In­ter­cept ‘low­ers the bar’ on its piv­otal PhI­II OCA study for NASH

In­ter­cept CEO Mark Pruzan­s­ki

In­ter­cept CEO Mark Pruzan­s­ki has re­drawn the rules on their piv­otal Phase III study of obeti­cholic acid (OCA).

Af­ter talks with the FDA, Pruzan­s­ki said to­day that in­ves­ti­ga­tors can re­duce their tar­get on the num­ber of pa­tients need­ed for an in­ter­im analy­sis of the FXR ag­o­nist in 2019, drop­ping the tar­get from 1,400 to 750. And the agency has signed off on chang­ing the end­point from fi­bro­sis and NASH res­o­lu­tion to ei­ther fi­bro­sis or NASH while ac­cept­ing an ob­jec­tive de­f­i­n­i­tion of NASH res­o­lu­tion to clar­i­fy suc­cess.

“We now on­ly need to achieve one end­point for the tri­al to be con­sid­ered suc­cess­ful,” the CEO said on a call with an­a­lysts Fri­day morn­ing, say­ing that the bar has been low­ered on suc­cess, in­creas­ing their odds of suc­cess. And if the drug hits both end­points, he added, In­ter­cept $ICPT will be able to “clear­ly dif­fer­en­ti­ate this drug from oth­ers.”

“We be­lieve we now have the best case sce­nario,” he ad­ed, gain­ing a low­er hur­dle while keep­ing their sights set on both end­points.

“We con­tin­ue to be con­fi­dent in both end­points,” said Pruzan­s­ki. And the in­ter­im read­out on the close­ly watched study is ex­pect­ed in 2019 re­mains the same. “We’ve suc­cess­ful­ly de­risked the study” with­out risk­ing the in­tegri­ty of the tri­al, he said. Ri­vals in the field, he added, have field­ed da­ta on one or the oth­er end­point.

The switch at In­ter­cept could have big im­pli­ca­tions for Al­ler­gan, notes Ever­core ISI’s Umer Raf­fat, which is plan­ning its own Phase III af­ter buy­ing To­bi­ra. And it ap­pears con­sis­tent with Gilead’s plan for the “Ph 3 for selon­sert­ib (where pri­ma­ry end­point will be fi­bro­sis.)”

It’s clear that the change-up is oc­cur­ring af­ter In­ter­cept’s time­line on re­cruit­ing the pa­tients need­ed for an in­ter­im analy­sis fell well be­hind sched­ule. Ear­li­er, In­ter­cept had said that it could en­roll all 1,400 pa­tients in the first half of this year. Now it’s say­ing that the new goal of 750 will be com­plet­ed in mid-2017, mean­ing that the switch will help keep them on sched­ule in­stead of los­ing the edge they have with ri­vals.

In­ter­cept set up the call on the fly this morn­ing, with­out any ex­plain­er in a press re­lease. That move drove its stock in­to the red ahead of the an­nounce­ment, but shares snapped back in­to the green once the news be­came clear.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,400+ biopharma pros reading Endpoints daily — and it's free.

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.