Sage ap­pears set for a break­through FDA OK on post­par­tum de­pres­sion drug, but who’s go­ing to use it?

Sage can start this week with a fair­ly firm idea on the time­line for its ini­tial trans­for­ma­tion in­to a mar­ket­ing op­er­a­tion, with the FDA and its out­side ex­perts ap­pear­ing to bless their land­mark drug brex­anolone for post­par­tum de­pres­sion. What they don’t know is who is go­ing to buy it.

The FDA’s ex­pert pan­el vot­ed to back the drug’s risk/ben­e­fit pro­file by a wide mar­gin, 17 in fa­vor and on­ly 1 against, with sev­er­al of the votes cast with an en­thu­si­as­tic en­dorse­ment for the po­ten­tial here. Com­ing in the wake of an up­beat in­ter­nal as­sess­ment at the agency, the writ­ing ap­pears to be on the wall for an OK by the De­cem­ber 19 PDU­FA date.

But the same sources have al­so in­di­cat­ed that for a new drug like this, tied to episodes of un­con­scious­ness among the small group of pa­tients who have tak­en a drug de­signed for a very large pop­u­la­tion — some­thing that may well have scut­tled any ap­pli­ca­tion ahead of the lib­er­al reg­u­la­to­ry era we’re in now — it al­so ap­pears like­ly that the ap­proval will come with a re­quire­ment that pa­tients un­der­go the bur­den­some 72-hour in­fu­sion process un­der 24/7 med­ical care. And that’s like­ly to se­vere­ly rein in de­mand for the drug.

That sen­ti­ment may have weighed down the biotech’s share price. The stock was down 5% in Mon­day morn­ing trad­ing.

Paul Mat­teis

Stifel’s Paul Mat­teis was com­fort­able with the ap­par­ent reg­u­la­to­ry at­ti­tude on abuse po­ten­tial, leav­ing the door open to an ap­proach that works for sleep­ing pills. But the ex­perts balked at home in­fu­sion, want­i­ng a set of des­ig­nat­ed fa­cil­i­ties that can in­fuse the ther­a­py over 60 hours with a 12-hour fol­lowup on safe­ty.

Al­though it was clear that FDA (and of course Sage $SAGE) would have liked to be able to OK in-home in­fu­sions and min­i­mize the bur­den for post-par­tum moth­ers, the agency – and the pan­elists – saw (loss of con­scious­ness) risks as too salient and too un­pre­dictable to al­low for use with­out rig­or­ous mon­i­tor­ing. It re­mains to be seen how spe­cif­ic FDA will be with the type of cen­ter in which it al­lows for use, but over­all the dis­cus­sion here was con­sis­tent with what one would’ve ex­pect­ed from the brief­ing book. Pan­elists asked for a va­ri­ety of things like clear treat­ment pro­to­cols, specifics on mon­i­tor­ing, and a few even men­tioned a black box warn­ing for “falls” would be a smart add; FDA not­ed it still needs to ham­mer out the de­tails of a REMS/la­bel, but its pro­pos­al for the for­mer is to cer­ti­fy health­care fa­cil­i­ties and have a cer­tain list of cri­te­ria that a fa­cil­i­ty can at­test to (con­tin­u­ous mon­i­tor­ing through 12-hours post in­fu­sion). On what ad­di­tion­al da­ta could be col­lect­ed for home in­fu­sions the pan­el was pret­ty vague, hop­ing for a large post-mar­ket­ing safe­ty data­base and/or stud­ies that could look at the ef­fi­ca­cy of short­er du­ra­tion reg­i­mens.

Add it all up, and Mat­teis is mak­ing a pre­dic­tion of $300 mil­lion in peak sales.

The mar­ket, though, is less in­ter­est­ed in the strings put on this ap­proval than the un­der­stand­ing they can glean about the FDA’s ap­proach to their oral fol­lowup SAGE-217. That drug is the key to un­lock­ing the block­buster mar­ket avail­able here. And so far, so good, with reg­u­la­tors gear­ing up to re­view a drug that has — so far — not been tied to the same safe­ty is­sues. 

With safe­ty da­ta on 140 pa­tients, Sage is still walk­ing on an in­cred­i­bly risky tightrope as it nav­i­gates its way in­to the mar­ket. Once used in a big­ger pop­u­la­tion, the drug is ripe for con­tro­ver­sy if new is­sues arise. On the oth­er hand, if Sage mas­ters the change and gets green-light­ed for PPD, it will be well on its way to jus­ti­fy­ing its multi­bil­lion-dol­lar val­u­a­tion. 

The stakes are enor­mous.

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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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).