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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Bernat Olle, Vedanta Biosciences CEO

Cit­ing 'chal­leng­ing eco­nom­ic en­vi­ron­ment,' PhI­II-ready mi­cro­bio­me biotech lays off 20% of staffers

The market downturn isn’t just sweeping up public biotechs.

Vedanta Biosciences, a developer of oral drugs derived from the human microbiome, is laying off about 20% of its staff — an unfortunately common occurrence these days. But CEO Bernat Olle took the unusual step of sharing the decision on LinkedIn and offering to connect the employees being let go with any company that’s hiring in their areas.

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Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Saurabh Saha, Centessa CEO (BIO19)

One of 2021's star biotech play­ers flags an­oth­er big set­back for the pipeline

Two months after scuttling their lead drug, Centessa’s executive team is back with the latest in a series of setbacks that have tanked its stock and blown holes in its strategic lineup of biotech subs.

The company reported in its Q2 post today that it has decided to scrap ZF874 after a patient demonstrated elevated liver enzymes — a classic red safety flag — in a Phase I study for alpha-1-antitrypsin (A1AT).

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.