Pascal Prigent (Genfit)

Gen­fit prunes near­ly half its work­force af­ter NASH ex­it — but CEO promis­es a fu­ture in PBC, di­ag­nos­tics

Two months af­ter Gen­fit’s lead drug, elafi­bra­nor, failed a close­ly-watched Phase III show­down, the French biotech threw in the tow­el on NASH al­to­geth­er. An­oth­er two months lat­er, top ex­ecs are lay­ing off 40% of the work­force — 75 po­si­tions in to­tal — as they look to re­build.

The re­struc­tur­ing is part of a plan to re­duce the cash burn by more than half, ac­cord­ing to Gen­fit, from €110 mil­lion an­nu­al­ly be­fore the Phase III RE­SOLVE-IT da­ta dropped to around €45 mil­lion in 2022. With few­er than 125 staffers be­tween France and the US, the down­sized com­pa­ny will fo­cus on push­ing elafi­bra­nor in pri­ma­ry bil­iary cholan­gi­tis and com­mer­cial­iz­ing a di­ag­nos­tic tool for NASH.

It will take some time and ex­tra mon­ey to ful­ly close RE­SOLVE-IT, CEO Pas­cal Pri­gent not­ed, in ad­di­tion to shut­ting down pre­vi­ous com­mer­cial launch prepa­ra­tions.

In an an­a­lyst call of Gen­fit’s fu­ture pri­or­i­ties, he ex­plained that the de­ci­sion to ex­it NASH didn’t just have to do with the da­ta but lat­est de­vel­op­ments of the field:

We con­sid­ered the FDA’s feed­back re­gard­ing the oth­er com­plet­ed Phase 3 NASH pro­gram as well as new da­ta from sev­er­al on­go­ing NASH pro­grams, and we feel con­fi­dent we made the right de­ci­sion. In­deed, clin­i­cal tri­als in the NASH space are large, long and very ex­pen­sive. Con­sid­er­ing the evolv­ing NASH land­scape and the re­sults of the analy­sis from our com­plete dataset, we de­ter­mined that the cost to prob­a­bil­i­ty of suc­cess ra­tio was not ac­cept­able to con­tin­ue de­vel­op­ment of elafi­bra­nor in NASH, and of­fi­cial­ly ter­mi­nat­ed RE­SOLVE-IT, in line with our ear­li­er guid­ance in Ju­ly.

It is quite a damn­ing as­sess­ment com­ing from one of the two biotechs that, at the be­gin­ning of the year, were tapped as the play­ers to watch fol­low­ing a slew of fail­ures at oth­er com­pa­nies in 2019.

The oth­er, In­ter­cept, bit­ter­ly re­port­ed in June the FDA’s re­jec­tion of their NDA for obeti­cholic acid — a ma­jor set­back that they blamed on reg­u­la­tors’ change of heart re­gard­ing a sur­ro­gate end­point.

De­spite al­ready book­ing rev­enue from the drug, which is ap­proved as Ocali­va for PBC, In­ter­cept was still forced to chop 170 jobs — 25% of the head­count — and tight­en its belt while try­ing to win the FDA back.

For Gen­fit, the OK in PBC won’t come for quite some time. Hav­ing just start­ed pa­tient en­roll­ment, which they en­vi­sion will take 18 months af­ter fac­tor­ing in Covid-19 con­straints, ex­ecs are ex­pect­ing re­sults from the Phase III EL­A­TIVE study in 2023. Their drug is a dual ag­o­nist of PPAR-al­pha and PPAR-delta.

In the mean­time, though, it does ex­pect to start earn­ing mon­ey soon with its di­ag­nos­tic arm.

Gen­fit struck a deal with Lab­Corp just two days ago where the di­ag­nos­tics gi­ant would lead the mar­ket­ing ef­fort of its NIS4 test, de­signed to de­ter­mine whether a pa­tient needs to be treat­ed for NASH based on four blood-based bio­mark­ers.

While the di­ag­nos­tic was orig­i­nal­ly de­vel­oped as part of the elafi­bra­nor NASH pro­gram, Pri­gent be­lieves it has a fu­ture on its own. Biop­sies, af­ter all, are a way-too-ex­pen­sive, re­source-in­ten­sive and painful way to find out if pa­tients are at the stage of need­ing treat­ment. Launch­ing the test now, the rea­son­ing goes, paves the way for when the di­ag­nos­tic mar­ket takes off with the first NASH drugs.

Gen­fit’s blood test promis­es to be a sim­ple way to find pa­tients with at-risk NASH, mean­ing fi­bro­sis stage 2 above and a dis­ease ac­tiv­i­ty score of at least 4.

“As a re­minder, the scope of the ini­tial agree­ment was to pro­vide a so­lu­tion for spon­sors run­ning clin­i­cal tri­als in NASH,” Pri­gent said. “By us­ing NIS4 as a pre­screen­ing tool, spon­sors have been able to dras­ti­cal­ly re­duce the num­ber of screen fail­ures, thus ex­pe­dit­ing re­cruit­ment and sav­ing both, time and mon­ey, as well as bet­ter pro­tect­ing pa­tients.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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