Regulus

Aqilion announces that the company has decided to focus on two of its pipeline projects; drug candidates AQ280 for eosinophilic esophagitis (EoE) and AQ128 as a topical treatment for psoriasis. AQ280 – Eosinophilic Esophagitis (EoE) Aqilion is developing AQ280 as a new treatment for eosinophilic esophagitis (EoE), an allergic disease of the esophagus which primary symptom is severe swallowing difficulties. The incidence of the disease is rising rapidly, today approximately 1 in 700 people in the US are affected and there is a clear medical need for new alternative treatments based on novel mechanisms of action. AQ280 is an orally available, super-selective JAK1 inhibitor that reduces inflammation and disease progression in chronic inflammatory diseases. As EoE patients have difficulty swallowing, Aqilion has developed a water-soluble tablet formulation. Two successful Phase 1 studies (ARIA-1 and ARIA-2) have been completed, in which AQ280 was well tolerated and demonstrated promising effects on relevant biomarkers. The results now enable progression to Phase 2. AQ128 – Psoriasis Psoriasis is a chronic inflammatory disease affecting 2–4% of the population, currently treated primarily with corticosteroids, which can cause troublesome side effects with long-term use. AQ128 is a highly potent, selective inhibitor of the enzyme TAK1, a central node in several pro-inflammatory signaling pathways, with the potential to become first-in-class in topical dermatology. In a psoriasis-like disease model, AQ128 has demonstrated efficacy comparable to the superpotent corticosteroid clobetasol but without the systemic effects observed with clobetasol. AQ312 discontinued and PKCtheta on hold The company has simultaneously decided to discontinue project AQ312 and will for the time being put the early discovery project PKCtheta on hold. The decision frees up resources to progress the programs with the greatest potential to reach value-creating milestones. The decision to discontinue AQ312 is primarily based on the project’s development risk. CEO Sarah Fredriksson comments: focus to drive value "We are at an exciting stage for Aqilion. With AQ280 ready to advance into Phase 2 following successful Phase 1 studies, and AQ128 showing truly promising results in preclinical models, we have a pipeline with real potential to make a difference for patients with chronic inflammatory diseases. By focusing our resources on these two programs, we are creating the best conditions to reach important milestones, attract development partners, and strengthen the company’s position. We are convinced that this is the right path forward – for the company, for our shareholders, and not least for the patients who are waiting for better treatment options.” For more information, please contact: Sarah Fredriksson, CEO, AQILION AB, + 46 (0)70 261 4575, sarah.fredriksson@aqilion.com

Aqilion, a Swedish biopharma company developing therapies for diseases driven by chronic inflammation and dysregulated immune responses, has reported positive results from a pharmacokinetic study evaluating its investigational treatment for eosinophilic esophagitis (EoE). EoE is an allergic inflammatory disease of the oesophagus characterised by swallowing difficulties, the development of scar tissue and oesophageal strictures. To date, only two types of therapy have been approved for EoE: corticosteroids and biologic treatments targeting the interleukin (IL)-4 and IL-13 pathways. Aqilion’s oral candidate, AQ280, is a JAK1 inhibitor offering a potential new therapeutic approach for EoE with a distinct mechanism of action. By inhibiting JAK1, a kinase enzyme involved in inflammatory signalling, AQ280 aims to suppress inflammatory processes, reducing symptoms and disease progression. The ARIA-2 study assessed the pharmacokinetic profile of a novel, water-dissolvable tablet formulation of AQ280 designed to improve ease of administration for patients. The study compared the tablet’s pharmacokinetics with the capsule formulation previously evaluated in the phase 1 ARIA-1 study. Following the positive results from ARIA-1 and ARIA-2, Aqilion is preparing to initiate phase 2a and 2b clinical trials in patients with EoE across Europe, Canada and the United States. AQ280 received Investigational New Drug (IND) approval earlier this year. Sarah Fredriksson, CEO of Aqilion, said: “[With] the results from this pharmacokinetic bridging study, it is an important milestone to have the formulation ready with EoE patients in focus. Finalising ARIA-2 now means that we can fully focus on the phase 2 study.”