Fi­bro­Gen’s new PhI­II da­ta set it up for quick OK, an­a­lysts say. But can it avoid a black box warn­ing?

Less than two months out from its PDU­FA date, Fi­bro­Gen has rolled out a slate of Phase III da­ta on rox­adu­s­tat that an­a­lysts say all but seals the deal on a broad OK for ane­mia as­so­ci­at­ed with CKD. A black box warn­ing, though, may still dim its prospects.

Fi­bro­Gen pre­sent­ed da­ta from six dif­fer­ent Phase III stud­ies at the Amer­i­can So­ci­ety of Nephrol­o­gy Kid­ney Week, cov­er­ing a broad spec­trum of CKD pa­tients both dial­y­sis-de­pen­dent and non-dial­y­sis-de­pen­dent. No­tably, the As­traZeneca-part­nered drug scored some pos­i­tive num­bers on MACE and MACE+ rates in a pooled safe­ty analy­sis, sug­gest­ing non-in­fe­ri­or­i­ty to place­bo on car­dio out­come.

“Based on the da­ta, we think rox­adu­s­tat sets a high bar for both ef­fi­ca­cy and safe­ty as the po­ten­tial­ly first-in-class HIF-PHI drug,” Cowen an­a­lyst Yaron Wer­ber wrote in a note. “We have high con­vic­tion that rox­adu­s­tat will re­ceive broad ap­proval for both NDD- and DD-CKD by its 12/20/20 PDU­FA.”

Just weeks ago, Fi­bro­Gen’s ri­vals at Ake­bia were slammed de­spite hit­ting the pri­ma­ry and key sec­ondary end­points, pre­cise­ly be­cause of fail­ing the all-im­por­tant MACE non-in­fe­ri­or­i­ty stan­dard. As pa­tients’ ane­mia wors­ens over time, de­creased oxy­gena­tion of­ten re­sults in an in­creased risk of car­dio­vas­cu­lar com­pli­ca­tions and even death, Fi­bro­Gen CMO Pe­ony Yu said in a state­ment.

While the ery­thro­poi­etin stim­u­lat­ing agents cur­rent­ly tak­en by ane­mic CKD pa­tients are tied to in­creased CV risk at high­er he­mo­glo­bin lev­els, roxa-treat­ed pa­tients ex­pe­ri­enced low­er CV risks with high­er Hb lev­els.

“This is im­por­tant to roxa and its com­mer­cial out­look, since the in­creased CV risk as­so­ci­at­ed with high­er Hb cost ESAs a black­box in 2011 and re­duced rev­enue by more than a half,” Ge­of­frey Porges of SVB Leerink wrote. “This post-hoc analy­sis won’t nec­es­sar­i­ly guar­an­tee a black­box-free la­bel for roxa, but it demon­strat­ed very en­cour­ag­ing sig­nals that roxa is a safer oral agent for the treat­ment of chron­ic kid­ney dis­ease (CKD) ane­mia.”

Then there’s the re­duc­tion in to­tal cho­les­terol, LDL cho­les­terol and triglyc­erides, all hope­ful signs for a group at high risk of car­dio­vas­cu­lar com­pli­ca­tions and even death.

Some an­a­lysts had al­ready pro­ject­ed roxa’s peak sales at $2 bil­lion or more, with Jef­feries’ Michael Yee pre­dict­ing that Fi­bro­Gen “could have the whole $2-3B+ NDD mar­ket to it­self” giv­en Ake­bia’s flop.

Mizuho es­sen­tial­ly echoed that sen­ti­ment this morn­ing, cast­ing ap­proval in DD high­ly like­ly for Ake­bia but a more dif­fi­cult path for­ward for the NDD in­di­ca­tion. But the dial­y­sis set­ting still rep­re­sents a $2 bil­lion US mar­ket.

“New da­ta from Ake­bia sug­gests low­er MACE in US-treat­ed pa­tients not on dial­y­sis (vs. Ex-US) dri­ven by re­gion­al pro­to­col dif­fer­ences, and we ex­pect this to be a key dis­cus­sion point with FDA,” a note from the firm read.

Back in 2018, Fi­bro­Gen and As­traZeneca marked the first time a top glob­al phar­ma se­cured ap­proval for a po­ten­tial block­buster drug in Chi­na be­fore even fil­ing in the Unit­ed States or Eu­rope — al­though the Chi­nese OK on­ly cov­ered dial­y­sis-de­pen­dent pa­tients.

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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