Arrowhead Pharmaceuticals has completed enrolment of subjects for its Phase III PALISADE trial of ARO-APOC3 to treat familial chylomicronemia syndrome (FCS), an ultrarare genetic disease often caused by various monogenic mutations.
The international AROAPOC3-3001 PALISADE trial will assess the investigational RNAi therapeutic ARO-APOC3’s efficacy and safety in adult subjects with FCS.
Percent change from baseline in fasting triglycerides (TG) at month ten is the trial’s primary endpoint.
For this trial, 75 subjects were distributed across 39 different sites in 18 countries and randomised to receive 25mg and 50mg doses of ARO-APOC3, or matching placebos, once every three months.
After completing the randomised period, participants will be eligible for continuing in a two-part extension period and will receive ARO-APOC3.
The primary portion of the clinical trial is expected to be completed in the second quarter of next year, with a data readout shortly thereafter and subsequent submissions of applications for regulatory review and approval.
Arrowhead Pharmaceuticals chief medical officer Javier San Martin said: “Completing enrolment of the Phase III PALISADE study represents a significant milestone for Arrowhead and brings our potentially important RNAi-based medicines closer to the patients that may benefit from them.
“I am grateful to the FCS patient community and principal investigators for their trust and participation in the PALISADE study.”
The company is developing ARO-APOC3 as a treatment for patients with mixed dyslipidemia, severe hypertriglyceridemia, and FCS.
Extremely high triglyceride levels due to FCS can lead to different serious signs and symptoms, including chronic abdominal pain, acute and potentially fatal pancreatitis, and type 2 diabetes mellitus, among others.
In July last year, Arrowhead dosed the first subjects in two Phase I/IIa clinical trials of pulmonary candidates, ARO-MUC5AC and ARO-RAGE, to potentially treat various muco-obstructive and inflammatory pulmonary diseases, respectively.
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