi said. 

Me­di­an PFS was 8.8 months for the pem­brolizum­ab arm, ver­sus 4.9 months for the con­trol group.

Some an­a­lysts have been wait­ing to see if the ben­e­fits for the com­bo leaned pri­mar­i­ly to high PD-L1 ex­pressers, leav­ing ri­vals an open­ing in divvy­ing up the mar­ket on the rest. But Gand­hi says the sur­vival ad­van­tages are clear re­gard­less of PD-L1 sta­tus. That marks a trend that many are see­ing, where new ther­a­peu­tic ap­proach­es are de­liv­er­ing en­hanced re­sults for the high-PD-L1 group while re­tain­ing promis­ing re­sults for the full field.

“There was no group that didn’t get a sur­vival ben­e­fit in this study,” Gand­hi says, with the re­sults of­fer­ing a prac­tice-chang­ing set of re­sults. She al­so says she wants to ful­ly con­sid­er the fi­nal da­ta from Check­mate-227 (Bris­tol-My­ers) and IM­pow­er-150 (Roche) as she pon­ders the chang­ing scene for prac­ti­tion­ers.

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.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.