Arrowhead Hosts R&D Day Highlighting Its Pipeline of RNAi Therapeutics - Seite 2

The R&D Day will also feature presentations by the Arrowhead team who will provide updates on multiple programs, in the following order:

Central nervous system (CNS):

• Development of the TRiM platform for intrathecal administration for CNS delivery demonstrates 90-95% dose-dependent mRNA knockdown in disease-relevant spinal cord and cortex brain regions in non-human primates​.
• ARO-SOD1 for treatment of patients with ALS caused by SOD1 mutations. A CTA filing is planned for the third quarter of 2023.

TRiM platform expansion progress:

• TRiM platform for systemic administration delivered siRNA across the blood brain barrier and achieved knockdown in non-human primates in all brain regions, including deep brain​.
• TRiM platform for adipose tissue delivery achieved up to 98% knockdown and maintained greater than 85% knockdown over 31 weeks.
• TRiM dimer platform for delivery to hepatocytes achieved equivalent or better knockdown of two separate target genes with longer duration than monomer mixture.

Pulmonary programs:

• ARO-RAGE for the treatment of patients with asthma is currently in a Phase 1/2 study.
  • New clinical data demonstrate continued dose response with single inhaled dose of 184 mg achieving mean knockdown of 90% and maximum knockdown of 95%.
  • New clinical data also show ARO-RAGE achieved serum sRAGE reductions in asthma patients consistent with effects seen in healthy volunteers at the 44 mg dose, the only dose level currently available in asthma patient cohorts​.
• ARO-MUC5AC for the treatment of patients with muco-obstructive pulmonary diseases is currently in a Phase 1/2 study.
• ARO-MMP7 for the treatment of patients with idiopathic pulmonary fibrosis is currently in a Phase 1/2 study.

Early clinical stage liver targeted programs:

• ARO-C3 for treatment of patients with various complement mediated diseases is currently in a Phase 1/2 study.
• ARO-PNPLA3 for treatment of patients with non-alcoholic steatohepatitis is currently in a Phase 1/2 study.

Cardiometabolic programs:

• ARO-APOC3 for the treatment of patients with familial chylomicronemia syndrome, severe hypertriglyceridemia, and mixed dyslipidemia in atherosclerotic cardiovascular disease is currently in a Phase 3 study and multiple Phase 2b studies.
• ARO-ANG3 for the treatment of patients with familial hypercholesterolemia is currently in multiple Phase 2b studies.

About Arrowhead Pharmaceuticals