FDA spurns J&J's trou­bled rheuma­toid arthri­tis drug sirukum­ab

New­man Yeild­ing

J&J $JNJ played this one straight out to the end, but af­ter an over­whelm­ing­ly neg­a­tive ex­pert pan­el vote against an ap­proval of its trou­bled rheuma­toid arthri­tis drug sirukum­ab, the FDA has for­mal­ly re­ject­ed the mar­ket­ing ap­pli­ca­tion — omi­nous­ly de­mand­ing more da­ta for re­view.

J&J said af­ter the mar­ket closed Fri­day that it will study the sit­u­a­tion on this drug, which some an­a­lysts once count­ed as a like­ly block­buster-to-be.

Just ahead of the Au­gust 2 agency pan­el re­view, Glax­o­SmithK­line pro­vid­ed a pre­view of what was to come when it abrupt­ly aban­doned its 6-year part­ner­ship on this drug. Then the pan­el vot­ed 12 to 1 against the ther­a­py, af­ter re­view­ing some trou­bling safe­ty da­ta.

At the time, it was an­oth­er red flag for RA, fol­low­ing a sim­i­lar re­jec­tion for Eli Lil­ly’s baric­i­tinib. The FDA re­versed its po­si­tion on de­mand­ing a new study on Lil­ly’s drug af­ter Scott Got­tlieb took over as com­mis­sion­er. But this time there was a clear in­di­ca­tion that the FDA plans to fol­low through in keep­ing the light red on for sirukum­ab.

Sev­er­al of the FDA’s ex­perts shook their heads over what they agreed was trou­bling but some­what mys­te­ri­ous ev­i­dence of an im­bal­ance in deaths among the tri­al pa­tients tak­ing the drug. While J&J in­sist­ed that the over­all ev­i­dence in­di­cat­ed that the risk/ben­e­fit ra­tion jus­ti­fied an ap­proval, some of the ex­perts won­dered why they would ap­prove an­oth­er IL drug with trou­bling safe­ty sig­nals while two oth­er such drugs were al­ready on the mar­ket that didn’t have the same safe­ty prob­lems.

“We are dis­ap­point­ed by this de­vel­op­ment as we feel the da­ta ac­cu­mu­lat­ed to date sup­port the ef­fi­ca­cy and safe­ty of sirukum­ab in the treat­ment of mod­er­ate­ly to se­vere­ly ac­tive rheuma­toid arthri­tis,” said New­man Yeild­ing, head of im­munol­o­gy de­vel­op­ment at Janssen. “We be­lieve sirukum­ab rep­re­sents an im­por­tant ther­a­peu­tic op­tion for pa­tients liv­ing with rheuma­toid arthri­tis, es­pe­cial­ly for those in­di­vid­u­als who cy­cle through mul­ti­ple treat­ments and con­tin­ue to strug­gle to find an ef­fec­tive op­tion for a po­ten­tial­ly dis­abling dis­ease. We are re­view­ing the de­tails of the com­plete re­sponse let­ter and plan to have a fol­low-up dis­cus­sion with the agency to gain a full un­der­stand­ing of FDA re­quire­ments for U.S. ap­proval.”

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.

Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

It’s also likely to be a much less controversial post than his board position at Pfizer, which stirred criticism from Democratic presidential candidate Elizabeth Warren.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.