Arrowhead Pharmaceuticals (ARWR) Investor update summary

Cardiometabolic pipeline overview

• Broad pipeline of RNAi therapeutics for rare and high-prevalence cardiometabolic diseases, leveraging the TRiM platform for deep and durable gene silencing.

• REDEMPLO (plozasiran) approved for familial chylomicronemia syndrome (FCS) in multiple regions, with commercial launch and additional launches for SHTG and HoFH anticipated.

• Pipeline includes over 20 clinical-stage programs, spanning early to late-stage assets, supported by strong financial resources and partnerships.

Technology and R&D process

• TRiM platform enables modular, rapid development of siRNA drugs targeting various cell types, including extrahepatic tissues, with scalable and durable gene silencing.

• AI-driven target identification and translational genetics enhance discovery and prioritization of new gene targets for siRNA therapeutics.

• Average of two to three new INDs or CTAs per year, with all programs homegrown from the TRiM platform and consistent pipeline growth.

Clinical development highlights

• REDEMPLO (plozasiran) demonstrated robust, sustained triglyceride reductions and reduced acute pancreatitis risk in severe hypertriglyceridemia over two years, with no AP events observed in long-term extension studies.

• SHASTA-3 and SHASTA-4 phase III studies for plozasiran in severe hypertriglyceridemia are fully enrolled, with top-line data expected in Q3 2026 and sNDA filing planned by year-end.

• Zodasiran targets ANGPTL3 for HoFH, showing up to 50% LDL-C reduction and a strong safety profile in phase II, with the YOSEMITE phase III study nearing full enrollment and NDA submission targeted for the second half of 2027.

• Broader pediatric program (SPRUCE) for zodasiran to initiate soon.