Arrowhead's lead RNAi heart drug takes centre stage, the other deprioritised

2024-06-26

临床3期临床结果AHA会议

Arrowhead Pharmaceuticals said it is refining its cardiometabolic pipeline, choosing to focus its resources on the development of plozasiran, while seeking a partner for zodasiran. The decision comes as the company prepares to advance plozasiran into a Phase III cardiovascular outcomes trial.

Plozasiran, an RNAi therapeutic designed to reduce the production of apolipoprotein-CIII (ApoC3), has shown promising results in studies across three distinct patient populations: familial chylomicronaemia syndrome (FCS), severe hypertriglyceridaemia and mixed hyperlipidaemia.

The company recently announced positive top-line data from the Phase III PALISADE study, which evaluated plozasiran in patients with FCS. The trial met the primary endpoint of lowering triglycerides (TG) and met all key secondary goals, including reducing the incidence of acute pancreatitis, compared to placebo.

Durable triglyceride reductions

Arrowhead shared additional data from PALISADE at its R&D day this week. Building on results showing placebo-adjusted median TG reductions of -80% and -78% at month 10 with quarterly doses of 25 mg and 50 mg, respectively, Arrowhead presented curves showing quick and lasting reductions in TG compared to baseline. "Plozasiran, administered once every three months, consistently maintained the median and mean triglyceride levels over the study period with low variability," said chief medical scientist Bruce Given.

The company also provided detailed safety data indicating that the incidence of treatment-emergent adverse events (AEs) was comparable between the placebo and active groups, while the rate of serious and severe AEs was slightly higher among placebo patients.

"We view the incremental update as encouraging," commented analysts at Morgan Stanley. The company will present the full dataset at a future scientific conference.

Meanwhile, Arrowhead is preparing to start a Phase III cardiovascular outcomes trial, dubbed CAPITAN, to further evaluate plozasiran in a broader patient population. The study will enroll patients with mixed hyperlipidaemia and residual risk of atherosclerotic cardiovascular disease.

Resource considerations

As for zodasiran, which targets ANGPTL3, the company indicated that while the data generated so far has been "highly encouraging," and warrants further investigation in Phase III, future development will require a partner.

"Based on our various assessments for both candidates, and resource allocation considerations, we have elected to broadly advance plozasiran ourselves and, at this time, will only conduct further development of zodasiran if we identify a suitable development and commercialisation partner," Given said.

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