Non-alcoholic steatohepatitis biotechs are increasingly finding it difficult to make headway, and the lack of any approved therapy despite a slew of research is a testament to the challenge.
Two NASH biotechs offered contrasting updates on their drugs Monday.
Independent Panel Says CymaBay's Drug Caused No Liver Injury
Newark, California-based CymaBay Therapeutics Inc (NASDAQ: CBAY) said an independent expert panel that reviewed findings from the Phase 2b study of its lead asset seladelpar in patients with NASH found no clinical, biochemical or histological evidence of drug-induced liver injury in the study.
In November 2019, the company terminated the midstage study of seladelpar in NASH patients following initial histological assessments that revealed atypical findings.
Apart from halting the NASH study and a Phase 2 study in subjects with primary sclerosing cholangitis, CymaBay also said at the time that it is putting seldadelpar studies in subjects with primary biliary cholangitis on hold as well.
CymaBay said Monday that an independent expert panel has unanimously supported the lifting of the clinical hold and the re-initiation of clinical development.
"Given the benefit observed on both NASH resolution and fibrosis with seladelpar in the NASH Phase 2b study as well as data presented at multiple medical meetings from studies of seladelpar in PBC, I am pleased that the independent review panel is supportive of restarting clinical development pending approval from the FDA," Dr. Stephen Harrison, principal investigator of the NASH study, said in a statement.
The company said it plans to re-engage with the FDA as quickly as possible.
Genfit's NASH Drug Fails To Meet Primary, Secondary Goals
French biotech Genfit SA (NASDAQ: GNFT) said the Phase 3 RESOLVE-IT study that evaluated 120mg of elafibranor in adults with NASH did not meet the primary endpoint of NASH resolution without worsening of fibrosis.
Interim results showed that the response rate was 19.2% for patients in the treatment arm compared to 14.7% for the placebo arm.
Genfit said no significant differences relative to the placebo were achieved on the key secondary endpoints, including fibrosis improvement of at least one stage and changes in metabolic parameters.
The company said it is reviewing the full interim dataset and will conduct analysis to decipher the higher-than-expected response rates in the placebo arm.
"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," CEO Pascal Prigent said in a statement.
In parallel, the NIS4 and Phase 3 primary biliary cholangitis programs will continue as planned, the company said.
CBAY, GNFT Price Action
CymaBay shares were soaring 126.09% to $4.16 ahead of Tuesday's close, while Genfit shares were plunging 67.28% to $7.10.
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Photo by Nephron via Wikimedia.