With the ink still drying on Arrowhead Pharmaceuticals’ recent announcement of successful 48-week results in a phase 2 trial of its RNAi drug candidate plozasiran, analysts are already projecting much longer-term success for the drug.
If approved, plozasiran could reach sales of around $707 million by 2032, according to a recent report from GlobalData.
Those revenues would come from the drug’s ability to fill a current gap in the market: It’s shown promise in reducing triglycerides and the protein APOC3, meaning it could be used to treat many forms of dyslipidemia, a family of diseases in which lipids or lipoproteins are found in abnormally high or low amounts in the blood, the presence of which is a known risk factor for heart disease.
Arrowhead’s latest results came from a study focused on patients with severe hypertriglyceridemia (SHTG) who have limited treatment options, according to Shireen Mohammad, Ph.D., a cardiovascular and metabolic disorders analyst at GlobalData, citing key opinion leaders interviewed for the report.
Those leaders also suggested that the drug prospect could be used to treat severe genetic disorders associated with heightened levels of triglycerides, such as familial chylomicronemia syndrome (FCS) and familial hypercholesterolemia. As Mohammad noted in the report, "there are no FDA-approved therapies to treat FCS. Therefore, there is a critical unmet need for new FCS treatments that effectively lower triglyceride levels and prevent the complications associated with this disease.”
Other ongoing studies of plozasiran have recruited patients with mixed dyslipidemia—where a phase 2 trial recently wrapped up—and FCS, for which a phase 3 trial is currently underway.
Last year, Arrowhead received a fast-track designation from the FDA for plozasiran’s use in reducing triglycerides in people with FCS.
“If approved, plozasiran offers hope for the dyslipidemia drug market. According to GlobalData, plozasiran is forecasted to launch in 2026 and is a promising pipeline drug for the treatment of dyslipidemia,” Mohammad said.
The data Arrowhead shared earlier this month from the SHTG-focused trial demonstrated how participants fared at the 48-week mark after receiving a second and final dose of plozasiran at Week 12.
Though APOC3 and triglycerides both began to creep back up in the following weeks, they still remained lower in the plozasiran group than among those assigned to placebo, with a 58% drop in triglycerides at Week 48 compared to just a 7% drop among the placebo group. At 24 weeks in the study, the drop in triglycerides was 70%.
With the company now planning to move into phase 3 trials of plozasiran in SHTG and mixed dyslipidemia, and the FCS trial already in process, it’s hot on the heels of Ionis Pharmaceuticals. Ionis has its own phase 3 trials in SHTG underway and has already wrapped up a phase 3 study in FCS, all centering on its APOC3 RNA-targeted investigational Ligand Conjugated Antisense (LICA) medicine olezarsen.