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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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