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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Austin biotech Mol­e­c­u­lar Tem­plates lays off more than 100 staffers as pipeline nar­rows

Molecular Templates is ridding itself of a Phase I HER2 asset and fine-tuning its pipeline to focus on three programs and a preclinical Bristol Myers Squibb collaboration. With the narrowed scope on its so-called engineered toxin bodies, the Austin, TX biotech is laying off about half of its staff.

That’s a little more than 100 employees, per an SEC filing. Molecular’s layoffs, approved by its board Wednesday, add to the dozens of pullbacks in the industry in the first three months of 2023.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

Aptinyx eval­u­ates fu­ture of the com­pa­ny fol­low­ing two failed tri­als, 60% lay­offs

This year has been tough for Aptinyx — two failed trials, a 60% cut in its workforce, and now the company has brought on a firm to help evaluate the future of the company.

The press release noted it’s working with the firm Ladenburg Thalmann as its financial advisor to assist in exploring and evaluating “strategic alternatives” — a process that a growing group of struggling biotechs has embarked on, sometimes ending in a merger, asset sale or wind-down.

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Sar­to­rius to ac­quire French man­u­fac­tur­er for $2.6B+ in cell and gene ther­a­py play

The German life science group Sartorius will be picking up French contract manufacturer Polyplus for the price of €2.4 billion, or $2.6 billion.

On Friday, Sartorius announced the acquisition through its French subgroup, Sartorius Stedim Biotech, which will be acquiring Polyplus from private investors ARCHIMED and WP GG Holdings IV. Polyplus has 270 employees and produces materials and components that go into making viral vectors that are used in cell and gene therapies. This includes DNA/RNA reagents as well as plasmid DNA. Polyplus has locations in France, Belgium, China and the US.

TScan Therapeutics' departing CEO David Southwell and CSO/COO Gavin MacBeath

TCR up­start an­nounces CEO ex­it, with CSO now act­ing re­place­ment

A public T cell biotech’s chief executive has decided to leave the company.

TScan Therapeutics said Friday morning that CEO David Southwell stepped down earlier this week, leaving both his chief executive and board member roles. Filling in is Gavin MacBeath, the company’s CSO and COO. He became the acting CEO on Tuesday, and will continue to remain CSO and COO, TScan’s announcement read.