Press Release Details
Arrowhead Presents Additional Clinical Data on Investigational ARO-HSD Treatment at AASLD Liver Meeting
Key data presented include the following:
Pharmacodynamic Response
- Dose-dependent pharmacodynamic effect on hepatic HSD17B13 mRNA was observed in all patients
- At 200 mg, all patients showed greater than 90% mRNA reductions
- Hepatic HSD17B13 protein levels were reduced at all ARO-HSD dose levels, with multiple measurements below the assay’s lower limit of quantitation
- Decreases in ALT and AST were observed at doses of 100 mg ARO-HSD and greater
- 9 of 18 patients had liver fat reductions on MRI-PDFF of 4-41%
- 6 of 18 patients had reduction in liver stiffness (kPa) on FibroScan of 4-37%
- Pharmacodynamic effect was not impacted by HSD17B13 (
rs72613567 , T>TA) or PNPLA3 (rs738409 , C>G) mutations
Safety
- ARO-HSD was well-tolerated in patients, with no ARO-HSD-related serious adverse events reported, no adverse events leading to drug discontinuations, and no ARO-HSD-related clinically significant adverse laboratory trends observed
- One treatment emergent serious adverse event of soft tissue injury that required hospitalization was reported in cohort 4b. This event was considered unrelated to study drug
A copy of the poster may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
ARO-HSD is an investigational RNAi therapeutic targeting HSD17B13 as a potential treatment for patients with alcohol-related and nonalcohol related liver diseases, such as NASH. HSD17B13 is a member of the hydroxysteroid dehydrogenase family involved in the metabolism of hormones, fatty acids, and bile acids. Published human genetic data indicate that a loss of function mutation in HSD17B13 provides strong protection against alcoholic hepatitis, cirrhosis, and NASH, with approximately 30-50% risk reduction compared to non-carriers.1 ARO-HSD is being investigated in AROHSD1001 (NCT04202354), a Phase 1/2 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-HSD in up to 74 normal healthy volunteers and patients with NASH or suspected NASH. Additional exploratory objectives of AROHSD1001 include the assessment of various measures of drug activity using liver biopsy.
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