Arrowhead Pharma has the wind at its back and the sun on its face. What could possibly go wrong?

The RNAi pioneer is building the commercial infrastructure to support its first approved drug, Redemplo (plozasiran), which was approved to treat familial chylomicronemia syndrome (FCS). But it's not really about FCS. The ultimate goal is to win sweeping approvals to treat hypertriglyceridemia with fancy labels for things like reducing the risk of pancreatitis. That would catapult the market opportunity from thousands of patients to millions of patients – on paper, anyway.

Data shared to date bode well for Redemplo to eventually claim blockbuster status, although it's a difficult market that might not ramp overnight. No matter. Arrowhead Pharma corrected its initial commercial mistake of pricing the drug product too aggressively (join Discord you uncultured brute), dropping the wholesale acquisition cost (WAC) from $60,000 per year to $45,000 per year. That's much more competitive with Tryngolza, an antisense oligonucleotide (ASO) from Ionis Pharma priced at $40,000 per year on the same basis. Redemplo is dosed less frequently and drives better patient outcomes to boot, so a small price advantage can be justified.

A trio of pivotal studies that will decide Redemplo's fate are expected to have topline data readouts in mid-2026. Throw in development updates for complement mediated diseases, obesity, MASH, neuro, asthma, and cardiovascular disease – as well as updates from assets partnered or owned by Amgen, Sarepta, Takeda, Novartis, and GlaxoSmithKline – and there's plenty for investors to be excited about in the near and long term. The current model is desperately outdated, but will be updated this summer.