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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

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Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.

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Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

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The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

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Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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