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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.