RNAi Digest - August 2020

RNAi Digest - August 2020

Arrowhead Pharmaceuticals dosed first patient in Phase 1/2 dosing study of ARO-ENaC for Cystic fibrosis, and in a Phase 1b study of ARO-HIF2 in clear cell renal cell carcinoma (ccRCC) and both studies

18 Aug 2020

ARO-ENaC (siRNA targeting αENaC)

  • Dosed the first subject in AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC in healthy volunteers (N= 24 )and patients with cystic fibrosis (CF) (N=30)
  • This is a dose-escalating study to evaluate the safety, tolerability, and pharmacokinetic effects of ARO-ENaC

ARO-HIF2 (TRIM targeting HIF-2α)

  • Dosed first patient in AROHIF21001, a Phase 1b dose-finding study in advanced clear cell renal cell carcinoma (ccRCC) N=18
  • ARO-ENaC and ARO-HIF2 are designed by Arrowhead pharmaceuticals’ proprietary TRiM platform (produces a double stranded, siRNA duplex conjugated to an integrin avb6 epithelial targeting ligand)

First patients dosed with Arrowhead’s inhaled RNAi and RNAi for ccRCC

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CI Scientists Remarks: 

  • Arrowhead is moving forward with its 2 most innovative and lead compounds
    • ARO-ENaC is the first inhaled RNAi therapy in clinical development targeting a pulmonary indication
      • CFTR modulators like Trikafta are only effective in 85% of CF patients with single F508del mutation, Inhaled ENaC inhibitors have been unsuccessful due to short-lived effect and side effect on kidney ENaC
      • Arrowhead’s ARO-ENaC, is for patients without F508del mutation or have insufficient response to CFTR modulators; ARO-ENaC showed positive signals in preclinical models in CF and COPD; will explore the accelerated review approach for Class I patients (without CFTR proteins)
    • ARO-HIF2 is the first and only product in Arrowhead’s pipeline targeting cancer
      • About ccRCC – Patients have an inactivated mutation in the Von Hippel-Lindau gene – they are unable to degrade HIF-2α, which leads to an increase in activity during tumor hypoxia and promotes tumor growth
  • β-ENaC a likely a back-up asset

– Tarun Raisinghani, CI Scientists

For full story click here: ARO-ENaC, ARO-HIF2

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