Arrowhead Pharmaceuticals Hosts Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibrosis
The webinar features a presentation by
Key points discussed on the webinar include the following:
- The epithelial sodium channel (ENaC) plays an important role in the pathophysiology of CF lung disease and represents a promising alternative target to improve airway surface hydration and mucus clearance in patients, independent of their CFTR genotype
- A substantial number of CF patients (without F508del allele) cannot be treated with current CFTR modulators and could benefit from ENaC inhibition
- Partial rescue of CFTR with current CFTR modulators does not prevent progression of CF lung disease, resulting in an unmet medical need for further improvement of targeted CF therapy
- ENaC inhibition has potential to act synergistically with CFTR modulators by improving the driving force for chloride/fluid secretion mediated by mutant CFTR rescued by CFTR modulators
- ENaC inhibition has potential as a novel therapeutic approach to improve mucus clearance and provide clinical benefits to patients with other muco-obstructive lung diseases, including COPD
- ENaC is a well-validated therapeutic target for cystic fibrosis and muco-obstructive lung disease
- ARO-ENaC overcomes critical limitations of small molecule inhibitors
- ARO-ENaC inhalation silences ENaC expression selectively in the lung, doubling mucociliary clearance for weeks post-dose and preserving clearance in a sheep disease model of mucostasis with no evidence of systemic activity (e.g. electrolyte imbalance)
- Promising results observed in various preclinical toxicology studies
-
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)
- There is a significant and growing population of patients without adequate response to current CF treatment
- Arrowhead is exploring potential accelerated regulatory pathways for patients with the highest unmet need, including Class I patients and other patient subsets with insufficient response to standard of care
CF is a rare disease caused by genetic mutations in the CFTR gene that lead to progressive deterioration in lung function due to poor clearance of mucus and associated recurrent infections. ARO-ENaC is designed to reduce activity of the epithelial sodium channel alpha subunit in the airways of the lung. In patients with CF, CFTR dysfunction causes increased ENaC activity, which contributes to airway dehydration and reduced mucociliary transport. This predisposes patients to persistent lung infections, structural damage, and progressive loss of pulmonary function. ENaC has been extensively explored as a potential therapeutic target for CF, but the development of inhaled small molecule ENaC inhibitors has been limited by on-target renal toxicity and short duration of action in the lung.
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