Aqilion AB announced today that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for AQ280, a selective JAK1 inhibitor being developed for the treatment of eosinophilic esophagitis (EoE).
The approval marks a significant milestone for the Swedish biotech company as it prepares to initiate its first US-based clinical study. The ARIA-2 trial, scheduled to begin in June 2025, will evaluate a novel formulation of AQ280 specifically designed for patients with swallowing difficulties – a common symptom of EoE.
"It is strategically important for us to continue clinical development with a formulation that is specially adapted for patients who have difficulty swallowing," said Sarah Fredriksson, CEO of Aqilion. "With a positive response from the FDA, we look forward to completing the final preparations for the start of a Phase 2 study in EoE patients."
Novel Formulation Addresses Key Patient Need
The ARIA-2 study will focus on verifying that the pharmacokinetic profile of a new water-dissolving tablet formulation matches that of the capsule formulation used in the previous Phase 1 ARIA-1 trial. The study will be conducted in healthy volunteers with results expected after summer 2025.
This formulation innovation directly addresses a critical need for EoE patients, many of whom struggle with dysphagia (difficulty swallowing) as a primary symptom of their condition. Upon successful completion of ARIA-2, Aqilion plans to submit applications for a Phase 2 study in diagnosed EoE patients across multiple regions including the US, Canada, and Europe.
AQ280: A Targeted Approach to Inflammation
AQ280 represents a potentially significant advancement in EoE treatment as a super-selective JAK1 inhibitor. The drug candidate works by targeting Janus Kinase 1, an enzyme that accelerates inflammatory processes involved in allergic reactions and fibrosis development.
The mechanism differs from currently approved EoE treatments, which include corticosteroids in Europe and an anti-IL4/13 antibody biologic in both the US and Europe. No JAK1 inhibitor is currently approved for EoE treatment, potentially positioning AQ280 as a first-in-class therapy for this indication.
Promising Phase 1 Results Support Development
The Phase 1 ARIA-1 study, conducted in the United Kingdom with 66 healthy volunteers, demonstrated several encouraging findings that support continued development:
- Dosing achieved exposures within the estimated therapeutic range based on preclinical models
- No serious adverse events occurred during the trial
- Pharmacokinetic data supported once-daily dosing
- AQ280 demonstrated dose-dependent inhibition of CXCL10, a biomarker for JAK inhibition
- The drug showed selective JAK1 effects without detectable JAK2 activity on reticulocytes
These results provide a solid foundation for the upcoming ARIA-2 study and subsequent Phase 2 clinical trials.
Rising Prevalence Highlights Unmet Need
Eosinophilic esophagitis is a chronic allergic disease of the esophagus characterized by eosinophilic inflammation. The condition typically manifests between ages 20-40, though it can affect patients of any age. Primary symptoms include difficulty swallowing, and in advanced cases, the disease can lead to scar tissue development and esophageal strictures that cause food impaction.
The prevalence of EoE has been increasing rapidly as diagnostic awareness grows among healthcare providers. This rising incidence, combined with limited treatment options, underscores the significant unmet medical need that AQ280 aims to address.
Strategic Importance for Aqilion
The IND approval represents an important advancement for Aqilion's pipeline focused on chronic inflammatory diseases. The company specializes in developing innovative treatments for conditions caused by dysfunctional immunological reactions and autoimmune processes.
With this regulatory milestone achieved, Aqilion continues to execute its strategy of advancing promising drug candidates through early clinical development stages before potentially partnering for later-stage development and commercialization.
The company operates with a partially virtual organizational structure, collaborating with specialized partners to efficiently advance its development programs from concept to clinical proof-of-concept.
