Thumbs up: Can­cer drug R&D is ex­plod­ing, get ready for the pric­ing de­bate to fol­low

I ar­rived in Chica­go last night, still try­ing to sort out how to pri­or­i­tize the news com­ing out of AS­CO this week­end. In the lead up, I had a chance to do a pair of sto­ries that looked at new re­ports cov­er­ing the rapid pro­lif­er­a­tion of PD-(L)1 com­bos along with a 7.7% one-year growth rate in late-stage can­cer drug stud­ies, com­ing in at 631 piv­otal-stage ef­forts.

This didn’t hap­pen by ac­ci­dent.

Over the last few years we’ve seen a tru­ly rev­o­lu­tion­ary change in the way on­col­o­gy drugs are de­vel­oped. Backed in par­tic­u­lar by the FDA’s break­through pro­gram, now lib­er­al­ly ap­plied along with fast-track sta­tus and pri­or­i­ty re­views, the bio­phar­ma in­dus­try is com­pet­ing like nev­er be­fore to carve out new ter­ri­to­ry in can­cer treat­ment.

I well re­mem­ber a lot of the ini­tial hoot­ing from some an­a­lysts at the idea that the FDA’s BTD pro­gram would make a dif­fer­ence. But no one is hoot­ing now. The agency — and in par­tic­u­lar Richard Paz­dur — can­did­ly em­braced an open-door ap­proach on can­cer R&D. And that’s the path FDA com­mis­sion­er Scott Got­tlieb wants the rest of the FDA di­vi­sions to fol­low.

Richard Paz­dur

When a bu­reau­cra­cy sets new goals, things change. So I’ll be ex­pect­ing lots of fresh ac­tion on the BTD front over the next few years, for all kinds of dis­eases.

There are bil­lions of dol­lars at stake here, which is as it should be. The greater the pay­back, the greater the R&D in­vest­ment and the greater the re­wards for pa­tients, who are see­ing some im­por­tant and some­times dra­mat­ic im­prove­ment in the way many can­cers are treat­ed.

Out of this rev­o­lu­tion, though, will come plen­ty of added pres­sure on drug mak­ers to find a way to rea­son­ably price these new drugs and the com­bos com­ing down the pipeline. Af­ter get­ting side­lined at the Trump ad­min­is­tra­tion, changes on pub­lic pol­i­cy around drug pric­ing may well get lost in all the shout­ing in DC these days.

But you can be sure that pay­ers haven’t lost their fo­cus, es­pe­cial­ly as six-fig­ure com­bos start to make the rounds. So the in­dus­try will need to have a more co­her­ent ap­proach to pay for per­for­mance.

On­col­o­gy now rep­re­sents the po­ten­tial that drug R&D has in sig­nif­i­cant­ly im­prov­ing the stan­dard of care in a short pe­ri­od of time. Next, it has to lead the way on mar­ket-based so­lu­tions to the pric­ing de­bate.

That next part could prove to be much, much more chal­leng­ing.

Daniel O'Day [via AP Images]

UP­DAT­ED: Look­ing to re­solve lin­ger­ing doubts, Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

UP­DAT­ED: Am­gen, No­var­tis scrap Alzheimer's stud­ies — is BACE fi­nal­ly dead or will Bio­gen and Ei­sai car­ry on?

The BACE theory of controlling Alzheimer’s died with failed pivotal projects at Merck, Eli Lilly and their partners at AstraZeneca. Now Amgen and Novartis have come along to bulldoze it under a mound of safety threats — leaving only Biogen and Eisai to carry on with a less than zero chance of success — with the notable addition that they may actually be doing harm to patients.

After the market closed Thursday, Amgen and Novartis announced that they were dumping two pivotal programs underway with the Banner Alzheimer’s Institute on their BACE drug CNP520 (umibecestat) after an independent review of the data indicated that patients’ cognitive abilities were actually worsening at a faster pace than the placebo arm.

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Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Fresh out of Eli Lil­ly, Christi Shaw sur­faces as Daniel O'­Day's new CEO at CAR-T pi­o­neer Kite

Well, that didn’t take long. 

We found out Thursday evening that Christi Shaw has given up her top post as the head of the Bio-Medicines group at Eli Lilly for the helm at CAR-T pioneer Kite. New Gilead CEO Daniel O’Day, a Roche veteran, had made finding a Kite CEO a top priority on his arrival at Gilead. And he went right for a headliner.

O’Day was clearly excited about the coup.

“We conducted an extensive search for a new leader at Kite and we believe that Christi’s unique set of skills will allow us to continue to build on our leadership position in cell therapy,” he said in a prepared statement. “Christi’s vast experience across complex therapeutic areas, and particularly in oncology, will serve Kite very well. She is clearly a leader who will bring teams and individuals together and I am confident she will build upon the entrepreneurial spirit at Kite as we seek to help more people with cancer around the world.”

Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Eli Lil­ly's Christi Shaw bows out of top post at the Bio-Med­i­cines unit

Less than 3 years after Eli Lilly CEO David Ricks recruited Novartis vet Christi Shaw to run their big Bio-Medicines business, she’s out.

In a statement put out Thursday morning, Lilly said that Shaw’s last day will come at the end of August. Patrik Jonsson, currently president and general manager of Lilly Japan, will succeed Shaw once he gets the paperwork sorted out.

Lilly’s shares dropped 4% on the news.

Jeff Poulton

Al­ny­lam’s Maraganore switch­es ‘per­haps the best CFO in mid-cap biotech’ with Shire vet Jeff Poul­ton

There’s a new CFO taking charge of the numbers at RNAi pioneer Alnylam.

Alnylam chief John Maraganore says that CFO Manmeet S. Soni is leaving in the proverbial pursuit of new opportunities. And he’s being replaced by ex-Shire CFO Jeff Poulton, not long after the Takeda takeover obliterated that position.

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Analy­sis: In most of the Big Phar­ma world, R&D spend­ing tow­ers over rev­enue from new drugs. Guess who beat the odds

It’s always been the case that there’s no medicine less useful than one that doesn’t make it to patients (unless you regard the task of R&D to be perpetually learning about swimming, while under water…). Yet, launching new medicines that physicians want, that payers will cover, and that patients will take, is a discipline that is unevenly distributed among the big players.

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President Donald Trump at State of the Union. AP Images

White House changes course to kill re­bate re­form

So what exactly is the White House’s plan to tackle rising drug prices? It doesn’t look like we will get definitive answers anytime soon. On the heels of President Donald Trump’s surprise revelation that an executive order is in the works to implement a “favored nations clause,” his administration is now putting out word that it has abandoned an earlier proposal to overhaul rebates.

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Janet Woodcock, AP Images

The FDA's Janet Wood­cock talks about some big changes she's push­ing for in drug de­vel­op­ment, and agency re­views

Janet Woodcock is perhaps the most influential regulator at the FDA. And when the head of CDER talks about the changes being made at the agency when it comes to clinical trial designs, or the need to reorganize for a specific disease arena, an assessment of the expansion of gene therapy or I/O, common development mistakes, and so on, you can be sure the industry pays attention to every word.

So it was with some eagerness that I opened up Geoffrey Porges’ summary of their recent conversation about the FDA. And I wasn’t disappointed. In a wide-ranging exchange with the SVB Leerink analyst, Woodcock discussed the growing importance of patient-reported outcomes in clinical trials, a campaign underway now to see if CROs would help spur more basket studies to compare drugs head-to-head, and much, much more.

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Ex-DARPA di­rec­tor pur­sues all-in-one can­cer pill as NED CEO; Karyopharm los­es com­mer­cial chief ahead of drug roll­out

“Why not try?”

That’s what Geoffrey Ling told me over the phone when asked about what led him to his journey to the position of CEO at NED Biosciences — a company with a lofty goal of creating an all-in-one oral drug to treat all types of cancer and making this drug available to not only developed nations, but also the developing world. 

Ling comes from an extensive background in medicine and the government. He is the co-leader of The Brain Health Project, a professor of neurology and an attending neurocritical care physician at John Hopkins University and Hospital, as well as the assistant director for Medical Innovation of the Science Division in president Obama’s White House Office of Science and Technology Policy (OSTP).