R&D Day Highlights Potential Of Cardiometabolic Pipeline; Focus On Q3 Readouts

Research evidence excerpt

R&D Day Highlights Potential Of Cardiometabolic Pipeline; Focus On Q3 Readouts

d do not substantially lower LDL-C. When TGs

are elevated above 500mg/dL this increases the chance for acute pancreatitis.

The KOL was optimistic for the potential role of ARO-DIMER-PA in mixed hyperlipidemia, noting

that both PCSK9 and APOC3 inhibition are validated targets for lowering LDL-C and TGs,

respectively. Further, the preclinical NHP data are supportive with a 50% reduction in LDL-

C and 70% reduction in TGs. Thus, he believed ARO-DIMER-PA has the potential to reduce

cardiovascular morbidity and mortality over individual LDL-C and TG lowering therapies alone.

Finally, he noted that adherence to LDL-C and TG lowering medications is a major issue when

treating these patients, and ARWR's platform and potentially longer-term administration could

provide another benefit.

The program is still early in development, thus we don't currently include it in our model. That

said, mixed dyslipidemia represents a large population and would represent upside to our

current estimates, if ARO-DIMER-PA is successfully developed.

The Ph. III Study Of Zodasiran Is Expected To Complete Enrollment This Summer Leading To

Data Next Summer

Arrowhead is also developing zodasiran, a liver-targeted anti-ANGPTL3 siRNA for homozygous

familial hypercholesterolemia (HoFH). ARWR has completed several studies in mixed

hyperlipidemia and HoFH and is currently conducting the pivotal Ph. III "YOSEMITE" study

that will evaluate the effect of zodasiran in n=60 adolescent and adult HoFH patients (2:1

randomization). The primary endpoint is reduction of fasting LDL-C from baseline at month 12.

HoFH is a rare genetic disorder characterized by LDL-C levels ranging from 400-1,000 mg/dL