Ionis showcases mid-stage MASH win as competitive landscape heats up
13 Mar 2024
Clinical ResultPhase 2siRNAPhase 1
Ionis Pharmaceuticals staked its position in an increasingly-crowded liver disease field on Wednesday, revealing that its antisense inhibitor for metabolic dysfunction-associated steatohepatitis (MASH) met the primary endpoint and key secondary goal in a Phase II trial.
ION224 is an experimental ligand-conjugated antisense (LICA) treatment designed to reduce production of DGAT2, an enzyme that catalyses the final step in triglyceride synthesis in the liver, a hallmark of MASH. Boehringer Ingelheim is also taking an RNA-targeted approach for the disease; in January, the pharma partnered with Suzhou Ribo Life Science to use the latter’s small interfering RNA (siRNA) liver-targeting delivery technology to develop candidates for MASH.
The study randomised 160 participants to receive placebo or a once-monthly injection of ION224 at either the 60mg, 90mg or 120mg dose level. At 51 weeks, the middle and high doses achieved statistically significant liver histologic improvement as measured by a ≥2 stage reduction in non-alcoholic fatty liver disease (NAFLD) activity score, the primary endpoint.
Those two doses also achieved statistically significant MASH resolution without worsening of fibrosis, as measured by biopsy (p=0.039).
The study showed that ION224 appears to be safe as well, with no treatment-related serious adverse events or on-study deaths. Patients who received the antisense inhibitor had a lower early trial termination rate than the placebo arm, and did not experience any worsening of hepatic or renal function or gastrointestinal side effects.
Ionis expects to share the trial’s full results at an upcoming medical conference.
"This Phase II trial of ION224 is the first to demonstrate clinical evidence that the reduction of hepatic fat after DGAT2 inhibition correlates with improvements in MASH histological endpoints," said Rohit Loomba, the founding director of the MASLD Research Center at the University of California San Diego. He was not involved in the study.
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