ARO-ENaC / Arrowhead - LARVOL DELTA

Arrowhead Pauses ARO-ENaC Phase 1/2 Clinical Study (Businesswire) - "Arrowhead Pharmaceuticals...today notified regulatory agencies, institutional review boards, and investigators that effective immediately it is voluntarily pausing AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF), after receiving a preliminary update from an ongoing chronic toxicology study in rats that contained unexpected signals of local lung inflammation....While we have not seen any concerning safety or tolerability signals in subjects enrolled in the AROENaC1001 study, out of an abundance of caution we have decided to pause new screening, enrollment, and any further dosing of investigational ARO-ENaC in the study while we await additional information from ongoing nonclinical toxicology studies. After we receive the full data from these studies, we will assess whether there is an acceptable path forward for further..."

Trial suspension • Cystic Fibrosis • Genetic Disorders

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2 | N=43 | Terminated | Sponsor: Arrowhead Pharmaceuticals | N=92 ➔ 43 | Trial completion date: Dec 2022 ➔ Sep 2021 | Recruiting ➔ Terminated | Trial primary completion date: Jun 2022 ➔ Sep 2021; Business decision

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2; N=92; Recruiting; Sponsor: Arrowhead Pharmaceuticals; Trial completion date: Mar 2022 ➔ Dec 2022; Trial primary completion date: Dec 2021 ➔ Jun 2022

Clinical • Trial completion date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Arrowhead Pharmaceuticals, inc (ARWR) Q4 2021 Earnings Call Transcript (The Motley Fool) - "I'd like to provide a quick update on our pulmonary programs, including ARO-ENaC, which is currently voluntarily paused to new enrollment as we assess some potential preclinical toxicology findings. We are still conducting studies internally to understand the toxicology findings and we don't have clarity yet on the path forward. While we conduct those studies, we continue to make progress on our two new pulmonary programs, which are on track for CTA filings in the first half of 2022....We believe in ENaC as a target for cystic fibrosis and are confident that our pulmonary targeted TRiM platform has the potential to address multiple diseases in the lung without adequate treatment options."

New trial • Preclinical • Cystic Fibrosis

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2; N=92; Recruiting; Sponsor: Arrowhead Pharmaceuticals; N=54 ➔ 92; Trial completion date: Nov 2021 ➔ Mar 2022; Trial primary completion date: Aug 2021 ➔ Dec 2021

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Arrowhead Pharmaceuticals, Inc (ARWR) Q1 2021 Earnings Call Transcript (The Motley Fool) - “ARO-ENaC is in a Phase I/II dose-escalating study to evaluate the safety, tolerability and pharmacokinetic effects of ARO-ENaC in up to 24 normal healthy volunteers and to evaluate the safety, tolerability and efficacy in up to 24 patients with CF….We are now dosing CF patients in the first cohort, which we expect to be fully enrolled before the end of next month, at which time we plan to begin enrolling the second patient cohort.”

Enrollment status • Trial status • Cystic Fibrosis • Genetic Disorders

How Arrowhead Protects Its Intellectual Property (SeekingAlpha) - "There's an a-ENaC portfolio with 2 expiry dates, 2028 and 2038, and a b-ENaC portfolio with a 2031 expiry date...ARO-Lung2 is being developed against an undisclosed target as a potential treatment for chronic obstructive pulmonary disorder (COPD). Arrowhead intends to file a clinical trial application at the end of 2020 to begin a Phase 1/2 clinical trial."

New P1/2 trial • Patent • Chronic Obstructive Pulmonary Disease • Cystic Fibrosis • Respiratory Diseases

Arrowhead Pharmaceuticals Initiates Dosing Phase 1/2 Study of ARO-ENaC for Treatment of Cystic Fibrosis (GlobeNewswire) - "Arrowhead Pharmaceuticals...today announced that it has dosed the first subjects in AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF)...AROENaC1001 is a Phase 1/2 dose-escalating study to evaluate the safety, tolerability, and pharmacokinetic effects of ARO-ENaC in up to 24 normal healthy volunteers and to evaluate the safety, tolerability, and efficacy in up to 30 patients with CF..."

Trial status • Cystic Fibrosis

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2; N=54; Recruiting; Sponsor: Arrowhead Pharmaceuticals; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Arrowhead Pharmaceuticals Hosts Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibrosis (Businesswire) - "Arrowhead Pharmaceuticals...is hosting a key opinion leader webinar today at 12:00 PM EDT on ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF)....Key points discussed on the webinar include...AROENaC1001, a Phase 1/2 study in up to 24 normal healthy volunteers (NHVs) and up to 30 CF patients, is expected to begin dosing in August 2020. Potential Phase 1/2 readout in the first half of 2021 may include safety in NHVs and patients, and an assessment of lung function in patients measured by forced expiratory volume (FEV1) and lung clearance index (LCI)..."

Live event • P1/2 data • Trial initiation date • Cystic Fibrosis

Arrowhead Pharmaceuticals to Host Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibrosis (Businesswire) - "Arrowhead Pharmaceuticals...today announced that on July 28, 2020, at 12:00 p.m. EDT it will host a key opinion leader webinar on ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF). The webinar will feature a presentation by Marcus Mall, M.D., Professor and Director of the Department of Pediatric Pulmonology and Immunology at The Charité University Medical Center Berlin...discuss the current treatment landscape and unmet medical need in treating patients with CF...also provide a review of the ARO-ENaC program...The webinar may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website."

Live event • Cystic Fibrosis

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2; N=54; Not yet recruiting; Sponsor: Arrowhead Pharmaceuticals

Clinical • New P1/2 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Arrowhead Pharmaceuticals files for regulatory clearance to begin phase 1/2a study of ARO-ENaC for treatment of cystic fibrosis (Businesswire) - "Arrowhead Pharmaceuticals Inc....today announced that it has filed an application for clearance to begin a Phase 1/2a clinical trial of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF)...We are excited to begin, pending regulatory clearance, the AROENaC1001 clinical study, which is designed to assess safety, tolerability, and pharmacokinetics and potentially provide an accelerated assessment of efficacy in patients with CF.The application for approval of the clinical trial is being submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials."

New P1/2 trial • Regulatory