Gen­fit’s bid to make R&D his­to­ry with a sto­ry­book turn­around came to a crash­ing halt on Mon­day as the French biotech ac­knowl­edged that its Phase III study of elafi­bra­nor failed to dis­tin­guish it­self from place­bo in treat­ing NASH.

Pas­cal Pri­gent

The biotech had tried to prove that the drug would re­solve NASH with­out fi­bro­sis in a pop­u­la­tion of more than 1,000 pa­tients. But the da­ta came up with a 19.2% re­sponse rate for the drug arm com­pared to a 14.7% rate for the place­bo.

Gen­fit CEO Pas­cal Pri­gent put down the “dis­ap­point­ing” re­sult to a high place­bo re­sponse, though the drug arm didn’t ap­pear to score all that high in the study.

The CEO not­ed:

We plan to share these de­tailed find­ings with the reg­u­la­to­ry au­thor­i­ties in the com­ing months and with their guid­ance, de­ter­mine a fi­nal de­ci­sion re­gard­ing the con­tin­u­a­tion of the RE­SOLVE-IT tri­al. In par­al­lel, we con­tin­ue as planned with our NIS4TM and Phase 3 PBC (pri­ma­ry bil­iary cholan­gi­tis) pro­grams, which are in­de­pen­dent of our NASH pro­gram with elafi­bra­nor. We will pro­vide up­dat­ed guid­ance on our glob­al cor­po­rate strat­e­gy lat­er in the year, once we have more clar­i­ty on the reg­u­la­to­ry im­pli­ca­tions of the RE­SOLVE-IT in­ter­im read­out, as well as more vis­i­bil­i­ty on the evo­lu­tion of the im­pact of the world­wide pan­dem­ic on our on­go­ing stud­ies.

Last year saw mul­ti­ple set­backs in NASH, with In­ter­cept as the sole ex­cep­tion to the rule. That left some an­a­lysts cau­tious­ly en­cour­ag­ing in­vestors to look at Gen­fit, even though the odds were daunt­ing.

SVB Leerink’s Pasha Sar­raf had been on the pro-Gen­fit side­lines, call­ing this study “the most sig­nif­i­cant and volatile up­com­ing cat­a­lyst” they cov­er. “The risk/re­ward is not for the faint of heart.”

There were al­so plen­ty of skep­tics along the way, par­tic­u­lar­ly af­ter Gen­fit not­ed a Phase II NASH fail­ure for GOLD­EN-505 in 2015, then tried to ex­plain it all away as a fail­ure of the tri­al de­sign.

The fail­ure leaves In­ter­cept $ICPT as the sole win­ner in this field so far, with Ocali­va un­der re­view for a la­bel ex­pan­sion on NASH.

Iron­i­cal­ly, on the same day that Gen­fit raised fresh ques­tions about the PPAR class, a ri­val in the class — CymaBay — popped up look­ing for a sec­ond chance. Michael Yee at Jef­feries not­ed:

CBAY which has a Ph II PPAR-delta fo­cused mol­e­cule but pre­vi­ous­ly halt­ed the pro­gram due to liv­er-in­jury sig­nals & FDA hold, an­nounced on the same day that an in­de­pen­dent ex­pert pan­el unan­i­mous­ly con­clud­ed there is no ev­i­dence of drug-in­duced liv­er in­jury, & they will re-en­gage with the FDA to see if they can lift the hold. It’s un­clear to us what will hap­pen but over­all we would re­main cau­tious on the big­ger pic­ture view that a PPAR which was halt­ed in Ph II will com­plete­ly re­move the safe­ty ques­tions un­til a full Ph III would be com­plet­ed.

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.

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.

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

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.