Arrowhead Pharmaceuticals has reported interim top-line results from an ongoing Phase I/IIa clinical trial investigating ARO-DIMER-PA to treat mixed hyperlipidaemia.
ARO-DIMER-PA is being developed for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidaemia.
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The ARODIMER-PA-1001 Phase I/IIa trial is designed to assess the safety, pharmacokinetics, pharmacodynamics, and lipid effects in up to 78 adults with mixed hyperlipidaemia.
It is evaluating the company’s first investigational RNA interference (RNAi) therapeutic dimer intended to target both proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes.
The interim analysis of single doses in individuals with mixed hyperlipidaemia showed that ARO-DIMER-PA produced dose-dependent mean maximal reductions in serum PCSK9 of 72% and in APOC3 of 88%.
Gene silencing was accompanied by reductions in several lipid measures, with reported mean maximal decreases of 54% in low-density lipoprotein cholesterol (LDL-C), 73% in triglycerides, 61% in non-high-density lipoprotein cholesterol, and 50% in apolipoprotein B.
The single-dose escalation portion of the study has so far been completed through the 400mg dose. The most frequently reported treatment-emergent adverse events were injection site reactions and headaches.
No drug-related serious adverse events have been reported to date. The trial remains ongoing to further evaluate the safety and tolerability of repeat dosing.
Arrowhead Pharmaceuticals president and CEO Chris Anzalone said: “With these interim top-line results, Arrowhead’s innovative and proprietary Targeted RNAi Molecule (TRiM) platform has achieved clinical validation of its ability to target and silence two genes simultaneously in one molecule.
“This represents an important step forward and a first for the field of RNAi therapeutics. We look forward to presenting additional details on this groundbreaking advance at an upcoming medical congress.”
Mixed hyperlipidaemia is described as a common disorder with raised levels of both LDL-C and triglycerides, presenting as a significant risk factor for ASCVD.
In July, Arrowhead Pharmaceuticals completed patient enrolment in the global Phase III YOSEMITE study of zodasiran, an investigational treatment for homozygous familial hypercholesterolaemia (HoFH).
