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
- 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.
For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.
Safe Harbor Statement under the Private Securities Litigation Reform Act:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.